FDA and Patient-Focused Drug Development

Patients are very much at the center of what the Food & Drug Administration (FDA) does, and the agency has long sought input from patients and caregivers regarding their treatment options and needs. For many years, FDA has included the patient perspective at advisory committee meetings and during the review of new medical products in selected meetings with FDA and product developers.

More recently, FDA has encouraged and fostered the use of patient-reported outcome measures in clinical trials, such as impact on quality of life or pain control, to support labeling claims in medical product development. We are excited about our current efforts to expand the role of the patient perspective: the agency’s Patient-Focused Drug Development initiative.

Understanding a condition’s severity and its treatment options is a part of the benefit-risk assessments that help FDA make decisions about the medical products it regulates. As part of this commitment, FDA will hold at least 20 public meetings over the next 5 years, each focused on a different disease area. The information discussed in these meetings will be summarized for consideration by FDA review divisions when making regulatory decisions in that therapeutic area.

Richard M. Klein is Director of the Patient Liaison Program in FDA’s Office of Health and Constituent Affairs.