Determine Patients’ Needs

PFDD Initiative

The PFDD Initiative grew out of the realization that patients, their families, caregivers, and associated patient groups are experts in living with their condition and are uniquely positioned to inform the understanding of the therapeutic context for therapy development and evaluation. 

  • FDA-led PFDD public meetings: The FDA wanted to include information in their marketing review process from the patient and caregiver perspective, but needed the collection of this information to be systematic and representative of the whole patient population. FDA-led PFDD public meetings were piloted as a method to collect this data.
    • Patient perspectives were sought concerning 2 main areas:
      • Most bothersome signs/symptoms of their condition and the impact of the condition on daily life, including:
        • Burden of managing the disease.
        • Affected daily activities.
        • Unmet medical needs.
        • Barriers to participating in clinical studies.
      • Current and potential approaches to treatment, such as:
        • Impact on patient’s ability to perform daily activities.
        • Minimum expectations of benefits.
        • Tolerance for harms or risks.
        • Acceptable trade-offs of benefits and risks (i.e., patient preference).
        • Attitudes towards uncertainty.
    • The FDA hosted 24, mostly disease-specific, public PFDD Meetings between 2012 and 2017 to determine the feasibility of such meetings to achieve their goals.
      • Since 2018, the FDA continues to host periodic PFDD meetings, but the focus is on much broader public health issues, such as opioid use disorder, chronic pain, and stimulant use disorder.
    • Sample questions asked included: 
      • Which symptoms have the most significant impact on your daily life?
      • Which symptoms have the most significant impact on your ability to do specific activities?
      • How well does your current treatment regimen treat the most significant symptoms of your disease?
      • What specific things would you look for in an ideal treatment for your condition?
      • What factors do you take into account when making decisions about using treatments?
      • What factors do you take into account when deciding whether to participate in a clinical trial?
    • Additional information gained through the meetings included:
      • Outreach is the key to success.
      • Specific experiences that mattered most to patients.
      • Patient perspectives on meaningful treatment benefits.
      • Patient-focused methods for engaging them in the therapy development process.
    • Following each PFDD meeting, FDA summarizes the input shared by patients and patient representatives in a Voice of the Patient report.
    • You can learn more about these initial meetings and future meetings on the FDA-led Patient-Focused Drug Development (PFDD) Public Meetings webpage. For each meeting there are links to the:
      • Agenda
      • Discussion questions
      • Voice of the Patient report
    • During the period of the first PFDD meetings, the National Health Council developed a tool to help patient groups collect patient information. Although the actual tool is no longer available, the accompanying manual is. The manual describes how your group can gather, translate, and share information. It also includes the efforts of Unite Narcolepsy, one first disease communities to have an FDA-led PFDD, to prepare their community for the public meeting.
  • Externally-led PFDD: Since the number of public meetings the FDA can host is limited, the FDA encourages patient groups, in collaboration with other stakeholders, to host externally-led PFDD meetings.
    • When appropriately modeled after FDA-led PFDD meetings, externally-led PFDD meetings can inform the FDA and therapy development through the patient voice. 
    • Because the meetings take resources and effort, you may wish to consider whether there is an identified need for a better understanding of the patient perspective for purposes of therapy development for your disease.
    • Meetings may be most helpful for disease areas:  
      • That are chronic, symptomatic, or affect the functioning and activities of daily living.
      • For aspects of the disease that are not formally captured in clinical trials.
      • For which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.
      • That have a severe impact on identifiable subpopulations, such as children or the elderly. 
    • Externally-led PFDD meetings are appropriate for most rare diseases.
    • FasterCures offers a self-guided PFDD Readiness Assessment tool as one means to determine whether your group is ready to plan an externally-led PFDD.
  • General elements of PFDD meeting model:
    • Key participants: Patients, patient families and caregivers, patient advocates.
    • Target audience: FDA and other federal agencies, therapy developers, researchers, healthcare professionals.
    • Meetings or sessions are facilitator led discussions.
    • Interactive webcasts increase the diversity of patient voice.
    • Polling tools are used to gather answers to questions from both those in attendance and those attending via webcast.
    • Main discussion topics should include:
      • Symptoms and impacts that matter most to patients, families, and caregivers.
      • Challenges of current approaches to treatment.
    • Web recording of meeting and transcript may be posted on the group website.
    • Summary report (Voice of the Patient) can be submitted to be posted on External Resources or Information Related to Patients’ Experience  (updated periodically).


Natural History Study
FAQ Orphan Products Natural History Grants Program U. S. Food and Drug Administration (FDA) (link)
OOPD Grant Application Overview U. S. Food and Drug Administration (FDA) (link)
Building a Registry
Rare Diseases Registry Program (RaDaR) National Center for Advancing Translational Sciences (NCATS) (link)