FDA issues draft guidance on Patient-Focused Drug Development


FDA has just issued a new draft guidance to inform patients and medical product developers on how to gather information on patients’ views and needs in a way that provides meaningful data.

This is the first in a set of four guidance documents. Each will include what FDA learned from hosting “patient focused drug development” meetings. Each will focus on different aspects of collecting information from patients and caregivers for use in medical product development and regulatory decision-making.     

The first draft guidance, Patient-Focused Drug Development: Collecting Comprehensive and Representative Input, issued on June 12, addresses different sampling methods for collecting representative information on patient experience. And how the research question helps determine the best sample (that is, a group of patients or caregivers). A glossary, also included in this first draft guidance, provides common definitions to help with future research.

FDA developed this first draft guidance from discussions during and comments from the public after a December 18, 2017 workshop held by the FDA.  Here, the public means patients, patient advocacy groups, pharmaceutical companies, experts outside of the FDA, and other interested groups and individuals.  Members from PREFER also attended the workshop and gave comments. PREFER comments included a request to add other ways patient experience data can inform decision making, and to specify how FDA will update guidance documents when new information is available, such as results from PREFER. They also encouraged using clearly defined terms when referring to data collected from patients. 

The PREFER consortium plans to review and stay engaged with the developing guidelines.  We will also provide comments based on PREFER results to move forward this evolving field of work.

Find more information at FDA’s web site CDER Patient-Focused Drug Development.

Read statement from the FDA

By Kristin Bullok

Last modified: 2021-11-10