FDA releases fourth guidance document in its series on patient-focused drug development

On April 5, the FDA released draft guidance on incorporating clinical outcome assessments into endpoints for regulatory decision making; the fourth and final guidance in its patient-focused drug development series. 

This month has seen the release of the FDA’s draft guidance “Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making,” which is the fourth and final part of a series of guidance documents on patient-focused drug development (PFDD). As stated on the FDA website, it is: 

 “…developing a series of four methodological PFDD guidance documents to address, in a stepwise manner, how stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making.” 

 In total, the PFDD guidance series consists of: 

• Guidance 1: Collecting Comprehensive and Representative Input (final guidance)
• Guidance 2: Methods to Identify What is Important to Patients (final guidance)
• Guidance 3: Selecting, Developing or Modifying Fit-for-Purpose Clinical Outcomes Assessments (draft guidance)
• Guidance 4: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making (draft guidance) 

In this fourth document, the focus is on the methods, standards and technologies used for collecting and analyzing clinical outcome assessment (COA) data, including selecting a COA-based endpoint and determining clinically meaningful change in that endpoint. The guidance is aimed at those conducting trials of medicinal products (including drugs, devices and biological products for human use) using COAs, and analyzing and interpreting the subsequent data. 

The guidance is long-awaited, with patient-engagement in the drug development and regulatory process being a topic of high interest for several years. As stated in the document, the aim of COAs is to “reflect, directly or indirectly, how patients feel, function, or survive”. However, there are many challenges in achieving this, and the guidance provides a section on COA-based endpoint considerations including thinking about the endpoint of interest (“What are you measuring in the target study population?”) and statistical analysis considerations (“Estimation and Missing Data”). 

The PFDD guidance series is being developed under the 21st Century Cures Act, and comments on draft Guidance 4 are invited to be submitted by July 5. 

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• A perspective on the future of patient engagement – Sheila Khawaja
• The importance of patient engagement: an interview with Daniel De Schryver
• Patient engagement in comparative effectiveness research: an interview with Daniel Mullins