Currently, there is scant evidence of policies that adequately outline the process for therapies and technologies in the regenerative medicine (RM) field, known in the EU as Advanced Therapy Medicinal Products (ATMPs). Areas such as payment mechanisms, pricing and reimbursement schemes still remain largely elusive. Clear policies are pertinent due to ATMPs such as KYMRIAH™ and YESCARTA® that have been approved, but how they and future ATMPs will be sustainably paid for remains unclear for many healthcare systems globally. Also, their long-term effects are not yet known, and social, ethical and legal issues have not always been adequately considered.
The scarcity in defined reimbursement and adoption policies for ATMPs prompted the development of an international workshop on the ‘Challenges in the Adoption of Regenerative Medicine Therapies (CHART)’. Co-hosted by Medicine by Design (a regenerative medicine initiative at the University of Toronto and funded by the Canada First Research Excellence Fund), CCRM, Toronto Health Economics and Technology Assessment (THETA) Collaborative, and the Centre for Biological Engineering at Loughborough University UK, CHART focused on the post-market approval challenges associated with economic evaluation, reimbursement and adoption of regenerative medicine products. The challenges were explored in the context of the Canadian and UK healthcare systems as provision of health in both countries is largely via publicly funded systems. The workshop was attended by 37 experts from institutions and companies from Canada, the UK, and the USA that included representatives of the Ontario government, National Institute for Health and Care Excellence (NICE), Canadian Agency for Drugs and Technologies in Health (CADTH), Takeda Canada, Gilead Sciences Canada, Inc., Office of Health Economics (OHE), Centre for Health Economics (CHE), University of York, and Oxford Academic Health Science Network (OAHSN) amongst others.
Proceedings from the workshop demonstrated that challenges regarding ATMP adoption are not unique to the regenerative medicine field. (1) Current methods of evaluation are applicable to ATMPs, but the biggest challenge for these novel therapies is providing evidence that reduces the uncertainty of their long-term effects. (2) The generation of evidence of clinical effectiveness needs to be improved by ensuring clinical trial designs employ robust controls and increase their sample sizes while collecting data in the relevant setting. (3) Incentives for real-world evidence capture should be established to ensure that data are gathered and can be used to further evaluate the therapies for long-term clinical effectiveness and adverse effects. (4) The implementation of ATMPs will require concerted efforts from multiple stakeholders to ensure that the adoption pathway for ATMPs is efficient, effective and aligned with social values. (5) There needs to be greater interaction with policy makers, as political will is essential for research to progress into meaningful efforts. (6) There is a need to improve patient management and data management to aid in evidence generation and facilitate outcome-based payment mechanisms. (7) Payment mechanisms remain a challenge; the sustainability of current payment methods for expensive therapies will need to be evaluated, prior to the approval of more ATMPs. The next steps outlined from the workshop included: targeted policy maker engagement, relevant stakeholder engagement, addressing the evidence generation issues, and understanding future payment system mechanisms.