New ASCO Study Aims to Learn From Patient Access to Targeted Cancer Drugs Used Off-Label

ASCO plans to launch a first-ever study that will offer cancer patients access to molecularly targeted cancer drugs and collect “real-world” data on clinical outcomes to help oncologists learn the best uses of these drugs outside of approved indications.

“One of the major challenges to implementing personalized medicine is the lack of information about the risks and benefits of targeted drugs that are used off label to treat patients whose tumor harbors a genomic abnormality,” ASCO Chief Medical Officer Richard L. Schilsky, MD, FACP, FASCO, said at the Friends of Cancer Research-Brookings Institution Conference on Clinical Cancer Research. “Other difficulties are lack of access to these agents for patients and interpretation by oncologists of the complex genomic test results. This ASCO-led clinical trial will address both challenges.”

TAPUR Study

The ASCO project, called the Targeted Agent and Profiling Utilization Registry (TAPUR) Study, will be the first clinical trial ever led by the Society in its 50-year history.

The two primary study objectives are:

• To describe the antitumor activity and toxicity of commercially available, targeted cancer drugs used off label for treatment of patients with advanced solid tumors with a known genomic variant.
• To facilitate patient access to commercially available, targeted anticancer drugs of potential efficacy prescribed off label for treatment of patients with an advanced solid tumor with a known genomic variant.

The prospective, nonrandomized clinical trial would enroll patients who have an advanced solid tumor and are no longer benefitting from standard anticancer treatment or for whom no such treatment is available. Patients would be screened for acceptable performance status and organ function, and a tumor genomic test must identify at least one genomic variant that can be targeted with drugs that will be offered through the study.

The study will provide a list of eligible drug variant matches. The treating physician could propose treatment according to one of the matches or seek concurrence from the TAPUR molecular tumor board for a different treatment plan. Approved targeted therapies would be provided by participating pharmaceutical companies.

For additional information and new developments on the TAPUR study, please visit www.asco.org/TAPUR. ■