Equal access to innovative therapies and precision cancer care

Area of Science:

• Oncology
• Genetics
• Clinical Pharmacology

Background:

• Targeted therapies offer significant benefits for specific cancer biomarkers.
• Off-label use of targeted therapies is limited by drug indications, restricting patient access.
• The French National Cancer Institute established the AcSé Programme to address this gap.

Purpose of the Study:

• To facilitate treatment decisions based on biomarkers for rare malignancies.
• To improve the safety and efficacy evaluation of targeted drugs used off-label.
• To expand patient access to potentially beneficial targeted therapies.

Main Methods:

• Implementing a national program for biomarker testing in 28 centers.
• Providing access to targeted therapies through Phase II clinical trials.
• Including pediatric patients (under 18) when safety data is available.
• Investigating the efficacy of crizotinib and vemurafenib in diverse cancers.

Main Results:

• As of January 2016, 183 French clinical sites and over 7,000 patients were enrolled.
• The program demonstrates proof of concept for biomarker-guided therapy.
• Evidence of effectiveness for crizotinib and vemurafenib in various cancer types.

Conclusions:

• The AcSé Programme successfully expands access to targeted therapies based on biomarkers.
• This approach enables treatment for rare cancers lacking approved targeted options.
• The program enhances the evaluation of off-label targeted drug use in oncology.