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Project

Framework for value driven market access of gene therapy

Drug development today is increasingly focused on the development of biological products, including advanced therapy medicinal products (ATMPs). However, market uptake of these therapies has been limited in Europe due to difficulties in obtaining reimbursement. Governments experience decision-uncertainty due to persisting uncertainties caused by shortcomings in clinical trial design and the unavailability of long-term data on efficacy and safety. Furthermore, the sustainability of the healthcare budget is threatened by having to absorb potential peaks of treatment prices of €320,000 for CAR-T cell therapies up to €1,900,000 for gene therapy treating spinal muscular atrophy. The project will contribute to developing new methods to facilitate the reimbursement of innovative therapies and subsequently aid patients in gaining access to these transformative therapies. This research project will aim to tackle the most important challenges that ATMPs face when trying to obtain market access: - Study the possibility of using new payment structures for the reimbursement of innovative therapies focusing on overcoming challenges to enable real-life implementation in the healthcare system. - Investigate how the value of these innovative therapies can be correctly evaluated by healthcare payers.

Date:1 Sep 2021 →  Today
Keywords:gene therapy, value framework, market access, payment models, patient preferences
Disciplines:Health care financing, Primary health care, Pharmaco-economics
Project type:PhD project