Nermina Ferizovic | Health Economics Expert

For my current blog post, I review a recently published systematic literature review which considered the published literature on the challenges and opportunities surrounding the use of managed entry agreements (MEA) for advanced therapy medicinal products (ATMPs) – found at the link here: https://www.sciencedirect.com/science/article/pii/S0149291824003679. 

This research takes a logical approach to understanding how payment methods, specifically MEA, can support the reimbursement of ATMPs. That is, this study seeks to understand whether there are any ATMP-specific challenges (and/or opportunities) that may impede the use of MEAs in the reimbursement of ATMPs. To this end, a comprehensive literature search including snowballing was conducted in most main databases, however, it would have been helpful were a justification as to why databases such as Embase and EconLit were excluded had been provided. Of note, only studies explicitly mentioning ATMPs were included meaning that fair conclusions on the counterfactual might be harder to argue (i.e., the study argues there are no ATMP-specific challenges of MEAs but did not identify studies looking at challenges of MEAs in general before narrowing in on ATMPs specifically). 

The findings from the 42 included studies were thoroughly reviewed and thematically categorised into the following themes: evidence generation and data management, financial and reimbursement, administration and resources, negotiation, and governance, law and regulation. This means that all the challenges and opportunities were centred around these five themes. Findings are discussed for MEAs in general using these themes and then specifically for outcomes-based agreements (OBA) and financial-based agreements (FBA) as most of the findings were specific to type of MEA, as would be expected. Asymmetric information and lack of transparency, specifically, were found to be key drivers hampering implementation of MEAs with legal factors exacerbating these. However, in light of the overarching aim of the study – which was to understand how MEAs can be used to support the reimbursement of MEAs by understanding the specific challenges/opportunities as they relate to ATMPs – the rest of the blog will focus on this. 

The study concludes that there are no ATMP-specific challenges or opportunities impeding implementation of MEAs for these interventions (see point above regarding the counterfactual) which is broadly consistent with other similar studies that have explored whether ATMPs require any technology-specific considerations to be put in place to improve patient access to these therapies. The study argues that the ‘intrinsic nature of ATMPs’ exacerbates the challenges. A definition of ‘intrinsic’ is not provided but it is likely a stretch to argue that ‘biological knowledge further complicates endpoint identification’ is an example of an element which is ‘intrinsic’ to ATMPs, for instance. Presumably, this is intrinsic to the disease in question and not ATMPs. Regardless, it is generally thought that the ‘intrinsic nature of ATMPs’ does indeed impede reimbursement of these therapies but that current methods used for decision-making/reimbursement are suitable. 

OBAs were highlighted as troublesome for payers not least due to their data demands which place large – and expensive – administrative burdens on participating entities. FBAs are preferred but also pose challenges including administrative and financial risk management.   

Overall, this is a thorough study which adds to the general understanding that current reimbursement methods do not need to be made specific for ATMPs as all challenges/opportunities of MEA implementation are consistent with non-ATMP therapies (again, see above point). However, there are some timely general policy discussions. Could an OBA ever be deemed suitable for an ATMP – or other interventions – given their inherent challenges? Should the focus be on developing FBAs further to foster patient access as these seem to be preferred? It will be interesting to see how this review contributes to subsequent research by the authors and how it may impact on policy.