Industry Seeks Changes to Cancer Trials PROs Guidance

The Biotechnology Innovation Organization (BIO) is urging FDA to develop additional and broader guidance on patient-reported outcomes. In just-posted comments to the agency on a draft guidance entitled Core Patient-Reported Outcomes (PROs) in Cancer Clinical Trials, BIO said many of the concepts can and should be utilized more broadly beyond just cancer trials, and encouraged the agency to release additional guidance on broad use and applicability of various types of patient experience data, including PROs. “We also encourage the agency to solicit feedback from the patient community regarding patient-focused drug development related guidances,” the group said. “Their viewpoints on these guidances will be critical as we move from developing medicines for patients to developing medicines with patients.”

In comments submitted by Janssen, FDA was urged to clarify how PROs will complement safety data. “Our understanding from various public forums and publications by the FDA Oncology Center of Excellence (OCE) is that there is no regulatory requirement for PRO data to be reviewed or reconciled with safety data,” the comments said. “Further, there was acknowledgment of a clear distinction between these two sources of data and while complementary, [they] are expected to differ and may not correlate. It was also discussed in these forums that PROs should not inform gaps or errors in safety measurement. We recommend the FDA clarify in the final guidance that there is no regulatory requirement for PRO data to be reviewed to identify safety events at the subject-level or population-level during or at completion of the clinical trial.”

Janssen also recommended that FDA clearly communicate how the core PRO set will inform regulatory decision-making. “The draft guidance includes a section on labeling considerations but does not specify how the FDA will use the core PRO set for regulatory decision-making beyond a general statement for benefit/risk assessment,” the company said.

And, Janssen asked FDA to create new opportunities for early PRO interactions with sponsors. “Early consultations with FDA on PROs are necessary to evolve a sponsor’s measurement approach to successfully incorporate patient input into drug development,” the company said. “Consultations to specifically address topics
related to PROs and patient experience data (PED) occur within FDA/Sponsor traditional clinical development meetings. It would be helpful for the FDA to outline which meeting pathways sponsors should use to discuss PROs and the information that should be made available to the FDA prior to and in such meetings. New meeting formats to discuss PROs beyond the current FDA/sponsor clinical development meetings are welcome.

Additionally, the Pharmaceutical Research and Manufacturers of America (PhRMA) called on FDA to align the PRO guidance with an upcoming guidance on patient-focused drug development and analyzing clinical outcome assessment (COA) data for purposes of regulatory decision-making. PhRMA suggested FDA consider inclusion of applicable COA principles in the PRO draft guidance to align with other PFDD guidance documents. “This will help ensure that all PFDD principles are applied to COA development and implementation and that there is consistency in the guidance for core PROs in cancer clinical trials,” the group said.

Comments were also submitted by AstraZeneca, Takeda, Gilead, and Pfizer.