Special Treatment? Flexibilities in the Politics of Regenerative Medicine’s Gatekeeping Regimes in the UK

Abstract

Emerging flexibilities are apparent in gatekeeping regimes applicable to regenerative medicine products, raising issues about the extent to which and forms in which such flexibilities might promote emerging products as a sector warranting special treatment, in the context of recent policy developments in the UK and wider European Union. Concepts of 'gatekeeping', 'gatekeeping regimes' and 'gateways' can point to the ways in which regulatory institutions, health technology assessment organisations, and national planners and purchasers of health services together define and control entry to the medical product marketplace and the adoption of products into the public healthcare system. Flexibilities in existing regimes and new gateways are a way of maintaining 'connection' between gatekeeping regimes and technoscientific innovation in order to steer innovation pathways. The gateways concept has affinity with that of Callon's 'obligatory passage points'. A wide set of recent policy documents show that the measures promoted exhibit a range of alternative gateways that are being constructed around central, legal, restrictive gatekeeping regimes. However, it would be easy to overestimate the significance of these developments as relaxations that would favour innovative producers and their products on a large scale with wide public health impacts. The concepts of gatekeeping regimes and gateways enable understanding of hybrid developments of exceptions and exemptions to dominant regimes which bridge across the arenas of market regulation, health technology assessment and healthcare system planning. These arenas are being drawn closer together as a means of politically managing stakeholders' aims in the UK, EU and other innovating biomedical health systems globally.

Evolution of Business Models in Regenerative Medicine: Effects of a Disruptive Innovation on the Innovation Ecosystem

“…Small patient numbers are especially difficult for under-resourced SMEs running either the virtual or early exit Phase I/II business models because they cannot afford to recruit patients outside of the United Kingdom. However, Faulkner 16 reported that regulatory systems are now being adapted for rare indications, and Mittra et al 17 argue that the usual approach for indications such as b-thalassemia is to choose a country with a patient population sufficient for the clinical trial.…”

Alter-Standardizing Clinical Trials: The Gold Standard in the Crossfire

“…The flexible enforcement of regulatory standards in emerging fields of medicine research in China and India has also been interpreted as an attempt by national governments to stimulate domestic innovation, by facilitating clinical research that is affordable to local researchers and companies (Salter, 2008;Sleeboom-Faulkner et al, 2016;Sleeboom-Faulkner, 2016). Other developments, such as the surfacing of new types of regulatory exceptions and exemptions in the EU, and increasing access to experimental products in the USA, are partly the outcome of pressure from patient and consumer organizations, and partly a result of criticism from professional organizations that seek to enable more localized, physician-based forms of innovation (Cooper and Waldby, 2014;Faulkner, 2017;Rosemann et al, 2018).…”

“…These arguments, Montgomery suggests, have facilitated a move away from standardization as the central logic of clinical trials, towards an acceptance of adaptation, which promises 'predictable uncertainty' and the possibility of 'fixing the future' as it unfolds. Alex Faulkner's paper explores the extent to which and forms in which regulatory agencies in the European Union (EU) have started to introduce flexibilities to the standard regulatory pathway for market approval of medicinal products, which involves the mandatory use of multi-phase RCTs (Faulkner 2017; this special issue). Based on a case study of EU regulations for advanced cells and tissue products, he discusses a range of regulatory exceptions and exemptions that allow investigators and research sponsors either to shorten and accelerate the multi-phase RCT process, oras with the EU 'hospital exemption scheme'to provide access to innovative (but unproven) medical interventions without preceding trials.…”

The pluralization of the international: Resistance and alter-standardization in regenerative stem cell medicine

“…As the geographical ties of these organizations illustrate, transnational opposition to the use of RCTs as the obligatory passage-point for market approval of stem cell technologies is increasing, even in the United States and Western Europe. In a context of intense global competition over markets and technological innovation, concerns about losing out, along with increasing health-care costs and the recent economic crisis, have resulted in calls for deregulation, more flexible regulations, and new spaces of regulatory exceptions and exemptions ( Cooper and Waldby, 2014 ; Faulkner, 2014 ). Moreover, stem cell controversies and regulatory changes affect regulatory debates and processes in other areas of medical research.…”