If your email program has trouble displaying this email, view as a webpage.

CDER Patient-Focused Drug Development

FDA Publishes "Assessment of the Use of Patient Experience Data in Regulatory Decision-Making" Report

Yesterday, the U.S. Food and Drug Administration (FDA) published a report titled “Assessment of the Use of Patient Experience Data in Regulatory Decision-Making.” Section 3004 of the 21^st Century Cures Act (Cures Act) requires FDA to report on the use of patient experience data in regulatory decision-making. This is the first such report released by FDA as part of the implementation of the Cures Act and provides a snapshot of progress to date.

FDA contracted with Eastern Research Group, Inc. (ERG) to conduct this first assessment and to produce the report. ERG assessed document reviews for New Drug Applications (NDAs), Biologics License Applications (BLAs), and efficacy supplements and conducted interviews with FDA staff, applicants, and external stakeholders. The assessment gathered information to answer three questions:

• How does FDA use patient experience data in regulatory decision-making?
• How do FDA staff, applicants, and external stakeholders characterize (a) use of patient experience data in regulatory decision-making and (b) FDA communication about use of these data?
• What good practices and opportunities for improvement exist for (a) use of patient experience data in regulatory decision-making and (b) FDA communication about use of these data?

The report provides details regarding the methods used to identify, collect, and analyze data for this assessment, results, and key findings. The report includes recommendations to FDA on how to better communicate and expand the use of patient experience data in regulatory decision-making, and to external stakeholders regarding the use of good practices to aid in the acceptance of patient experience data by FDA. FDA will use this report to support its efforts to enhance the use of patient experience data in regulatory decision-making. FDA is committed to incorporating the patient’s voice in drug development and evaluation.

To view the report, and for more information, please click here.

FDA Publishes Voice of the Patient Report for Systemic Sclerosis

On October 13, 2020, FDA hosted a public meeting on Patient-Focused Drug Development for Systemic Sclerosis. FDA was interested in hearing perspectives from individuals with systemic sclerosis on the health effects and daily impacts of their systemic sclerosis, treatment goals, and decision factors considered when seeking out or selecting a treatment.

Today, the U.S. Food and Drug Administration (FDA) published the Voice of the Patient Report for Systemic Sclerosis. The Voice of the Patient Report summarizes the input provided by patients and patient representatives at the public meeting and as part of the public docket. FDA would like to thank everyone who took the time to share their perspectives during the meeting and as part of the public docket. 

To view the report, and for more information, please click here.

To contact FDA's CDER Patient-Focused Drug Development Program Staff, please email patientfocused@fda.hhs.gov. 

If this message was forwarded to you and you would like to subscribe to this free email subscription service, please visit our CDER PFDD Homepage and click the button "Subscribe to Email Updates" at the top of the page.