“Our work is taking place during an inflection point in both science and policy,” FDA Commissioner Scott Gottlieb said in a statement. “We have more opportunity to deliver on the promises of science than at any time before. Gene- and cell-based therapies hold the opportunity to treat a wider range of debilitating disorders, and deliver outright cures to intractable diseases.”
Opioids
As the number of opioids sold to pharmacies, hospitals and doctors’ offices nearly quadrupled from 1999 to 2010, and deaths from immediate-release opioids have more than quadrupled since 1999, FDA said in the roadmap that it needs “to make sure that patients are prescribed these drugs only when properly indicated, and that when prescriptions are written, they are for dosages and durations of use that comport closely with the purpose of the prescription.”
The agency will also look to help push the US to use opioid drugs with improved formulations that are harder to manipulate and abuse, advance other drugs and devices that can treat pain and are less likely to be addictive, and create new paths for the development and approval of better treatments for addiction.
Generic Competition
In addition to establishing an inter-agency working group with the Federal Trade Commission to better stop companies from gaming the patent and exclusivity system, the agency said it will issue new draft guidance in the first quarter of this year on its process for determining whether to grant a waiver for adopting a single, shared-system risk evaluation and mitigation strategy (REMS).
“We expect that this guidance will be helpful in situations where the negotiations of a single, shared-system is lengthy, complex, or otherwise creates a burden for stakeholders. We will also advance draft guidance on the development of shared-system REMS, to make the process for developing a shared REMS more efficient,” the roadmap said.
FDA also said it will also issue new guidance on how to more efficiently handle citizen petitions, including so-called “blocking” petitions that are subject to section 505(q) of the Food, Drug & Cosmetics Act. In the past week, the agency has denied three such petitions.
“While the record shows that citizen petitions have rarely delayed specific generic drug approvals, there is no doubt that the deadlines associated with 505(q) petitions can add to resource burdens on the generic review process. We expect our new guidance will allow for a more efficient approach to 505(q) petitions, and allow us to focus more reviewer resources on the approval of generic drugs,” the roadmap said.
And as previously discussed in November, FDA is also looking to address complex generics, and to focus “on the overall efficiency of our generic drug review program.”
On the biosimilars front, FDA noted that it will make the process for developing and approving biosimilar drugs more efficient.
“As part of these efforts, we will create better incentives for the adoption of safe, effective, and high-quality biosimilar drugs. These policies will be part of a new Biosimilar Innovation Plan (BIP) that we will advance over the coming year,” the roadmap said.
Devices
The roadmap’s highlights for medical device companies include: Creating a new Total Product Life Cycle Office in CDRH, first discussed in September, to minimize “organizational layers of review throughout the device life-cycle, achieving more efficient work processes and allowing employees to better leverage their knowledge of pre- and post-market information to optimize decision-making.”
The roadmap also discusses the advancement of an alternative 510(k) pathway for reviewing medical device submissions, through legal experts have questioned the legality of such a pathway.
The agency also said it will advance a new framework to balance pre- versus post-market requirements for devices targeted to significant unmet medical needs and it will develop and advance the use of in silico tools and models to evaluate device performance and patient outcomes as part of the Medical Device Innovation Consortium.
Digital Health and Workforce
Building off its work on the precertification pilot project, FDA said it is seeking to develop a more tailored approach to accommodate the rapid pace of digital innovation while still taking a risk-based approach.
In addition, FDA said it will embrace new predictive toxicology methods and computational modeling across the agency’s product centers and making new investments in the FDA’s high-performance, scientific computing.
And the roadmap noted several efforts already underway to build and retain FDA’s workforce.
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