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FDA Continues Important Work to Advance Medical Products for Patients with Rare Diseases

Graphic with dark blue background and White cross walk lines. It also has white text, aligned to the left of the image, that reads "FDA's Rare Disease Day 2023."

By: Robert M. Califf, M.D., Commissioner of Food and Drugs and Sandy Retzky, D.O., J.D., M.P.H., Director, Office of Orphan Products Development 

Patients with rare diseases are experts in their health condition. They provide a unique perspective, and understanding their experience is an essential element to our public health mission.  

The U.S. Food and Drug Administration is committed to advancing treatment options for patients with rare diseases. More than 7,000 rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S. Approximately half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments. 

Robert M. Califf M.D., MACC
Robert M. Califf, M.D.

Each year, Rare Disease Week is observed during the last week of February. The FDA will host its virtual Rare Disease Day on Monday, February 27, 2023. The goal of this year’s Rare Disease Day is to explore ways to engage and collaborate with patients and patient advocates to support the development of medical products for rare diseases. We want patients to connect with the FDA and stay involved with our outreach and engagement programs to ensure that the patient voice informs medical product development and regulation. 

The FDA’s theme this year is “Intersections with Rare Diseases – A Patient-Focused Event.” We will share four different topics that intersect with rare diseases underscoring the FDA’s agency-wide approach to engaging stakeholders and advancing medical product development for rare diseases:

  • Clinical trial challenges with rare diseases focusing on enrolling children and improving diversity.
  • FDA initiatives to advance product development for rare diseases.
  • Opportunities for patient engagement with the FDA.
  • The role for medical students in shaping rare disease care.

Patient Engagement and Progress in Rare Disease Treatments

This year marks two important milestones for the rare disease programs at the FDA. First, it’s been five years since the agency signed a memorandum of understanding with the National Organization for Rare Disorders (NORD) and launched the FDA Patient Listening Sessions program. Over the last five years, the Patient Listening Sessions program has helped the agency gain a greater understanding of the experiences, needs and priorities of patients and caregivers. Going forward, this program will continue to provide a platform for patients’ voices to be heard and help the FDA better understand patients’ priorities. Patients play a unique role by sharing their experiences to help inform rare disease medical product development and regulation.

The second milestone this year is the 40th anniversary of the Orphan Drug Act. This law provides incentives for the development of drugs and therapeutics for patients with rare diseases, and it also provides for government grants to help develop medical devices, including diagnostics, for rare diseases. 

The Orphan Drug Act has changed rare disease research and product development. Since the law was enacted, the agency has approved more than 550 unique drugs and biologics for over 1,100 rare disease indications. 

Bringing the Rare Disease Community Together

Sandra Retzky, D.O., J.D., MPH
Sandra Retzky, D.O., J.D., MPH

Rare Disease Week is an important opportunity to reflect on the great progress we have made and the important work ahead. The FDA will once again participate in NORD’s Light Up for Rare campaign by illuminating Building 1 at the FDA headquarters and Building 50 at the FDA’s National Center for Toxicological Research in Jefferson, AK, during Rare Disease Week. This symbolic activity is designed to shine a light on the experiences and needs of the rare disease community. 

While the rare disease community has made significant strides, at the FDA we recognize that thousands of rare diseases still do not have approved treatments. We remain committed to supporting rare disease research, engaging patients and caregivers, and continuing to enhance our review processes to help advance medical products for rare disease patients. The FDA will continue our critical work with all stakeholders, including patients, patient advocates, product developers and researchers, to help speed the development and review of safe and effective drugs, biologics, and devices for rare diseases. 

The FDA’s Rare Disease Day will be an important opportunity to engage with patients and caregivers, and to hear from panel experts on a range of topics related to rare diseases. Please register today to participate in this virtual event.


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