PFDD is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.

The primary goal of patient-focused drug development is to better incorporate the patient’s voice in drug development and evaluation, including but not limited to:

• Facilitating and advancing the use of systematic approaches to collect and utilize robust and meaningful patient and caregiver input that can better inform medical product development and regulatory decision-making.

• Encouraging identification and use of best practices to facilitate patient enrollment and minimize 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.

• During the post-marketing period, identifying the information that is most important to patients related to treatment benefits, risks, and burden, and determining effective methods for communicating this information to support their decision-making.

In 2012, the U.S. Food and Drug Administration (FDA) launched its PFDD initiative with the goal of making patient and care partner input a structured part of medical product review and development guidance. Initially, the FDA organized 24 public meetings on specific diseases and conditions. People living with these conditions shared their experiences, described their most challenging symptoms, and discussed their preferences for treatments.

Starting in 2015, patient communities have directly organized meetings, which are referred to as “externally-led” PFDD meetings (EL PFDD). These are one-day events held in person or virtually. They feature representative panels of patients and care partners discussing their experiences, followed by moderated discussions involving the larger community. Meetings are planned with input from FDA staff and follow the process established by FDA-led PFDD meetings as a model.

Historically, patient and care partner perspectives were underrepresented in the development and evaluation of medical treatments. Before launching its PFDD initiative, the FDA recognized the need to incorporate these perspectives into its decision-making, but lacked a structured and representative process for collecting and applying this input.

Today, PFDD is an established, FDA-recognized process for systematically incorporating patient and care partner perspectives into medical product development and review. Meetings are designed to capture patient experience data—covering daily impacts, treatment experiences, and meaningful tradeoffs—using a standardized, discussion-based format.

Each meeting produces a public Voice of the Patient Report, which can be included on the FDA’s condition-specific page and can be referenced in benefit–risk assessments, clinical trial design guidance, and labeling discussions. This structured process can help ensure that patients' experiences and priorities inform the development, evaluation, and review of new therapies.

Spinal cord injury (SCI) is a devastating, lifelong condition that profoundly affects mobility, bladder and bowel control, pain, and overall quality of life—yet there are currently no FDA-approved therapies to repair it. While research has advanced, clinical trials often fail to measure outcomes that matter most to people with SCI, focusing instead on narrow or misaligned endpoints shaped by historical regulatory expectations. As a result, critical priorities such as bladder and bowel control, arm and hand function, and pain reduction remain underrepresented in treatment development.

At the same time, people living with SCI feel their voices aren’t being heard. Surveys show that while more than 9 in 10 U.S. adults with SCI want to be involved in setting research priorities, 8 in 10 feel excluded from the process.

FDA’s Patient-Focused Drug Development (PFDD) initiative offers a formal, structured process to address these issues by systematically gathering and incorporating patient perspectives into regulatory and clinical decision-making. Through an Externally-Led PFDD meeting, the SCI community can communicate its most pressing needs and meaningful tradeoffs directly to regulators and developers. Insights from the meeting’s Voice of the Patient Report will help ensure that future clinical trials, treatments, and regulatory evaluations better align with what truly improves daily life for people living with SCI.

PFDD can help ensure that the voices of people living with spinal cord injury are treated as essential evidence in shaping how therapies are developed, evaluated, and approved. By systematically capturing lived experience perspectives, PFDD can help define the outcomes that matter most, guide clinical trial design, and ensure that new therapies target the priorities people with SCI truly care about.

Through PFDD, regulators can gain clearer insight into the community’s benefit–risk perspectives, and developers receive structured, citable input that can inform product design and regulatory submissions. Together, these efforts can help drive more meaningful innovation and improve the quality of life for those affected.

• For people living with SCI: Provides a formal role in defining the benefits, burdens, and daily impacts that should shape research and treatment development.

• For the FDA: Offers a structured, evidence-based model for integrating patient experience into regulatory review, labeling, and benefit–risk assessments.

• For medical product developers: Can produce actionable insights to guide endpoint selection, clinical trial design, and investment decisions.