Aurélie Mahalatchimy

The emerging landscape of reimbursement of regenerative medicine products in the UK: publications, policies and politics

AIM This paper aims to map the trends and analyze key institutional dynamics that constitute the policies for reimbursement of regenerative medicine (RM), especially in the UK. MATERIALS & METHODS Two quantitative publications studies using Google Scholar and a qualitative study based on a larger study of 43 semi-structured interviews. RESULTS Reimbursement has been a growing topic of publications specific to RM and independent from orphan drugs. Risk-sharing schemes receive attention among others for dealing with RM reimbursement. Trade organizations have been especially involved on RM reimbursement issues and have proposed solutions. CONCLUSION The policy and institutional landscape of reimbursement studies in RM is a highly variegated and conflictual one and in its infancy.

The impact of European embryonic stem cell patent decisions on research strategies

Recent restrictions on the patentability of human embryonic stem cell inventions in Europe necessitate a shift in R&D strategies.

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.

EU law and policy on new health technologies

The evocation of « new health technologies » takes us straight away into a strange world where reality and fantasies are intertwined, in which connected wristbands can be found next to gene therapy medicinal products, where bionic prosthetics herald 3D-printed prosthetics or in which human eggs are activated through parthenogenesis while human genome sequencing can be achieved within a few minutes. In order to review the state of European Union law on new health technologies and its prospects, we must first try to define these technologies and discuss a number of preliminary points regarding EU law which can apply before presenting the structure of this chapter.

The politics of valuation and payment for regenerative medicine products in the UK

The field of regenerative medicine (RM) faces many challenges, including funding. Framing the analysis in terms of institutional politics, valuation studies and “technologies of knowledge”, the paper highlights growing debates about payment for RM in the UK, setting this alongside escalating policy debates about “value”. We draw on interviews and publicly available material to identify the interacting and conflicting positions of institutional stakeholders. It is concluded that while there is some common ground between institutional stakeholders such as industry and health system gatekeepers, there is significant conflict about reward systems, technology assessment methodologies and payment scenarios; a range of mostly conditional payment schemes and non-mainstream routes are being experimented with. We argue that current developments highlight a fundamental conflict between a concern for the societal value of medical technologies in a resource-limited system and a concern for engineering new reward and payment models to accommodate RM innovations.

Producing ‘Human Elements Based Medical Technologies’ in Biotech Companies: Some Ethical and Organisational Ingredients for Innovative Cooking

This article is based on the findings of an EU-funded qualitative research project, entitled ‘From GMP to GBP: Fostering good bioethics practices [GBP] among the European biotechnology industry’, which seeks to improve the understanding of bioethical issues through the observation of the daily practices in European biotechnology companies and proposes a methodology approaching ethical issues. The comparative study was carried out in biotech companies in France, Italy, Sweden, Hungary and Belgium which develop a wide range of new technologies, all of them involving human materials or where human subjects participate (in clinical trials). Based on our findings in these local settings, we suggest that the notion of bioethics and the way its production is theorised need to be re-conceptualised. We argue that material practices and moral statements are intermingled in inextricable ways that render the formation of bioethical concerns fully dependent on the organisational landscape in which it is embedded. More precisely, the here presented co-production model of moral statements and organisational practices presents a set of common factors that influence how bioethical discourses are shaped, despite the heterogeneity of their epistemic cultures. For example, the procedural design of cell-based-products, the modes of collecting and storing biological specimen, the relationship between patients and companies and technological transfers to emerging countries are defining components that contribute to the shaping process of bioethical concerns. Thus, the path dependency of bioethical concerns relies on an already existing, specific infrastructure and existing relationships within and outside a company rather than on external judgement subsequently applied to its objects, or a collection of processes of reasoning coming from external institutions.