- October 15-16, 2018
05:00 AM - 01:00 PM EDT
On October 15-16th, the Food and Drug Administration (FDA) is hosting a two-day public workshop to convene a discussion on: 1) methodological approaches that may be used to develop and identify what is most important to patients and caregivers with respect to burden of disease, burden of treatment, and the benefits and risks in the management of the patient’s disease; and 2) best practices for selecting, developing or modifying fit-for-purpose Clinical Outcome Assessments (COAs) to measure the patient experience in clinical trials. This workshop will inform development of patient-focused drug development guidance as required by Section 3002 of the 21st Century Cures Act of 2016, and as part of commitments made by FDA under the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI).
The purpose of this public workshop is to obtain feedback from stakeholders on considerations for:
- Methods to identify what is important to patients with respect to burden of disease, burden of treatment, and the benefits and risks in management of the patient’s disease
- Best practices for eliciting information on which aspects of disease symptoms, signs, impacts and other issues are important and meaningful to patients
- Measuring symptoms, signs, impacts and other issues of a disease or condition in a meaningful way
- Selecting, developing or modifying fit-for-purpose COAs to measure the patient experience in clinical trials
FDA is seeking information and comments from a broad range of stakeholders, including patients, patient advocates, academic and medical researchers, expert practitioners, drug developers, and other interested persons. FDA will publish discussion documents outlining the topic areas that will be addressed in the draft guidance(s) approximately 1 month before the workshop.
This website will be updated as workshop materials are developed.
October 15-16, 2018
9:00 a.m. – 5:00 p.m. EST (Registration will begin at 8:00 am)
FDA White Oak Campus
10903 New Hampshire Avenue
Building 31, Room 1503 (Great Room)
Silver Spring, MD 20993
(Information about arrival to FDA’s White Oak campus)
To register for this meeting, visit: https://pfddmethods.eventbrite.com
Registration will close on October 8, 2018.
If you are unable to attend the workshop in person, you can register to view a live webcast of the workshop. You will be asked to indicate in your registration if you plan to attend in person or via the webcast. Seating will be limited, so early registration is recommended. Registration is free and will be on a first-come, first-served basis. However, FDA may limit the number of participants from each organization based on space limitations. Onsite registration on the day of the workshop will be based on space availability.
In addition to providing input at the public workshop, stakeholders are invited to submit comments through the public docket. The public docket will close on December 14, 2018.
Persons with disabilities having problems accessing the PDF file(s) below may call (301) 796-3634 for assistance.
- Discussion Documents
Attachment to Guidance 2 Discussion Document – Appendices (PDF - 472 KB)
Attachment to Guidance 3 Discussion Document – Appendices (PDF - 219 KB)
Legislation Background and Glossary (PDF - 138 KB)
- Additional Background Material
- Clinician-Reported Outcome Assessments of Treatment Benefit: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force (PDF - 530 KB)
- Developing and Implementing Performance Outcome Assessments: Evidentiary, Methodologic, and Operational Considerations (PDF - 237 KB)
- FDA Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims
- CDER Patient-Focused Drug Development Homepage
- FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making
- Plan for Issuance of Patient-Focused Drug Development Guidance (PDF - 146 KB)