RAPS

Patient Focused Drug Development: FDA Issues Guidance on Gathering Patient Input

In a draft guidance issued Tuesday, the US Food and Drug Administration (FDA) details how drugmakers can gather comprehensive and representative input from patients. "FDA recognizes the need to engage ...
RAPS

Fourth Patient-Focused Drug Development Meeting Announced With Focus on Common Questions

The US Food and Drug Administration (FDA) has today announced the details of its fourth patient-focused drug development meeting, this time focusing on narcolepsy, a condition characterized by an ...
JD Supra

FDA Issued Updated Patient-Focused Drug Development Guidance on Clinical Outcome Assessments

The U.S. Food and Drug Administration (FDA) issued its third Patient-Focused Drug Development (PFDD) guidance titled, “Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments ...
The American Journal of Managed Care

How Patient Voices Shape Regulatory Drug Decisions: Eleanor Perfetto, PhD

Explore how patient experience data shapes regulatory drug decisions, enhancing the patient-focused drug development initiative for better treatment outcomes. Patient experience data are critical to ...
JD Supra

FDA Presses Forward on Patient-Focused Drug Development, Releases Final Guidance Focused on Fit-For-Purpose Clinical Outcome Assessments

On October 23, 2025, FDA released its final guidance regarding Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. The guidance is the ...
Nature

A guide to effective drug development through nonprofit models

Patient organizations are reshaping rare disease drug development where conventional systems falter by integrating patient expertise, scientific stewardship, R&D infrastructure and cross-sector ...