Regenerative medicine is one of the rapidly developing areas of healthcare. However, due to the unique character of such medicines, questions have arisen regarding regulation. These tensions are intensified by the technical novelty, evidential uncertainty and high promise of such technologies. Therefore many have placed forward the argument for regenerative medicine to be granted special treatment with respect to regulation.
A recent paper by Alex Faulkner examines how such medicines can be more readily available to patients, whilst exploring whether or not they should be accorded special treatment. Faulkner assesses how regenerative technologies can be made more readily available to patients by looking at the roles of the market/practice entry regulators and the healthcare system assessment and adoption agencies on the other.
In the UK, ‘gatekeeping’ of regenerative medicines is largely shaped by its role in the EU, although this has become more relaxed in recent years. In turn, three key licensing flexibilities have been introduced in order to provide greater incentives to medicine developers. One of the key flexibilities is conditional approval which essentially allows a product to jump to Phase 4 study as long as its safety has been proven through Phase 1 and Phase 2 clinical trials. This conditional marketing license thus bypasses Phase 3 study. However, this flexibility is typically only allowed for life-threatening diseases. A second flexibility is Exceptional Circumstances which is designed to meet the circumstance where sufficient data can never been generated, for instance disease is rare, research is limited or it is unethical to subject seriously-ill patients to long and intensive testing. The final flexibility is Accelerated Assessment which takes into account the rapid progression of science and responds the expectation of patients. All the three flexibilities have been integrated into the adaptive licensing programme launched by European Medicines Agency (EMA) in early 2014.
The UK has introduced the Early Access to Medicines Scheme (EAMS) cohering with Medicines Adaptive Pathways to Patients of the EU Committee for Advanced Therapies. Unlike above flexible licensing strategies, the EAMS operates outside of marketplace if a clear unmet medical need is perceived. In this regards, the EAMS recognised not only the early role of patients in the process but also the changes driven by genomics, data and personalised medicines. However, there are still conflicts and a need to find a balance between commercial interests, clinician decision-making and national system-level evidence appraisal.
While these incentives and flexibilities are presented as solutions to the issue of ‘special treatment,’ they appear to be lacking. Such relaxations in regulation are often unable to increase incentives, and many do not create faster approval times. Furthermore, the limited number of disease exemptions is preventing such initiatives from having an impact across the board. The rise of regenerative medicine is thus constrained due to existing institutional regimes, epistemologies and methodologies.
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