Nermina Ferizovic | Health Economics Expert

For my latest blog post, I briefly review a recently published study which provides a policy framework for the implementation of outcomes-based agreements for innovative therapies: https://www.valueinhealthjournal.com/article/S1098-3015(24)02788-8/fulltext?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1098301524027888%3Fshowall%3Dtrue

This recently published study explores the use of outcomes-based agreements in health technology assessment (HTA) as a way to improve patient access to innovative health interventions, including advanced therapy medicinal products (ATMPs). The premise of the study is that gaining access to innovative therapies presents many challenges with one of the potential solutions being outcomes-based agreements. As given by the authors ‘outcomes-based agreements are one type of managed entry agreements that link reimbursement decisions to health and cost outcomes, with the aim of reducing the risk to payers’. As discussed in previous blog posts, ATMPs present healthcare decision makers with large uncertainty due to several factors including a lack of long-term data on safety and efficacy as well as a lack of comparative effectiveness data combined with high prices.

Some of these challenges are highlighted and ranked by the authors as part of the pre-workshop survey conducted in this study. The authors included this pre-workshop survey to ‘enable HTA and payer representatives to consider some of the key challenges of assessment and reimbursements of ATMPs’ ahead of two in-depth workshop discussions ultimately aiming to provide recommendations on how outcomes-based agreements can be implemented by HTA bodies to support reimbursement, thereby meeting the challenges of reimbursement. It is important to note that the National Institute of Health and Care Excellence led this study. The ultimate output of the study is a policy framework for the implementation of outcomes-based agreements in HTA in Europe. 

The study provides some helpful insight into the implementation of outcomes-based agreements to foster patient access to ATMPs in Europe. Although the study has potential to prove useful in the future, one of the key drawbacks of the study questions the applicability of the findings to ATMP reimbursement decision making. For instance, the lack of a representative sample of stakeholders in the workshops limits generalisability. An additional point is that there is no precedent for outcomes-based agreements having been implemented for ATMPs in the UK; however, the authors highlight that outcomes-based agreements may be a suitable solution for risk mitigation in decision making. However, the acknowledgement that outcomes-based agreements ‘should be considered only if a simpler solution cannot be reached’ reduces the applicability of the study’s policy framework further. Indeed, no justification is provided as to why outcomes-based agreements are the focus of this study given their complexity and unlikelihood of implementation by HTA bodies including NICE, not least due to the health services adversity to such arrangements. 

Overall, this is a helpful study which provides a policy framework to potentially support reimbursement of ATMPs, however, its findings arguably lack applicability.