Access to Regenerative Medicine in the NHS: Regulation and Reimbursement
by Aurélie Mahalatchimy and Alex Faulkner
The complexity of tissue, cell and gene therapies means that few have been evaluated for entry into the marketplace and possible NHS adoption. The strict safety, efficacy and quality regulations and the normal evidence requirements governing medicines and medical devices are challenged by these therapies. Likewise, their specialised nature, high promise but uncertain health impact mean that the issue of how to pay for them is crucial to the development of the field and to patients’ access to therapies.
Regulation of the field is complex. The main applicable law is the European Union Advanced Therapy Medicinal Products (ATMP) Regulation whose decision committee recommends to the European Medicines Agency. However, few products have been approved through this system, none of which to date has originated from the UK. This and other applicable regulations are implemented by the MHRA. The system allows for a variety of flexibilities in the form of exceptions and exemptions to the centralised procedure especially where a producer can claim to be addressing an unmet medical need or a rare disease where ‘orphan’ drug status can provide incentives. Therapies require scientific assessment by gatekeeper NICE appraising clinical and cost evidence that is typically highly uncertain, and payment for such therapies would be a question for NHS England’s central Specialised Commissioning processes.
Producers in the UK are trying to take advantage of the flexibilities in the system, especially the so-called ‘Specials’ scheme to bring products to the clinic for individual patients, which does not require marketing approval. The MHRA has introduced the ‘Promising Innovative Medicine’ designation as an incentive, under an Early Access to Medicines initiative, where producers have reduced data requirements and provide the therapy to small number of patients at no cost. NICE has introduced its Office for Market Access, to inform producers what likely data will be required by NICE for its clinical and cost effectiveness appraisals; the applicability of NICE’s usual appraisal methods is contested by stakeholders. Discussion of many possible payment models is under way, with various forms of financial ‘risk-sharing’ being widely debated.
The regulatory pathways remain complex and impending Brexit introduces further uncertainties. The appropriate and feasible forms of data for assessment of regenerative medicine therapies remain under debate, with pressure to adopt more ‘real world’ evidence. It is unclear whether ‘regenerative medicine’ can be treated as a category for regulatory and especially reimbursement purposes, in spite of the UK government’s highlighting of the field.
A plethora of flexible regulatory and commissioning pathways is developing, but the evidence for the effects of acceleration schemes on time to the clinic is sparse and equivocal. Trade associations are particularly active at national level in trying to promote use of real world (e.g. registry) evidence. ‘Value’ has emerged as a key term in appraisal and reimbursement debates, highlighting value conflicts between patients, national gatekeepers and producers. The provisional nature of new designation and incentive schemes is a sociological phenomenon of broad significance in biomedical innovation.