Comparative study on anticancer drug access times between FDA, EMA and the French temporary authorisation for use program over 13 years

Introduction: The cancer incidence continues to rise worldwide. Medical innova-tion has a major impact on patient survival, but within drug development, it can take more than 10 years to obtain marketing authorisation (MA). The time required for access to ther-apeutic innovation remains critical, so France has developed a specific expanded access pro-gram named ATU, which allows the administration of drugs before the European Medicines Agency (EMA) approval. The purpose of this study is to put in perspective the average time to access antineoplastic drugs worldwide, taking into account ATU, US Food and Drug Administration (FDA) and EMA approvals. Methods: The ATU system allows the use of a medicine before its MA, under exceptional con-ditions. All antineoplastic drugs in oncology that have benefited from the ATU system are analysed in terms of tumour site, biomarkers and number of patients who have access to the drug. Results: Between 1st January 2007 and 31st December 2019, 36 of 64 drugs ( 56.2%) that received MA in oncology were assigned an ATU, to the benefit of 16,927 patients. Thanks to the ATU, 25 of 36 drugs (69.4%) were made available early, on average 203 d (95% CI, 76-330) before FDA approval and on average 428 d (95% CI, 272-583) before EMA approval. Only three of 36 drugs were approved by the EMA before the FDA, and the average time lapse between European MA and FDA approval for these 36 drugs was 216 d (95% CI, 140-293). Conclusion: This article demonstrates that the ATU system allows patients to benefit from therapeutic innovations before MA in Europe and USA, with full coverage by the healthcare system. (c) 2021 The Author(s). Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

Automated summary

This study examines the use of the ATU system in oncology, showing that 56.2% of drugs that received European MA were granted ATU, benefiting 16,927 patients. Through ATU, 69.4% of the drugs were made available early, an average of 203 days before FDA approval and 428 days before EMA approval.