Reimbursement of cell- based regenerative therapy in the UK and France

Cell- based regenerative therapies are presented as being able to cure the diseases of the XXI century, especially those coming from the degeneration of the aging human body. But their specific nature based on biological materials raises particular challenging issues on how regulation should frame biomedical innovation for society’s benefit regarding public health. The European Union (EU) supports the development of cell- based regenerative therapies that are medicinal products with a specific regulation providing their wide access to the European market for European patients. However, once these medicinal products have obtained a European marketing authorisation, they are still far away from being fully accessible to European patients in all EU Member States. Whereas there is much written on the EU regulatory system for new biotechnologies, there is no systematic legal study comparing the insurance provisions in two EU countries. Focusing on the situation in the United Kingdom and France that are based on two different healthcare systems, this paper is based on a comparative methodological approach. It raises the question of regulatory reimbursement mechanisms that determine access to innovative treatments, and their consequences for social protection systems in the general context of public health. After having compared the French and English regulations of cell- based regenerative therapy regarding pricing and reimbursement, this papers analyses how England and France are addressing two main challenges of cell- based regenerative therapy, to take into account their long- term benefit through their potential curative nature and their high up front cost, towards their adoption within the English and French healthcare systems. It concludes that England and France have different general legal frameworks that are not specific to the reimbursement of cell- based regenerative therapy although their two current and respective trends would bring more convergence between the two systems while addressing the main challenges for the reimbursement of these therapies. Nevertheless, despite their current differences neither the English nor the French national healthcare systems have yet approved the reimbursement of cell- based regenerative therapies. The paper highlights where both systems could be learning from each other experiences to favour the adoption of cell- based regenerative therapies through the adaptation of their reimbursement methodologies. It also emphasizes the gap between market access and patients’ access and, it calls for research and discussions through reflexive agencies such as Regenerative Medicine Expert Group in the UK

EU law and policy on new health technologies

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Stretching and challenging the boundaries of law: varieties of knowledge in biotechnologies regulation

The paper addresses the question of adaptation of existing regulatory frameworks in the face of innovation in biotechnologies, and specifically the roles played in this by various expert knowledge practices. We identify two overlapping ideal types of adaptation: first, the stretching and maintenance of a pre-existing legal framework, and second, a breaking of existing classifications and establishment of a novel regime. We approach this issue by focusing on varieties of regulatory knowledge which, contributing to and parting of political legitimacy, in principle enable the making of legally binding decisions about risks and benefits of technologies. We base the discussion around two case studies, one of animal biotechnology ethical regulation, the other of ‘advanced therapy’ medicinal product regulation, both in the context of European Union frameworks. Specifically, we explore the knowledge configurations constituting expert committees and other institutional formations of expert regulatory knowledge in their political context. We show that where sectoral and moral boundaries are challenged, different modes of regulatory knowledge beyond scientific forms – legal, procedural, moral, economic and industrial – can shape regulatory innovations either by maintenance of regimes through commensuration and stretching, or through differentiation and separation creating new frameworks. We conclude that establishing an essential techno-scientific difference between pre-existing and novel technologies does not in itself require new regulatory structures, and that the regulatory strategy that is followed will be determined by a combination of different forms of knowledge

The Legal Landscape for Advanced Therapies: Material and Institutional Implementation of European Union Rules in France and the United Kingdom

In 2007, the European Union adopted a lex specialis, Regulation (EC) No. 1394/2007 on advanced therapy medicinal products (ATMPs), a new legal category of medical product in regenerative medicine. The regulation applies to ATMPs prepared industrially or manufactured by a method involving an industrial process. It also provides a hospital exemption, which means that medicinal products not regulated by EU law do not benefit from a harmonized regime across the European Union but have to respect national laws. This article describes the recent EU laws, and contrasts two national regimes, asking how France and the United Kingdom regulate ATMPs which do and do not fall under the scope of Regulation (EC) No. 1394/2007. What are the different legal categories and their enforceable regimes, and how does the evolution of these highly complex regimes interact with the material world of regenerative medicine and the regulatory bodies and socioeconomic actors participating in it

Accreditation and regulations in cell therapy

Advances in stem cell biology and immunology raise hopes for new developments in regenerative medicine as well as cell-based immune intervention, with potential applications in many medical disciplines. Emerging from the medical practices of organ, tissue and cell transplantations over several decades, the development of cellular therapies nowadays enter a new era in which the industry may take cell manufacturing activities away from academic facilities, using complex procedures for cell engineering and large-scale production at centralized facilities. Recent changes in regulations push towards this historical transition; however, a pragmatic analysis of met and unmet clinical needs, as well as of specific procedural and organizational aspects that govern human cell procurement and processing suggest that a significant role will remain for academic facilities in the near future. Taking advantage of the large experience in the field of haematopoietic stem cell transplantation, we here review actual and potential consequences of regulatory changes in these medical practices, with a focus on the unique system for quality management that has been established in Europe and the United States of America to address specific risks associated with these therapeutic procedures