Innovation Expanded access Early drug access Cancer FDA EMA

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

by Emmanuelle Jacqueta, Ghania Kerouani-Lafayeb, Francoise Grudeb, Sergio Goncalvesb, Annie Lorenced, Florence Turcryb, Liora Brunelb, Laetitia Belgodereb, Adrien Monardc, Gaëlle Guyaderb, Lotfi Boudalib, Nicolas Albin

Apr 7^th 2021
12 mins
EJC: Volume 149, p82-90, May 2021

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Summary

Cancer incidence is increasing globally, and while medical innovation significantly impacts patient survival, the drug development process is lengthy, often exceeding 10 years for marketing authorization (MA).
France has implemented the ATU (Temporary Authorization for Use) program to facilitate earlier access to therapeutic innovations, allowing drug administration prior to European Medicines Agency (EMA) approval.
The study compares the average time for accessing anti-neoplastic drugs globally, considering ATU, FDA, and EMA approvals.
From 2007 to 2019, 36 out of 64 oncology drugs with MA used the ATU system, benefiting 16,927 patients. The ATU enabled earlier access to 25 of these drugs, on average 203 days before FDA and 428 days before EMA approvals.
Only 3 of the 36 drugs received EMA approval before FDA approval, with an average delay of 216 days between European MA and FDA approval.
The ATU system ensures full healthcare coverage for patients receiving therapeutic innovations before official MA in Europe and the USA.

The cancer incidence continues to rise worldwide. There were about 18,000,000 new cases in 2018 and about 9,500,000 deaths. Despite therapeutic advances, cancer has become the leading cause of death since 2004 in France, with 157,000 deaths in 2018. To cope with this challenge, societies need to adapt their approach to cancer prevention and treatment, with changes in the development and use of innovative anti-neoplastic drugs playing an important role. Medical innovation has a major impact on patient survival and longevity. The joint assessment of incidence, mortality and survival shows that real progress has been made in the management of many cancers. Overall, patient survival continues to increase, although there is a wide disparity between countries. Although few studies have addressed the issue because of the many variables and confounding factors, the time required for access to treatment appears to have an impact on survival.

In the development of a drug, from the laboratory research phase to the end of clinical trials, it can take more than 10 years to obtain marketing authorization (MA). The MA should guarantee the quality, efficacy and safety of the product. In addition, there are procedures for access to the medicinal products market before the marketing of the medicinal product is possible. Access to cancer drugs, especially new innovative drugs, varies wor

ldwide and depends on the country's economic strength and policy choices based on scientific evidence and cost-effectiveness. In the European Union (EU), access to new cancer drugs involves centralized licensing decisions by regulators and then each country decides on repayment according to its nati