New Indications for Oncology Drugs

Proposed Recommendations for Designing Clinical Trials for “New Indications” of Approved Oncology Drugs for Treatment of Late Stage Disease

This Effectiveness Guidance Document (EGD) provides a framework to guide the design and implementation of prospective clinical trials for FDA-approved oncology drugs used outside their current labeled indications to treat late stage cancer. Currently, there are evidence gaps that make it difficult to obtain sufficient information to make informed decisions regarding treatment decisions. The aim of this EGD is to reflect the desires of patients, prescribers, payers, and compendia developers so that those who design future post-approval clinical trials will do so with these stakeholders in mind. Rather than providing an exhaustive list of all aspects of prospective clinical trial design, the EGD focuses on those aspects that will be most impactful in making the results that emerge from future trials in this area most meaningful and actionable.

The research that grows out of this EGD will ultimately be more informative to:

  • Patients making decisions to enter, continue, or switch treatment
  • Physicians making treatment recommendations
  • Investigators designing clinical trials to improve treatment
  • Professional societies developing clinical guidelines
  • Producers of compendia drug information
  • Expert panels making decisions about compendia listing
  • Payers making coverage and reimbursement decisions

Documents

  • Proposed Recommendations for Designing Clinical Trials for “New Indications” of Approved Oncology Drugs for Treatment of Late Stage Disease. pdf New Indications EGD (pdf) (795.35 kB)