Definitions of payment models to address market access and affordability of innovative medicinal products.

Abstract

Innovative therapies can treat conditions and diseases that were previously considered incurable. There is a class of biopharmaceuticals, called Advanced Therapy Medicinal Products (ATMPs), that include somatic cell therapies, gene therapies and tissue-engineered products. ATMPs offer potentially life-changing cures for patients, as well as therapeutic alternatives for conditions with limited clinical options. Despite this, pricing and reimbursement authorities are confronted with extremely high prices and significant clinical uncertainty, rendering their reimbursement particularly challenging.

ATMP development is expensive due to the many years of research and development required, accompanied by a complicated production process. Manufacturers are unable to recoup production costs with lower prices due to a limited patient population, in the case of rare diseases. Manufacturers also take advantage of the higher willingness to pay for innovative therapies targeting diseases with no current treatment options. Clinical uncertainty is expected because the ability to conduct clinical trials is limited by a small patient population. ATMPs are often supported by single-arm clinical trials, and no long-term data.

In response to reimbursement issues, managed entry agreements (MEAs) have been proposed and implemented. MEAs are defined as ‘any agreement beyond a yes/no decision on reimbursement between the marketing authorization holder (hereafter called manufacturer) of a therapy and a payer’. MEAs are typically categorised either as financial-based agreements (FBAs) or outcome-based agreements (OBAs). FBAs aim to contain costs and make a product more affordable. OBAs aim to reduce uncertainties surrounding the effectiveness of a product by holding manufacturers accountable for their outcomes in the real world.

There is increased discussion in the literature surrounding MEAs, in order to facilitate the reimbursement of the growing number of ATMPs in the drug development pipeline. There are however several barriers impeding their widespread application. Among these, is the necessity of a standardised negotiation framework, which is further complicated by the lack of a consensus-based MEA taxonomy and definitions. Thus, this work aims to create a taxonomy of the payment models described in the literature, and the definitions used. This will accommodate further research, design, assessment, and implementation of these models in health care systems.

A literature review of articles published in PubMed, Medline, and Embase between 2010-2023 was conducted. Articles containing MEAs and their definitions related to ATMP reimbursement were selected. The availability of full texts was required and the articles had to be written in English. The search queries retrieved 3442 results. Removal of duplicates, followed by title and abstract screening, and full-text screening yielded a final selection of 64 articles to be included in the Microsoft Excel datasheet.

In total, 255 MEAs and definitions were identified. A comprehensive table of MEA types and their definitions was created. Categorisation of MEA types was displayed in both a clustered table, and in a unique taxonomy tree. A lack of consensus regarding MEA taxonomy and definitions is evident. A common language between payers and manufacturers is needed to facilitate effective reimbursement negotiations. To conclude, a call to action for the establishment of a standardised negotiation framework, accompanied by consensus-based MEA terminology is made.