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

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. EL-PFDD meetings are about patient experiences, perspectives, and needs related to their health or a disease, and not about specific medical products (drug, biologic, or device).

Consider the following 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)?

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.

The letter of intent should be submitted to patientfocused@fda.hhs.gov.

Guidelines for developing a letter of intent are provided here: Guidelines for Developing a Letter of Intent (LOI) for Externally-Led Patient Focused Drug Development Meetings

FDA’s CDER Patient-Focused Drug Development Program Staff leads the externally-led PFDD program and will coordinate an internal review of submitted LOIs on a quarterly schedule as follows:

• LOIs submitted December 1 – February 28 will be reviewed starting March 1
• LOIs submitted March 1 – May 31 will be reviewed starting June 1
• LOIs submitted June 1 – August 31 will be reviewed starting September 1
• LOIs submitted September – November 30 will be reviewed starting December 1

How can stakeholders plan and prepare for an externally-led PFDD meeting?

• Collaboration:

• Partner with the other patient organizations associated with the disease/condition

• Coordinate planning, execution, and developing meeting deliverables together

• Ensure there is broad patient community awareness and participation

• Agenda and Meeting Format:

• The format for a PFDD meeting has been refined over the conduct of many meetings and through significant input from FDA staff, medical product developers, and patient groups. For this reason, FDA strongly recommends that groups planning an EL-PFDD meeting follow the FDA PFDD format provided below.

• The process, materials, and deliverables from FDA-led PFDD meetings should serve as a model for engaging patients in a way that is useful within the drug regulatory context.

• Consider various hosting formats (hybrid or virtual)

• Patient Outreach:

• Conduct active community outreach to ensure a representative group of patient perspectives

• Resources:

• The patient organization is responsible for determining its capabilities and resources for planning an externally-led PFDD meeting. The planning of an externally-led PFDD meeting can be done without being resource intensive. FDA does not encourage enlisting event planners, consultants, scientific writers, or other external resources on your team, especially when resources may be limited.

• The key is to begin planning 9-12 months in advance. FDA encourages patient organizations to consider including externally-led PFDD sessions as part of annual meetings or symposiums to help maximize resources.

• A patient organization may seek financial sponsors (e.g., medical product developers, larger patient organizations) for their externally-led PFDD meeting. To facilitate transparency, the patient organization should identify any financial sponsors in their LOI and any subsequent meeting materials and deliverables.

• The patient organization and any other meeting planning team members and collaborators are also encouraged to disclose any relationships (financial and non-financial) that could be considered relevant to the planning and conduct of the meeting.

• All decision-making related to the externally-led PFDD meeting (e.g., development of agenda, discussion/polling questions, selection of patient panelists) should be done independent of medical product sponsor input.

What does a typical PFDD meeting look like?

• Welcome (5 min)

• Opening Remarks (5 min) (FDA Speaker)

• Clinical Overview and Background on the condition (10 min)

• Overview of Discussion Format (10 min)

• Topic 1: Health Effects and Daily Impacts

• Panel (30 min)

• A panel of 4-5 patients and/or caregivers provide comments to start the discussion on health effects and daily impacts of the condition.

• Large-Group Facilitated Discussion on Topic 1 (65 min)

• Patients and patient representatives in the audience are invited to add to the dialogue.

• Questions:

1. Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact on your life? Examples may include chronic pain, constipation, difficulty concentrating, etc.

2. Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition? Examples of activities may include sleeping through the night, daily hygiene, participation in sports or social activities, intimacy with a spouse or partner, etc.

3. As it relates to your condition, what does a good day look like? What does a bad day look like?

4. How have your condition and its symptoms changed over time?

5. Would you define your condition today as being well managed?

6. What worries you most about your condition?

• Break (30 min)

• Topic 2: Current Approaches to Treatment

• Panel (30 min)

• A panel of 4-5 patients and/or caregivers will provide comments to start the discussion on current approaches to treatment for the condition.

• Large-Group Facilitated Discussion on Topic 2 (65 min)

• Patients or patient representatives in the audience are invited to add to the dialogue.

• Questions:

1. What are you currently doing to help treat your condition or its symptoms? Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modification.

2. How has your treatment regimen changed over time, and why?

3. How well does your current treatment regimen treat the most significant symptoms of your disease? For example, how well do your treatments improve your ability to do specific activities?

4. How well have these treatments worked for you as your condition has changed over time?

5. What are the most significant downsides to your current treatments, and how do they affect your daily life? Examples of downsides may include going to the hospital or clinic for treatment, time devoted to treatment, restrictions on driving, etc.

6. Short of a complete cure, what specific things would you look for in an ideal treatment for your condition?

7. What would you consider to be a meaningful improvement (for example symptom improvements or functional improvements) in your condition that a treatment could provide?

Where can stakeholders find additional resources to use in preparation for an externally-led PFDD meeting?

The Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience webpage provides an alphabetical listing of condition-specific meeting reports and other information related to patients’ experience. Please refer to resources and deliverables from FDA-led PFDD Meetings including:

• Meeting agendas and discussion questions
• Meeting slides
• Meeting recordings
• Meeting transcripts
• Voice of the Patient Reports

What are some valuable meeting deliverables?

Useful meeting deliverables that can inform the FDA and other stakeholders include:

• Webcast Recording
• Meeting Transcript
• Summary Meeting Report (Note: Voice of the Patient Reports from FDA-led PFDD meetings can serve as good examples in terms of scope, content, and level of detail expected in a summary report.)

FDA welcomes submission of summary meeting reports from externally-led PFDD meetings to the Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience webpage. For more information on how to submit a publicly available website link to FDA, please review the Frequently Asked Questions and Guidelines for Cover Page. An externally-led PFDD meeting and any resulting products (e.g., meeting reports) will not be considered FDA-sponsored or FDA-endorsed.

What topics are outside the scope of PFDD?

The purpose of this meeting is to gather a variety of patient perspectives. These topics, while important, are outside the scope of a PFDD meeting:

• The cost or price of treatments and insurance coverage
• In-depth discussions of specific clinical trials or medical products
• Advocating for specific regulatory actions or drug approvals

Will FDA attend an externally-led PFDD meeting?

FDA will be open to attending a well-designed and well-conducted meeting held in the DC Metro area (Virginia, Maryland, Washington D.C.). For meetings conducted outside the DC Metro area, FDA may attend remotely (e.g., webcast). FDA will determine its level of involvement on a case-by-case basis, taking into account a number of factors, including any identified need for a better understanding of patient perspective, recent interactions with patient stakeholders, proposed meeting details, and FDA staff and resource capacity. Consider your organization’s resources and capacity when choosing between virtual and hybrid formats.

The primary purpose of PFDD meetings is to hear directly from patients, their families, caregivers and patient advocates; therefore, as with FDA-led PFDD meetings, any FDA staff in attendance will be primarily in listening-mode throughout the meeting.

Will FDA help plan an externally-led PFDD meeting?

FDA may provide specific recommendations on the planning of the meeting (e.g., development of agenda, discussion/polling questions). After a review and assessment of an LOI, FDA’s CDER Patient-Focused Drug Development Staff will reach out and liaison directly with the host organization. During the planning of an externally-led PFDD meeting, FDA’s CDER PFDD Staff will serve as your primary point of contact and be a helpful resource (e.g., monthly check-in calls).

FDA will not provide meeting space, day-of staff support (e.g., meeting facilitator), or financial support for externally-led PFDD meetings.