Access to Oncology Drugs: Insights From France's Early Access Reform

Speaker(s)

Borget I1, Bayle A2, Marabelle A3, Massard C3, Martin T4
1Institut Gustave Roussy and Paris Saclay University, ORSAY, 91, France, 2Institut Gustave Roussy and Paris Saclay University, Villejuif, Ile de France, France, 3Gustave Roussy, VILLEJUIF, 94, France, 4Faculty of Pharmacy - GRADES Health Economics Department / Hospital Pharmacy, Paris-Saclay University / AP-HP (Hôpital Européen Georges Pompidou), Orsay, France,, Orsay, France

Presentation Documents

OBJECTIVES: In July 2021, France implemented an exhaustive Early Access (EA) reform, aiming to simplify procedures and accelerate access to innovative drugs. This study examined characteristics of oncology (including hematology) drug approvals through AE process and the evolution in number of patients treated by cancer type.

METHODS: All oncology applications submitted to the Health Authority (HAS) since the EA reform (from July 1, 2021) until December 31, 2023 were identified, reviewing approval decisions, timelines, cancer localization, and treatment line. A longitudinal retrospective study on patients treated with an EA oncology drug between January 1, 2019, and December 31, 2023, was also performed using the French Nationwide Claims Database (SNDS) to assess the impact of the reform in the number of patients treated by cancer type.

RESULTS: Among the 106 EA applications related on oncological indications, 88 (83%) were approved (including renewals) and 18 were refused, with an average evaluation time of 71 days. For the 53 new indications (excluding renewals), they predominantly concerned monoclonal antibodies or antibody drug conjugates (49%) and CAR-T cell therapies (17%). Most approved indications (N= 35, 66%), concerned solid tumor mainly in digestive (23%), urological (23%), and breast cancers (20%), whereas other authorizations (N=18, 34%) concerned onco-hematology. In the SNDS data, the number of cancer patients treated with an EA drug strongly increased from 2019 to 2022 (respectively 3,137 to 18,341, +484%), and become stable in 2023. In 2023, it mainly concerned patients with breast cancer (48%), stomach cancer (14%) and multiple myeloma (7%), in 2nd line of metastatic treatment or more (50% of patients) or in adjuvant setting (25%).

CONCLUSIONS: The French EA reform has expedited approval across various indications, facilitating access to innovative drugs like monoclonal antibodies or antibody-drug conjugates, notably in solid oncology for metastatic indications, even if an evaluation with a longer time horizon is necessary

Code

HPR223

Topic

Health Policy & Regulatory, Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes, Reimbursement & Access Policy

Disease

Oncology