- The Food and Drug Administration a few months ago kicked off a plan for sifting through its large backlog of orphan drug designations. On Tuesday, the agency announced it had completed that task.
- Regulators cleared the backlog, which amounted to roughly 200 requests, on Aug. 28 — nearly a month ahead of the anticipated Sept. 21 deadline, according to a blog post from FDA Commissioner Scott Gottlieb. The initiative fits into broader goals that Gottlieb set when he took control of the agency, namely optimizing its drug review processes.
- Gottlieb also unveiled new measures for preventing requests from piling up in the future. They included a revamped workflow for reviewers to curb inefficiencies, as well as an upcoming public meeting that will help the FDA evaluate how it grants orphan drug designations and whether those procedures align with the aims of the Orphan Drug Act (ODA).
Rare disease drug development has revved up in recent years, bringing with it more orphan drug designation requests to the FDA. The requests have increased steadily over the last five years, hitting a record high in 2016 when the agency received nearly 570 of them.
The uptick is due to several factors. Drugmakers often claim that it stems from the pharmaceutical industry's dedication to patient populations with high unmet medical needs. But opponents argue that the real incentives lie with the high price tags that companies can stick on their rare disease products or the medley of tax and exclusivity perks granted to those products through the ODA.
Gottlieb, since taking helm at the FDA, has made reforming orphan drug review processes one of his central focuses. And so far, that focus has manifested in the Orphan Drug Modernization Plan. Established in late June, the plan sought to accomplish two goals: addressing the backlog of orphan drug designation requests and ensuring the agency responds to all new requests within 90 days of receipt.
With the first goal taken care of and the FDA starting to tackle the second, Gottlieb is taking a closer look at the means through which companies may be gaming orphan drug development system.
"For all the success of the ODA, there’s been criticism that some sponsors are using designations as a way to sidestep other important public health goals set out by Congress," Gottlieb wrote in the Sept. 12 blog post. "We need to make sure our policies take notice of all of these new challenges and opportunities."
In response, Gottlieb said the FDA will issue guidance about such abuses, including some about a discrepancy between the ODA and the Pediatric Research Equity Act that allowed drugmakers to exploit a loophole for skirting their obligations to study drugs in the pediatric setting.
"Consider a condition like inflammatory bowel disease," Gottlieb wrote. "A drug may be approved to treat the large population of adults with the condition. Then the same drug may be granted an orphan designation to treat the much smaller population of a subset of children suffering from the same condition."
"But once a drug receives an orphan designation for a pediatric population of the adult disease, the drug then becomes statutorily exempt from the requirements of PREA," he added. "This occurs even in cases where the sponsor never goes on to develop the drug for this pediatric use."