Regenerative medicine and its development and implementation: an analysis of emergent value systems and health service readiness

There has been considerable interest developing over recent years in regard to the therapies that have become and may become available based on what is known as 'regenerative medicine'. This is an important emerging field in clinical research, and one that has attracted considerable investment in the UK and internationally. The main areas of development include gene therapy medicinal products, cell therapy medicinal products and tissue engineered products. Its importance lies in its promise to transform healthcare by offering curative, not palliative, therapeutics through the regeneration of tissue within the body. This in itself may reshape healthcare provision though it is equally important to understand how the existing healthcare system will influence the pathways through which the field develops. In addition, the field poses new challenges for regulators because of the ways in which the 'mode of action' of tissue/cell therapies is quite different from the most common innovation the regulator has to understand and approve (or not), the new pharmaceutical drug. In short, the project seeks to understand the complex relationship between these clinical and wider social, organisational, regulatory and economic issues.

It does so through a series of Workpackages (WP). The first maps the field in the UK (within its wider international - principally EU/US context), especially in regard to current patterns of investment, clinical trials, product development, the intellectual property strategies of companies and the specific regulatory processes shaping these. The second WP examines the role of a range of intermediary agencies, such as the National Institute for Health and Clinical Excellence (NICE), patient groups and health insurance companies, in determining what can be called 'healthcare readiness' for the field, that is, how the field aligns with and can be embedded in existing practice and how far changes need to be made. The third WP involves detailed case studies of firms and hospital sites currently engaged in developing/using cell therapies chosen to reflect a) different regulatory classifications by product area b) the main areas of current activity by disease that are closely linked, and the sites that we would access to track product development. We will use a methodology called Analysis of Life Science Innovation Systems (ALSIS) modelling which captures different types of business models, value-chains and their relationship to the wider innovation environment. This allows us to examine the full range of activities required to bring a product from conception to market or to direct application in a hospital within the wider economic, regulatory, societal and political contexts that play a key role in determining the scale and scope of the innovation.

In the fourth WP the ALSIS method which allows us to map and explain innovation paths will be integrated with and triangulated against our modelling of the potential future of the field using horizon scanning techniques developed by one of the co-applicants based at the National Institute of Health Research Horizon Scanning Centre to interrogate likely diffusion paths within health care, and the expectations surrounding regenerative medicine among public and private actors (including government and private agencies).

Throughout the project we will work with collaborating parties, including the participating companies, hospitals, regulatory and reimbursement agencies (notably the Medicines and Healthcare products Regulatory Agency and NICE), patient groups, Research Councils UK, the Technology Strategy Board's Cell Therapy Catapult, the NIHR's i4i Secretariat, the Science and Technology Select Committee and the All-Party Parliamentary Group for Social Science and Policy.

The project will make an important contribution towards understanding the likely pathways that regenerative medicine takes within the UK and other health care systems, by identifying distinct commercial and non-commercial (e.g. hospital) routes to its application, including in particular cell therapies but in other domains too. It will also benefit regulatory and HTA agencies for their own formal regulatory models as well as more informal deliberations with those in the field seeking advice about regulation, product development and uptake within the NHS. Bio-clinical researchers participating in the regional stem cell and tissue engineering networks and patient groups keen to understand the likely availability of therapies will have a much clearer understanding of the trends ahead through the horizon-scanning element of the project. The research will clarify likely scenarios of adoption and diffusion for NHS policymakers and commissioners in different clinical sectors. Our case studies' descriptions and analysis will provide examples of RM development models and their challenges that will be of direct value to the Technology Strategy Board's Catapult Centre, to companies and public researchers developing their own innovation strategies. Patient groups likely to be most interested in the outcome of the research are those associated with the principal areas of inquiry where trialling and product development is underway, such as in musculoskeletal, stroke, and cardiovascular areas of research.

These outcomes of the research address the needs identified in the UK government's very recent review of its current investment in the life-sciences, most importantly to understand investment trends and the reimbursement landscape and to use social science to identify the value and likely market penetration of regenerative medical products. Moreover, our work on comparing hospital-based innovation via the 'exemption' or 'specials' route with the product-driven trials process addresses the government's desire to show how the NHS and commercial activities might be more effectively integrated. NICE is charged with the assessment of new products, requiring information about comparators, cost-consequence analysis, impact on patient pathways, and workflows in the NHS. The results of our work feed directly into these concerns and we will be able to help NICE, the new NHS Commissioning Board and the recently established Emerging Science and Bioethics Advisory Committee (ESBAC - Tait is leading ESBACs Regulatory Issues Working Group) develop new policy for the field, including informing the possible establishment of a national commissioning agency for RM.

The research also addresses a major issue identified by the Ministerial Industry Strategy Group (MISG) in a report that the applicants helped to prepare on the regulation and development of regenerative medicine, which has been passed to the House of Lords Inquiry due to report in 2013. There it is argued that social science research is needed to understand the 'institutional readiness' of the healthcare system, to complement the 'technological readiness' which the major science funding agencies are working to secure.

Finally, the research will contribute to debates at a European level about core problems of the RM market that have been raised in the European Medicines Agency and the European Parliament and which need to be addressed over the next few years as products come to regulators for market approval: these include the form that clinical trials and postmarket surveillance will take, clarification of the use of the hospital exemption, orphan medicine and other routes to therapy to ensure an appropriate balance is struck between commercial incentivisation and non-commercial development, and the pattern of reimbursement regimes and whether new models are needed to foster the development of the field.