Sponsors Weigh in on Proposed FDA Patient-Focused Drug Development Guidance

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The jury is still out for drug manufacturers when it comes to the U.S. Food and Drug Administration’s (FDA’s) parameters for its in-progress, patient-focused drug development guidance. However, the steady stream of early industry comments skews mostly positive.

FDA says it will unveil additional draft guidances on patient-focused drug development over the next four years, as part of its Prescription Drug User Fee Act (PDUFA) commitments to increase efforts to collect feedback from caregivers and patients. The FDA issued a discussion document on patient-focused drug development in October and held a public meeting to gather stakeholder input in December.

“We applaud the Agency’s continued efforts under the 21st Century Cures Act and PDUFA VI to establish a systematic framework to facilitate the collection of patient input to inform drug development and the development of methodologically sounds tools to measure patient-relevant data in clinical trials,” Roche and Genentech commented in a February 16 response..


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To be most useful to sponsors, Roche/Genentech believe “the guidance should consider the evolving objectives of patient-focused research throughout the drug development and provide practical guidance as to how to choose a fit-for-purpose method to gather patient input for different objectives.” Further, while acknowledging the new guidance is designed to be broad, it calls on the FDA to “produce a follow-up guidance to articulate when and how the agency will use this information as part of a benefit-risk [assessment] and provide clarity regarding the evidentiary standards to support a claim of clinical benefit.”

Pfizer echoed many of the issues raised by Roche/Genentech. It also called on the agency to provide more detail down the line in several areas. For example, “it would be helpful to know what level of evidence would be required to incorporate patient experience information into a label, as opposed to…using patient perceptions and experience to inform procedures in a clinical trial protocol.”

Allergan also asked the FDA to “consider the need for flexibility with response to the methodological requirements and opportunities for engagement to provide sponsors with clarity to inform evidence generation efforts.”

To access all posted comments, go to https://www.regulations.gov/document?D=FDA-2017-N-5896-0001.

Author: Michael Causey