The Chairperson (Ms Kimmins): In attendance we have Chris Garland, senior principal pharmaceutical officer, and Eddie Murphy, deputy principal, pharmacy directorate. You are welcome, and I thank you for your patience, as we have been significantly delayed. I ask you to make your opening remarks, and, if there are any questions, we will come in with them afterwards.
Mr Eddie Murphy (Department of Health): Thanks, Chair. I will try keep this short and sweet. I will provide some brief background and detail around the policy intent of the regulations in question. When I have finished, if anybody wants to ask any questions, we will be happy to answer them.
Thank you very much for the opportunity to brief the Health Committee on the proposals set out in a recent SL1 letter to the Committee on plans to make a UK-wide statutory instrument entitled the Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations. As the Chair and Committee members will be aware, the human medicines regulations (HMRs) govern the arrangements for the licensing, manufacture, wholesale dealing and supply of human medicines for human use. The medicines set out in the Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations will amend the Medicines for Human Use (Clinical Trials) Regulations 2004 and the HMRs to enable the introduction of a new tailored regulatory framework to support point-of-care and modular manufacturing, enabling decentralised methods of medicine manufacturing and moving innovative medicines closer to the patient while maintaining robust regulatory standards and ensuring patient safety.
Introduction of this framework is intended to proactively support the development of new medical advancements on the cutting edge of technology and new means of medicine manufacturing, facilitating the development of highly specialised medicines where they are most needed for patients, whether that may be in hospital wards, operating theatres, community centres or even patients' homes. These products are in the early stages of development, but one example is a diabetic foot-ulcer treatment using blood-derived products obtained from the patient and manufactured at their bedside. Modular manufacture can support early-stage vaccinations deployment by allowing vaccines to be locally filled and finished, ensuring efficient supply to mass vaccination centres.
These amendments are needed as, increasingly, with advanced health technology, innovative medicines are now being developed that will need to be manufactured close to patients and healthcare settings. The traditional approach by the UK medicines regulator, the Medicines and Healthcare products Regulatory Agency (MHRA), therefore needs to adapt to support the development of new technologies. Current arrangements are suited to centralised, factory-based manufacture, with a small number of fixed manufacturing sites named on manufacturing licences and on marketing authorisations.
Point-of-care and modular-manufacture products may potentially be manufactured at multiple sites across the country. Some of these products are developed to meet the unique needs of each patient when those patients need them and are often derived from the patient's own cells or blood. Indeed, some products must be administered within an hour, or even minutes, of being manufactured. This creates an urgency and specificity that traditional manufacturing cannot easily accommodate. It would be extremely challenging for these innovative products to be regulated in accordance with current regulations, resulting in significant regulatory and financial hurdles. The objective of this instrument, therefore, is to provide an enabling framework to allow for the safe and efficient manufacture and supply of emerging medicines still in early development and to provide the regulatory clarity necessary to encourage development of these new products and approaches.
The benefits of this new approach will extend to patients and carers, who will benefit from access to new and more personalised medicines in a timely and convenient manner, with potential for some patients to be treated with medicines manufactured at home. It will help move care from hospitals to communities, reducing the need for patients to stay in hospitals. Additionally, healthcare professionals will benefit from the ability to provide a greater range and more effective treatment options, which can improve patients' response to treatment. Furthermore, innovators and industry will benefit from these clear regulatory expectations and the enabling of speedier product development. The new framework will remove regulatory barriers that are not suited to novel manufacturing methods. This benefit will be seen across all sizes of companies in large, small and medium-sized enterprises. It is vital that the medicines regulatory framework is flexible for new innovations but does not compromise on patient safety.
I will now set out briefly how the MHRA will ensure that point-of-care manufacturing products will meet the necessary standards of safety, effectiveness and quality that are expected of all medicines. The new framework is centred on a hub-and-spoke model, with a single control site as hub for each product, overseeing all aspects of the point-of-care and modular manufacture system, including the spokes of the individual manufacturing locations and their activities. The control site will be the only named manufacturing site on the manufacturing licence, clinical trial and marketing authorisation application. The holder of the control site, as its name signifies, will be responsible for ensuring product quality across all manufacturing sites and notifying the MHRA of reportable incidents. The diligence of the control site in overseeing the manufacturing locations will be scrutinised at routine MHRA inspections, and arrangements for oversight will be scrutinised as part of the licensing process. A number of manufacturing spoke locations will also be sampled and subject to inspections to ensure that the oversight claimed by the control site can be independently supported by inspection findings.
The new framework is modified from the current regulatory system for the evaluation of regulatory compliance at manufacturing sites and safety monitoring. There will be no change in the expected standards that must be met for the safety, quality and efficiency of the products. There will not be an increased risk to patient safety, either, with the MHRA's retaining regulatory oversight in that regard.
The MHRA led on a joint public consultation with the Department in accordance with section 45(1) of the Medicines and Medical Devices Act 2021 to seek views on the legislative proposals to introduce a new regulatory framework for the manufacture and supply of point-of-care products. Comments and views were expressed by a variety of stakeholders across the UK and internationally. There was significant support for the creation of a new UK regulatory framework for manufacture and supply at the point of care. The overwhelming majority of responses were positive, with 91% of respondents agreeing that a new framework was required and 94% agreeing with the framework that was proposed. Many responders also supported the framework's having a wider scope of manufacturing and supply to include modular manufacture and home-based manufacture.
Amendments to the HMRs are made under powers contained in the Medicines and Medical Devices Act. The Department of Health in Northern Ireland is named in that Act as an appropriate authority, in recognition of the fact that the regulation of medicines is a devolved matter to Northern Ireland. The regulations will be subject to the draft affirmative procedure and will be laid before the Assembly and approved by resolution of the Assembly. Therefore, an Assembly debate on the regulations will be required.
As the Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations are a joint statutory instrument with the MHRA, they were laid in draft form in Westminster last month and are due for debate and approval in both the UK Houses of Parliament later this month. The House of Commons debate is scheduled for 27 November, and the House of Lords debate is scheduled for 28 November. Following consideration of the SL1 by the Health Committee on the policy intent of the amendment, and if the Committee is content, the Department will seek to lay the legislation in draft form in the Assembly Business Office at the end of this month, and then provisionally schedule the necessary Assembly debate on the regulations in early 2025, following the Christmas recess. That will allow the Committee the opportunity to further consider the legislation, alongside any views that the Examiner of Statutory Rules may have.
I am happy to take questions.
Mr Garland: This is an enabling framework that is intended to incentivise industry and provide a supportive regulatory environment for the development of new products. We expect that the number of patients who will benefit will be quite small in the beginning, because the medicines will still need to go through the normal stages of clinical trials and manufacturing before licensing before any decisions are made about health service access. They are products that are in the early stages of development, so it is likely to take a few years before we start to see them becoming available. However, the pace of development in that area is phenomenal, so, over time, we will, hopefully, see more and more patients benefiting, particularly those who currently have limited therapeutic options for rare diseases and the like. It is potentially transformative.