June 21, 2021 – Amid increased scrutiny of its accelerated approval pathway – partly prompted by the recently rescinded approval for two oncology product indications and the controversial approval of Biogen’s aducanamab for Alzheimer’s disease – the FDA’s Office of Prescription Drug Promotion (OPDP) has announced plans to study how well consumers understand the limitations of drug products approved through the accelerated pathway and what mechanisms improve that understanding.

Under FDA labeling regulations, the indications and usage section of physician labeling for products granted accelerated approval must include not only the indication but also a description of the limitations of usefulness of the drug and any uncertainty about anticipated clinical benefits. Further, FDA guidance suggests that labeling include a disclosure that continued approval of the drug may be contingent on whether confirmatory trials verify the clinical benefit of the drug, according to a June 11 notice in the Federal Register about the proposed OPDP studies. OPDP will accept comments on these studies until Aug. 10.

OPDP’s previous content analysis of direct-to-consumer (DTC) websites for accelerated approval products found that:

• 21 percent of the disclosures used language directly from the approved physician labeling;
• 79 percent of the disclosures used at least some medical language; and
• 27 percent of the websites did not include any disclosure that the products “attained approval through this pathway.”

“The same analysis found that 84 percent of accelerated approval disclosures on DTC websites mentioned the approval basis, 68 percent mentioned unknown outcomes, and 47 percent mentioned confirmatory trials,” OPDP stated in the notice. In prior research, OPDP also found that although comprehension of consumer-friendly disclosures helped participants understand accelerated approval information, “participants’ understanding was low overall.”

The goal of the new studies is “to replicate and extend our prior research … by: (1) testing the same experimental conditions with a different study population (cancer survivors and cancer caregivers in study 1 and (2) testing additional consumer-friendly disclosures in study 2,” the notice states.

As suggested in public comments on the prior analysis, the new Study 1, in cancer survivors and caregivers, will test the prominence of the disclosure and will track comprehension for an accelerated approval drug based on no disclosure, physician labeling disclosure or a consumer-friendly disclosure. For Study 1, OPDP hypothesizes that “participants will be more likely to notice the disclosure when it is presented more, rather than less prominently,” and that participants will be more likely to “notice and understand” the consumer-friendly disclosure.

Study 2 will attempt to address public comments expressing concern that “over-disclosure could dissuade consumers from considering accelerated approval products.” OPDP will test four versions of a consumer-friendly disclosure. For Study 2, OPDP expected that participants “will be more likely to understand each accelerated approval concept (i.e., confirmatory trials, unknown outcomes) when the disclosure directly addresses the concept, compared with when the disclosure does not directly address the concept.” This study will also examine whether information about the concepts affects participants’ perceived risk, perceived benefit-risk tradeoff, perceptions of the website, or information-seeking intentions, according to the notice.