On this page:
- FDA's Rare Disease Day Activities
- Personal stories about rare diseases
- Artfully Rare - Beyond the Diagnosis
- Other FDA Activities Relevant to Rare Diseases
- Contact Information
On March 5, 2021, FDA joined the global observance of Rare Disease Day, which was created to raise awareness about the 7,000 known rare diseases, many of which have no treatment. Although each rare disease affects fewer than 200,000 Americans, in total, these disease affect an estimated 30 million people in the United States.
See FDA Shows Sustained Support of Rare Disease Product Development During the Public Health Emergency for more information.
FDA Public Meeting: FDA hosted a virtual public meeting on Friday, March 5, 2021. The purpose of this meeting was to highlight strategies to support rare disease product development. Patients, patient advocates, researchers and medical product developers attended this virtual public meeting on rare disease product development. During presentations and panel discussions various stakeholders shared their perspectives on and experiences in rare disease product development.
Patients with rare diseases offer a unique perspective about their condition and their experience is important to understand to help advance therapies for rare diseases. See Personal stories about rare diseases for videos capturing patients’ stories and FDA experts’ work on rare diseases.
FDA’s Rare Disease Day 2021 public meeting included portraits from the Beyond the Diagnosis Art Exhibit to showcase portraits of diverse children with rare diseases. This exhibit has traveled to medical schools, research institutes and hospitals around the globe to encourage the medical community to look "beyond the diagnoses" of patients. Beyond the Diagnosis unites art and science to inspire research and innovations of treatments for people living with rare and neglected diseases.
SHIP-MD 2021 (Virtual Workshop): The Critical Path Institute (C-Path) in collaboration with FDA's Center for Devices and Radiological Health (CDRH), AdvaMed, the American Academy of Pediatrics (AAP) and multiple pediatric health system leaders, hosted the System of Hospitals for Innovation in Pediatrics – Medical Devices (SHIP-MD) Virtual Workshop held in February 2021. The workshop focused on the acceleration of medical device development for the unique needs of children.
Evaluating RWE from Observational Studies in Regulatory Decision Making (Virtual Workshop): The FDA and the Duke-Margolis Center for Health Policy will hosted a two-day virtual workshop on February 16-17, 2021, focused on considerations about observational (non-interventional) study design and conduct when using evidence from observational studies to inform regulatory decisions. See the workshop recording for more information.
Patient listening sessions: Listening sessions are one of many ways patients, caregivers and their advocates can share their experience with a disease or condition by talking directly with FDA staff. See FDA patient listening sessions for more information.
Contact OOPDorphanevents@fda.hhs.gov or 301-796-7634 for more information.