Rare disease meeting gets pushed off by HHS
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As hundreds of researchers, patient advocates and policymakers gather in Washington, D.C., this week for an advocacy fly-in on rare diseases, a notable gap has appeared on their schedule: an annual gathering hosted by the Food and Drug Administration and the National Institutes of Health.
Why it matters: The annual two-day gathering known as Rare Disease Day is among the agencies' largest public engagements, and its postponement is raising questions about the agency's priorities.
- It is the second event to be postponed by a health agency since Robert F. Kennedy Jr. took the role of Health and Human Services secretary. Last week, the Centers for Disease Control and Prevention's vaccine advisory committee meeting was indefinitely postponed.
The big picture: Individual rare diseases are, by definition, uncommon. But there are thousands of them, and they affect a large group of patients — more than 30 million in the U.S.
- Rare diseases include: cystic fibrosis, Huntington's disease, muscular dystrophy, sickle cell anemia and Turner's syndrome.
- Just 5% of rare diseases have FDA-approved treatments, and research assessing new drugs' effectiveness faces unique challenges in gaining approval under FDA trial guidelines due to the small number of people impacted by a particular disease.
More than 800 rare disease advocates will already be in D.C. for Rare Diseases Week, and many were planning to attend the FDA-NIH event, said Will Nolan, a spokesperson for EveryLife Foundation for Rare Diseases.
- "The cancelation of this important event for our community underscores the consequences of removing key experts at Health and Human Services Agencies including NIH and FDA," he told Axios in an email.
- "For children and adults with rare diseases, even a momentary pause in funding for these programs or agencies has devastating, life-altering consequences."
- In January, the FDA announced the launch of its Rare Disease Hub under the Biden administration.
HHS, FDA and NIH officials did not respond to requests for comment.
- "Rare Disease Day is important to all of us, and we want to make sure we can fully focus on the event to make it the best that it can be," officials said in a statement posted on the event's website.
- They said the event will be rescheduled in the coming months.
