FDA Commissioner Vows to Eliminate Backlog of Orphan Drug Designation Requests

Regulatory NewsRegulatory News | 20 June 2017 |  By 

At a Senate hearing on President Donald Trump's FY2018 budget request for the US Food and Drug Administration (FDA), Commissioner Scott Gottlieb vowed to eliminate the backlog of orphan drug designation requests and said he will soon release "modern and risk-based" tools for assessing new treatments, especially for rare diseases and conditions with no effective treatments.

"Right now we have a backlog of about 200 orphan drug designation requests where we haven't responded to sponsors," Gottlieb said, noting that the agency plans to "completely eliminate" this backlog within 90 days and will strive to address all future orphan designation requests within 90 days.

Gottlieb said the agency plans to streamline its processes for reviewing orphan drug designation requests by developing an improved request template.

In addition, Gottlieb said the agency will update a number of its drug development guidance documents on targeted therapies, including "guidance on clinical trial enrichment strategies to improve efficiency and adaptive trial designs."

And, within six months, Gottlieb said the agency will issue new guidance discussing the agency's approach to evaluating targeted therapies for rare disease subsets.

"This new policy will address targeted drugs and how we can simplify the development of drugs targeted to rare disorders that are driven by genetic variations, and where diseases all have a similar genetic fingerprint even if they have a slightly different clinical expression," Gottlieb said.

Anti-Competitive Practices

During the hearing, Gottlieb also addressed questions on practices that delay generic competition, such as risk evaluation and mitigation strategy (REMS) abuse and distribution contracts that prevent generic drugmakers from obtaining drugs for bioequivalence testing.

"There is no question there are places where companies do take advantage of rules meant for one purpose as a way to gain commercial advantage," Gottlieb said, noting that he believes the agency can address the issues through administrative action.

"I don't want to be playing whack-a-mole with companies either," he added. "I want to have in place a consistent framework and a consistent set of rules that prevent these kinds of abuses."

In the near term, Gottlieb said the agency is planning to hold a public workshop to get insight on "where other people believe there are practices that could be forestalling access to generic competition [and] where branded companies might be taking advantage of certain rules."

Trump FDA Budget

Sen. Jon Tester (D-MT) asked Gottlieb how the FDA will manage to fulfill new generic drug review provisions included in the Senate's user fee reauthorization bill under the President's budget proposal, noting that the Congressional Budget Office (CBO) estimated the agency would need to hire an additional 500 full-time-equivalent employees to meet those mandates.

"I wasn't involved with the formulation of the budget. I would have to obviously make it work if the budget were passed as it was proposed," Gottlieb responded.

Sen. John Hoeven (R-ND), meanwhile, said he was "concerned that [Trump's budget] request relies on a significant increase in user fees that is not feasible and unlikely to gain congressional approval," later noting that he believes Congress will ultimately pass the previously negotiated user fees.



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