The FDA is scheduled to have a closed-door roundtable this week with over a dozen groups advocating for rare disease treatments, according to a senior agency official.
The meeting, scheduled for Wednesday, will connect Food and Drug Administration Acting Commissioner Kyle Diamantas and other senior agency officials with at least 10 rare disease groups for a private conversation about advancing treatments that often struggle to meet the agency’s rigorous approval process, said the official, who requested to speak anonymously because the information isn’t public.
Among the groups invited was the National Organization for Rare Disorders, EveryLife Foundation for Rare Diseases, Friends of Cancer Research, and the Foundation for Angelman Syndrome Therapeutics.
Acting Commissioner Diamantas and FDA leadership constantly meets with stakeholders, the official said, adding that Diamantas has made it a priority to meet with as many parties as possible, especially rare disease groups.
The National Organization for Rare Disorders confirmed that it will be participating in the roundtable.
“NORD has a long history of collaboration and constructive engagement with the FDA and routinely participates in discussions and roundtables with agency leadership and stakeholders across the rare disease and greater healthcare community,” Cheryl Herbert, vice president of marketing a communications, said in an email. “We look forward to continuing to work with the Acting Commissioner and providing a patient-centered perspective on issues important to the rare disease community.”
Other invited groups did not immediately respond to requests for comment.
The meeting comes as the rare disease community has sought more clarity from regulators amid turmoil over recent decisions for rare treatments.
Under former Commissioner Marty Makary, who resigned from his post in May, several initial rejections were issued to companies seeking to market rare disease drugs and gene therapies.
The agency has approved hundreds of drugs to treat rare diseases, but developing them remains a challenge due to regulatory requirements sponsors must meet. Some of the obstacles relate to data collection, limited patient pools and trial designs, and limitations to natural history studies that would show the progression of rare diseases.
Among the pushback against the FDA this year, groups and patients seeking treatments for Huntington’s disease criticized an agency decision that said a biopharmaceutical company needed to conduct another study before its gene therapy is approved.
The company, UniQure NV, said the FDA’s decision was a shift from past guidance.
“We’re seeking consistency,” Amy Gray, president and CEO of the Huntington’s Disease Society of America, said in a press call Monday. “We need consistent decisions about trial design and we need to consider the benefit risk of patients.”
At the same time, drugmakers and groups have felt some optimism about the FDA’s vision to be more flexible with rare treatments.
The agency in February released a new pathway that would allow companies to bypass certain clinical trial requirements for drugs treating ultra-rare conditions.