CREATIC ATMP newsletter

Managed entry agreements (MEA) as pathway to access: a win for all?

Managed entry agreements (MEA) such as performance-based payment schemes (P4P) are widely perceived to be one of the potential solutions to the priority setting problem of ATMPs: little evidence and a high upfront price. But, this solution may also bring about new problems to be solved by frontline professionals.

By Anna Brueckner Johansen, PRIME – Prioritization in Precision Medicine

When the gene therapy Luxturna was approved by the FDA in 2017 as the first gene therapy to treat an inherited eye disease, candidates for this long-awaited therapy were excited. But for patients in Denmark, hope soured when it was rejected due to what the health technology assessment (HTA) agency in Denmark — the Danish Medicines Council — called an "unreasonably high" price. After continuing negotiations, an agreement about a performance-based payment scheme (P4P) concluded by Novartis and the Danish procurement organization for public medicines (Amgros) eventually enabled patient access. In the P4P-model, payment for the one-time treatment is split up into several instalments, contingent on the treatment outcomes of individual patients. If the predefined outcomes are not met, payment stops. The five-year duration of the agreement served to reduce uncertainty about long‑term effects. In this way, the P4P temporally enlarges the moment that matters – from treatment administration to years of follow-up. The Danish state and Novartis thus shared the risk associated with the treatment's actual effect through a flexibility of the final price, what has popularly been called ‘no cure, no pay'.

A central argument made by stakeholders as to why the P4P was successfully agreed was the ability to settle on an outcome measure "that was completely objective, not open for interpretation or disagreement," (ISPOR, 2022)- Merely the "click of a button". However, my observations of the annual, clinical tests of patients used to evaluate the treatment effects of Luxturna reveals that it takes much more than the click of a button to establish these outcome measures: it is tedious work. Not only are the infrastructuring efforts of putting this MEA in place huge. It also requires careful labor by frontline professionals, patients and caregivers as they go through hours and hours of exhausting and dull work to produce data for the P4P, even after treatment administration. If performance-based payment agreements are to constitute a more widespread solution to the clinical implementation of ATMPs, it is important to consider not only the alignment of price and effect, but also the costs of making such an agreement work in practice. Notably, human resources, infrastructure, work and logistics needed for the establishment and implementation of P4P schemes, data and money flows, as well as annual testing in the clinic of patients both laborious to patients themselves and health care staff are to be considered, as they may burden already resource constrained departments.

Moreover, a P4P may benefit industry more than payers. Instead of providing a flat rebate, which can leak and affect price negotiations in other countries, a P4P seems like an innovative solution while concealing and obscuring price as an issue, shifting focus to whether the product has the effect it promises to have. As prices for new ATMPs continue to rise, we may ask ourselves whether such a shift of focus is the solution to the problem of little evidence and high pricing. Instead, we could raise the question whether the logic of pharmaceutical businesses is really geared to a market of one-time treatments. After all, as critical pharmaceutical studies scholar, Victor Roy notes, "cures carry the possibility of eliminating the very potential for continual revenue growth on which their value as assets in financial markets rely" (Roy 2020:99).

Since the payment agreement was settled with Luxturna, another P4P for the gene therapy, Hemgenix, was launched in 2024. Time will tell whether these agreements are a viable solution of the priority setting problem of precision medicine or merely a method of turning the conversation away from the problem of ATMPs high prices to their potential future effects.

This opinion is based on ethnographic fieldwork conducted in connection to the PhD project "Valuing visibility: Rationing dilemmas in rare eye disease" (ongoing, link: https://www.vive.dk/en/project-descriptions/prime/ ), which explores how different notions of value are enacted among patients and relatives, health care professionals, regulators, patient organizations, researchers, and manufacturers when a new gene therapy enters the Danish welfare state.

Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them.