Clinical relevance of reports on early access programs for checkpoint inhibitors in cancer patients: a French retrospective nationwide cohort study.

Background: To accelerate access to new drugs, France operated an early access program known as Temporary Authorizations for Use (ATUs) until 2021. We analyzed clinical reports submitted under ATUs for immune checkpoint inhibitors (ICIs) and assessed their clinical relevance regarding the approval of ICIs in oncology.

Methods: We included all ICIs granted an ATU by the French drug safety agency, Agence nationale de sécurité du médicament et des produits de santé (ANSM; French National Agency for the Safety of Medicines and Health Products), for patients with cancer between 1 January 2010 and 31 December 2020.

Trastuzumab Deruxtecan in Human Epidermal Growth Factor Receptor 2-Positive Metastatic Gastric Cancer in a Real-World Setting: A Nationwide Cohort Study.

Introduction: Trastuzumab deruxtecan (T-DXd) has been approved for human epidermal growth factor receptor 2-positive locally advanced or metastatic gastric and gastroesophageal junction (HER2+ mG/GEJ) cancer since July 2022 in France, through an accelerated approval. The aim of this study was to evaluate its real-world use.

Methods: We characterized T-DXd users treated for HER2+ mG/GEJ cancer using data from the French National Health Insurance database.

Prevention and management of health products shortages by the French national agency (ANSM), 10 years of experience.

Shortages of drugs and medical devices have tended to increase in France and worldwide, with consequences for patients and healthcare professionals. Preventing shortages of health products has become a priority for regulatory authorities, including the French National Agency for Medicines and Health Products Safety (ANSM). To highlight perspectives for a better prevention, we described and analyzed the management of shortages in the availability of health products in France over the last 10 years.

Access to innovation through clinical trials and the national early access program for patients with lung cancer in France: focus on atezolizumab and durvalumab.

Purpose: Tumor genomic profiling and PD-L1 testing mean lung cancer management can be tackled through a personalized approach. Targeted therapies and immunotherapy are necessary to improve survival and preserve the patients' quality of life. Early access to innovation before marketing authorization (MA) is possible in France through clinical trials and an early-access program called a Temporary Authorization for Use (ATU), which is a unique regulatory system in Europe.

[Early access to innovative drugs, ethical impacts].

EARLY ACCESS TO INNOVATIVE DRUGS, ETHICAL IMPACTS In France, the average time between marketing authorisation and patient access to drugs is 530 days, this period takes into account the period of price negotiation and reimbursement specific to countries that provide medical coverage to the entire population. Regarding innovation, since 1994, the introduction of the ATU system has made it possible to reduce this delay. The new system set up in July 2021 includes the ANSM and HAS with the objective of linking the access decision and the reimbursement decision of the innovation.