NICE Appraisals: Rare Diseases Treatments

Part of Backbench Business - Services for People with Autismbackbench Business – in the House of Commons at 4:48 pm on 21st March 2019.

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Photo of Steve Brine Steve Brine The Parliamentary Under-Secretary for Health and Social Care 4:48 pm, 21st March 2019

Indeed, Mr Deputy Speaker; some people just cannot help themselves.

NICE operates two separate programmes for the assessment of new medicines. First, there is a technology appraisal programme through which NICE assesses the vast majority of new medicines. Secondly, as has been said, there is a highly specialised technologies, or HST, programme that is reserved for the evaluation of very high-cost drugs for the treatment of the very small number of patients in England treated in a handful of centres in the NHS. Decisions on whether a medicine should be routed to NICE’s mainstream technology appraisal, or the HST programme are taken through an established topic selection process that includes consideration against published criteria and engagement with a wide range of stakeholders. When NICE recommends a drug for use through either route, NHS organisations are legally required to provide funding so that it is made available to patients.

Today, we have heard concerns that NICE’s technology appraisal programme is not suited to the assessment of medicines for rare diseases, with some calls for individual drugs to be assessed through the HST programme instead. We have also heard calls for a third appraisal route for rare diseases not eligible for the HST programme. I have listened very carefully to all of them and will reflect on them all. There is some sense in a lot of what has been said. Indeed, over the last 20 years, NICE has made positive recommendations in 75% of its appraisals of orphan medicines. By comparison, NICE recommends around 80% of medicines for more common diseases.

I shall give the House two recent examples, because of course we only ever hear about the examples that are stuck or refused. NICE has been able to recommend orphan medicines for neuroblastoma, a cancer of the nerve cells that affects children—this has been widely welcomed—and for primary biliary cirrhosis, a progressive liver disease. Moreover, through its HST programme, NICE has to date been able to recommend a further eight medicines for NHS patients outside of the standard appraisal route. In each case, NICE’s recommendation is subject to a managed access agreement negotiated between the drug company and NHS England.

There will always be cases where NICE is unable to recommend a medicine because the price set by the company does not reflect the benefits that it brings. That is a fact. Hon. Members have of course spoken about the rare diseases of people in their constituencies—they are doing their job—but NICE is an independent body and it should be allowed to develop its guidance free from politicians. The hon. Member for Heywood and Middleton said that that was her initial instinct before she became a politician. That is the foundation of NICE’s reputation as a world leader in its field, and it is in the best interests of patients that it does that.

The hon. Member for Blaydon, in introducing the debate, raised concerns about Kuvan, the treatment for phenylketonuria. NICE has initiated an appraisal of Kuvan, and officials from NICE, NHS England and our Department have been reconsidering the appropriate assessment route in the light of the new available information that the hon. Lady mentioned. Riley is right: we have to make this fair. I am told that a final decision will be taken promptly—I urge that again from the Dispatch Box today—and with the minimum impact on the timescale for NICE’s assessment.

The hon. Members for Strangford, for Bristol East and for Dudley North have all spoken about the issue of Orkambi so many times and so well. It is incredibly frustrating and disappointing to Ministers, just as it is to them and everyone else, that Orkambi is not available to NHS patients in England at the moment. I understand and share that frustration. This is why my right hon. Friend the Secretary of State held a meeting with Vertex, NHS England and NICE a couple of weeks ago. I was at that meeting, at which the parties again discussed how best to reach a conclusion. I am pleased to say that they are meeting again today to continue the discussions and decide on the next steps. Decisions about the availability of drugs in Scotland are of course a devolved matter, and that is up to Scotland. I understand that no decision has been taken on routine funding for NHS patients in Scotland, but the hon. Member for Motherwell and Wishaw asked me to look again at the Scottish system. I will do that and I will ask the Minister responsible for this policy area to do so.

The hon. Member for North Tyneside and others raised the issue of the drug Spinraza for the treatment of spinal muscular atrophy. I understand that NICE’s independent appraisal committee met earlier this month to consider its recommendation on Spinraza following new evidence being put forward by the company. NICE wrote to the company and patient groups last week to say that it was not yet able to provide an update on the outcome of the meeting, but that it would provide an update soon. Again, I encourage that to happen even sooner. I recognise that the protracted process in this instance is hugely frustrating for patients and their families and, whatever our differences across the Dispatch Box, of course I feel the deep hurt that the hon. Ladies who spoke on the subject have laid out. I hope they will appreciate that a final decision has not yet been made and that NICE must be allowed to complete its work free from political interference.

I do not have time to go into a huge amount of detail. I have been asked lots of questions during the debate, but I have little more time than the people who have spoken today. I thank Members for speaking so passionately and I hope that they will welcome the forthcoming review of NICE’s methods and processes over the course of this year for both its technology appraisal and its highly specialised technologies programme, which is at least partly what today’s motion calls for. It would not be appropriate to pre-empt the review by commenting in detail on what it should look at, but I will ensure that it is directed towards the motion before us today and to the transcript of today’s deliberations. I now want to give time to the hon. Member for Blaydon, who introduced the debate, to close it in the time that we have left.