Should Regenerative Medicines be Awarded 'Special Treatment' in Regulation?

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. 

To read the full paper click here. 

Neoliberal Technocracy: The US FDA and Pharmacogenomics

The relaxation of regulation in pharmaceuticals has been linked to an increasing role of regulatory agencies as facilitators of innovation as opposed to simply gate-keepers. The US Food and Drug Administration has been at the debate since the beginning. A recent paper by Stuart Hogarth (drawing on the work of Williams et.al, 2011) highlights the recent ‘innovative’ turn of the US FDA, its vision of a genomic future for pharmaceutical Research and Development in the USA and the wider neoliberal policy agenda of permissive regulation.

There have been many differing opinions on the way that the US’s pharmaceutical policy has become more neoliberal in character. On the one hand Abraham and Davis (2013) argue that there has been an emergence of ‘corporate bias theory’ in which reforms calling for less stringent regulations are pursued under the belief that patients and industry both seek less regulations and faster drug discovery processes. In contrast, Carpenter (2011) takes a pluralist perspective focussing on the impact of proposed reforms on the 'interests' of a wider group of actors ranging from specialist interest groups, civic organisations, media syndicates, corporations and wider society. As reputation management is crucial to the organisational behaviour of regulatory agencies, Carpenter suggests that by according importance to the interests of such audiences the FDA has manage to retain such power.  

The push for relaxing regulation in the US FDA can be traced to the years following tightening of FDA processes in the wake of the thalidomide crisis.  In 1962, The Kefauver Amendment to the Federal Food, Drug and Cosmetic Act (in response to the Thalidomide crisis) was enacted to tighten (i) pre-market review of safety and efficacy of every new drug candidate, (ii) through a three-phase clinical trial process, (iii) using statistical methods popularly known as Randomised Control Trials (RCTs). By the end of the 60s and 70s, these regulations were held primarily responsible for slowing the FDA's approval processes and came under considerable fire for slowing the US drug discovery pipeline compared to counterparts in other countries. In response, the Reagan administration sought to speed up US drug discovery with a ‘radical deregulatory agenda’ and in 1992 signed the Prescription Drug User Fee Act into law. The Prescription Drug User Fee Act (since amended numerous times) was intended to allow the FDA to collect feeds on human drug and biological products in order to expedite the drug approval process.

At the same time, 'deregulation' has not been without its critics. For instance, some have blamed the the Vioxx crisis (2004) as a negative fallout of FDA's deregulatory turn.  A study of the Vioxx episode by Woodcock (Clinton and Wechsler 2006) highlights the inadequacy science in general and the existing drug discovery methods in particular, to provide a credible solution to similar crises. For Woodcock, the key need is to shift away from existing statistics-dependent testing methods on broad pools of patients towards targeted clinical development on individual patients likeliest to benefit from a drug (Clinton and Wechsler, 2006). Increasingly, it is the latter 'individual patient focussed' solution -popularly called Pharmacogenomics that presents a credible drug discovery alternative as the best way if you will to ‘establish unmet medical needs, and identify patients who are predisposed to adverse events’’ (Woodcock 2011, p15).

In summary, while pharmacogenomics is often viewed as a result of the neoliberal turn for deregulation, at the same time it holds considerable promise as a better vehicle (than existing drug discovery processes) for delivering vital public goods like cheaper and faster drug development etc. 

To read the full paper click here