New Framework for Early Access to Drugs in France: Feedback After a One-Year Use in University Public Hospitals of Paris

Speaker(s)

Denoual C1, Siorat V2, Fusier I3, Mougenot P2, Paubel P4
1General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Orsay, 91, France, 2General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France, 3General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), PARIS, France, 4General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP) ; Law and Health Economics Department, Faculty of Pharmacy & Health Law Institute (INSERM UMR S1145) University of Paris Cité, Paris, Paris, France

Presentation Documents

OBJECTIVES: France has been a pioneer in setting up regulations allowing patients to be offered early access to drugs with a potential benefit. A new framework for early access to drugs was implemented in France in July 2021. The aim of this study is to review the use of early access drugs (EAD) at Public Hospitals of Paris (AP-HP), in terms of consumption, types of hospitals, and therapeutic areas involved.

METHODS: PMSI hospital database (French medical information system program) was used to collect data, over the July 2021 - June 2022 period, on both the EAD use (quantities, expenditures and Anatomical Therapeutic Chemical Classification (ATC)) and consumers (hospitals and hospital wards).

RESULTS: During the study period, 53 medicines of EAD were used in AP-HP, representing €34,115 million of expenditures, 52 % of which were due to a gene therapy drug and a CAR-T cell.

Moreover, 21 out of 37 hospitals in AP-HP used EAD. All types of hospitals are represented, 19 general hospitals (medicine-surgery-obstetrics wards), 3 paediatric hospitals, and 2 follow-up care and rehabilitation hospitals. Intensive care, nephrology, and oncology-haematology departments were the main users of EAD, accounting for 95 % of expenditures.

The three most represented ATC classes are M - Musculo-skeletal system (35 % of expenditures and <1 % quantities), L - Antineoplastic and immunomodulating agents (31 %, 20 %) and A - Alimentary tract and metabolism (24 %, 60 %).

CONCLUSIONS: EAD are widely used, in nearly two-thirds of AP-HP hospitals. The three main therapeutics areas involved (90 % of expenditures for M, L and A classes) are representative of EAD used in the three main departments mentioned. EAD represent significant expenditures, but low quantities, due to the high cost of innovative drugs.

Maintenance of price and innovative nature will have to be confirmed after the French Health-Technology-Assessment process.

Code

HPR194

Topic

Health Policy & Regulatory

Topic Subcategory

Public Spending & National Health Expenditures, Reimbursement & Access Policy

Disease

Drugs, Rare & Orphan Diseases