What is Patient-focused Drug Development?

As experts in what it is like to live with their condition, patients should play a major role in helping to define the therapeutic context for drug development and evaluation. Patient-focused drug development (PFDD) ensures that patients’ experiences, perspectives, needs, and priorities are prioritized when developing and evaluating drugs.

The FDA describes several requirements of PFDD:

  • Facilitating and advancing use of systematic approaches to collecting and utilizing robust and meaningful patient and caregiver input to more consistently inform drug development and regulatory decision-making

  • Encouraging identification and use of approaches and best practices to facilitate patient enrollment and minimizing the burden of patient participation in clinical trials

  • Enhancing understanding and appropriate use of methods to capture information on patient preferences and the potential acceptability of tradeoffs between treatment benefit and risk outcomes


  • Identifying the information that is most important to patients related to treatment benefits, risks, and burden, and how to best communicate the information to support their decision making.

 FDA’s Efforts to Advance PFDD

The FDA’s Patient-Focused Drug Development Program includes several initiatives to support PFDD in drug development and evaluation.  These include:

Links to FDA resources and public meetings