Scott Gottlieb has not shied away from tough drug development and access issues since taking the helm of FDA in May 2017. To help mitigate the nation’s opioid drug epidemic, he has backed policies to reduce inappropriate prescribing and internet marketing of illegal drugs. And to continue last year’s notable gains in bringing important new drugs and gene therapies to market, he has promoted innovative clinical research methods and strategies to make biopharmaceutical testing more efficient.
Gottlieb’s overriding goal is for these and other initiatives to achieve “a good balance between innovation and access,” he told Pharm Exec in a recent interview. The commissioner has loudly challenged high drug prices, blasting brand manufacturer “shenanigans” for blocking generic drug development. While there are more things that FDA can do to create a more competitive drug market place, “there’s no one silver bullet,” he commented, noting that FDA will continue to promote reforms “that will have an impact” (see sidebar at bottom).
In tackling these and other contentious issues, Gottlieb has quieted critics on all sides. Democrats initially feared an industry bias, but have been impressed by his initiatives to advance public health and challenge drug prices. Republicans hoping for a free-market deregulator support his attack on opioid abuse and efforts to speed innovative therapies, devices, and diagnostics to patients.
Gottlieb also has built support in Congress, as seen in the timely enactment of new user fee programs last year and slight modifications in “right-to-try” legislation to lessen its erosion of the FDA approval process and maintain patient protections. Certainly, many of Gottlieb’s initiatives are not new and reflect years of hard work by FDA staff. But vocal support from the commissioner has accelerated action on many fronts.
A clear sign of achievement for Gottlieb is the $400 million boost in FDA’s budget plan for 2019, a notable shift from earlier administration proposals to sharply cut agency appropriations. To convince the legislators to approve the requested funds, FDA has outlined how the added resources will advance biomedical innovation (view the agency’s budget plan for 2019 here: https://bit.ly/2HqnDj0). There’s a prime initiative to develop data and analytical tools to better utilize real-world evidence in accelerating medical product development, plus an expanded “knowledge management system” for evaluating new drugs more rapidly and consistently. Additional funds will support FDA’s Oncology Center of Excellence and advance new treatments for rare diseases. And
access to generic drugs will gain from further automation of the review process and a proposal for legislation to prevent “first filers” of generic drug applications from blocking subsequent competitors.
Gottlieb also highlights the importance of modernizing the manufacture of drugs and biologics and vaccines to produce needed therapies more reliably and at lower cost. A related initiative is to ensure quality production of safe compounded drugs from outsourcing facilities to provide more reliable sources of needed products, particularly in shortage situations.
“We’re now at a tipping point” for advancing drug quality, Gottlieb told Pharm Exec. The agency will develop further guidance and standards to reduce uncertainty for industry in adopting high-technology platforms. And more highly trained field investigators will inspect for quality and those aspects of manufacturing that create risks.
This focus on modern manufacturing fits Trump administration efforts to encourage “re-domesticating some of the manufacturing that has moved overseas,” Gottlieb explained. He noted that high-quality, small-footprint manufacturing platforms have lower labor costs more suited to US operations. Advances in how FDA oversees medical device production similarly bolsters a “Bring Medtech Manufacturing Home” initiative that encourages firms to retool production operations in the US.
FDA will be hard pressed in the coming months to realize last year’s gains in drug development and approvals. But the commissioner shows no sign of sidestepping hot issues, such as nicotine levels in cigarettes, oversight of independent testing labs, and food contamination outbreaks. FDA faces numerous deadlines for implementing the 21st Century Cures legislation, including provisions to support regenerative medicine and to speed the development of new cancer treatments, personalized medicines, and gene therapies. At the other end of the spectrum is a proposal for new user fees to support more efficient oversight and approval of over-the-counter medicines.
Transparency remains a thorny issue, as FDA promotes wider disclosure of data and analyses related to regulatory, safety, and enforcement decisions. Yet complete response letters issued when FDA delays an approval remain under wraps, as industry seeks to keep confidential certain regulatory decisions. And although Gottlieb has supported more liberal FDA regulation of commercial speech in the past, he has said little on this topic as commissioner. Gottlieb expects final guidance on communicating healthcare economic information to payers will encourage “more vigorous discussion” of the economic value of medicines with less FDA oversight, reducing the need for the agency “to police those discussions involving sophisticated parties.”
Combating the opioid epidemic remains a prime challenge, as physicians push back on proposals to limit opioid prescribing and mandate provider education. FDA has a key role in developing less addictive pain medicines and new treatments for substance abuse and has gained added resources to detect and block imports of counterfeit or illegal controlled substances at the border. New legislation may strengthen the agency’s authority in these areas and in requiring limited-dose packaging.
An intriguing issue for Gottlieb is how current policies and practices encourage global “free riding” on US biopharmaceutical R&D. New FDA data indicates that other industrial nations pay more for generic drugs than the US and less for innovator therapies—“but not a lot less” when adjusted for net price, he points out. The payments should be reversed, Gottlieb says, as the current situation is a “recipe for destroying innovation.
Jill Wechsler is Pharmaceutical Executive’s Washington Correspondent. She can be reached at [email protected]