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Access to New Medicines

– in the Scottish Parliament on 9th October 2013.

Alert me about debates like this

Photo of John Scott John Scott Conservative

The next item of business is a Health and Sport Committee debate on access to new medicines. I invite Mr McNeil to open the debate on behalf of the Health and Sport committee. You have 14 minutes.

Photo of Duncan McNeil Duncan McNeil Labour

First, I thank the clerks, the Scottish Parliament information centre and all those who contributed written evidence and gave of their time to participate in the committee’s public sessions. It is just as important to thank all those people who came forward over the period of the committee’s inquiry at a difficult and vulnerable time in their life and spoke out about the failings in terms of the patient experience. I am speaking on behalf of the Juszczak family in my constituency and the family of Anne Fisher, who is sadly deceased, who spoke out at a very difficult time. All those contributions ensured that the Health and Sport Committee approached the whole issue with sensitivity and consensus.

Access to medicines is a big, complex and deeply emotive issue. The committee first looked at the matter in March 2012 when we received a trio of petitions on orphan medicines. Now, as they say, for the science bit: orphan medicines are those used to treat very rare diseases. We have learned a lot in the past 18 months and I hope to share some of it with members in the next 13 minutes.

I want to retrace our steps as a reminder of why we are discussing the issue today. Along with the usual who, what and when, I will set out the main findings of the report that we published in early July, which is the how. I shall also offer some thoughts on yesterday’s statement from the cabinet secretary in the where-we-are-headed bit.

My colleagues on the Public Petitions Committee deserve credit for their role in this story. It was with their committee that the petitioners first raised their concerns. The petitioners were Alastair Kent, Allan Muir, Lesley Loeliger and Professor Peter Hillmen, individuals working on behalf of Rare Disease UK, the Association for Glycogen Storage Disease, and PNH Scotland, respectively. I am sure that they would all acknowledge the work on an earlier petition of January 2008 by Tina McGeever, on behalf of the late Mike Gray, which resulted in revision of the guidelines to the end-to-end process, which is the licensing of medicines through to individual patient treatment requests, or IPTRs.

The petitioners argued that the revision had not resulted in improved access to orphan medicines for patients with rare diseases. The committee took evidence last March from the petitioners and then from the Scottish Medicines Consortium, the SMC, and the Association of the British Pharmaceutical Industry, the ABPI. I apologise for all the acronyms—it is like a secret services convention.

We followed up the earlier evidence with evidence from clinicians and patient representative bodies. On 14 November 2012, the cabinet secretary announced the Routledge and Swainson reviews. The committee heard from the authors of the reviews and from the cabinet secretary and the chief pharmaceutical officer on 7 May 2013. On 21 May, we held a further round-table session with interested parties to gauge reaction to the twin reviews. The report of the committee’s findings was published on 3 July. The committee found that there is enthusiasm from all quarters to work together to improve the system for accessing new medicines and create a system that enables a wider assessment of their value with more of what might be termed a societal dimension.

Our report welcomes the recommendations from Swainson and Routledge, but we want both the IPTR and the SMC processes to be improved to ensure that we have a more transparent system for accessing new medicines. In short, we want more yeses. Many of the suggestions in the reviews are welcome, including those on meetings being held in public, the standardisation of paperwork, the monitoring of applications and the publication of decisions, but they are about process and would do little to improve access.

One of the difficulties with the IPTR system lies in establishing the exceptionality of the patient’s circumstances. We said that the Scottish Government must outline the steps that it will take to improve the system. Decisions on whether to recommend a medicine for use in Scotland depend on the cost of the additional quality-adjusted life years—a system known as QALY. I hope that the official reporters have a glossary, because there are a lot of acronyms in this area. Nobody told us of a better system than QALY for assessing the value of competing treatments. Who knew that an equation could be as brutal as cost divided by the number of weeks for which a life might be extended?

However, the way in which so-called modifiers are applied is crucial in determining the cost effectiveness of medicines. We asked the SMC and the Scottish Government to review how modifiers and thresholds are applied to take better account of orphan and ultra-orphan conditions, end of life and innovation. After all, our work began with the petitions on orphan and ultra-orphan medicines.

We welcomed the interim £21 million rare conditions medicines fund, but questions remain about the extent to which it can improve access to medicines. The committee said that clear guidance should be published and that decisions about specific cancer medicines should be made on the same basis as decisions on medicines for other conditions. We said that cancer should not be singled out in comparison with other life-shortening conditions. The committee accepted that this was a difficult issue. Nevertheless, we did not believe that a cancer drugs fund was the Scottish answer.

The committee recognised that there were concerns about the impact of innovative medicines not routinely being available in Scotland and we asked the Scottish Government to investigate. Likewise, we said that developments with value-based pricing, or VBP, should be monitored.

I am afraid that I do not have time to tell you about ADTCs, which are area drug and therapeutics committees, about PPRS, which is the pharmaceutical price regulation scheme or about NICE, which is the National Institute for Health and Care Excellence. I could say “NICE but naughty”—speaking of which, I note that it was only yesterday morning when we received a copy of the Scottish Government’s response to our report. That is a shame, as we would have liked to have fully considered it and come to a view as a committee.

What I can say is that I appreciate the language and the intention of what is proposed. The Cabinet Secretary for Health and Wellbeing wants to move to a more flexible approach to the evaluation of medicines for end-of-life care and rare conditions and he wants to increase access to new medicines, which is good. The committee and the cabinet secretary are on the same page, but we need to see the detail, which is where the devil lurks, as always.

The Scottish Government says that value-based pricing will not be delivered. It believes that the pricing element of pharmaceutical price regulation is a reserved matter but medicines assessment is devolved, and it will develop a new value-based assessment, or VBA, process for Scotland.

As a first step, the SMC has begun to look at the evaluation of orphan, ultra-orphan and end-of-life medicines. That is to include a review of the wider aspects of value and QALYs to increase access to those medicines. The report states that the SMC is due to report its findings to the cabinet secretary before Christmas. As somebody once said,

“I love deadlines. I like the whooshing sound they make as they fly by”.

Let this please be one of those deadlines that we are able to stick to. That is particularly important for those who have been diagnosed with these conditions last week or today, or who will be diagnosed with them tomorrow or next week. Some consideration should be given to how the system operates in the transitional phase, but we need to stick to the deadline.

The IPTR system is being replaced with a new peer approved clinical system—PACS. We are told that guidance will be published shortly and I seek assurances that the old double act of “postcode” and “lottery” are not reunited by PACS.

In all honesty, it is hard to tell whether the committee’s recommendations will be matched by the new systems, as we are short of information. I would be grateful if the cabinet secretary could offer some clues about timescales.

We are told that the rare conditions medicines fund will continue until 2016, but it is still unclear whether the £20 million is an annual budget or total funding until 2016. I would be obliged if the cabinet secretary could elaborate.

The Scottish Government agrees that there should be scope for a temporary pause in the appraisal process to permit further dialogue with the manufacturer. That was a recommendation from Routledge and the committee welcomed it. The Scottish Government says that a pause would allow a confidential discussion with the manufacturer about cost through a new or improved patient access scheme. However, no picture has yet emerged of how the scheme will look.

I have another one for the cabinet secretary, who I appreciate is listening patiently—I hope that he is not getting writer’s cramp as a result of taking notes.

There is to be £1 million of funding for the SMC’s engagement with the public and the pharmaceutical industry, but it is not clear whether more money would be required following the development of VBA.

Scepticism is a good thing, but let us give credit where credit is due. I sense that the direction in which we are heading is the right one. The frustration, for me, is that we have yet to arrive.

I want to say something more about the cost side. It is clear that the Scottish Government considers there to be a devolved element in that regard, and I want to make a couple of observations. In March, the chief executive officer of GlaxoSmithKline described the often-mentioned $1 billion research and development price tag as

“one of the great myths of the industry”.

I think that that is significant and interesting. Doctors Without Borders said:

“It is true that innovative new drugs can change the way we treat people and we need more of them. But innovation is of little use if people cannot access new treatments because they are so expensive.”

The pricing of medicines is, in many ways, a global issue. The issue is big, complex and deeply emotive—we had better believe it.

I commend the inclusive and listening approach of the person who will speak next in the debate. We have come a long way, policy-wise, in the past 18 months. Things have moved relatively quickly since the committee took evidence, and we welcome announcements. However, things can never move quickly enough for people who are diagnosed with rare conditions and terminal diseases. Three months is a lifetime to such people.

I hope that the cabinet secretary will report back to us on progress by December. Perhaps he can give us that undertaking. The committee believes that we can improve the processes. We can remove some of the bumps in what clinicians call the patient journey, and we can devise a system that is fair, objective, transparent, robust and within our means.

We need to ensure that there is access for all people who have orphan and ultra-orphan and life-threatening diseases. That is what people petitioned the Parliament for. We owe them nothing less and they deserve nothing less.

Photo of Alex Neil Alex Neil Scottish National Party

I welcome this important debate on the highly complex and difficult issue of access to new medicines.

As Duncan McNeil pointed out, it is worth remembering that the issue was originally highlighted in the experience of patients through the Public Petitions Committee. Their voice was put directly to the Parliament in the way that it should be. After consideration by the Public Petitions Committee, the matter was passed to the Health and Sport Committee for further detailed consideration. As Duncan McNeil said, its inquiry took evidence and took time to fully explore the position. I thank the Public Petitions Committee and the Health and Sport Committee—in particular, I thank Duncan McNeil for his chairing of that committee’s inquiry—for the rigorous and serious manner in which they approached this wide-ranging subject.

Through the Health and Sport Committee’s questioning, a number of important aspects were crystallised, not least the desire for the development of a Scottish model of value and the shortcomings of the IPTR process. I commend the committee’s consensual and sensitive approach and feel sure that we can continue that approach right across the chamber today and beyond.

The committee heard a wide range of views from the pharmaceutical industry, clinicians, patient charities, patients and their families, some of whom are with us in the gallery. Members will see from our written response to the committee that we have accepted almost all of its recommendations and those of Professor Routledge and Professor Swainson. I thank Professor Routledge and Professor Swainson for their efforts and suggestions, which will, I believe, make the system much more transparent. The committee broadly welcomed their recommendations but recognised that more would have to be done to increase access, as spelled out by Duncan McNeil. I agreed with that assessment.

As time is short, I will focus my response on giving a brief overview of some of the steps that we propose to take.

First, I want to describe the introduction of flexible decision making for medicines that are licensed to treat patients at the end of life and medicines that are licensed to treat very rare conditions. I have directed the Scottish Medicines Consortium, which is the body that approves medicines for use in the national health service in Scotland, to apply a more flexible approach to evaluating medicines for end-of-life care and treating very rare conditions to increase access to them. The SMC will carry out a review by Christmas at the latest and establish new approaches to facilitate improved access to those medicines for patients in Scotland. That will be the first step in a wider process to determine Scotland’s requirement to create a value-based approach to new medicines assessment.

The second action is the introduction of a new peer-approved clinical system to replace current individual or group patient treatment requests. New detailed guidance for NHS boards on the new arrangements is being developed and will be issued shortly. The guidance will clarify that the peer-approved clinical system is a single national system—I emphasise that—that will be delivered locally.

Photo of Christine Grahame Christine Grahame Scottish National Party

For the sake of people who are involved or are becoming involved in the IPTR process, it would be helpful if it could be given more clarity shortly. Are we talking about months? When will the new system be up and running? I appreciate that it is early days, but it is important to narrow that down.

Photo of Alex Neil Alex Neil Scottish National Party

My intention is to do this very early—certainly within the next couple of months or so—because it is clear, as Duncan McNeil pointed out, that people could be caught in the transition, which would be very unfair. We are cognisant of that particular situation. However, I should point out that 60-plus per cent of IPTRs are accepted, so we are talking about the 40 per cent of applications that are not accepted.

In brief, the new system will be predicated on clinical opinion. Where all other treatment options have been exhausted, a lead clinician can make the case to clinical peers for a treatment that has either not been recommended for general use by the SMC or not yet been submitted for approval.

The new system will for the first time introduce standardised paperwork as a requirement. Healthcare Improvement Scotland will continue robust auditing of decision making about medicines considered under the new system, and will facilitate the regular sharing of expertise between NHS boards across Scotland to eliminate unwarranted variation.

The new system will be underpinned by a centralised patient support team to advise, support and advocate for patients and families to make it easier to understand and use. I am pleased to confirm the extension of the £20 million per annum rare conditions medicines fund. It was due to conclude in April 2014 but will now run until at least April 2016.

Photo of Kenneth Macintosh Kenneth Macintosh Labour

Is the minister adding extra funds—£20 million per year for each year to 2016—or is it simply the case that the current £20 million will be used for the fund until 2016?

Photo of Alex Neil Alex Neil Scottish National Party

I will make available up to £20 million for this fund every year, but to date only £6.5 million of the £20 million that is currently available has been spent. In each fiscal year there will be up to £20 million available.

The Scottish Government believes that the introduction of value-based assessments of new medicines, and the new interim arrangements that I have described for medicines that are to be used to treat very rare diseases, will significantly reduce the use of individual patient treatment requests currently and peer review in the future. In recognition, however, of the fact that medicines within those categories are often not routinely available, extending the rare conditions medicines fund will provide access to medicines to treat very rare diseases for people who make a successful request under the IPTR process or its successor, the peer-review approach.

In talking about improvements to SMC submission arrangements, let me first state that the SMC is a globally respected health technology assessment organisation, which considers and weighs the evidence that it receives very carefully. I pay tribute to the tremendous work of that body. I want to build on that reputation and ensure that the SMC is as transparent as possible about its decision making. I have asked the SMC to transition and to hold its first public meeting by May 2014.

The role of the SMC’s patient and public involvement group will be extended and supported to engage proactively with patient representative organisations. Patient representative groups will also be able to attend SMC meetings and to provide evidence, much as they have the ability to do within NICE’s process.

This increase in the pace and depth of engagement with patients and the public will significantly contribute to the transition towards a wider assessment of value in relation to new medicines. The SMC will also develop processes and protocols to support the attendance at SMC meetings of a representative from the manufacturers of medicines that are being appraised in order that they may answer any questions that the committee has on the evidence submitted.

In the longer term, the SMC will work with industry partners to establish scoping meetings with manufacturers, prior to a submission for a newly licensed medicine. That will ensure that pharmaceutical companies fully understand the SMC submission process and have the best possible chance to submit high-quality evidence to the SMC first time round. Protocols will be developed to implement a temporary pause in the SMC’s appraisal process, which will be instigated where a medicine’s cost effectiveness poses a stumbling block to its acceptance. The pause will facilitate a confidential discussion with the manufacturer, through an external negotiator, about improving the medicine’s cost effectiveness, through a new or improved patient access scheme. Consideration will also be given to the benefits and challenges of establishing arrangements to carry out external evaluation of medicines where no submission has been instigated by the manufacturer.

The Scottish Government currently supports the retention of NHS board area drug and therapeutics committees as a tool to ensure safe and effective prescribing practices and ownership of the formulary by local clinicians. That will be contingent on NHS board ADTCs being able to demonstrate over the next three years that they are working well and are ensuring optimal clinical outcomes for their patient population. That is an indication of the Government’s commitment not only to medicines that improve people’s quality of life on a day-to-day basis but to medicines that sustain life or improve the quality of end-of-life care.

The Government is committed to pursuing continuously improving, high-quality health and care services for the people of Scotland, with a focus on equity and clinical need. I believe that the measures that I have outlined will help us to secure that vision.

Photo of Neil Findlay Neil Findlay Labour

I would very much like to thank the Health and Sport Committee for its report, and I commend its convener, Duncan McNeil, for his excellent speech. The report is a very good one that makes an informed contribution to what is a vital debate that is being watched by many people in the chamber, across Scotland and beyond.

For many of those who have tried to navigate the current system of accessing new treatments, it is at best confusing and at worst downright frustrating and unfair. The former health secretary defended the IPTR system by saying:

“We already have a very fair, rigorous and quick system for appraising NHS drugs in Scotland. The Scottish Medicines Consortium considers whether newly-licensed drugs should be used nationwide and recommends them for use where they are proved to be safe, clinically effective and cost-effective.

Even where a medicine isn’t recommended by the SMC for general use, patients in Scotland can still get it on the NHS if their clinician believes it is appropriate and obtains permission from their local health board.”

For many patients, that description simply did not reflect the reality on the ground. In case after case, patients with serious conditions such as cancer and multiple sclerosis were denied access to new-generation drugs. As the committee’s report states, they were often denied access

“despite them submitting clinical and expert evidence as part of the request.”

High-profile cases emerged as my colleagues Jackie Baillie, Graeme Pearson and Johann Lamont, along with other MSPs, represented their constituents in Parliament. The patients who were involved in those cases did not seek the limelight or public exposure—all that they wanted was the chance of an extended life for themselves and their loved ones.

In support of those brave individuals, we had the campaigning work of the people who took petitions through the Parliament’s public petitions system. They are the people whom we must commend for getting us to where we are today. It is their petitions and campaigning that have influenced the committee’s inquiry and forced the Government’s hand. They have done the Parliament and the people of Scotland a great service.

Although the Health and Sport Committee welcomed the previous Swainson and Routledge reviews, it said that they would

“do very little to improve access to new medicines in any meaningful way and that more fundamental changes were needed.”

Those changes must, of course, be fair and transparent but, ultimately, they must provide greater access to medicines that have not gone through the system.

There is no simple solution—none of us is suggesting that there is. It is a complex and highly sensitive area in which answers do not come easily. The committee captured those difficulties well when it said:

“decisions need to be made about the value of treatments in relation to their effectiveness, cost and wider societal benefits, but within the context of a public sector under increasing budgetary pressures. When these issues combine with personal circumstances and experiences of individual patients and their loved ones and the impact that decisions can have on their length and quality of life, it is probably not surprising that the answers to the questions posed by this issue are not easily found.”

A good example of the failings of the existing system was provided in the evidence of Dr Stephen Harrow from the Beatson west of Scotland cancer centre. He recounted how he had made an individual patient treatment request that was refused not once but three times, despite its being supported by clinical evidence. That was a situation that clinicians, patients and stakeholders found difficult to understand.

There are always ways of doing things better, and the system has to support patients in getting access to the medicines that they need when that request for access is supported by clinical judgment and expertise.

The Health and Sport Committee’s report has identified many areas where things could and should be done better, including improving transparency, patient involvement, data collection, sharing expertise and standardising paperwork. However, although such steps are welcome, the report also makes it clear that

“urgent consideration should be given to encouraging greater flexibility in the IPTR process to approve drugs where there is clear, clinical evidence that a particular patient would derive material benefit from such a drug even if existing IPTR criteria had not been met fully.”

I am pleased that the Scottish Government has accepted the need to improve access and that, in its words,

“the procedure for accessing drugs in exceptional prescribing circumstances, when all other treatments have been exhausted, should be clearly linked to clinical opinion.”

Although the replacement of the IPTR system with a new, peer-approved clinical system is a move in the right direction, the proof of the pudding will be in how that impacts on patients and whether more people are able to access the life-saving or life-extending drugs that they so desperately need.

I ask that, in summing up, the cabinet secretary responds to the following questions. First, how will the new system be implemented and rolled out? How will it be monitored to ensure that guidelines are not ignored? How does the fund fit with the statement of intent on innovation? How will he avoid the new locally run PAC system becoming another postcode lottery? I know that the cabinet secretary has said that he wants a national system, but I point out that it will be run locally. Finally, when will he publish the timescale for implementing the new system?

Sadly, these changes have come too late for many, but I hope that a new supportive system will help save and extend the lives of many other very needy people.

Photo of Jackson Carlaw Jackson Carlaw Conservative

In welcoming today’s debate, I, too, thank Professors Swainson and Routledge, the Health and Sport Committee and the cabinet secretary for the focus and energy that has been brought to this issue in the past year.

In a Parliament that has a number of progressive legislative health and care achievements to its collective credit, I make no apology for saying that our inability hitherto to agree an effective route for the access to new medicines for those in need of life support—and particularly, but not exclusively, those suffering from cancer—has, in my opinion, been a stain on the face of devolution.

Scottish Conservatives also make no apology for our repeated determination to have this chamber return to the issue. As we stated when, earlier this year, we lodged a motion on this subject for debate, we have done so in the understanding that the political will to establish a cancer drugs fund does not exist in this place. Mindful of that, we have repeatedly offered to work to find an alternative solution by whatever name that nonetheless allows Scotland to recover its pre-eminent reputation in cancer care.

In our view and indeed in the view of many clinicians, pharmaceutical companies and others, the existence of a cancer drugs fund in England and the lack of one in Scotland was, notwithstanding the fact that some 34,000 patients had benefited from access to pioneering drugs south of the border, an impediment to current clinical experience and practice and, potentially, to future cancer research and development in Scotland.

Photo of Jackson Carlaw Jackson Carlaw Conservative

Not just now.

That some 3,500 Scots, some inevitably no longer with us, were unable to access drugs in their home country is a regret that I feel very personally. I also know of several members on all sides of the chamber who, notwithstanding the battery hen-like utterances that we customarily make, understood that the status quo was not an option and, indeed, that the cancer drugs fund in England made the status quo progressively impossible, and I pay tribute to their willingness to reach across the political divide, quietly but determinedly, to make progress. They know who they are and cancer sufferers should be grateful to them.

Without beating about the bush, I thank Alex Neil for having the courage, will and determination to pursue a potential solution of which this Parliament can be proud. In their essential ambition with regard to a significantly higher QALY, a properly resourced SMC with a new ability to negotiate on price, and a dynamic clinical trials register, these proposals set out a package that Scottish Conservatives welcome and which we will work with the cabinet secretary in any way we can to advance and secure.

Party politics aside, this is a good day for Mr Neil and his officials but, much more importantly, it is possibly a breakthrough day for cancer treatment in Scotland. A lifetime of witnessing the hardest progress in the battle against cancer being fought and won is being rewarded today by a rush of technological breakthroughs, and—let us be clear—Scots and Scotland must aspire to offer the very best that is available.

We have an unenviable record of cancer incidence. Although much has been done—and should be applauded—on the early detection of a range of cancers from breast to bowel and all body points north and south, and on the investment in facilities, that national effort must be backed by the latest in life-extending and life-enhancing drug technology. There must be no more of politicians queasily debating whether extra months of life for a young mother or father with skin cancer are worth the price to the public purse. That is what I believe the cabinet secretary aspires to achieve, and I applaud him for it. We have worked constructively with him and will continue to do so.

Yet, to borrow Mr Findlay’s cliché, the proof of the proverbial pudding will be in the eating, and the cabinet secretary must ensure that the short time between now and the end of the year is not wasted. In a constructive spirit, I ask him the following. The Scottish Medicines Consortium has been reluctant to change, and it would be a tragedy if the proposals that subsequently emerged did not improve access. Will the cabinet secretary offer clear direction? We know that many medicines are not available today. Does the cabinet secretary envisage that the new medical individual application process will be used for patients who need such medicines now?

Photo of Jackson Carlaw Jackson Carlaw Conservative

I need to make progress.

Will the current catch-22 situation, whereby a medicine must be within licence but outside an SMC restriction, be resolved? How does the cabinet secretary anticipate that the new systems will catch up with all the medicines that are currently denied to patients in Scotland? A resubmission process could take a considerable time, given that the SMC may have a workload bulge as a result. How will ministers ensure that patients who are suffering as we speak do not miss out on new drugs?

The fact that the new PAC system is to be locally run is welcome but, as others have said, it must not be allowed to recreate a system of inequitable patient access throughout Scotland, as happened before. Can the cabinet secretary assure me that the Scottish Government’s new guidance will, therefore, be published and implemented swiftly, with health boards held to account through the strict auditing arrangements that have been promised?

I have deliberately asked technical and searching questions, not to confuse the issue but because I believe that this week’s announcement by the cabinet secretary is a genuine and sincere initiative. For success to be achieved, those questions and others need to be considered and answered.

A year ago, I asked the cabinet secretary to set aside any entrenched position that he may have inherited and to use his capacity to reach for, identify and implement practical solutions—to use his talent in that regard and to bring all his energy to bear on cancer. There may yet be much to secure and improve, but in his response to the Health and Sport Committee’s report Alex Neil has not been found wanting. Today, let us agree that, although we have not embraced a cancer drugs fund, the Scottish Parliament has embraced the funding of cancer drugs.

Photo of Aileen McLeod Aileen McLeod Scottish National Party

As a member of the Health and Sport Committee, I am delighted to speak in this important debate.

I acknowledge the cross-party consensus that underpins the committee’s report. There is common ground across all political parties in the belief that access to new medicines is one of the most pressing and sensitive issues confronting the NHS. I therefore hope that the Government will be able to implement the report’s recommendations in the same spirit of consensus to create a better and more transparent set of criteria for accessing new medicines that will lead to improved outcomes for Scottish patients, particularly those suffering from rare and very rare medical conditions.

I record my thanks to the many expert witnesses, clinicians, industry representatives, patient groups and charities who gave evidence. In particular, I thank Breakthrough Breast Cancer, Myeloma UK, Marie Curie Cancer Care and Beating Bowel Cancer. I also thank the private individuals who offered the committee both expert scientific evidence and evidence drawn from their experiences. I am pleased that so many of them are in the public gallery to listen to the debate.

I welcome the cabinet secretary’s remarks in setting out the Government’s response to the committee’s report, which tackle head on many of the key issues and recommendations in the report. I hope that my committee colleagues will also welcome his remarks as a significant response to our work.

In the time available, it is not possible for me to cover every aspect of the committee’s report, so I will focus my remarks on a few key areas.

The commitment to increased transparency in Scotland’s medicines assessment system, which the cabinet secretary mentioned, is particularly welcome, as is the additional £1 million investment to support the SMC to hold its meetings in public from May next year. Greater transparency in the process should help to increase patient confidence that the systems that are used to decide access to medicines are both scientifically sound and fair.

In our report, the committee made various recommendations that seek a greater involvement by patients and patient representatives in the SMC’s work, so I very much welcome the improvements that are to be made in supporting patients to engage in the process.

Welcome as these changes are, they will only take us so far. A bigger challenge recognised by the committee was to reform the SMC’s decision-making process to ensure a better assessment of medicines’ cost-effectiveness and wider societal impact, in particular for end-of-life care and for treating very rare conditions. The committee called on the Scottish Government and the SMC to review that issue as a matter of priority, and I am delighted therefore that the cabinet secretary has taken on board that recommendation and that the SMC is expected to conclude its review on establishing a more flexible approach to the evaluation of new medicines and ensure that there is a wider assessment of value before Christmas.

Similarly, I am sure that all members of the committee will welcome the proposal to replace the IPTR system with a new peer-approved clinical system that will oversee individual or group requests for medicines not recommended by the SMC, led by local consultants and local consultants and linked to clinical opinion. I am particularly pleased that the Government has listened to the committee’s concerns on the IPTR exceptionality criteria and our calls for the approval of particular drugs where there is clear clinical evidence that a patient would derive material benefit from them.

It is also welcome that the NHS will be able to refer for consideration by the SMC new medicines that have not been submitted by a drugs company, when they are considered clinically important to NHS Scotland.

The committee also considered the issue of clinical research trials and encouraging greater participation by patients in Scotland where appropriate. In that respect, I very much welcome the cabinet secretary’s response: for the Government’s chief scientist office to look at the creation of a Scottish clinical trial register, which will, I hope, help to raise awareness among patients and clinicians of on-going trials.

The Health and Sport Committee’s report is a thorough and far-sighted document. It addresses one of the most complex and sensitive matters that anyone involved in public policy is likely to encounter. However, the very nature of the issue makes it equally important that the Government exercises care and performs the very highest level of due diligence at each stage in the process of constructing the new system—nowhere more so than in the development of a revised value-based assessment, in which there is a clear opportunity to develop a Scottish model of value. That will take time, but, as the cabinet secretary indicated, that does not mean that the Government cannot act in the meantime, as it has done through the changes outlined in its response. I am delighted that the rare conditions medicine fund will be extended to 2016, which provides an interim measure that should alleviate dependence on IPTRs.

I firmly believe that the committee’s recommendations, which commanded cross-party consensus, together with the Scottish Government’s response plot a course towards a better and more transparent system for access to new medicines that is based on equity and clinical need.

Photo of Malcolm Chisholm Malcolm Chisholm Labour

I join the petitioners and external organisations in welcoming the announcement that was made yesterday, although, as Beat Bowel Cancer said, the devil is in the detail. Clearly most of that is still to come, but we should certainly pay tribute to those organisations and petitioners for setting the process going. We should also pay tribute to the Parliament’s committee system, because it has been seen at its best in the way that it has engaged with this subject.

Clearly, the system for access to new medicines had to change, but we should join the cabinet secretary in paying tribute to the SMC, which over the past 12 years has been a globally respected organisation. I was privileged in the first years of its existence to be Minister for Health and Community Care, so I followed it closely at that time. More recently I have taken an interest, particularly as co-convener of the cross-party group on cancer and the cross-party group on rare diseases. Those two groups of illnesses have been very much to the fore in recent discussions.

Before I concentrate on some of the announcements made by the Government yesterday, I will make some general points. It seems that the elephant in the room is value-based pricing. It is not clear to me how that will interrelate with the SMC—I am not sure whether it is clear to the minister or anybody else—but it would be good to hear about that in the winding-up speech if possible. We are certainly moving towards an era of more personalised medicine, which is good news not only because individual patients will get better targeted drugs, but because it will make many medicines more cost effective.

Everybody knows what the problems were, and I hope that members will forgive me for concentrating only on cancer and rare diseases. Many of the witnesses noted that it was difficult to generate robust and cost-effective data for rare diseases, and the system must be more flexible in that respect. On cancer, the committee was told that the SMC accepted two thirds of non-cancer drugs but only one third of cancer drugs. It is clear that there were particular problems in that area, particularly in relation to end-of-life criteria, and we were told that other health technology organisations have more flexibility in that respect. Those were the key issues that needed to be addressed.

The other big issue that was very much the focus of attention was the individual patient treatment request system. Again, we heard in committee—and I have begun to hear at the cross-party group—about the particular problems with regard to rare diseases. The referral criteria are extremely difficult to satisfy and I am told that it has been difficult for those organisations that are involved with rare diseases to access the new fund over the past year, because much of it was available through the IPTR process, which—as is well known—was already not working for cancer patients.

I welcome the Government’s acceptance that the existing cost-effective thresholds are not appropriate for end-of-life medicines or medicines to treat rare diseases. That was the Government’s central recommendation yesterday, and it is a positive response to the committee’s recommendation for modification of the quality-adjusted life years in those two circumstances.

The other key announcement yesterday concerned the peer-approved clinical system. The Government said that it wants to link access to those drugs that are to be prescribed in exceptional circumstances with clinical opinion. That met the demands of those involved with rare diseases, cancer and other diseases, and was consistent with the committee’s recommendation that we could not have a system that was based only on exceptionality.

That is all very positive, although some concerns have been raised about the idea of a national system that is delivered locally, because in some sense the whole system, since its inception, was supposed to have been a national system that is delivered locally. I remember that, in its first year, I had to intervene to say, “Boards, you have to follow these SMC recommendations”, and there have been continuing problems and doubts about whether the boards have always been doing that.

There are two further recommendations that interest me. One is the recommendation that the SMC should engage proactively with patient representative organisations. I wrote to the minister—although I do not know whether he has seen my letter yet—a week or two ago on behalf of a constituent from one of the prostate cancer organisations. The SMC was revisiting a prostate cancer drug, and he said that his organisation and the patient organisations did not know about it but would like to have been involved. The Government’s recommendation in that respect should address that particular concern. In addition, Myeloma UK said that it would like the SMC to explain how it takes into account patient groups’ submissions, so it is clear that the whole area is important.

I was interested to read at the very end of the Government’s response to the committee’s report that there is no

“robust evidence to suggest a decline in Phase III commercial clinical trials.”

That seems to contradict Professor Gourley’s evidence to the committee, in which he stated:

“there is absolutely no doubt that a number of examples of clinical studies cannot be done in Scotland”—[Official Report, Health and Sport Committee, 21 May 2013; c 3826.]

because of the standard drugs factor. Professor David Cameron said the same, both in his written submission and to the cross-party group on cancer, as well as in other places. There has been a genuine threat to Scotland’s great reputation for clinical research because the standard drugs that were the comparators in clinical trials have often not been available to cancer clinicians in Scotland. I hope that yesterday’s announcement will deal with that problem too. That will benefit not only patients, who are fundamentally what the system is about, but clinical research and the life sciences industry, which is an important part of the Scottish economy.

Photo of Gil Paterson Gil Paterson Scottish National Party

I will start by thanking the convener of the Health and Sport Committee, Duncan McNeil, who encapsulated the committee’s thinking in his opening speech. He certainly represented the views of the committee extremely well, so I thank him for that. I am pleased to speak in the debate as a member of the committee—that is an added bonus.

The committee’s inquiry into access to new medicines presented me with the difficulties involved in providing individuals and families with comfort and in satisfying what, on the surface, might seem to be an expectation that every drug and treatment on offer should be made available on demand. During the inquiry, we heard stories from individuals who require a particular drug for themselves, from family members who wanted to extend the life of a loved one and from people who wanted their quality of life to be just that bit better.

Much though I would like or prefer to agree that there should be no barriers to receiving help, no matter the cost or the perceived benefit to the individual, we must look at the broader picture. Unfortunately, as much as I might have empathy for those families and individuals, I am curtailed in my support by the realisation that tough choices need to be made. Even though the Scottish Government has protected the health budget from Westminster cuts, the prospect of being in a position to provide unlimited funds for every new drug, no matter the cost, is sadly not an option simply because of finite resources.

When it comes to choices, I very much support the establishment of the £20 million rare conditions medicines fund, which was welcomed by the committee. The fund helps to improve access to orphan and ultra-orphan drugs—medicines for illnesses that affect fewer than 1 in 2,000 people—including those that deal with different categories of cancer. We recommended that approach rather than a specific fund for cancer. Indeed, in our deliberations we found little support for the establishment of a separate and exclusive cancer drugs fund in Scotland.

Although cancer can be, and is, a devastating illness, singling out cancer from all other devastating conditions makes me uneasy. I suppose that my experience gathering evidence for my member’s bill on palliative care in the previous parliamentary session has coloured my views. I found that cancer sufferers have a 90 per cent chance of receiving high-quality palliative care, whereas those with other life-threatening conditions have a 90 per cent chance of not receiving the same quality of care. That is a situation that I thought was just wrong, so I am very much in favour of a fund or scheme that is of equal status and effect across illnesses, rather than a preferential or exclusive fund for one category of illness. Our service should never be partial.

Choices also need to be made on where resources should be deployed. If we were to fund all available treatments and drugs, no matter their effectiveness or cost, the ability to provide early treatment that may lead to a cure could be diminished. My choice is to provide both early treatment and treatment later on in an illness. It should be recognised that that sentiment is at the heart of the Scottish Medicines Consortium, which has been world renowned for a considerable time and commands global support.

The Scottish Government is striving to ensure that Scotland’s drug approval system both becomes even more transparent and increases access to medicines for end-of-life care and treatment of very rare conditions. The Scottish Government has therefore directed the SMC to apply different and more flexible approaches in the evaluation of medicines in that category. The SMC will have to conclude by the end of this year a review on how to establish that more flexible approach. Also, to replace individual patient treatment requests, a new peer-approval system will be introduced to allow clinicians to prescribe medicines that are not accepted for routine use by the SMC. That process will be led by local consultants. Further, NHS Scotland will be able to refer to the SMC for consideration new medicines that have not yet been submitted by a pharmaceutical company, where those are considered clinically important to the NHS in Scotland, a system that was recommended by the Routledge review.

I believe that the additional measures on the SMC will do two things. First, they will enhance a system that is already highly regarded and build on its undoubted success. Secondly, and perhaps more important, they will give comfort to the families and individuals whom I mentioned at the start of my speech. Of course, the changes will increase transparency in the approval process, particularly for patients, as the meetings of the SMC will be held in public and patients will be invited to attend peer review decision-making meetings. I welcome the additional investment of £1 million to ensure that the work of the SMC becomes more transparent.

I am pleased to have been part of the committee’s inquiry and to have had the opportunity to make recommendations to the Scottish Government on how to move forward. It is pleasing that, although our system is envied the world over, the Parliament and our Government are big enough not to rest on their laurels and brave enough to inquire into our delivery system for new medicines with a view to making it even better. I believe that we have achieved our goals, but at the same time we must always strive for improvement.

Photo of Elaine Smith Elaine Smith Labour

I ask members to ensure that their mobile devices are switched off, unless they are being used to deliver a speech, in which case they should be on silent.

Photo of Kenneth Macintosh Kenneth Macintosh Labour

I will begin with a tale of two constituents who live less than two miles apart in Newton Mearns and who both suffer from the very rare blood disease paroxysmal nocturnal haemoglobinuria, or PNH, as some members might know it. One of those constituents, Mr Bill Devine, was denied access to the one drug that could have helped. He contacted me in October 2010, weak and unable to leave his house other than for blood transfusions. I wrote to the local health board, but the only route to access the drug was through the individual patient treatment request, or IPTR, system. His case was “not proven”. Mr Devine died in April 2011.

The other constituent will be known to some colleagues from her evidence to parliamentary committees. Mrs Lesley Loeliger was granted access to the same drug that was denied to Mr Devine and it has transformed her life. She is raising her young family and is an active member of the local community. The drug has even allowed her the energy to campaign on behalf of others—she has petitioned the Parliament to fight for access and fairness for all patients.

That same story is being played out across Scotland and across different health conditions and drug treatments. Too many families are being denied treatments that their clinicians recommend for them. Their feelings of dismay and frustration are compounded by the knowledge that others are accessing those very drugs.

No one in this Parliament has a monopoly on compassion, and I certainly do not wish to portray either the current health secretary or his predecessor as cold or heartless but, for several years, we have been told repeatedly that Scotland has a robust system in place and that we would not go down the route of a drugs fund for one particular condition, as has been pursued in England. The cabinet secretary, in his response to the work of the Parliament’s Health and Sport Committee, now acknowledges that our system does not work and, of course, he has introduced a Scottish rare conditions fund. I simply seek reassurance that the system that the Scottish Government intends to put in place to approve medicines will be effective and that all patients in Scotland will have equitable access to the care that they need.

At the heart of the issue is how we measure effectiveness. In a cash-limited world, how do we balance clinical effectiveness with cost effectiveness? How do we measure the quality of life?

I am encouraged that the SMC is to be asked to broaden its approach to take into account issues such as the burden of illness and the wider societal impact. For the often very young patients who struggle with skin cancer, such an approach holds out the hope that the first new treatments in more than three decades will be available to them.

I am pleased that the SMC is looking at the benefits of a different evaluation of end-of-life treatments. I am sure that none of us underestimates the difficult task that faces the SMC in trying to improve on the quality-adjusted life year system and set new approval thresholds. A number of recommendations have been made to improve the transparency of the process. That will help, even if it does not make a substantive difference to the number of approvals.

Let us make no mistake; as the Health and Sport Committee said in its report,

“The challenge is not only to improve transparency and consistency within the IPTR process but to ensure that the SMC process in the first instance better assesses the cost effectiveness of medicines.”

For drugs that the SMC has not approved but which could benefit some patients in some cases—as with the two examples that I highlighted—the IPTR system should have offered at least some hope. However, as we all now know, not only was the test of exceptionality—that every case had to be virtually unique—too severe for most patients to pass but it was interpreted differently in different parts of the country.

I hope that the new clinically led system will mark an improvement by moving away from decisions that are taken solely on the ground of cost. I understand that the exceptionality test is to be replaced by the criterion that all other treatments have been exhausted, which gives me hope.

The drug treatment for PNH is expensive but, for the few who need it, it works. It is life transforming and does not demand an open-ended cheque from the health service, because PNH affects only up to two people in every million of the population.

Where are we at the moment? Members might be interested to know that, following the Health and Sport Committee meeting at which the cabinet secretary said that patients would be turned down only if it was believed that a drug would not work and no one would be refused solely on the ground of cost, three patients who were trying to access treatment for PNH had their applications approved—two applications were approved on appeal and the other decision was reversed after the treatment had initially been refused in the week before the cabinet secretary gave evidence.

We are here primarily because of patient protest, but also partly because of decisions that have been taken in Europe. As far back as June 2009, the European Council published a recommendation on rare diseases that recommended a national plan for rare diseases. We know from experience that, if we manage rare conditions only locally, there is a lack of specialist knowledge, long delays are created with on-going bureaucratic and complex referrals, and health boards sometimes refuse to share risks and costs, which leads to a postcode lottery.

Scotland is one of the last countries to comply with the European recommendation on rare diseases, which insisted on compliance by the end of 2013 at the latest. Given that directive, I am still a little concerned by the guidance that the new peer-approval system is to be locally run.

I ask the cabinet secretary to clarify the Scottish Government’s approach to the rare diseases drugs fund. For several years, the Government has opposed the cancer drugs fund, which I believe that it still opposes. However, we now have the rare diseases drugs fund. I have constituents who have children with cystic fibrosis and I am incredibly pleased and relieved that they have access to the drug Kalydeco to treat that terrible condition. However, none of the other health cases that I have dealt with has qualified for the fund. Eculizumab, which is used to treat PNH, does not qualify, and neither does ipilimumab, which is used to treat skin cancer.

As the cabinet secretary clarified, the rare conditions fund has been extended, but only a third of the money has been spent so far. Given that, will the fund’s criteria be extended to cover more conditions until the new SMC criteria and the new peer-approval clinical system are in place?

In the end, the test will be whether more patients receive treatment that is currently denied them and whether they do so equitably. Mrs Loeliger told me last night that a new PNH patient was seen at Monklands hospital last week and that she does not know whether they will require eculizumab. She said:

“I want a proper system in place so I do not have to fight for each patient individually. Please do not think I minded fighting for the patients that got their drug, it just should not come down to someone shouting loudly.”

Photo of Mark McDonald Mark McDonald Scottish National Party

I pay tribute to the petitioners who came to the Public Petitions Committee to raise their concerns in Parliament. I have played quite a unique role in the process, as I was a member of that committee when the petitions were lodged. I then became a member of the Health and Sport Committee when it was undertaking its inquiry into the issue, so I have been involved from the beginning in the parliamentary process, although I missed the writing of the report due to being engaged in a by-election up in Aberdeen. The point is that the process has demonstrated a concern being brought to Parliament and being given the serious treatment that it requires.

I want to consider the issues around rare diseases and orphan conditions, following on from Ken Macintosh’s points. I admit that I was familiar with the issues prior to the petitions being lodged, but I would not previously have professed to know as much as I have since learned about rare diseases and orphan conditions.

In the Routledge report, the recommendation was made that the SMC

“should develop a policy specifically relating to ultra-orphan medicines to guide the process of consideration of all available evidence”.

That is one of the difficulties that has been faced in relation to ultra-orphan medicines. The fact is that there is a very small patient cohort not necessarily just in Scotland, or just the United Kingdom, but sometimes pan-Europe; we are talking about a very small number of people. It can therefore be difficult to ensure that there are robust trial data for medicines. Also—and most crucially, in terms of some of the points that have been raised—there is an issue around affordability, within QALY, of the medicines that are available for ultra-orphan conditions. That is why I welcome Professor Routledge’s further recommendation about having a temporary pause in the appraisal process. The Scottish Government has accepted that recommendation and it is one that the Health and Sport Committee welcomed in its report, saying that

“such a pause could also create an opportunity for discussion on, for example, whether there was scope to develop a reimbursement rate which could into account various factors such as supplying post licensing data or assessed benefit of medicines post approval.”

To me, the moves around the threshold of QALY and around the pause in the process create what could be described as a double-lock opportunity, in that those moves allow for much wider consideration of medicines that previously would simply not have been considered. Also, they allow for further discussion of medicines that are still not captured by that process, in order to allow them to be so captured. That is encouraging.

I am sure that members will be aware of the AllTrials campaign: it has been running for a significant time and has a number of high-profile supporters. It seeks to ensure that trial data that are made available at the point at which drugs are submitted for approval are as transparent and robust as possible. I believe that, as well as there being role for the SMC, and a role for the decision-making process, there is a role for the pharmaceutical industry.

Eric Low, the chief executive of Myeloma UK, has said that he is

“very encouraged that the SMC will be given additional resources and a revised remit so that they can further build on and evolve their already excellent appraisal processes.”

However, he goes on to say that

“it is critically important that while improvements to access to medicines are made we also ensure that we are getting genuine value for money, and that the pharmaceutical industry improves the type and quality of information it provides to get their medicines funded on the NHS. I strongly believe that the SMC are best placed to help make this happen.”

There is an issue about pharmaceutical companies fulfilling their role not just on transparency but on affordability, because we cannot escape the fact that we live in a world of finite resource within the health service. It is therefore imperative that the drugs companies show willing to come to the table and negotiate on prices. That point was reflected in a lot of the evidence that the committee received during its deliberations.

The SMC investment is very welcome. We need to make the SMC process not just more transparent, but more publicly accessible. It is one thing to publish the findings of the SMC, but to the individual on the street—indeed, even to the MSP—a lot of decisions are published that are very difficult to translate. Making that information more publicly accessible and explaining it to the public would be helpful. All too often we read stories about drugs that have not been approved, but we do not always see or understand the reasons that lie behind the decisions, so making that information more accessible would be beneficial.

I am not sure whether the cabinet secretary has been made aware of it, but the MS Society has raised a number of questions. My colleague, George Adam, who has a strong link to the MS Society, asked me to bring them to the debate, to which I agreed. I will forward the briefing to the cabinet secretary so that he can reply more fully. The questions are about the timescale for introducing value-based assessment. The crux of the MS Society’s concern is the Scottish Government’s plan to involve stakeholders in developing the reforms. I suspect that the crux of the concerns of many other organisations would be how stakeholder organisations will feed into the reform process and feel fully involved. A point that has been raised repeatedly is that we should, as well as making the process being more transparent and open, ensure that organisations and individuals who have a concern or an interest can feed in their views and have them taken into account. Perhaps the cabinet secretary will take a moment in his closing remarks to reflect on that.

Photo of Christine Grahame Christine Grahame Scottish National Party

As other members have done, I welcome the reforms that have been announced and, in particular, I welcome the tone of the debate. Members have mentioned the £20 million for the rare conditions medicines fund, and how the SMC is to be more transparent and flexible in its approach by holding its meetings in public, and so on.

However, I want to focus particularly on the demise of the IPTR process, and the introduction of the peer approved clinical system. It is incumbent on the cabinet secretary to give deadlines or timescales for that, because time is of the essence for people who are listening to the debate.

I welcome the more flexible approach to the critical test for authorising drugs that are not readily available on the NHS. Ken Macintosh and other members mentioned the postcode lottery of IPTR, and the critical test moves away from that. The process causes difficulties for people, but there will be advocacy in the new system. The test of exceptional circumstances, which is almost impossible to meet, is moving to what I believe—and hope—will be a person-centred approach that will be of more benefit to applications for drugs that fall outwith what is usually available.

The debate shows Parliament at its thoughtful, compassionate and responsible best, but we have not shed our critical faculties—we need to ensure that what the Government has announced actually happens. We owe it to all the people outside here.

I have to say to Duncan McNeil that the Health and Sport Committee’s report is excellent. I am glad that I came into the debate because I have read it from cover to cover. It is sound and it tackles some hard issues. The area is a sensitive one and the committee did not shy away from it. More important is that the report was unanimously agreed.

The Public Petitions Committee here started out being more of a meet and greet, but it now has substance and gets things done. It got the Borders railway agreed, and it has brought this announcement. It really has arrived; other Parliaments should look to what it achieves for the people by bypassing the politicians and making them pay attention.

I am not looking for promotion, but the cabinet secretary is a man who gets things done. He has got into the job and is achieving things, but we still have to find out whether he will get a good report card.

Most of all, I congratulate the constituents who have turned up at all our surgeries who have been looking for IPTRs and have had to struggle against the system. I will talk about one of my constituents—Ian Morrison. He will not mind my mentioning him, because he has been mentioned in Parliament before. He tried everything on his own. Like others, he trawled the internet and websites, and went on a very steep learning curve. He went to politicians from across the political spectrum. I do not blame him—who would not do that? He had to find out for himself how to deal with the situation. His application was at first rejected, but it was successfully reviewed. Having been diagnosed with bowel cancer two years previously, he eventually accessed cetuximab. Where is he today? His wife is in the gallery and he is in the shop, minding it. Ian Morrison had to go about it the hard way, including going to websites, finding out about voluntary organisations, going to every politician he could think of and so on. He is now directing other people who are in the same situation to politicians and others who might help. Of course, it should not be like that, and we all know that.

I thought that it was a case of “Well done, Ian Morrison”, and that was it. However, on Friday, a man of 40-odd walked into my surgery with his wife, with a three-month-old baby in a carrier and a toddler at home. I thought that it was just another ordinary meeting and asked what was happening, but we had a wee bit of chat and I heard that he had been diagnosed with bowel cancer on 1 August this year and his life had been turned around. From having an ordinary life in which he built houses, he now scans the internet and websites, and is going to everybody, including me, to try to find out what he can have from the system.

What struck me was that his surgeon and consultant did not tell him anything about IPTRs. He is now fighting the system to try to get second opinions and to see what is available to him. That is what would happen to any of us, but it should not be like that. I therefore very much welcome the change in culture that the new system will bring, so that people who suddenly have such news landing on them out of the blue will find something in place that is helpful, accessible, fair, just and open. We know that it will not mean that everybody will get everything they want, but at least that will be the case for the right reasons and the position will be the same across Scotland.

What I want to hear from the cabinet secretary in his closing speech is what will happen to people like my constituent between now and when the new system is in place, because every week and every month counts for such people, as the cabinet secretary and I both know. In the transition period, we need to know whether the new system’s culture and way of looking at applications will be applied to IPTRs. Will we find it being individual-centred? Will we find the test being material benefit, not exceptional circumstances? If that starts from the minute we walk out of here, this Parliament will have achieved something.

Photo of Nanette Milne Nanette Milne Conservative

We live in a time when, increasingly, new drugs are coming on stream that can not only prolong the lives of patients with metastatic cancer and other terminal conditions, but can enhance their quality of life so that they can enjoy precious additional time with their nearest and dearest. However, those new drugs tend to be expensive and might not be approved for use by the NHS because the appraisal process does not find them to be cost-effective. Patients who might benefit from them either have to pay for them themselves or appeal to their health board to consider their case for treatment.

I first became aware of the difficulties that are encountered by such patients in 2008, when the late Mike Gray, who was a victim of advanced bowel cancer, brought his experiences of what was then the exceptional circumstances prescribing procedure to the notice of the Public Petitions Committee because he wanted to spare future patients from what he had gone through in his efforts to be prescribed cetuximab by NHS Grampian. Sadly, Mike Gray died while his petition was still being considered, but his widow, Tina McGeever, continued his work, which resulted in new Scottish Government guidelines and the introduction of the IPTR process.

The new system still relied, of course, on proving exceptionality from the general population of sufferers of a disease or condition. So, further petitions were brought to Parliament by representatives of patients with rare and orphan diseases who were still unable to access appropriate treatment. Moreover, the IPTR, as we have heard, was not resulting in easier access to new medicines for terminal cancer patients; hence the Health and Sport Committee’s inquiry. All the committee’s members, including me, felt strongly that a better way had to be found for patients to access medicines that are clinically justified, even if they are not recommended for NHS use because they have not been assessed by the Scottish Medicines Consortium as being cost effective.

Until a better value-based system can be devised, my party—in particular, my colleague Jackson Carlaw—continues to campaign for a cancer drugs fund in Scotland to replicate what was set up in England three years ago. Although some patients are now benefiting from the rare diseases fund that the cabinet secretary initiated, it is a matter of regret to us that more than 3,000 cancer patients in Scotland have already missed out on accessing the drugs that would have been available to them had such a fund been in place here.

Concerns have been raised with us by clinical academics that there is a real risk that we will lose our place at the cutting edge of research because new-generation drugs are assessed against current state-of-the-art products—the knock-on effect for Scottish patients being that they may not receive still newer drugs that are being trialled against those that are not currently recommended to the NHS in Scotland, and the effect for clinical research being a significant negative impact. I hope that the creation of a dynamic Scottish clinical trials register will allow Scotland’s involvement in such research to be accurately monitored in the future, because it is crucial not only to patients but to our economy, which is heavily dependent on our good reputation in life sciences.

However, we are now moving on, and the Government’s response to the Health and Sport Committee’s recommendations gives us hope that cancer patients in Scotland will soon have access to new drugs, when they are justified on clinical grounds, via what I hope will be a robust and sustainable procedure for both the appraisal of new drugs and their prescription to NHS patients, based on expert clinical judgment.

It became clear to the committee that two main issues had to be tackled—the appraisal process by the SMC, and the need to reform the IPTR process. The Routledge and Swainson reviews were therefore welcome. As others have said this afternoon, the SMC has gained international respect for its work over the past 12 years in evaluating relative to other available drugs the clinical and cost effectiveness of all new licensed medicines that the pharma industry presents to it, and then deciding whether they should be recommended for use in the NHS in Scotland.

When the SMC considers a drug’s cost effectiveness, its yardstick is the cost per quality-adjusted life year, or QALY, but when that is relatively high it may use modifiers to allow flexibility in its decision making. Although that generally works well, the committee considered that existing assessments are not always appropriate for medicines for use at the end of life or to treat rare diseases. The Government’s determination to take action on that by instructing the SMC to review its procedures to allow greater flexibility in evaluation of such drugs is good news, and I have no doubt that the SMC will welcome the opportunity to do that. There is a degree of urgency about it, of course, and I am pleased that we will hear the SMC’s proposals by the end of the year.

The committee also agreed with the Routledge review that the system of SMC appraisal could be more transparent and publicly available, and I also welcome the allocation of £1 million a year to support the SMC in achieving that.

The proposed replacement of the increasingly discredited IPTR system with a peer-approved clinical system has been widely welcomed by the groups who gave evidence to the committee, because it should enable patients to access such drugs when they are recommended on clinical grounds by experts, who will be called on to give their judgment. However, I would like an assurance from the cabinet secretary that he anticipates that increasing numbers of patients will at last be able to access new medicines, and that he will ensure that the funding of NHS boards in Scotland will not be a barrier to achieving that.

There is a limit to what I can comment on in six minutes, but the Scottish Government has responded positively to most of the recommendations in the Health and Sport Committee’s report. Scottish Conservatives appreciated the opportunities to contribute to the Routledge and Swainson reviews and to present our views to the cabinet secretary, and we welcome his consensual approach to such an important and sensitive issue.

We still have to learn more of the all-important detail, but I think that, at last, we are seeing progress on widening access to new medicines as a result of painstaking work by both the committee and the Scottish Government. However, I ask the cabinet secretary, at the end of the debate, to commit to reporting on progress to the Health and Sport Committee within six months of the new systems being set up.

Photo of Richard Lyle Richard Lyle Scottish National Party

As a member of the Health and Sport Committee, I too am happy to take part in this debate, which is on a matter of great importance to the people of Scotland. I note the petitions that were lodged with the Public Petitions Committee, in response to which the Health and Sport Committee made it a priority to explore the issue. As my colleagues have done, I record my thanks to the convener, Duncan McNeil; to the witnesses who came to give evidence, including the cancer groups; and to the committee’s staff, who helped to move the inquiry along.

The committee’s ambition, which I am sure is shared by all parties in the chamber, is to improve access in the NHS in Scotland to newly licensed medicines that provide the best outcomes for patients and enable them to lead as healthy a life as possible. I am pleased that the Scottish Government’s proposals for change have been made with that ambition in mind, and developed with reference to the Health and Sport Committee’s recommendations, in consultation with stakeholders.

I am also pleased that the £20 million fund to cover the cost of medicines for patients who have very rare conditions has been introduced, as a result of interim recommendations from Professor Charles Swainson and Professor Routledge. It was initially intended that the fund would run until April 2014, but it has been extended to 2016.

As the cabinet secretary said, an extra £20 million each year will help to improve access to orphan drugs, for illnesses that affect fewer than one in 2,000 people, which have not been recommended for routine use by the Scottish Medicines Consortium. I am pleased that by 5 May, 71 patients in Scotland had benefited from the new funding, of whom 24 were between the ages of six and 16.

The SMC is an effective and comprehensive organisation, given its timeliness in providing advice to NHS boards in Scotland on the status of newly-licensed medicines. It is my hope that when the committee’s recommendations are implemented, the SMC will become more transparent and will increase access to medicines for end-of-life care and very rare conditions.

I am pleased that the health secretary has directed the SMC to apply a more flexible approach in the evaluation of medicines for end-of-life care, and to complete its review on the matter by the end of the year. I hope that the SMC will consider how it can implement a more flexible approach in the new year. The aim is to ensure that the process happens as quickly as possible, while maintaining attention to detail.

Quick implementation of the recommendations will help to make the medical approval process more transparent for patients, because the SMC will meet in public. Patients will be invited to attend peer-approval decision-making meetings, and a further £1 million will be invested to help to increase the transparency of the SMC’s work.

It is right that we continually examine all aspects of our health service; there is always room for improvement. However, the SMC should be commended. It is respected globally and has the fastest and most efficient medicines review process in the UK. During a meeting of the Health and Sport Committee in December, broad support was expressed for the SMC, which of course does very difficult work. Great support was expressed in relation to the SMC’s timescale for producing guidance on new drugs. On average, assessments are completed in 7.4 months, compared with an average of 21.4 months for NICE in England. SMC achieves that without sacrificing rigour in its methods.

Many members have mentioned cases in their areas, and I, too, have been asked to highlight a case. Earlier this week I was contacted by a constituent; I have her permission to name her. Ms Geraldine Ward needs the drug Fampyra, which helps MS sufferers to walk. The drug is not currently prescribed by Lanarkshire NHS Board. I hope that when the new arrangements come into effect, they will help Ms Ward to get the medicine that she requires to improve her quality of life. I intend to pursue that brave lady’s case to a resolution with NHS Lanarkshire. As Christine Grahame said, we must help our constituents where possible.

I commend the cabinet secretary, Alex Neil, for his commitment to the NHS in Scotland and for his quick action with regard to the committee’s inquiry.

Photo of David Stewart David Stewart Labour

I congratulate Duncan McNeil and the Health and Sport Committee on their excellent report and I commend the Routledge and Swainson reviews, which made a helpful contribution to the debate.

I will focus on patient access to medicines for orphan diseases. The issue was highlighted in the three petitions that served as a springboard for the Health and Sport Committee’s report.

As members have said, as convener of the Public Petitions Committee, with my colleagues I took evidence from a variety of groups, such as Rare Disease UK. I associate myself with Nanette Milne’s comments on the great work on drugs that Tina McGeever from Moray did, and thank Christine Grahame for her very kind remarks about the Public Petitions Committee’s work.

Orphan medicines are, of course, for the treatment of life-threatening conditions that affect no more than five in 10,000 people. Ultra-orphan medication is licensed for the treatment of diseases with a prevalence of less than one in 50,000.

During evidence to my committee, Alastair Kent of Rare Disease UK said:

“People with rare diseases do not choose to have a rare disease. There is no kudos attached to having something that is difficult to diagnose, expensive to treat and about which little might be known.”—[Official Report, Public Petitions Committee, 4 October 2011; c 154.]

As others have done, Mr Kent praised the European Union’s orphan medicinal products policy, which has led to therapies being produced for diseases that have been untreatable and have led to consistent and chronic ill health.

Market access to new drugs is one dimension, of course, but direct access for individual patients is another. I welcome the Scottish Government’s £20 million a year rare conditions medicines fund, which should provide access for patients who suffer from orphan and ultra-orphan conditions to medicines that are not approved by the Scottish Medicines Consortium. Initially, the fund was to last to 2013, but I think that the cabinet secretary said that it will last until 2016. Will he say in his winding-up speech whether he is confident that value-based pricing will be in place when the fund comes to an end? Will he also say what the timescale is for the peer-approved clinical system to take over from the individual patient treatment request scheme?

Many treatments for orphan conditions make a tiny physical difference to the patient but a massive change to their quality of life. I will give one example, which Mr Kent gave my committee. He described the story of a young woman who has an inborn error of metabolism. By having enzyme replacement therapy, her lung function went up by 4 per cent. Members may think that that is not a lot, and they would be right to think that. It is enough for a short intake of breath for a healthy person—or perhaps a longer intake of breath for someone who is giving a speech. However, it meant that she could come off artificial respiration for most of the day, which was a huge improvement to her quality of life. Members can imagine the frustration of families when they see a new therapy that they cannot access, even when their own clinicians have recommended it.

We have heard that health economics play a huge role in the debate, and we have heard a lot about the SMC. Just the other morning, I heard it described on the radio as a group of hard-headed economists and statisticians who have the QALY assessment tool, which we have heard a lot about, as their second language. However, many of the treatments for rare diseases come above the £30,000 per QALY limit because they are novel therapies at the cutting edge of innovative research and development.

Photo of Bob Doris Bob Doris Scottish National Party

I have a point of information, which Mr Stewart was perhaps going to address. I am sure that he is about to highlight that there are difficulties with the thresholds and modifiers that are applied, but the £30,000 threshold is not an automatic cut-off. Medicines are approved at a far higher threshold than that—although perhaps not enough; I accept that point.

Photo of David Stewart David Stewart Labour

The member is quite right about modifiers and in the point that he made, which I was going to touch on.

NICE looked at extending the QALY system to ultra-orphan conditions, but ruled that out because it would require such a high level of assumptions that it would leave no confidence in the outcome of the process.

I was struck to find that there are more than 6,000 rare diseases, which affect one in 17 people. That is around 300,000 people in Scotland. Stephen Nutt, who gave evidence on rare diseases to the Health and Sport Committee in March 2012, said:

“we do not think that patients in Scotland currently have fair access to treatment.”—[Official Report, Health and Sport Committee, 27 March 2012; c 2025.]

The key point that he stressed was that patients sought equity, not preferential access to treatment.

Ken Macintosh spoke about the PNH Alliance, which represents those who suffer from an ultra-rare bone marrow disease, which is another orphan condition. The key drug, which Ken Macintosh talked about, is Eculizumab, which can change life expectancy to normal from a median survival rate of 10 years. Last year, Lesley Loeliger from PNH Scotland told the Health and Sport Committee:

“In my health board area, five patients have been recommended for Eculizumab. Of the five, I have been granted funding but the other four have not been so fortunate. One gentleman was refused funding several times. When I spoke to him, he told me that all he wanted was a life. Sadly, he died the next day.”—[Official Report, Health and Sport Committee, 27 March 2012; c 2026.]

I draw members’ attention to sub-orphan diseases, such as underactive thyroid, which affects 15 in every 1,000 women in the UK. My committee is considering a petition on that condition, for which there is one recommended medicine—in relation to other options, either they are not licensed or there is not enough research to establish their long-term safety.

I ask the cabinet secretary to address the concern that, as we heard from Malcolm Chisholm, Scotland is in danger of losing its position at the forefront of clinical research. In particular, Scotland has gone down the league table for the provision of large-scale phase 3 commercial trials. The cabinet secretary knows well that those trials are crucial for the scientific community and for patients who participate in trials.

The debate has given us a useful reminder of the often-forgotten sufferers of orphan diseases, who feel that they are the Cinderellas of the health service. I welcome the Scottish Government’s initiative in setting up the new rare conditions medicines fund. However, careful evaluation is needed over the three years of the fund’s life to ensure that it works for those who suffer the most.

The Deputy Presiding Officer:

Before I call Willie Coffey, I remind members that after his speech we will move to the closing speeches. I expect all members who participated in the debate to return to the chamber for the closing speeches.

Photo of Willie Coffey Willie Coffey Scottish National Party

In more than 20 years as an elected member trying to help local people with a variety of issues, I think that I can say that this issue has proved to be the most challenging, frustrating and heartbreaking of all.

When Janice and Alan Glasswell first came to see me at my surgery in Darvel, I had no idea of the battles that they faced. Janice faced a battle with cancer, but the family faced another battle—one with the system, to try to get treatment that they hoped would help. It was a battle to get basic information, to get explanations, to meet the people at the centre of the case and ultimately to understand the reasons why the system rejected her application for the drug Cetuximab.

Even after Janice sadly died, and despite my best efforts, I could not understand why a person who, opinion suggested, might benefit from the drug was still refused it. None of us could understand the criteria that applied in the IPTR process. Nobody explained it, and the appeal did nothing to alter the outcome. There was clear clinical opinion in favour of giving Janice the treatment but that was not enough. I still do not know why, and I suspect that to this day Alan Glasswell does not know either.

Many of the issues that made matters worse were not even clinical issues. They were about communication, getting simple explanations, being allowed to meet people and feeling that the system was trying to help rather than hinder. The family became medical research experts, looking for comparable cases to help their argument—and they found them. However, they should not have had to do any of that—so much of the family’s remaining precious time together was taken up with that activity.

I am grateful to the cabinet secretary for personally meeting Mr and Mrs Glasswell during her illness. I am grateful that he listened to their story and took the action that I and many people hope will go a long way towards making sure that families do not have to face such circumstances again at a time when they are at their weakest and when they need most help.

The IPTR process is to go and will be replaced by a new system that will be clinically driven, will consider those medicines not yet approved by the SMC and will put the patients at the heart of the process, involving them in matters that affect their health. The decision on whether to grant a medicine will now be taken by clinicians who agree that benefit can derive from it, rather than people having to prove exceptionality, as happens at present. The Glasswell family found that perhaps the most torturous part of the experience was knowing that one clinician thought that the drug could help—only for them to be demoralised by refusal on the basis of something called “exceptionality”.

One crucial element of any new system is this thing called “timeliness”—the need to conduct reviews and to come to decisions as quickly as possible.

As I understand it, the SMC’s review, which is to be completed by Christmas, will look at cost-effectiveness and at what kind of cost thresholds, or QALYs, should be in place. Such a value-based pricing policy appears to have been dropped by the UK, to which overall powers in this area were reserved.

As the Health and Sport Committee recommends, whatever we do with the QALYs, we must ensure that we do not simply apply the same criteria and end up with the same outcome, which disregards clinical benefit. The committee’s report and the witness evidence covered that issue, and I am pleased that the Scottish Government is to introduce a range of measures to make all the necessary improvements. Members have mentioned that the extension of the £20 million rare conditions medicines fund to 2016 will help many people who are suffering from cancer and other diseases for which medicines have not yet been approved. The cabinet secretary has acted positively to address those difficult situations.

It is important that any new system that we introduce is transparent and allows people to be held to account for the decisions that are made. An apparent lack of accountability was one of the main difficulties reported to me. Therefore, I hope that the additional £1 million that is being made available to assist with transparency will in some way involve HIS in its wider audit role, as well as helping to keep patients in the loop.

As we know, healthcare does not come cheap. It costs nearly £12 billion, which means that about a third of the entire Scottish Government budget is aimed at supporting our national health service. It is a service that we are proud of and one that we are determined to maintain and improve.

Ultimately, although a new system for providing access to new medicines will lead to yeses, sadly, it will still lead to some noes. The crucial element that has been missing until now is confidence—families such as the Glasswells had lost confidence in the previous system and they had to fight on another front, which they should not have had to do. We now have the chance to restore that confidence.

I think that we owe a debt of gratitude to the cabinet secretary, Alex Neil, not only for listening with sympathy and understanding to the plight of many families, but for acting when he was asked to do so. I think that he deserves our full support to make the new process one that we can all be proud of.

The Deputy Presiding Officer:

We come to the closing speeches.

Photo of Jackson Carlaw Jackson Carlaw Conservative

I begin by congratulating Duncan McNeil on the way in which he spoke to the motion and—as others have done—on the way in which he has convened the Health and Sport Committee and managed the evidence-hearing process. He said that the official reporters might get a bit lost with all the acronyms; he said that they would be bedazzled, bewitched and bewildered by them. I am not sure that, at one point, Duncan McNeil did not drop in “AC/DC” among all the acronyms that he deployed.

That illustrates the fact that this is a complicated subject. It is also a very serious subject. As numerous members have said, there is a lot of detail, but I do not think that we need to be po-faced. I hope that I am not being unnaturally celebratory about the whole thing, but I approach the debate on the report and everything that has happened not as a cock-eyed optimist, but as a cold-eyed optimist. A lot of detail needs to be developed and established, but I think—I hope—that today is a landmark day. We must ask whether the many questions that members across the chamber have raised have been posed out of a suspicion that an end result may not arise from everything that has been announced today, or because we believe that there is a determination to arrive at an end result. It is the latter that I think that we can have confidence in.

When I was having a coffee, an aide texted me to say, “You’ve been far too kind to the cabinet secretary—yuck!” That sentiment has been ladled on by other members. The door into the chamber is wide, but I hope that the cabinet secretary can still fit through it at the end of the debate.

I think that Mr Findlay is going to remind me of that fact.

Photo of Neil Findlay Neil Findlay Labour

I think that one of the lessons that we can take from today is that when people at the grass roots tell us that there is a problem with a system or policy, we are duty-bound to listen and correct it.

Photo of Jackson Carlaw Jackson Carlaw Conservative

I very much agree. Indeed, as Neil Findlay, Malcolm Chisholm, Christine Grahame and several other members have pointed out, the whole process began with petitioners raising the issue for us. From that, we got a far better understanding of the reality of what was happening and, as politicians, we had to confront that truth. Mr McDonald said that he was unique in being a member of the Health and Sport Committee and the Public Petitions Committee but I can assure him that Nanette Milne was bristling with impatience at that point, having also been a member of both when these matters were being discussed. However, although his experience might not have been unique, we can agree that Mr McDonald is and leave it at that.

I particularly liked two speeches in the debate. First, I was very impressed with Ken Macintosh’s personalised speech, which detailed individuals’ very practical experience and really spoke to the point that I tried to make, which is that we are living in an era of rapidly advancing technology, to which we must be able to respond quickly. We must not forget that every sufferer is an individual.

Mr Macintosh mentioned ipilimumab, and the effectiveness of the new PAC system will be determined by whether it improves patient access to innovative treatments. An early test of the system might lie in whether patients are able to access a new drug for melanoma. Ipilimumab is expected to receive a European licence next month for first-line treatment of patients with melanoma, having already been approved by the SMC for patients requiring second-line treatment. When the drug first received a licence in 2011 for patients who already received therapy, it represented the first advance in treating the disease in 30 years. Several of us attended an excellent briefing at which we met relations of those who suffered and died from that cancer but who had an extraordinary end-of-life improvement as a result of access to the drug through trials elsewhere. In fact, some using the drug survived well past the original expectations. Despite that, however, all 30 IPTR applications that clinicians made for the drug were turned down. We must not see history repeating itself over the coming months.

Christine Grahame said that some constituents were not told about the IPTR process. I think that some clinicians and GPs simply lost faith in the process. Indeed, some will say that they were told not to bother making an application, as a determination had already been made and there was no point. Such things must be overcome. Ms Grahame also made points similar to mine about the importance of knowing what is going to happen in the here and now, between today and the end of the year, to ensure that, after all the positive stuff that we have discussed today, people do not find themselves left in the wash.

Presiding Officer, you have given me an extra minute, which I was not expecting. I will conclude by saying that I was not unduly or unnecessarily ladling on the applause earlier. The Scottish Conservatives feel very passionate about what we believe to be a terribly important issue. We recognise—Christine Grahame was keen to nobble me on this point on the way out of the chamber—that there was no support for a cancer drugs fund either in the chamber or from those who gave evidence to the committee, but we still believe that the existence of such a fund in England meant that we in Scotland could not carry on without making a qualitative step forward. The cabinet secretary’s qualitative step forward is full of promise and, as I said earlier, I do not believe that this announcement is designed to create a smokescreen from which genuine progress will not emerge. In supporting—as I believe we will—the cabinet secretary, the Parliament is empowering him to ensure that what he has announced today becomes a reality sooner rather than later.

Photo of Rhoda Grant Rhoda Grant Labour

Like many others who have spoken this afternoon, I welcome the committee’s report and the Government’s response. However, as many have pointed out, the credit for these changes should go to the petitioners who petitioned the Parliament on the subject and the very many people who lobbied their MSPs and had the bravery to fight for others while their own lives were ending.

Nanette Milne and David Stewart paid tribute to Mike Gray and Tina McGeever, who started the campaign five years ago—a campaign that has been joined by so many. If, as Duncan McNeil said, three months is a lifetime to many people who are looking for the drugs, that tells us how many lifetimes have passed in the five years since we started discussing the matter and looking for solutions to the problem.

Decisions on access to medicines need to have empathy and compassion at their core and must be based firmly on clinical judgment and effectiveness. A system that depends on people’s ability to pay flies in the face of an NHS that is free at the point of use. A system that depends on people being able to make the case for their treatment is equally wrong. Everyone, regardless of their wealth or ability to make their case, must be treated the same. We must have a system that is fair and transparent, which we can all sign up to.

Although, like others, I welcome the cabinet secretary’s announcement, the devil is in the detail, as members have said, and I look forward to getting more information when it comes to hand. The test, as Malcolm Chisholm said, will be whether there are more yeses. However, they must be based on clinical assessment with the patient at the centre of the decision making.

Much of the discussion has surrounded access to cancer treatments, mainly because it is a field in which there are rapid changes in medication. As Malcolm Chisholm said, treatments are becoming more individualised and we can tell which medicines will be of benefit to individuals on the basis of their DNA. That means that the choices that we face may not be so tough, as clinicians can burrow down into whom the treatments will be effective for and whom they will benefit. It is not a case of making very expensive drugs available to everyone on a hit-and-miss basis; we can choose people for whom those drugs will be effective and make them available to them.

In order to do that, we must be able to test the effectiveness of the drugs on tumours, and that work is done only when there is a recognised SMC-approved drug treatment regime. If tumours are not tested against all available medicines, clinicians will not have the opportunity to look at alternative drug treatments or apply under the IPTR system. The cabinet secretary said that 40 per cent of applicants are turned down, but we do not know the figure for those who do not apply, perhaps because they do not have the information to enable them to talk about the efficacy of the treatment because it has not been carried out. Also, as others have said, it could be because they know that other applications that have been made have failed. We need to find a fairer process.

If we focused only on cancer treatments in the debate, that would do a disservice to many people out there who have life-threatening conditions. Many conditions that were life threatening in the past are now termed treatable chronic diseases because of medical intervention. As Gil Paterson said, we need to ensure that there is not two-tier access to treatments, whereby those with cancer go ahead of those with other conditions. We need a system that works for all treatments and diagnoses.

Many members have spoken about the cost of drugs for orphan and ultra-orphan diseases, which can be hugely expensive because there is little use for such medicines due to the rarity of the illnesses on which they impact. We may need a different funding formula that reflects that and ensures that those who suffer from those rare diseases are not discriminated against.

Malcolm Chisholm made the point that the fund is difficult to access. I hope that the cabinet secretary will consider improving how people access the fund in the short term. The £20 million per annum is welcome but, as he said, only £6.5 million was drawn down from that this year, which indicates that there may be a gap in how people can access the fund.

David Stewart mentioned that the QALY system does not work with rare diseases. Perhaps we need a quick review of the system to see how we can improve it in the short term until we come up with a long-lasting system that addresses all conditions.

There are also changes in medicines that are available for chronic diseases. Arthritis, for example, can be totally debilitating and have an enormous effect on a person’s life, wellbeing and independence. In such cases we have to weigh the cost of the treatment against the value to not only the patient but society. I spoke to somebody who is looking for treatment for arthritis, which would have kept her independent and at work and stopped her being a burden to society—as she would have seen it—by needing full-time care and depending on benefits for the rest of her life. That cost did not seem to have been taken into account when the decision was made on whether she could access the treatment. That is really important.

Availability of drugs is also an important issue. Recognised drug treatments do not always work for everyone. Lesser-known treatments are out there, but because they are not fully recognised it can be difficult sometimes to persuade clinicians to use them. When clinicians are persuaded, supplies are not readily available. All those issues need to be looked at in the round.

A number of people—Malcolm Chisholm and Nanette Milne were among them—mentioned access to drug trials. That should be talked about, because we lag behind in drug treatments and we are not seen as a preferred site for drug trials because we cannot compare new data with the most up-to-date treatment data available. We need to look at that and ensure that it does not become a problem.

I see that my time is running out, Presiding Officer. I welcome the debate and I hope that it will lead to a system in which we do not have to pay or lobby to get the treatment that we need. We look forward to seeing the detail of the proposals.

The Deputy Presiding Officer:

I call Alex Neil. Cabinet secretary, I can give you up to nine minutes.

Photo of Alex Neil Alex Neil Scottish National Party

I thank every member who has taken part in the debate, which has shown the Parliament very much at its best in responding to the needs of the people. We should remind ourselves that the shapes in the windows are not whisky bottles; the concept is that they are the people looking in on their Parliament to see whether we are responding to their needs. We have done that this afternoon.

I particularly thank those who mentioned me—Jackson Carlaw was extremely kind—but let me say that I have a first-class team in St Andrew’s house, some of whom are sitting here in the civil service gallery. Without them, we would not be where we are today.

One of the key themes running through the debate, from all sides of the chamber, has been the need to spell out the detail much more, so let me make two pledges to the Parliament—well, three pledges really. First, in the time allotted to me—which includes an extra minute—I will try to answer as many of the questions that members posed in the debate as I possibly can. However, I will not be able to answer them all—we would be here until about 7 o’clock tonight if I was able to do that. We will go through the Official Report tomorrow morning and look at all the questions that have been posed by members; next week, we will place our responses in one document in SPICe, which members can access to see answers to the questions that we can answer at this stage. Obviously, there are some questions that we cannot answer yet.

The third pledge—I have made this pledge already to Duncan McNeil as committee convener—is that I will report regularly to the committee before six months are up. In my first report, which I hope will be at the end of November or beginning of December, I will advise the committee of progress on all fronts, so that we are absolutely sure across the chamber that we are all moving together on this and that the committee is fully informed and able to get the information that it needs to monitor our performance in implementing the policy decisions that we have taken.

Photo of Christine Grahame Christine Grahame Scottish National Party

I very much welcome the cabinet secretary’s commitment, but I do not think that the public have access to SPICe. Many questions have been posed, so as far as the cabinet secretary is able to answer them in that timescale, it is very important to put the answers in a publicly accessible area—perhaps the Government’s website—so that those who are interested in the debate, including the many beyond here, can see those answers for themselves.

Photo of Alex Neil Alex Neil Scottish National Party

That would be no problem whatsoever—Christine Grahame makes a very good point, and we will do as she suggests.

I will try to answer as many questions as I can in the six minutes that I have left.

Christine Grahame raised the issue of what happens in the interim period. I have asked Sir Harry Burns, the chief medical officer, and Professor Bill Scott, the chief pharmacist, to provide the guidelines for the new PACS as a matter of urgency.

I stress the urgency: setting up the PACS is not a long-term but a short-term game, and the quicker that we get it set up, the better. We will issue interim guidance on the IPTRs; the single biggest difference between the IPTR system and the new PACS is that the former had to show exceptionality vis-à-vis the population measured by the SMC in considering the drug’s approval, while the PACS criteria relate to the anticipated clinical outcome for the patient. That makes a difference not only to the decision itself, but to the way in which it is reached, as the process will be much more clinically led.

In our statement yesterday, we accepted the recommendations in Tina McGeever’s original petition on the need to ensure that patients are properly informed at every stage of what their options are and how they can appeal a decision; to improve the turn-around time for decisions, which we will monitor very closely; and to improve the NHS inform website so that patients and their carers, friends and GPs and others can very quickly find out what they can do to appeal and find their way around the system. We will implement all those detailed proposals. Taken together as a package, they represent a very substantive improvement in how the individual patient is dealt with in the appeal process.

We understand fully the need to implement the PACS guidance and to get the system set up quickly, and we will do that. I have directed the SMC to come back to me by Christmas at the latest on its review of the QALY thresholds—modifying the modifiers, if you like. I will stick to that timetable, because I honestly believe that we as a Parliament do not want to get into a position in which people who may be caught between the existing system and the new system pay a penalty because they are in the transition phase.

Photo of Duncan McNeil Duncan McNeil Labour

How does the cabinet secretary expect to overcome that problem when those decisions will be constrained by the current modifiers and QALY thresholds during an interim period of up to three months?

Photo of Alex Neil Alex Neil Scottish National Party

The SMC is a very intelligent organisation—it is aware of the Scottish Government’s desire and policy intention, and I am sure that it will take that into consideration in the meantime.

What has happened this week is a very good example of the public interest in the matter. The publicity focused on the SMC’s decisions in its latest round on three particular drugs. One was a lung cancer drug, which it approved. Another was a drug to reduce the craving for alcohol—we are the first country in the whole of Europe, not just the UK, to approve that particular drug. The third was a breast cancer drug, which was turned down.

First, I cannot comment on whether, under the new system, the SMC would have turned down or approved that particular drug. It would be wrong of me to make a clinical judgment in that respect, and I have no intention of doing so. The SMC may have approved it, or it may have paused, or got the drug through the patient access scheme, which it was not able to do before. A whole host of things might have happened under the new system that would not have happened under the existing system.

Secondly, even if that particular drug had been turned down under the new system, the understanding of the public, the wider medical world and the manufacturer regarding the reasons for that decision should now be much greater because of the increased transparency and openness. That is very important indeed.

As Willie Coffey rightly said, even under the new more flexible system, there will still be drugs that are turned down. However, if people at least know the reasons why new drugs are turned down—in much more detail than is the case today—there will, I hope, be greater public, political and media understanding about why such a decision was reached. That is an important part of the process.

Photo of Duncan McNeil Duncan McNeil Labour

I appreciate the cabinet secretary’s answer, but the issue whether, during the transitional period, drugs will be turned down under the new regime or the old regime has not been resolved. The cabinet secretary has said that he would expect the SMC to take into cognisance his current position. Has he made his current position clear to the SMC?

The Deputy Presiding Officer:

Cabinet secretary, your time is nearly up.

Photo of Alex Neil Alex Neil Scottish National Party

Let me say that I have absolutely, totally and unequivocally made my position clear to the SMC. Obviously, we will work with the SMC. The chairman of the SMC has made a public statement endorsing the statement that we made yesterday, so we are talking to people who are on our side on this issue. As I said earlier, the SMC is an intelligent and world-leading organisation and it recognises that the additional changes that I have outlined are required to make the system even more robust.

Unfortunately, that brings me to the end of my time. We will place in SPICe next week detailed responses, where we can, to the questions that have been raised but which I have not had time to answer today. Again, I thank members for their kind compliments.

Photo of Bob Doris Bob Doris Scottish National Party

It is a privilege to sum up the debate on behalf of the Health and Sport Committee. Let me begin by echoing the thanks that many members have given to all those who provided written and verbal evidence to our committee. I also thank the Cabinet Secretary for Health and Wellbeing, Alex Neil, and his team for their open, constructive and listening approach to the committee’s work. Those sentiments were echoed by both Jackson Carlaw and Willie Coffey during the debate.

The Deputy Presiding Officer:

Sorry, Mr Doris, can you move your microphone closer?

Photo of Bob Doris Bob Doris Scottish National Party

Yes, of course.

The process by which Scotland’s Parliament has dealt with the access to medicines issue perhaps demonstrates our Parliament at its best. From what we have heard, the cabinet secretary will need to ensure that the door is large enough to fit his head through when he leaves the chamber, but I ask Mr Findlay not to leave the chamber at the same time. I thought that the tone of the speech from Mr Findlay, who leads for the Labour Party on health, shows that we have a real cross-party consensus to move forward on the issue together. I thank Mr Findlay for his speech. There is light and shade to Mr Findlay’s speeches in this Parliament.

Photo of Neil Findlay Neil Findlay Labour

I will stand up to respond, but I may collapse after hearing that. I congratulate Mr Doris on giving me what I think is the first compliment that I have ever had from an SNP member.

Photo of Bob Doris Bob Doris Scottish National Party

I will hold a sweepstake among the back benchers on how long it will be before Mr Findlay gets his second compliment. I will let him know who wins that.

On a more serious note, let us not forget that the Health and Sport Committee’s report is a substantial body of work that, as the committee convener said, follows on from a number of petitions that the committee received via the parliamentary petitions system. Our findings are based on robust scrutiny and evidence.

Although our committee has not formally considered the Scottish Government’s response to our recommendations, given that many of our recommendations seem to have been accepted by the Scottish Government, I am sure that—in general terms if not on all the specifics—committee members will welcome much of the cabinet secretary’s response. Of course, as the cabinet secretary would expect, detailed scrutiny by our committee will follow. We will also look through the Official Report of today’s debate to see how best to take matters forward as a committee.

On the SMC system more generally, in my view our inquiry’s aim was not to fix a system that was broken but to improve a system for approval and access to medicines that was already considered world leading. Malcolm Chisholm also made that point. We looked at the quality-adjusted life year, which we have heard much about during today’s debate. In paragraph 71 of our report, we said:

“No one argued that there was a better system than the QALY for assessing the value offered by competing treatments”.

However, despite that assertion by our committee, the next three words in the quote, which is incomplete, are vital—they are “despite its limitations.” The committee sought to move forward beyond those limitations, which is why we urged the Scottish Government to

“review as a matter of priority how modifiers and thresholds are applied to better take account of orphan and ultra-orphan conditions, end of life and innovation”.

Of course we want the details of that review, but I am delighted to hear that the SMC has already started the process and that we expect a report back to the cabinet secretary before Christmas. The committee decided that the matter is pressing and I am glad that the Government appears to have moved speedily on it.

I want to raise the matter of modifiers and thresholds, because to focus only on the IPTR system would be to ignore a key issue in our inquiry, which is why the SMC was turning down certain medicines for approval in the first place. In my opinion, that led to the IPTR process being used as a backdoor approval mechanism, for which it was never designed.

In giving evidence to the committee, Eric Low of Myeloma UK repeatedly made the point—I am not quoting directly; rather, I am going from memory—that we need to design a system that gets more yeses by the SMC first time but in a fair and consistent manner. I believe that we will achieve that through the review of the QALY modifiers, combined with a number of other matters. The first of those is having a pause in the SMC process. In England, if it looks as though discussions between the pharmaceutical companies and NICE are not going to lead to a yes, there is a pause in the process to allow people to reconsider rather than take entrenched positions. That is a sensible proposal and I am glad that the Government is taking it forward. There are also recommendations, to which I will return if there is time, on taking a wider view of value and cost effectiveness in relation to approval of medicines in Scotland.

An issue that could have been controversial but which did not turn out to be because of the sensitive way in which our committee handled it was the matter of the cancer drugs fund in England and end-of-life conditions. I was going to read some quotes from the report but, because of time constraints, I will not do so. However, the committee unanimously ruled out a cancer drugs fund in Scotland. I pay tribute to Nanette Milne for sensitively handling the way in which we got to that cross-party consensus. Needless to say, the committee agreed that we should not single out any end-of-life condition but that we should instead improve our system in a fair, consistent and evidence-based manner. That is why the committee ruled out a cancer drugs fund.

I have spoken about the enhanced modifier for the SMC to take greater cognisance of end-of-life conditions. Of course, that is only part of the solution to dealing with end-of-life conditions and, in particular, cancer. As we have heard, no system will always say yes, so individual patient treatment requests become crucial. Paragraph 62 in our report makes a number of recommendations that lean towards a national system of individual patient treatment requests, with a desire to move away from exceptionality, which has been mentioned. We believe that exceptionality was a flawed basis on which to move forward with patient treatment requests.

The cabinet secretary’s response to those recommendations is perhaps to rip up the IPTR system, so the committee will now have to take a decision on the peer-approved clinical system. Intuitively, I am drawn towards that. In the debate, we have heard of a number of examples of clinicians saying that there was a strong evidence base for an end-of-life medicine and that it would make a material difference to their patient, yet the request was still refused. If the peer-approved clinical system does what it appears to say on the tin, that situation will become a thing of the past, which I warmly welcome. Of course, the committee will have to hold back on taking a position, to allow us to consider in more detail how the new approach will be rolled out.

As the convener, Duncan McNeil, did, I ask the cabinet secretary to consider the use of modifiers in the SMC process before the new system comes into place and to consider how area drug and therapeutics committees will view exceptionality in the transitional period until the peer-approved clinical system comes into place. My strong call—I do not have the convener’s permission to make it, but I am sure that I speak with the committee’s consent—is that we would expect every area drug and therapeutics committee to take cognisance of peer-approved clinical recommendations for drugs now and not to wait until the formal system comes into place. I think that I am on safe ground with the committee in saying that.

A delay can occur in medicines that the SMC has approved finding their way on to local formularies. On my reading of the cabinet secretary’s response to the committee, that is one of the few instances when he is not on the same ground as the committee. The committee looked at the idea of making a national formulary available locally and making medicines available consistently and readily within the 30-day period that is set out. The cabinet secretary has given assurances about the speed with which medicines will be put on local formularies but has not necessarily accepted the idea of a national formulary. However, I note that the Scottish Government’s response talked about

“the implementation of certain medicines of key clinical importance though discussion and agreement of the relevant specialists within NHSScotland to ensure ... outcomes for patients in all parts of Scotland are optimised”, which will be made available on a national basis outwith local formularies. That moves part of the way towards the committee’s recommendation. The committee will of course have to look at that.

The Deputy Presiding Officer:

You are in your final minute, Mr Doris.

Photo of Bob Doris Bob Doris Scottish National Party

In the time that is left, I will talk about value-based pricing. That has unravelled slightly—to be kind to the UK Government—but we should not dwell on that. We should look forward as a Parliament, a committee and a Government. We must capture better the value of medicines in Scotland. When we give a patient a medicine, that has a benefit. That patient might benefit—as one of my constituents could—by no longer being in a wheelchair. If that is the case, what are the savings to the local authority and to the NHS? What are the benefits to society? Right now, none of that is captured.

I am delighted that the cabinet secretary has indicated that we will move to a Scottish model of value to capture the value in what is cost effective, beneficial and desirable for society.

The Deputy Presiding Officer:

Mr Doris, I need you to finish.

Photo of Bob Doris Bob Doris Scottish National Party

I see this as a groundbreaking moment in how the Parliament and the Government address access to medicines. Like Jackson Carlaw, I think that what is proposed will endure and will make a substantial difference to our constituents’ lives.

It is a pleasure to close the debate. I look forward to on-going scrutiny on behalf of and with the Health and Sport Committee.