Support for regenerative medicines – can the Government and the NHS deliver?
Following the surprise announcement of a general election on 8th June, there has been a flurry of activity from the Commons' and Lords' committees, which have had to wind-up their current inquiries much sooner than expected and release reports on their findings so far. The House of Commons Science and Technology Committee (STC) has had a busy session and their report on regenerative medicine was one of three rapidly released at the end of April.
Despite an early finish to the inquiry, the STC made the most of the three oral evidence sessions and numerous written submissions (including from PHG Foundation) and have developed a number of recommendations to support research, innovation and implementation of regenerative medicines.
The key message from the report, which echoed many of the evidence submissions, was that of leadership: from NHSE by including regenerative medicines in the personalised medicine strategy, and from the Government to work with UK Research and Innovation (UKRI) to develop a strategy for advanced therapies, aligned to its response to the Accelerated Access Review and the Industrial Strategy Green Paper. Both organisations were also tasked with working collaboratively with a number of stakeholders to support various aspects of the regenerative medicine workflow, and to strengthen current efforts and widen their scope. The report also highlighted that the regulatory 'one-stop shop' support from the Medicines and Health Regulatory Agency (MHRA) and the regulatory/manufacturing support offered by the Cell and Gene Therapy Catapult are vital to current efforts however both could extend their reach.
A solid research foundation
The report reiterated what many contributors stated – that the basic research base in the UK is currently very strong and that this provides a solid foundation for the development of regenerative medicines. The STC recommended that UKRI should work with the Government to ensure that in the rapidly changing regenerative medicine field there is "…a balance of investment in both basic scientific research and the translational research that it underpins, and to identify any research gaps…". It will be interesting to see how this fits into UKRI's busy agenda when it starts operating in early 2018.
Regulation did not escape the committee's notice, however it was not given as much emphasis as the number of complaints about it in the contributions suggested. In addition to the MHRA's efforts, it was acknowledged that more needs to be done particularly in maintaining robust and flexible regulation. The STC recognised an opportunity presented by Brexit, to assess how the EU Advanced Therapy Medicinal Products regulations might be adapted for UK use to achieve the balance between safety and accelerated access to cutting-edge technologies. A concern raised in the evidence submissions pointed out that hospital exemptions for ATMPs mean that decisions on whether to fund are made at a hospital level, possibly resulting in regional inequalities in availability, particularly if there are inconsistencies in how special cases are defined and guidelines applied.
The NHS as a research resource
The report and many of the contributors recognised the value of the NHS as a resource. Having a universal health service not only facilitates the ability to carry out clinical research – a patient can be followed all the way through their journey – but also a unified service facilitates the sharing of data, information and expertise. However concerns were raised about the challenges of doing clinical research in the NHS. Many cited staff pressures, reluctance to interact and the lack of an obvious 'way in'. The report recommended that the Government work with NHSE and clinical commissioning groups to provide financial incentives to carry out research, and to encourage more clinicians to become involved in research. In addition, NHSE and NICE should be supported to deliver a fast track appraisal system for regenerative medicine therapies. However the challenges of funding clinical research and the delivery of therapies are ubiquitous; the recommendation that effective reimbursement models are developed to pay for regenerative medicines will require creative and long-term thinking from NHSE, NICE and commissioners.
A funding reality check
While these recommendations are all sensible and practical, the challenges facing the NHS will have a significant impact on whether any of them come to fruition. Given the political uncertainty caused by Brexit, funding issues and a preoccupation with the more fundamental challenges facing the delivery of basic care, regenerative medicines are in danger of falling down the 'to do' list. With many NHS staff facing pay cuts in real-terms, investing in expensive therapeutics that benefit the few, admittedly with severe conditions, will be a hard sell. There is likely work to be done in terms of making current processes more efficient and making easy gains in terms of facilitating collaborations between different parts of the health system. However it is not worth developing processes and collaborations if there are not the staff available to make clinical research happen, or staff who are interested cannot take part because of their current workload. With this in mind, promoting the agenda of regenerative medicines will require clear-headed and creative thinking, enthusiasm and a smattering of luck.