FDA Patient-Focused Drug Development (PFDD) Guidance - Part 3

June 22, 2023

Back to all webinars

Open to all ISPOR Members and Non-members

Title: FDA Patient-Focused Drug Development (PFDD) Guidance - Part 3

Thursday, June 22, 2023
10:00AM EDT | 2:00PM UTC | 4:00PM CEST 

Click here for time zone conversion

Register Now


The US Food and Drug Administration (FDA) has drafted a fourth Patient-Focused Drug Development (PFDD) guidance document which focuses on “Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making.” The FDA has developed four guidance documents to inform the inclusion of patient experience when developing new medical therapies and products. The PFDD guidance documents are anticipated to be influential in the field of health economics and outcomes research.

In part 1 and 2 of this webinar series, patient representatives and FDA staff presented on the first 3 finalized guidances. In part 3 of the webinar series, an overview of the draft guidance 4 document will be provided including the methods, standards, and technologies for collecting and analyzing clinical outcome assessment (COA) data. Guidance document 4 was recently released and is open for comments. With the public comment period open until July 2023, this webinar will also present ISPOR members with the opportunity to ask questions and provide comments that will help inform ISPOR’s response on the draft guidance 4 document.

Speakers will include an FDA representative to inform on draft guidance 4 and Richard Willke, ISPOR's Chief Science Officer, who will address ISPOR's role and response to the document.

The draft guidance 4 titled “Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making” can be accessed hereIn case you missed it, webinar part 1 and part 2 of the series are available on-demand.

Learning Objectives

  • Inform the ISPOR membership about the FDA PFDD initiative in general and the four PFDD Guidances more specifically, and the various ways PFDD might impact the work they do in HEOR, COA, regulatory science, and other areas.
  • Detail newly released FDA PFDD draft guidance 4.
  • Provide key considerations on how draft guidance 4 can be used and on the future of PFDD.

Reference Materials
An overview of the FDA Patient-Focused Drug Development Guidance (PFDD) Series and guidance documents 1-4 can be found here.


    Eleanor Perfetto, MS, RPh, PhD, Professor, Department of Practice, Science, and Health Outcomes Research, University of Maryland, Baltimore, MD, USA

    Monica Morell, PhD, Statistician, Patient-Focused Statistical Support Team, Division of Biometrics III, Office of Biostatistics, Center for Drug Evaluation and Research (CDER), US FDA, Silver Spring, MD, USA

    Richard Willke, PhD, Chief Science Officer, ISPOR, Lawrenceville, NJ, USA


    Brought to you by:  ISPOR Patient-Centered Special Interest Group

    Please note:
    On the day of the scheduled webinar, the first 1000 registered participants will be accepted into the webinar. For those who are unable to attend, or would like to review the webinar at a later date, the full-length webinar recording will be made available at the ISPOR Educational Webinar Series webpage approximately 2 days after the scheduled Webinar.

    Reservations are on a first-come, first-served basis.

    Your browser is out-of-date

    ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now