Abstract 1549P
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.
Resources from the same session
1199P - Developing and systematically validating homologous recombination repair gene detection method based on next-generation sequencing
Presenter: Yi Sun
Session: Poster session 10
1200P - Investigation of multiphoton microscopy as an innovative tool for intraoperative section-free histologic investigations in just a few minutes
Presenter: Martí Homs Soler
Session: Poster session 10
1201P - Novel deep learning model and validation of whole slide images in lung cancer diagnosis
Presenter: Alhassan Ahmed
Session: Poster session 10
Resources:
Abstract
1202P - A deep learning approach using routine pathology images to guide precision medicine in metastatic CRC
Presenter: Chaitanya Parmar
Session: Poster session 10
1203P - Analytical evaluation of whole genome sequencing for acute myeloid leukemia
Presenter: Guidantonio Malagoli Tagliazucchi
Session: Poster session 10
1204P - Real-world utility of whole genome sequencing for patients with cancer: Evaluation of a regional implementation of the 100,000 genomes project
Presenter: Helen Robbins
Session: Poster session 10
1205P - A retrospective machine learning-based analysis of nationwide cancer CGP data across cancer types to identify features associated with recommendation of mutation-based therapy
Presenter: Hiroaki Ikushima
Session: Poster session 10
1478P - Dual single-nucleotide polymorphism biomarker combination to select opioid for cancer pain management
Presenter: Yoshihiko Fujita
Session: Poster session 10
1479P - Use of rescue opioids and pain control after ketamine initiation in refractory cancer pain: A multicentric observational study
Presenter: Pablo Gallardo Melo
Session: Poster session 10
1480P - Long term therapy with denosumab and zoledronic acid: A comparative real-world retrospective observational study on skeletal-related events and pain in patients with metastatic breast cancer
Presenter: Giacomo Massa
Session: Poster session 10