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  4. Public Workshop on Patient-Focused Drug Development: Guidance 4 – Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making - 12/06/2019 - 12/06/2019
  1. Development & Approval Process | Drugs

Workshop | In Person

Event Title
Public Workshop on Patient-Focused Drug Development: Guidance 4 – Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making
December 6, 2019


December 6, 2019
Event Location
White Oak Campus: The Great Room
Conference Center

10903 New Hampshire Ave
Building 31, Room 1503
Silver Spring, MD 20993
United States

The U.S Food and Drug Administration (FDA) is hosting a public workshop to gather input from the wider community of patients, patient advocates, academic researchers, expert practitioners, drug developers, and other stakeholders. The workshop will address a range of issues and considerations related to the collection and analysis of clinical outcome assessment (COA) data for drug development and regulatory decision making.
The purpose of this public workshop is to obtain feedback from stakeholders on considerations for:

  1. Endpoint development
  2. Estimands and analysis models
  3. Addressing heterogeneity in disease symptoms and functional status between patients and within the same patient over time
  4. Data collection, storage, transmission, and analysis

This public workshop is intended to support FDA implementation of requirements for guidance development under section 3002 of the 21st Century Cures Act (Pub. L. 114-255) and to meet a performance goal included in the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI). Section 3002 of Title III Subtitle A of the 21st Century Cures Act directs FDA to develop patient-focused drug development guidance to address several areas including methodologies, standards, and technologies to collect and analyze COA data for purposes of regulatory decision making.

In addition, FDA is committed to meet certain performance goals under PDUFA VI. As part of the FDA Reauthorization Act of 2017 signed by the President on August 18, 2017, PDUFA VI includes a number of performance goals and procedures that are documented in the PDUFA VI Commitment Letter.


Registration is free. To register for the public workshop, please visit the following website: patientfocuseddrugdevelopment.eventbrite.com. Please provide complete contact information for each attendee, including name, title, affiliation, address, email, and telephone.

Registration for in-person attendance is required and will close on December 3, 2019. Persons without access to the internet can call (301) 796-0621 to register. Early registration for in-person attendance is recommended because seating is limited. Priority is given to early registrants and FDA may limit the number of participants from each organization. Onsite check-in will begin at 8:00 am on December 6, 2019. Onsite registration for in-person attendance on the day of the workshop will be based on space availability.

If you are unable to attend the workshop in person, you can register to view a live webcast of the workshop. Registration for online attendance via webcast will remain open throughout the day of the workshop.


If you need special accommodations due to a disability, please contact the Patient-Focused Drug Development Staff at (240) 402-6525 or PatientFocused@fda.hhs.gov at least 7 days before the workshop.


Public Workshop Agenda


This meeting will be available for online viewing, on the day of the meeting via the following link: Patient-Focused Drug Development Guidance: Collection and Analysis of Clinical Outcome Assessment Data for Drug Development and Regulatory Decision Making; Public Workshop. If you have used Connect Pro before, test your connection at Meeting Connection Diagnostic. To get a quick overview of the Connect Pro program, visit Adobe Connect Pro Overview.


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 February 4, 2020.


There will be time allotted during the workshop for open public comments. Sign-up for this session will be on a first-come, first-served basis on the day of the workshop. Individuals and organizations with common interests are urged to consolidate or coordinate and request a time for a joint presentation. No commercial or promotional material will be permitted to be presented or distributed at the public workshop.


Entrance for the public meeting participants (non-FDA employees) is through Building 1. Please allow time for routine security checks, as screening procedures will be performed. For parking and security information, please refer to the following link: Public Meetings at the FDA White Oak Campus.


Patient-Focused Drug Development Staff
Center for Drug Evaluation and Research
Office of the Center Director


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