During a public hearing Wednesday to discuss a newly proposed rare disease framework, FDA center director Michael Davis, M.D., Ph.D., said the agency aims to design a scheme that “stands the test of time.”
Earlier this month, the FDA announced the Rare Disease Framework Initiative to reimagine the development of drugs and biologics for rare diseases. The agency's new framework is designed to speed the development of safe and effective therapies through clearer guidance, greater patient engagement, expert collaboration, and innovative regulatory and scientific approaches.
The initiative is spearheaded by the FDA’s newly created Rare Disease Innovation Hub, a point of collaboration between the Center for Devices and Radiological Health, the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER), which Davis leads.
Although more details of the framework are still in the works, Karim Mikahil, director of CBER, said: “The vision here is for this to be a true quantum leap, not another program, not another step, but an actual true built from scratch all the way bottom up with every stakeholder step.”
When thinking about rare disease regulation, the term flexibility gets thrown around often, but Mikhail argued that “you cannot use flexibility to deliver on predictability and consistency.”
“When that term is used, we feel like we're driving out of the highway on the shoulder, and we're going to hit a concrete wall,” he added during the Oct. 28 meeting. “It feels like you're making a very big exception, but it's not an exception. It's just the reality of that rare disease.”
For biotechs developing drugs for rare and ultra-rare diseases, regulatory predictability is key. With this new framework, the FDA hopes to provide clear guidance and clinical milestones for drugs in development for rare disease so that by the time the drug faces an advisory committee, there is no need for flexibility.
“We're not ripping up and throwing away what we've done in the past. We've done some fantastic things,” Amy Comstock Rick, director of the FDA's rare disease innovation hub, said during the meeting.
“But with the innovation of science, evolution of FDA, strong support from leadership right now, it really is time to take a fresh look and begin to weave the many rare disease programs that we have together and make sure that FDA's regulatory process really aligns with what's possible scientifically,” she added.
This summer, the FDA was busy with advisory committee meetings. In as little as 48 hours in July, a panel of experts voted against Capricor’s cell therapy for Duchenne muscular dystrophy (DMD) and voted for Replimune’s previously rejected oncolytic virus, RP1, to be approved in melanoma.
When a proposal makes it to an advisory committee meeting, the patient voice input is often too late to be considered, Rick explained.
With the new framework, the FDA is considering when the right time is to bring that patient voice in to talk about risk tolerance, burden of disease and to help educate a review team on the rare disease in question.
“These are big questions, and I think we'll have to give a lot of thought to how to manage this, but it is an absolute must-have,” Rick said.