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# PhRMA Seeks More FDA Info on Rare Disease Steps
- URL: https://www.fdaweb.com/phrma-seeks-more-fda-info-on-rare-disease-steps/
- Published: 2024-02-15T12:00:00.000Z
- Updated: 2026-09-14T14:23:11.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156370

Pharmaceutical Research and Manufacturers of America (PhRMA) says an FDA virtual meeting on advancing the development of therapeutics through rare disease patient community engagement “provided a critical forum to discuss best practices and opportunities for engaging with rare disease patients and other key stakeholders throughout the rare disease drug development process.” The association’s comment [letter](https://downloads.regulations.gov/FDA-2023-N-4718-0005/attachment%5F1.pdf?ref=fdaweb.com) says PhRMA encourages FDA to provide information about how the agency leverages patient experience data (PED) in regulatory decision-making.

“We request that the agency provide additional details regarding how PED,” it says, “including but not limited to clinical outcome assessments used in clinical trials and patient preference information collected within or outside clinical trials, is used in the context of benefit-risk decisions and overall totality of evidence regarding patient experience in drug development.”

The trade group also wants FDA to clarify how PED may be incorporated into product labeling and the agency’s expectations for PED-related discussions with industry during product development.

The letter also includes specific comments.