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Implementing Outcomes‐Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

Karen M. Facey, Jaime Espin, Emma Kent, Angèl Link, Elena Nicod, Aisling O’Leary, Entela Xoxi, Inneke van de Vijver, Anna Zaremba, Tatyana Benisheva, Andrius Vagoras, Sheela Upadhyaya
July 2021

Outcomes-based managed entry agreements (OBMEAs) have gained renewed interest as a potential solution to managing uncertainties surrounding innovative treatments for rare diseases, which have high therapeutic potential but significant uncertainties in clinical evidence. To investigate how OBMEAs are being implemented for innovative therapies, this paper reviewed their application in the case of two orphan medicines (nusinersen and tisagenlecleucel), using data from HTA/payer/academic experts in Europe, Australia and Canada. The findings demonstrate that OBMEAs could play an important role in reimbursement and treatment optimization of rare disease treatments. At the same time, due to the complexities and administrative burden of implementation, it is not recommended that such agreements should become the standard approach.

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