A new FDA strategy is targeting total elimination of the existing orphan designation request backlog by mid-September and committing to respond to all new requests for it within 90 days of receipt. To get there, Gayatri Rao, director of the Office of Orphan Products Development (OOPD), told BioWorld, her group is reorganizing review staff, working to educate sponsors about how to file better designation requests, and establishing a new FDA Orphan Products Council.

The effort is the first of several to improve the agency's support for rare disease drug development and review under the agency's new Medical Innovation Development Plan, an effort that is also expected to, within the next six months, yield new guidance on the clinical evaluation of targeted therapies for rare disease subsets.

The number of orphan drug designation requests made under the Orphan Drug Act has steadily increased over the past five years. In 2016, the OOPD received 568 new requests for orphan status – more than double the number of requests received in 2012. This year, the number of incoming applications in down a bit, but not by much, Rao said. (See chart, below.)

Orphan designation qualifies the sponsor of a drug for various development incentives, including tax credits for clinical trial costs, relief from the prescription drug user fee if the indication is for a rare disease or condition, and eligibility for seven years of marketing exclusivity upon approval. Those perks, as well as the high market prices an approved orphan drug can command, have driven growing interest in securing the status.

But even as demand for the designation has climbed, other responsibilities for OOPD's "lean-staffed" office of 33 have increased, too, Rao said. Two of the six programs her office runs — the rare pediatric disease designation program in 2012 and last year's orphan products natural history grant program — were added to its plate in just the past five years.

All along, science that allows researchers to target rare diseases that were previously not readily amenable to therapy has driven greater activity in the rare disease space. The scientific progress is good news, the report says, calling it "a reflection of substantial medical progress" and on "our better understanding of the genetic basis of diseases, which unlocks our ability to define and target rare disorders."

"Like any other organization," she said, we "really have to take an internal look to make sure we efficiently staff folks to work on the programs that best marry their expertise." The reorganization process, already underway, has led to tasking some OOPD employees to focus just on one program rather that a matrix of responsibilities, she said.

The office is also working to better educate sponsors about how to file quality orphan designation applications in an effort to reduce the number of review cycles needed for each request by developing web-based training for sponsors. Last summer, Rao said that, on average, a request for designation went through two such review cycles, meaning OOPD staff needed additional information from the sponsor prior to determining the outcome of the request. "I can't tell you the number of applications we get that are just not good quality," she said.

Other work included in the new Orphan Drug Modernization Plan includes the creation of a "SWAT Team" of senior, experienced, and proficient OOPD reviewers to focus on designation requests as well as the creation of a new streamlined "Designation Review Template" to facilitate consistent and efficient reviews of new designation requests.

A new Orphan Products Council will address scientific and regulatory issues related to orphan products to ensure a consistent approach to regulation. It will include participation from senior management across the FDA, Rao said. Depending on what the issue is, it might draw in the directors of CDER, CBER, CDRH, as well as other agency officials for consideration and decision-making.

There are no statutory or regulatory deadlines for review of requests for the industry-coveted orphan drug designation, nor are any being created by the new plan. So the new goals are guides rather than requirements. But they appear to reaffirm and recommit OOPD to its aspirations to speed up clearance of its backlog. The group had earlier sought to review three-quarters of orphan designation requests within 90 days, but was forced in July 2016 to move the goalposts back a bit to be within 120 days of receipt instead.

How well OOPD does in reaching the new more stringent goals should be readily evaluable once a new "tracking dashboard" it calls for is implemented. A full time line of progress on the planned activities is also due by the end of August.

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