Advanced Therapy Medicinal Products (ATMPS) Market Access Challenges in France

OBJECTIVES: ATMPs are defined by European Medicines Agency (EMA) as medicines for human use that are based on genes, tissues or cells. Patient expectations are high in front of those cutting-edge therapies for high-burden diseases. ATMPs face considerable market access difficulties notably due to clinical uncertainties and affordability challenges. The objective of this retrospective review of health technology assessment (HTA) outcomes is to determine the key challenges related to the ATMPs market access in France.

METHODS: ATMPs approval data were extracted from the EMA website, reimbursement and pricing drivers were retrieved from the opinions of the French Health Authority (HAS), details on early access authorizations came from the French Medicine Agency (ANSM) and the therapies public prices were collected in the Official Journal of the French Republic if necessary. The timeframe of the study was 1st January 2017-17^th december 2021.

RESULTS: Since 2017, 12 ATMPs have been authorized in Europe. Nine ATMPs were assessed for reimbursement by the HAS among which 8 had a positive reimbursement decision: 3 are still in the market access process and are covered by the early access authorization, 4 are covered outside the diagnosis-related group and 1 was withdrawn from the market. For 4/9 ATMPs, the clinical evidence was based on phase I/II clinical trials. HAS invalidated 3/6 economic evaluations of ATMPs. ATMPs have a median price of €350K and can cost up to €2M (Zolgensma®). The target population varies between 34 and 800 patients. The average time between market authorization and price setting is 20 months.

CONCLUSIONS: Reimbursement of ATMPs achieved a high level in France despite high prices and other challenges like the generation of robust clinical evidence. The issue of sustainability for the French health insurance remains outstanding and there is still progress to be made in improving patient access time.