As part of FDA’s commitments under the Prescription Drug User Fee Act reauthorization of 2012, FDA has taken several steps to inform assessment of benefit-risk in CDER’s regulatory decisions concerning new drugs. The Patient-Focused Drug Development (PFDD) initiative is part of FDA commitments under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The 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 an important opportunity to hear directly from patients, patient advocates, and caretakers 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 recognizes that there are many more disease areas than can be addressed in the planned FDA meetings under PDUFA V. There has been growing external interest in expanding efforts to gather patient input in support of drug development and evaluation.
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 through Patient-Focused Drug Development as a model.
How might FDA participate in an externally-led meeting?
An externally-led PFDD meeting and any resulting products (e.g., surveys or reports) will not be considered FDA-sponsored or FDA-endorsed, and FDA does not guarantee specific involvement in such meetings. However, FDA will be open to participating in a well-designed and well-conducted meeting. FDA may consider in-person attendance for meetings held in the DC Metro area. FDA may also be willing to provide specific recommendations on the planning of the meeting and the development of any meeting outputs. 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 capacity.
What should stakeholders consider when deciding to pursue an externally-led 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 drug 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 outlined in its PDUFA V PFDD disease area meeting identification process:
- 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)
- 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?
- 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?
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 (recommended approximately 5 pages) and submitted approximately 1 year before the anticipated meeting date. The letter of intent should be submitted to email@example.com. FDA’s CDER Office of Strategic Programs 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
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
How can stakeholders plan and prepare for an externally-led PFDD meeting?
The success of an externally-led patient-focused meeting will typically require a joint, aligned effort by multiple patient advocacy groups 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 believes that the process, materials, and deliverables of its FDA PFDD meetings can serve as a model for engaging patients in a way that is useful within the drug regulatory context. Patients groups 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.
The meetings conducted to-date have followed a similar but tailored design, which takes into account the current state of drug development, specific interests of FDA review division, and the needs of the patient population.
What are some valuable meeting deliverables?
Useful meeting deliverables that can inform the FDA and other stakeholders include:
- Webcast Recordings
- 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.)
Where can stakeholders find resources to use in preparation for the 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:
- Webinar: Background on FDA and Patient-Focused Drug Development
- Federal Register Notice, particularly discussion questions
- Meeting agenda
- Meeting slides
- Webcast recordings
- Meeting transcript
- Voice of the Patient Report