In an effort to broaden the eligibility criteria of noncurable cancer trials, the FDA has published final guidance recommending that sponsors and investigators allow patients who have not taken existing treatment options to participate.

The short guidance, which defines noncurative cancer as unresectable, locally advanced or metastatic disease in solid tumors or hematologic malignances with unfavorable long-term overall survival, explains that patients who haven’t received available therapy should be able to receive investigational drugs as long as they’re adequately informed about their existing options prior to consenting.

To accommodate this group, the guidance offers three considerations for trial design. First, sponsors should ensure that all aspects of informed consent are addressed when consenting participants, including alerting them to “appropriate alternative procedures or courses of treatment, if any, that might be advantageous.”

In addition, patients who have and haven’t received available therapies should be split into separate cohorts if interpreting the efficacy results requires a homogenous patient population. Efficacy analyses could also be done in prespecified subgroup analyses defined by prior receipt of available therapy.

Lastly, early dose-escalation trials may evaluate safety without considering participants’ prior therapies as long as the drug’s toxicities aren’t expected to differ across patient subgroups, the agency said.

The draft form of the guidance was issued a year ago (CenterWatch Weekly, June 28, 2021).

Read the final guidance here: https://bit.ly/3OI7xjh.