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Regulatory Focus™ > News Articles > 2020 > 2 > Hahn, FDA Center Directors Discuss What’s Necessary to Better Address Rare Diseases

Hahn, FDA Center Directors Discuss What’s Necessary to Better Address Rare Diseases

Posted 24 February 2020 | By Zachary Brennan 

Hahn, FDA Center Directors Discuss What’s Necessary to Better Address Rare Diseases

The US Food and Drug Administration (FDA) on Monday held a public meeting on rare disease product development, and senior leaders provided some perspective on what their respective centers are working on to help those with rare diseases.

FDA Commissioner Stephen Hahn opened the afternoon discussions by explaining how important it is to drive rare disease drug development by unleashing the data from very small populations. He addressed the need to integrate more data sources to modernize FDA’s approach and how sometimes FDA has to revisit an initial decision based on new data, which is a sign of a healthy, learning organization.

Echoing comments made last month, Hahn also stressed the importance of the patient voice and ensuring FDA is aligned with what patients want. “We will incorporate your input into our decision making,” he said.

The comments come as approvals of orphan drugs, defined as drugs that treat a condition that affects less than 200,000 persons in the US, have risen significantly over the past two years. And the Alliance for Regenerative Medicine said in a report released Monday that there are currently 647 ongoing clinical trials in rare diseases, including more than 500 for gene therapies and gene-modified and cell-modified immunotherapies.

After Hahn spoke, Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, and Jeff Shuren, director of FDA’s Center for Devices and Radiological Health, discussed what their centers are working on with respect to rare diseases.

Marks discussed some of the difficulties companies have in manufacturing cell and gene therapies for such small populations and how even the not-so-rare diseases become rare when you split them apart into their different genetic mutations.

He also noted that CBER is working on guidance to help companies in cases where a product made with one manufacturing technique can be slightly modified to address a different disease, and whether the drugmaker will be able to just modify that initial application.

Dialogue between FDA and the European Medicines Agency on rare diseases is progressing, although Marks said, “It’s not perfect yet.” However, he said he attended a meeting last week with the World Health Organization to discuss more opportunities to harmonize regulations in the rare disease space.

Both Hahn and Marks also mentioned Novartis’ spinal muscular atrophy therapy Zolgensma (onasemnogene abeparvovec) as “truly incredible” and a clear sign of progress.

Woodcock, meanwhile, stressed that the greatest challenge is still that, “We don’t know enough about rare diseases and we can’t find enough patients to enroll for trials.” She also stressed the need to develop more registries and natural history studies.

She noted that FDA has received a number of applications with a single patient in mind (discussed further in this editorial) and that new policies are coming.

On the device side, Shuren stressed the need for new, progressive approvals, which is an idea that’s been floated since at least May 2017. He explained how companies’ return on investments can be difficult in the rare disease space because as soon as a new device is brought to market, other companies can re-engineer around intellectual property, making exclusivity periods “generally meaningless.”

He also noted that there have not been a lot of devices developed under the Humanitarian Device Exemption pathway and “we need to find ways to fix it.” And he stressed that there has been “very little innovation” in the pediatric device space.

FDA Rare Disease Day 2020: Supporting the Future of Rare Disease Product Development


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