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 in helping FDA understand the context in which regulatory decisions are made 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)?
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 firstname.lastname@example.org.
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
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 will be open to attending 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.
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.
How can stakeholders plan and prepare for an externally-led PFDD meeting?
- Collaboration: The success of an externally-led PFDD meeting will require a joint, aligned effort by multiple patient organizations associated with the disease area, and other interested stakeholders. This effort helps to ensure awareness and increased participation in the meeting by the patient community, enhancing the value of the meeting as an opportunity to hear from the community. FDA encourages multiple patient groups and other stakeholders in a disease space to collaborate in planning, executing, and developing deliverables from the meeting.
- Agenda and Meeting Format: FDA believes that the process, materials, and deliverables of its FDA-led PFDD meetings can serve as a model for engaging patients in a way that is useful within the drug regulatory context. Patients organizations can explore different mechanisms to organize and host these meetings (e.g., public meetings, web-only meetings, and other possible mechanisms to collect public input).
- The FDA PFDD meetings conducted to date have covered a spectrum of disease areas (e.g., fibromyalgia, lung cancer, sickle cell disease) that have tested and support the applicability of this design across a wide range of diseases and related considerations. We recommend reviewing the meeting materials and discussion questions for a variety of the PFDD meetings when planning the design for your meeting.
- Patient Outreach: The key to an insightful, robust, and informative externally-led PFDD meeting is active community outreach to ensure a representative group of patient perspectives.
- Resources: It is at the patient organization’s discretion to determine 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 (e.g, 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 early. FDA does encourage 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 interactions (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.
Will FDA help plan an externally-led PFDD meeting?
FDA is open to providing 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 Program Staff will reach out and liaison directly with the host organization. During the planning of an externally-led PFDD meeting, FDA’s CDER Patient-Focused Drug Development Program 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.
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: FDA’s Voice of the Patient Reports can serve as good examples in terms of scope, content, and level of detail expected in a summary report.)
FDA is piloting an External Resources or Information Related to Patients’ Experience Webpage. FDA welcomes submission of summary meeting reports from externally-led PFDD meetings to this webpage. For more information on this pilot effort, 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.
Where can stakeholders find resources to use in preparation for an externally-led PFDD meeting?
Please visit the Patient-Focused Drug Development meeting page to find the various meeting materials supporting FDA’s PFDD initiative including:
- Meeting agendas and discussion questions (e.g., Autism, Alopecia Areata)
- Several FDA-led PFDD meetings have been paired with a scientific session. For an example of this meeting format, please visit the FDA-led PFDD meeting for patients who have received an organ transplant webpage. The agenda for this meeting can be found here.
- Meeting slides (e.g., Autism, Alopecia Areata)
- Webcast recordings (e.g., Autism, Alopecia Areata)
- Meeting transcripts (e.g., Autism, Alopecia Areata)
- Voice of the Patient Reports (e.g., Autism, Alopecia Areata)
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