Industry Backs FDA Guidance on Patient-Focused Drug Development

February 12, 2020 - Biotechnology and pharmaceutical stakeholders largely supported guidance from the FDA on patient-focused drug development, but some sought some clarification about FDA decision-making.

The agency held a public workshop last December to inform the development of the fourth draft guidance to come out under the patient-focused drug development program of FDA’s Center for Drug Evaluation and Research. It engaged drug developers and industry groups, among others.

Holding the public workshop completes a portion of FDA’s commitments under the latest reauthorization of the Prescription Drug User Fee Amendments (PDUFA VI). The agency issued guidance documents outlining consideration factors for sponsors when developing endpoints based on fit-for-purpose COAs. It was the fourth and last in the string of such public workshops.

An FDA discussion document accompanying the last workshop explains what FDA considers when a COA will be used to support regulatory decision-making.

Past workshops discussed collecting comprehensive and representative input, methods to identify what is important to patients and selecting, developing, or modifying a fit-for-purpose COA. FDA issued draft guidance on the first two topics in June 2018 and September 2019. It has until the end of this fiscal year and FY 20201 to draft and finalize the remaining documents.

The public comment period that FDA opened with the notice of the workshop to discuss the fourth guidance closed earlier this month, with 20 submissions. The docket allowed commenters to address certain topics not discussed during the workshop, including meaningful within-patient change. A consensus among commenters seeks clarity and cross-referencing.

Detail is needed on the types of regulatory decisions for which FDA considers patient experience data and how COAs inform decision-making, PhRMA and BIO argue in letters to FDA.

Danielle Friend, BIO science and regulatory affairs senior director, writes, “BIO requests that FDA expand the guidance to provide the agency’s views on how and where information COAs can be best incorporated in the label and/or be included in other communications to disseminate patient-centric outcomes to other key stakeholders. FDA may also consider publishing assessment reports explaining why certain PROs are/are not included in product labels”

PhRMA senior director of science and regulatory advocacy, Maria Apostolaros, says the group “strongly recommends cross-referencing all the guidance documents in each of the four guidances that are part of the PFDD series.” She adds in PhRMA’s February 4 comment letter that “This will ensure all PFDD principles are applied to COA development and implementation.

The groups ask FDA to encourage early interaction between agency staff and sponsors on COAs. BIO and PhRMA also share their recommendation for FDA, outlining its current thinking on challenges facing COA development and approaches to help sponsors mitigate these well.

On the topic of within-patient change, BIO asks for information on methods in a triangulation approach. Apostolaros adds that “PhRMA believes that additional detail on the FDA’s preferred methods for developing interpretation thresholds for within-patient change and on the context of use of these methods might help mitigate risk and encourage COA development, with the intended goal of benefiting patients and accelerating product development.”