ISPOR 2020: do managed entry agreements for ATMPs exist in Germany? Curative and genetic therapies abstract highlights
What research was due to have been presented at the 2020 ISPOR Annual Meeting (18–20 May)? We summarize our abstract highlights, available to view online, focusing on curative, regenerative and genetic therapies.
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Here, we share our abstract highlights, due to have been presented at the annual meeting, focusing on curative, regenerative and genetic therapies! All abstracts are available to view online here>>
Do managed entry agreements for ATMPs exist in Germany?
Achieving reimbursement for cell and gene therapies: what evidence is considered?
How are clinical outcome assessments utilized in gene therapy development?
Traditionally, managed entry agreements (MEAs) are defined as arrangements between manufacturers and payers for enabling access to a health technology – such as advanced therapies – under specific conditions. A recent working paper by the Organisation for Economic Co-operation and Development (Paris, France) observed no MEAs that affect coverage to exist in Germany. In this study, by employing a more comprehensive definition of MEAs, investigators from Ecker + Ecker GmbH (Hamburg, Germany) evaluate which MEAs exist in Germany and what implications these may have on understanding of MEAs, focusing on EMA-approved advanced therapy medicinal products (ATMPs).
For nine identified EMA-approved ATMPs available in Germany, researchers also identified MEAs existing for five of these, with risk–share arrangements with individual sick funds existing for three of these and performance-based agreements linked to duration of treatment or need for re-intervention existing for the other two products.
In the abstract, the authors concluded: “For this sample more than 50% of all ATMPs have MEA in Germany, each linked to product performance.” This may indicate that the term ‘MEA has broader meaning than is traditionally defined.
The potentially curative benefits of novel cell and gene therapies make achieving reimbursement and access for these imperative; however, these are associated with significant challenges, for example, due to orphan populations, poor long-term data availability and large one-time expenses, which can exceed US$100,000. In this study, researchers from Purple Squirrel Economics (QC, Canada and NY, USA) sought to determine the quality of evidence reviewed by health technology assessment agencies during the reimbursement decision making process for cell and gene therapies.
Investigators observed that less than 1% of all submissions to health technology assessment bodies were for cell and gene therapies, indicating that this treatment class is relatively small. Agencies accepted data from Phase II trials – considered lower-level data – in support of products when considering reimbursement, as well as generally high incremental cost–effectiveness ratios.
Researchers concluded: “Transparency regarding risk-sharing agreements and price reductions would help manufacturers assess economic viability and potentially spur greater investment in cell and gene therapies.”
With gene therapies offering the potential to transform the lives of patients, it is essential that appropriate assessment methods are used throughout their development, in order to accurately capture patient experience and the benefits of these therapies. In this study, researchers from Adelphi Values (MA, USA) evaluated the extent to which clinical outcome assessments are used in the development and obtainment of regulatory approval for gene therapies approved by the US FDA and EMA.
Investigators observed that, whilst only a limited number of gene therapies have, to date, been approved by the FDA and EMA, clinical outcome assessments were used in drug development trials and in support of label claims for two FDA-approved gene therapy products and during drug development for two out of four EMA-approved products. The authors conclude that future research should continue to explore ways for clinical outcome assessments to capture the treatment benefit for these potentially transformative therapies.