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As we prepare to apply for a PFDD with the FDA we thought it would be a good idea to explain to the community what a PFDD is.

What is Patient-Focused Drug Development?

Patient-focused drug development (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. As experts in what it is like to live with their condition, patients are uniquely positioned to inform the understanding of the therapeutic context for 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 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.

The Patient-Focused Drug Development (PFDD) initiative aims to more systematically obtain the patient perspective on specific diseases and their treatments. The patient perspective is critical to help provide context when FDA makes regulatory decisions for new drugs. PFDD meetings give FDA and other key stakeholders, including medical product developers, health care providers, federal partners, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application.

What is the opportunity for an externally-led PFDD meeting?

FDA has conducted over 25 disease-specific PFDD meetings to more systematically obtain the patient perspective on specific diseases and their treatments. FDA recognizes that there are many more disease areas that can be addressed beyond the PFDD meetings planned and conducted by the FDA.

To help expand the benefits of FDA’s PFDD initiative, FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas, using the process established by FDA-led PFDD meetings as a model.

What should stakeholders consider when deciding to pursue an externally-led PFDD meeting?

Given the expanse of diseases affecting the U.S. patient population and the effort required to conduct a successful PFDD meeting, externally-led PFDD meetings should target disease areas where there is an identified need for patient input on topics related to medical product development.

Some considerations to take into account when deciding to plan an externally-led PFDD meeting:

  • Is there an identified need for better understanding, for purposes of drug development, the patient perspective on a disease area? FDA recommends considering the following criteria:
    • Disease area that is chronic, symptomatic, or affects functioning and activities of daily living;
    • Disease area for which aspects of the disease are not formally captured in clinical trials;
    • Disease area 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;
    • Disease areas that have a severe impact on identifiable subpopulations (such as children or the elderly)
  • Are you interested in collecting patient perspectives to inform the therapeutic context of the disease area in the early stages of development (e.g., symptoms and impacts of disease on daily life and current treatment options)?
  • Who would be the target patient population? What are the important disease subpopulations or patient characteristics that should be represented (e.g., variations of the disease, the spectrum of severity, and the spectrum of experiences with current treatments)? Is it valuable to focus the meeting on any particular subpopulation(s), such as children, people with metastatic forms of the disease, etc.?
  • Have there been recent interactions with patient stakeholders and the FDA? If so, how might that inform the focus of a proposed meeting?
  • To ensure a joint and aligned effort, how will you collaborate with other stakeholders in the given disease area?
  • Is a scientific meeting already being planned (by your organization or by other stakeholders) for the identified disease area? If so, could the planned meeting be informed by an adjacent meeting or session focused on patient input (e.g., PFDD panel during an annual conference)?

THIS NEXT PART IS WHAT HPA HAS ALREADY BEGUN WORKING ON FOR OUR COMMUNITY

How can stakeholders contact the FDA regarding their interest to conduct an externally-led PFDD meeting?

FDA recommends that patient organizations who are interested in conducting an externally-led PFDD meeting initially engage FDA by submitting a Letter of Intent (LOI) that communicates (1) the importance of the meeting in the context of the disease area, and (2) important details regarding the meeting plan. The letter of intent (LOI) should be brief (approximately 3 pages) and submitted approximately 1 year before the anticipated meeting date.