I beg to move

That the draft Human Medicines (Amendments Relating to the Early Access to Medicines Scheme) Regulations 2022 be approved.

The Business Committee has agreed that there should be no time limit on this debate.

I am seeking the Assembly's approval of the regulations, which will put the existing early access to medicines scheme (EAMS) on a statutory footing.

The EAMS aims to give patients with life-threatening or serious debilitating conditions early access to promising new medicines that do not yet have marketing authorisation. Under the scheme, the Medicines and Healthcare products Regulatory Agency (MHRA) gives a scientific opinion on the benefit-risk balance of using a medicine. That opinion provides independent assurance to prescribers, who may wish to prescribe an unlicensed or off-label medicine in cases where there is an unmet medical need.

Since its inception in 2014, the EAMS has acted as an important regulatory flexibility for early access. Significant numbers of patients in all parts of the UK, across a range of conditions including cancers and rare diseases, have benefited from more than 40 scientific opinions issued by the regulator. During the EAMS period, the EAMS medicines are provided by the company free of charge to Health and Social Care (HSC). Once a marketing authorisation has been granted, arrangements are put in place for existing patients to continue to receive supplies free of charge. Access to new patients is governed by existing HSC processes for the managed entry of new medicines into the health service.

Even just one new product made available through the EAMS can benefit hundreds of patients. For example, through EAMS, UK patients were amongst some of the first in the world to access the breakthrough treatment of pembrolizumab. Approximately 500 patients with advanced melanoma received that medicine when no other treatment was available to them.

It is, however, the case that, at present, there is no UK legislation that specifically covers EAMS, and the scheme is entirely non-statutory. The supply of EAMS products is regulated by exemptions in the Human Medicines Regulations 2012 that govern the supply of unlicensed medicines in certain circumstances. Those regulations have a much wider remit and are not tailored to EAMS specifically. There are some aspects of EAMS that would benefit from clarification in law.

Placing the EAMS on a statutory footing allows us to maximise the benefits of this valuable scheme for patients and pharmaceutical companies, ensuring that the EAMS remains an attractive option for companies to provide medicines to patients prior to licensing. It will also make the EAMS more visible to those who are developing medicines and will ensure that the UK remains internationally competitive in the pre-market medicines access landscape.

It is proposed, therefore, to mandate the scheme by making specific legislative provisions for the EAMS in the human medicines regulations (HMRs). Following extensive stakeholder engagement and a public consultation, it is now proposed to amend the HMRs in order to, first, provide clarity around EAMS medicinal products and establish EAMS licensing authority functions; secondly, to allow for the manufacture, assembly, importation, distribution and supply of EAMS medicinal products; thirdly, to set out pharmacovigilance compliances; fourthly, to introduce the new and important arrangements for the collection of real-world data; and, fifthly, to provide for transitional arrangements prior to the regulations coming into force.

A UK-wide consultation took place on the proposed amending regulations during August and September 2021. My Department ensured that the consultation was circulated as widely as possible in Northern Ireland. Overall, there was broad agreement, including from Northern Ireland stakeholders, that introducing the core principles of the EAMS on a statutory basis would provide legal clarity, which would benefit industry, patients and healthcare professionals alike. While some respondents focused on the procedural elements of the scheme, it is intended that those will be addressed through the provision of updated, bespoke EAMS guidance to supplement the new provisions.

The EAMS has already demonstrated its enormous value in transforming the lives and health outcomes for hundreds of patients throughout the UK as a whole. Already this year, scientific opinions for new, innovative medicines to treat sickle cell disease and chronic myeloid leukaemia have been issued. The new regulations allow us to build on that solid foundation and to deliver a scheme that goes even further in boosting access to life-changing treatments while, at the same time, enhancing safeguards to protect patients and driving forward opportunities for innovation and the development of new medicines.

At an evidence session with my officials on 3 February 2022, the Health Committee was content with the policy intent of the draft regulations, and on 24 February 2022, the Committee raised no issues with the content of the regulations. With the Committee's support, I commend the draft regulations to the House.

I will make some brief remarks as Chair and then some comments as my party's health spokesperson. The early access to medicines scheme is important because it allows patients in the North of Ireland with a life-threatening or seriously debilitating condition to get access to a medicine before it has gained approval from the UK's Medicines and Healthcare products Regulatory Agency.

As the Minister has outlined, however, the early access scheme currently operates on a non-statutory basis. The new regulations seek to put the scheme on a statutory footing, and that is to be welcomed. The Committee was briefed by departmental officials on the regulation on 3 February 2022. Officials outlined to Committee members the processes that are in place to allow for early access to medicines, and they provided further detail on the consultation that has taken place on the new regulation.

The Committee sought further information on the number of patients here who have benefited from the scheme. The Department advised in writing that 45 patients from the North had applied to the scheme over the last three years. Officials advised the Committee that, by placing the scheme on a statutory footing, they would expect the number of applicants to increase as the scheme should simplify the process. I thank the officials for engaging with the Committee in relation to that. The Committee considered the statutory instrument, and members were content that it be approved by the Assembly.

Very briefly, as Sinn Féin's health spokesperson, I will say that the scheme offers a small degree of flexibility to address unmet clinical need in rare cases or diseases by allowing access to potentially life-saving or life-changing medication. The small number of cases and the impact of costs should be relatively low. However, future widespread use will still need to be considered by the appropriate authority, which is the National Institute for Health and Care Excellence (NICE) at this time.

The scheme will, hopefully, help those hard and rare cases to get help and support sooner, and I know that, as MLAs, we have all been involved in cases or campaigns where that has been an issue. Sinn Féin supports improving access to medicines.

Again, I support the motion before us. This time, it is the draft Human Medicines (Amendments Relating to the Early Access to Medicines Scheme) Regulations 2022. We understand, as we did before, that the Human Medicines Regulations 2012 govern the arrangements across the UK for the licensing, manufacture, wholesale dealing and sale or supply of human medicines for human use. Rather than repeat the Chairman's remarks, I will simply say that this is a sensible move and that, as a party, we welcome the new provisions, which will provide clarity and practical solutions for dealing with EAMS, medicinal products and licensing authority functions.

The regulations aim to provide the early access to medicines scheme with a legal basis for delivering the relevant medicines. Patients with the rarest conditions are facing challenges about which we are still very much learning. Very often, those conditions are identified in young children, so it is only right and proper that we do all in our power to try to make life easier for those children and their families, whose lives are turned upside down as a result of some genetic conditions. The hope for us, however, is that many new medicines are being developed and that they have the promise of being genuinely life-changing for those who suffer from such conditions. In fact, I understand that about 40% of the medicines currently in development are for rare or very rare conditions, and they include many cell and gene therapies that are personalised to individual patients. We therefore cannot afford to be left behind in the fight against such diseases. Developing new and better medicines for such debilitating and genetic conditions takes time and effort, to say nothing of funding. We must place that task in the hands of our scientists and virologists, with trust and optimism that they know what they are doing and what they are trained to do, which is to improve the lives of people everywhere. The SDLP supports the motion.

Again, I will say a few words in support of what is, in effect, a complicated but necessary set of regulations that will amend existing law to put the early access to medicines scheme on a statutory footing. Over the past two years or so, we have seen how essential clinical trials are to our common well-being. In 2014, the early access to medicines scheme was developed as a further lifeline. There is no doubt about the positive impact that it has had, not least for people with rare diseases, who would otherwise simply never have had access to medicines. It is also good news for those of us who sit on the newly formed all-party group (APG) on rare disease, because it is something on which we will be working.

There have, however, been occasions on which the scheme has been a cause of frustration to people, including some who have contacted my constituency office. No doubt, part of the reason for that is because the scheme remained non-statutory. It is evident that placing the scheme on a statutory footing will provide legal clarity for pharmaceutical companies, as they now have a clear framework for operation in Northern Ireland and across the UK, as well as clarity around data collection. It does much more than that, however. There is a significant element of tidying up which licences are necessary. That will do much not just for the providers but, more importantly, for people, by getting medicines out to them faster. There is also clarity around the prohibition of the advertising of unlicensed products and around monitoring the risks of medicines that are relatively rarely prescribed.

Uniquely in the UK, for Northern Ireland the regulation of medicines is a devolved matter. I trust that that will enable us to respond swiftly to any concerns pharmacists or other healthcare professionals raise about how the scheme will be continued here once it is put on a statutory footing. We must therefore proceed with appropriate caution. Overall, however, this step makes a lot of sense and provides welcome clarity.

I call the Minister of Health to conclude and make a winding-up speech.

I thank the Chair, Deputy Chair and members of the Health Committee for their contribution to the debate. I will make a few comments on the contributions made. Mr McGrath said that we must put our trust and optimism in the scientists and virologists as they seek to develop our medications, as we did especially during the development of our COVID vaccines. Others talked about the ability to improve access to the specific medications that are being brought forward. My officials and I look forward to engaging with Ms Bradshaw, through the APG on rare disease, on how moving EAMS on to a statutory footing can make a difference. The statutory scheme will continue to be open to any medicines that meet the EAMS criteria. To date, those have predominantly been treatments for cancer, but other examples include heart conditions and chronic hepatitis. The scheme is equally open to all treatments that may be available in the future for different conditions.

I commend the motion to the House.

Question put and agreed to. Resolved:

That the draft Human Medicines (Amendments Relating to the Early Access to Medicines Scheme) Regulations 2022 be approved.