Gottlieb Faces Multiple Challenges at FDA

The Senate moved quickly to confirm Scott Gottlieb this month as FDA commissioner, deciding that his strong understanding of agency policies and programs and impressive medical and policy credentials outweighed his close ties to industry that raised conflict-of-interest concerns. Gottlieb comes to FDA as agency leaders begin the considerable task of implementing important policy changes authorized by the 21st Century Cures legislation. But he benefits for now from a notable budget increase for the coming months, and indications that Congress will move to quickly pass legislation reauthorizing critical new FDA user fee programs for drugs, medical devices, generic drugs, and biosimilars. Leading Senators are pushing for a “clean” measure that will move through the legislative process before summer.

That said, Gottlieb faces enormous challenges at FDA. The White House has announced a federal hiring freeze and stiff deregulatory policies that threaten FDA efforts to attract the scientists and experts it needs to efficiently evaluate ever-more-complex medical products and raise uncertainty about which standards and rules will remain in effect. Outside pressure for FDA to do more to reduce high drug costs will focus more attention on agency efforts to bring more innovative generic drugs and other competitive medicines to market, which may stretch FDA’s authority and capabilities.

The opioid epidemic is a major challenge for everyone, as FDA seeks to balance the critical need for effective treatments for patients living with pain with efforts to limit public access to medicines subject to abuse and misuse; more abuse-resistant formulations are in the works, as well as formulations able to prevent death from overdose. Similarly, FDA is at the forefront of efforts to develop new vaccines and therapies to combat deadline infectious diseases, a role that requires new research capacity and regulatory flexibility. FDA officials are about to implement a major overhaul of its field force as part of the Program Alignment initiative in the works for several years. At the same time, industry critics oppose recent proposals for FDA regulation of tobacco and laboratory-based diagnostic tests.

During the confirmation process, Gottlieb noted an interest in greater FDA transparency, which could involve public release of “complete response” letters on nonapproval of  drug applications. He voiced support for policies to bring more innovative generic drugs and biosimilars to market, and for loosening restrictions on the communication of off-label information. He earned points by discounting claims of a link between vaccines and autism, as expressed by the White House, and opened the door to more modern clinical trials that may not always require randomization, but will maintain FDA’s gold standard for safety and efficacy.

As acting FDA commissioner Stephen Ostroff noted in a speech before the annual meeting of the Food and Drug Law Institute (FDLI) last week, FDA faces “many uncertainties going forward.” He cited a “charged regulatory environment” and anticipated threats to FDA’s budget. At the same time, he acknowledged an abundance of scientific opportunities for FDA programs to support a healthier future of all. Ostroff indicated that he was only too happy to step aside, which leaves Gottlieb front and center in efforts to realize those hopes and expectations.