FDA introduces orphan drug pilot scheme
The US Food and Drug Administration (FDA) has introduced a pilot scheme for processing orphan designation requests more efficiently, as part of its commitment to support and expedite the development of rare disease products.
Scott Gottlieb, commissioner of the FDA, announced the agency’s actions yesterday, February 27, a day ahead of “Rare Disease Day”, which is aimed at raising awareness about rare diseases and their impact on patients’ lives.
The pilot scheme includes a new form intended to make the submission process easier for sponsors to complete designation requests and make it more efficient for the FDA to review submissions. This includes an online tutorial to guide sponsors through the process.
The FDA is also entering into a memorandum of understanding with the National Organization for Rare Disorders to conduct outreach with the FDA’s new patient affairs staff on “ways to enhance the incorporation of patient experience into regulatory discussions”.
As part of this, the FDA is planning a joint series of listening sessions on rare diseases to provide a common understanding of patient needs.
The agency is also planning a public meeting to help it “prepare for the changing landscape of orphan drug development posed by the growth in targeted therapies and molecularly defined diseases”.
In September last year, LSIPR reported that the FDA had cleared the backlog of orphan drug designation requests, one month early.
Three months before, in June, the agency promised to eliminate the backlog of around 200 orphan drug designation requests that were older than 120 days by September 21, 2017. It also promised that this backlog would never occur again.
The agency is also implementing a 90-day timetable for processing new designation requests.
“Over the course of 2018 we’ll continue our efforts to increase the consistency and efficiency of our reviews of rare disease products. We remain committed to supporting rare disease research on diagnostics, therapies, and potential cures,” said Gottlieb.
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