For Industry

Rare Disease Day 2019

Rare Disease Day 2018

FDA Recognizes Rare Disease Day

On February 28, 2019, FDA joins 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 illnesses affect an estimated 30 million people in the United States. This year’s Rare Disease Day theme is Bridging Health and Social Care in recognition that rare diseases deeply impact patients, caregivers and their families.

FDA's Rare Disease Day Activities

  • FDA Voices: View the blog post,'FDA is Working to Bridge Gaps and Meet Needs for Rare Disease Product Development' by Dr.Janet Maynard from the Office of Orphan Products and Development and Andrea-Furia Helms from the Patient Affairs Staff.  
  • FDA Public Meeting: On Rare Disease Day, FDA is announcing a public meeting to be held Monday, April 29, 2019. This meeting will obtain patients' and caregivers' perspectives on the impacts of rare diseases on daily life and to assess their common experiences that may help the agency and medical product developers further understand and advance the development of treatments for rare diseases, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare disease. View links below for more information and to sign up!
  • Beyond the Diagnosis Art Exhibit: FDA is hosting 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" RDof patients. FDA employees may find the exhibit at White Oak Campus on the rear wall of Building 32 by the badging office from Wednesday, February 13 - Friday, March 29, 2019. Due to campus security restrictions, access to this exhibit is limited to FDA employees and scheduled visitors. 
  • FDA Discusses Rare Disease Natural History Studies: FDA will be joining NIH and others on February 28, 2019 to exchange the latest rare disease information to advance research and therapeutic efforts.

For Rare Disease Day questions, email orphan@fda.hhs.gov

 

Recent FDA Rare Disease Day Activities

  • Twitter Chat: FDA joined the Rare Disease Day community through NIH Rare Disease Twitter Chat. View the Twitter conversation from Friday, February 22, 2019 with #NIHchat or click here to view the thread.
  • Patient Advocacy Sessions: FDA participated in patient advocacy sessions to further discuss ways to promote development of products for rare diseases.  

On Video: Remarks from Patients with Rare Diseases

These patient videos reflect the views of those in the videos and should not be construed to represent FDA's views or policies.

 
 

 

Page Last Updated: 02/28/2019
Note: If you need help accessing information in different file formats, see Instructions for Downloading Viewers and Players.
Language Assistance Available: Español | 繁體中文 | Tiếng Việt | 한국어 | Tagalog | Русский | العربية | Kreyòl Ayisyen | Français | Polski | Português | Italiano | Deutsch | 日本語 | فارسی | English