Cancer Survivors' and Caregivers' Understanding of FDA Accelerated Approval Disclosures

The US Food and Drug Administration (FDA)'s accelerated approval pathway is intended to facilitate and expedite development and review for prescription drugs that treat serious or life-threatening illnesses such as cancer. Three elements of this pathway are often disclosed on prescription drug websites for oncology prescription drugs: the basis for the drug's approval, that some clinical benefits are unknown, and that confirmatory trials are ongoing. To assess their retention and understanding of these elements, we randomly assigned 202 cancer survivors and 199 caregivers to view a website for a fictitious acute lymphoblastic leukemia prescription drug with one of four accelerated approval disclosures. All disclosures stated, "In a clinical trial, Altisol returned blood counts to normal." We randomly assigned participants to the presence or absence of an "unknown outcomes" disclosure: "However, we currently do not know if Altisol helps people live longer or feel better." We also randomly assigned participants to the presence or absence of a "confirmatory-trials" disclosure: "We continue to study Altisol in clinical trials to learn more about Altisol's benefits." Participants noticed the disclosures, recognizing the three elements when asked. Regardless of whether participants saw the confirmatory-trials disclosure, participants agreed that more data is needed to learn about the drug's efficacy. The unknown-outcomes disclosure increased comprehension across a wide range of measures. These results suggest that an accelerated approval disclosure (in particular, a statement about unknown outcomes) can help patients and caregivers understand a prescription drug's benefits, which can be critical for their participation in treatment decisions.