Performance-based managed entry agreements

Managed entry agreements (MEA) are arrangements that address uncertainty about the performance of technologies (mainly medicines) and simultaneously manage their adoption in order to maximize their effective use, or limit their budget impact. Basically, MEAs can be understood as a tool that gives patients an access to new medicines, while the risk of uncertain effectiveness is shared between healthcare authorities and the producer. Even if financial MEAs have become quite common, the use of performance-based MEAs remains limited.

There are generally two types of MEAs: financial and performance-based. In both types, the agreements can be further distinguished according to whether the evaluation takes place at the level of one patient or the treated population. Economic agreements can include confidential agreements on discounts or prices depending on the volume and agreements on financial caps. This also includes agreements on free initial treatment with subsequent funding, if successful after evaluating the aggregated data. The last-mentioned type of agreements may not include an element of performance monitoring

A 2019 OECD survey by Wenzl and Chapman indicated that MEAs were used in at least 28 of 41 countries that are members of the OECD and/or the European Union. Beyond medicines, MEAs have also been used for various types of non-pharmaceutical health technologies including medical devices and diagnostic or surgical procedures. There is a wide range of types of MEA used across countries, as well as a variety of different names given to such arrangements (for example deeds, special pricing arrangements or risk-sharing agreements in Australia, conventions in Belgium, or patient access schemes in the United Kingdom). Payers employ MEAs to achieve a variety of goals. However, financial objectives, such as limiting budget impact and achieving a desired level of cost-effectiveness predominate.

It is the last point that leads to an interesting consideration. Could well-defined performance parameter, which determines the payment of available innovation, help to motivate doctors and patients to chieve results? The idea, that this leads to a better use of resources, and a personalization of the selection of patients after the diagnostic is in place, is appropriate. However, there is not much evidence to support this hypothesis. However, with the increasing number of highly personalized and expensive drugs with often high subjective variability in efficacy (which is the essence of personalized treatment), this method of combined reimbursement and risk-sharing appears a fair one.

The content of a performance-based MEA is often confidential, including information on health outcome measures used to evaluate the product performance. Generally, there is still limited experience with performance-based MEA, as they often face technical challenges. Existing hospital information systems often do not provide sufficient flexibility to establish an observation infrastructure available to payer. National registries may be rigid in their structure, as to the new drug to be observed in each performance endpoint. In addition, literature mentions challenges of MEAs, such as significant administrative burden and costs for providers, companies, or payers involved in executing the agreement and collecting and analysing performance data. Payers may also see it difficult to reduce prices, recouping payments already made to companies, or de-list treatments from coverage, if the data analysed under MEA show that the treatment is less effective than expected. Defining and obtaining data informative about relevant health outcomes and surrogate endpoints or biomarkers requires a deep knowledge of the real-world performance of the drug, which may be missing at the time of planning.

In our team, we address the above-mentioned issues and we also examines, whether there is a sufficient legal framework to implement the above presented performance-based agreements.