1549P - Accelerating access to innovative oncology drugs: Insights from France's early access reform

Background

In July 2021, France initiated a significant overhaul of the Early Access (EA) process, replacing the former Authorization for Use (ATU) system. This reform aimed to simplify procedures and expedite access to innovative drugs. This study examined the characteristics of oncology (including onco-hematology) drug approvals through the AE process and assessed the methodological quality of clinical trials supporting these requests.

Methods

All oncology applications submitted to the National Health Authority (HAS) since the EA reform (from July 1, 2021) until December 31, 2023 were identified, reviewing approval decisions, timelines, and reasons for acceptance or rejection. The therapeutic class (ATC) of drugs, cancer site and stage were identified, and the data characterizing clinical trials supporting the requests from reviews provided by HAS and clinicaltrial.gov.

Results

Out of 171 EA approvals (including renewal), 88 (51%) were granted for oncologic indications, with evaluation time by HAS of 71 days on average. Excluding renewals, 48 drugs for 53 indications in oncology received EA approval, predominantly monoclonal antibodies or antibody drug conjugates (49%) and CAR-T cell therapies (17%). Most approved indications were in solid oncology (35, 66%), mainly in digestive (23%), urological (23%), or breast cancers (20%), in metastatic indications in the first-line (31%) or second-line (23%). Onco-hematology authorizations accounted for 18 indications (34%), mostly in the third line and beyond (56%). Oncology drugs approved through the EA pathway were primarily backed by phase III trials (70%), including direct comparison (72%) and/or randomization (70%) whereas refused EA drugs were frequently supported by indirect comparison or non-comparative trials (50%). Overall survival (OS) was included as primary or secondary endpoints in 55% of approved EAs, with mature data available in 36% of trials.

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. Ensuring high-quality studies is crucial for EA success, with mature data frequently provided.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.