Patient Engagement Opportunities With the FDA

The Federal Drug Administration (FDA) seeks to engage with patients, families, and the general public in many ways. Since the establishment of the Patient Affairs Staff in 2017, the FDA has actively been expanding its patient reach and including more opportunities for patients to make their voices heard.

The patient voice is a very important aspect of consideration during decision-making processes within the FDA. As this institution seeks to keep patients at the heart of their work, opportunities are expanding to allow patients to make their voices heard. This mission allows the FDA to better understand the priorities and needs of patients from diverse disease communities and backgrounds. Patients can make a direct impact on how the FDA considers an issue and work towards a solution by providing personal experiences and opinions on their unique illness journey.

The FDA currently has established the following programs to provide patients with the opportunity to interact and share their insight.

Patient Engagement Collaborative

This is a collaboration with the Clinical Trials Transformation Initiative that consists of individual and patient organization representatives. This external group comes together to discuss topics about enhancing patient engagement in regulatory discussions with the FDA. The inaugural group of representatives was chosen following an official call for nominations through the Federal Register. These individuals are expected to serve for two to three years.

FDA Rare Disease Patient Listening Sessions

The division of patient engagement began with the National Organization for Rare Disorders (NORD) and is intended to educate FDA review staff about rare diseases. This opportunity allows patients to directly share their experiences with regulatory decision-makers at FDA while educating themselves on the work and mission of FDA. This opportunity is solely open to patients, caregivers, and advocates. The discussion allows those in attendance to share their unique perspectives on treatment, priorities, daily living, burdens, and needs of those living with the rare disease in discussion. Each meeting lasts one to two hours and can be conducted in-person, over the phone, or a combination of the two. A unique topic is chosen for each session which depends on the type of listening session and requestor.
There are two divisions of this new program providing patients and caregivers with the opportunity to speak of their experiences living with rare diseases. In the United States, A rare disease is defined by the Orphan Drug Act of 1983 as a condition that affects fewer than 200,000 people.

1. Patient Requested

This listening session can be requested by patients and patient organizations through the Patients Ask FDA portal here. These are especially helpful when there is an increased and/or common need among patient members within a rare disease community. Meetings such as this can be organized through patient organizations, such as IFFGD. It is best to include a broad range of patients and/or caregivers, providing FDA staff with as well-rounded of a perspective of the rare disease in discussion as possible.

2. FDA Requested

These listening sessions are requested by decision-makers within the FDA when they feel the patient voice is necessary. This can be the result of the FDA deciding they need to increase their understanding at the early stages of research and development. Many rare diseases are being newly researched, and the best way to guide this research is to learn where to best delve into those efforts based on patient and caregiver experience.

When the FDA wishes to conduct a specific rare disease listening session, they will request patients to share their experiences. This may be through a patient organization and will have specific requirements for engagement opportunities. The FDA requested one of these rare disease listening sessions to better understand gastroparesis, this meeting was held in December 2019. To learn more about these previously conducted sessions click here.

FDA staff and patient/caregiver participants have expressed positive feedback as a result of sessions they have conducted thus far.

For Patients Education

On the FDA’s website, the For Patients section is dedicated to educating and informing patients on how they can get involved and make a difference. This includes the Patients Matter Video Series which are short videos created to teach patients and community members about the FDA and ongoing efforts to engage patients. To learn more information on the many ways you can engage with the FDA, please continue to explore the For Patients section of

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IFFGD is a nonprofit education and research organization. Our mission is to inform, assist, and support people affected by gastrointestinal disorders.

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