FDA Seeks Reliable Patient Information to Support Regulatory Decisions

Oct 05, 2018

As FDA continues to advance the role of patients in designing clinical trials with meaningful and accurate endpoints, it is seeking more input from stakeholders on how best to identify clinical outcomes most important to patients and caregivers. The aim is to reduce uncertainty about the reliability of patient reported outcomes (PROs) and encourage the sustained use of patient experience data in drug development. More consistent standards and new FDA guidances should make it “standard practice” to incorporate patient experience in drug development and regulatory decision making, says Theresa Mullin, Associate Director for Strategic Initiatives, who heads up the Patient-Focused Drug Development (PFDD) program at the Center for Drug  Evaluation and Research (CDER). 

To advance this initiative, FDA officials will discuss best practices for collecting comprehensive and representative patient input, methods for identifying what’s most important to patients, and selection of fit-for-purpose clinical outcomes assessments (COAs) with patients, sponsors, the research community, and others at a public meeting October 15-16, 2018 [see https://www.fda.gov/Drugs/NewsEvents/ucm607276.htm for workshop details]. The aim is to advance a series of PFDD guidances, Mullin explained at a recent conference on “Advancing the Science of Study Endpoints” sponsored by the Drug Information Association. The meeting addressed the complexities of developing appropriate patient-focused endpoints in clinical trials, including how measures may evolve during development and the use of exit interviews to gain useful input on PROs.

Mullin described at the DIA conference how FDA is developing four methodological PFDD guidance documents to clarify how to collect and submit patient experience data, as authorized by the 21st Century Cures Act to help advance medical product development and regulatory decision making [see https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm610279.htm]. She emphasized the importance of patient input in supporting benefit-risk assessment in FDA regulatory decisions, and how that may vary related to the severity and burden of disease and the availability of treatment. FDA also is developing a compendium of COAs to provide basic information on what outcome assessments have been used in studies or have been qualified for potential use to further support the development of measures and tools that can systematically capture key data during clinical trials. 

To help stakeholders prepare for the October public meeting, FDA has issued two discussion documents and background information on methods for identifying what’s most important to patients [see https://www.fda.gov/downloads/Drugs/NewsEvents/UCM620707.pdf] and on developing fit-for-purpose COAs [see https://www.fda.gov/downloads/Drugs/NewsEvents/UCM620708.pdf]. Another public workshop in 2019 will aim to gather recommendations from patients and caregivers on ways to enhance engagement in clinical trials.


Jill Wechsler is the Washington Correspondent for Applied Clinical Trials.

lorem ipsum