This is an update to a story El Tejano first reported on Oct. 17.
https://www.eltejanorgv.com/only-5-of-rare-diseases-have-an-approved-drug-the-fda-says-it-will-rethink-the-rules/
The U.S. Food and Drug Administration has a new plan for rare diseases. The man in charge says it is at the top of his list. His name is Kyle Diamantas. He is the acting FDA commissioner. He spoke on Monday, Oct. 26.
He was at a summit in Washington, D.C. A group called the National Organization for Rare Disorders, or NORD, holds it. The summit runs Oct. 25 to Oct. 27. NORD says more than 900 people are there.
"We've got to make sure that that process is in place," Diamantas said. "We're working really hard to do it." He also said, "It's a top priority of mine."
The plan is called the Rare Disease Framework Initiative. A framework is a set of rules and steps. The FDA announced it on Oct. 14. The agency says it wants to rethink how drugs for rare diseases are made. It also put out a report that explains the plan.
Why does this matter? The FDA says only about 5 percent of rare conditions have an approved treatment. He spoke about it on Oct. 14 too. He said, "Patients and families living with rare diseases have few or no approved treatment options."
NORD has a number too. It says more than 30 million children and adults live with rare diseases.
Now the FDA wants to hear from the public. It will hold a meeting on Wednesday, Oct. 28. It will be at the FDA's White Oak campus. The agency wants to hear from patients. It wants to hear from advocates. It wants to hear from the drug industry too. The Reagan-Udall Foundation is helping to run it.
