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Volume 31, Issue 5
FDA’s commissioner addresses opioid abuse, drug costs, and manufacturing quality.
Scott Gottlieb has seen a whirlwind of activity since taking the helm of FDA in May 2017. He blasted brand manufacturer “shenanigans” for blocking generic-drug development and cited “pernicious” rebating schemes that expose consumers to high out-of-pocket costs for medicines. To help mitigate the nation’s deadly prescription drug epidemic, he has backed policies to reduce opioid prescribing and to develop less addictive pain treatments. And to continue last year’s notable gains in bringing important new drugs and gene therapies to market, Gottlieb has promoted innovative clinical research methods and other strategies to make drug testing more efficient.
The commissioner also told BioPharm International that he is revving up FDA efforts to encourage industry conversion to advanced manufacturing systems able to scale up quickly to address public health issues. New policies to ensure quality manufacturing can facilitate the development of competitive biosimilars and prevent shortages of critical medicines to protect public health.
These and other initiatives have quieted critics on all sides. Democrats initially feared an industry bias but have been impressed by the commissioner’s criticism of drug pricing and his campaign to reduce nicotine in cigarettes. Republicans hoping for a free-market deregulator admire his efforts to combat opioid abuse and his support for innovation to speed new medicines, devices, and diagnostics to patients. Certainly, many of Gottlieb’s initiatives are not new and reflect years of hard work by FDA’s professional staff. But vocal support from the commissioner has accelerated action on many fronts.
A clear achievement is a proposed $400-million boost in FDA’s budget for 2019, as compared to the administration’s plan to cut agency appropriations by $700 million a year ago. The commissioner has gained support for FDA in Congress, which helped enact crucial user fee reauthorizations last year and blocked the Department of Defense from taking control of approving new treatments for the military. FDA also negotiated some modifications in “right-to-try” legislation to lessen its erosion of the FDA approval process and maintain patient protections.
To convince the legislators to approve FDA’s new budget plan, Gottlieb outlined proposals for utilizing added resources (1, 2). Several initiatives advance the testing and production of new therapies, including greater use of real-world evidence and knowledge management systems to facilitate regulatory decisions, particularly for new treatments for rare diseases.
FDA also highlights the importance of modernizing drug manufacturing in proposing to spend $35 million to produce novel technologies, vaccines, and personalized medicines more reliably and at lower cost. Gottlieb now sees a convergence in regulatory resources, industry frustration with traditional production methods, and wider availability of new technology to advance more reliable and flexible advanced and continuous manufacturing systems.
“We’re now at a tipping point,” says Gottlieb. 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, Gottlieb explains, and move away from a checklist system for uncovering violations. Further innovation may come from FDA funding of advanced manufacturing R&D projects to assess new quality manufacturing and analytical methods (3).
This focus on quality manufacturing fits well with Trump administration efforts to encourage “re-domesticating some of the manufacturing that has moved overseas,” Gottlieb explains. He notes 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 re-tool manufacturing processes in the United States. And streamlined oversight of software-based devices aims to lower the time and cost of market entry.
FDA also seeks to build the largely domestic pharmacy outsourcing industry through clearer manufacturing standards and policies. And more modern production methods for biologics are on the agenda to improve the agility, flexibility, and reliability of manufacturing processes for vaccines and cell and gene-based therapies. In the wake of this year’s severe influenza outbreak, Gottlieb has urged shifting from an egg-based to cellular system for seasonal flu vaccines, along with efforts to develop a universal influenza vaccine to provide multi-year protection, and ensure US access to vaccines to protect against a pandemic.
More advanced manufacturing methods also support more reliable production of biosimilars. Gottlieb plans to issue new guidance documents on reducing lot-to-lot variability over time for biologics by utilizing new tools better able to identify where lot variation raises potential risks to patients, along with more standard reference products that are easier to test and copy as biosimilars.
Despite a year of considerable success in broadening support for FDA initiatives and policies, the commissioner shows no sign of slowing down. He continues to tackle controversial issues, such as limits on flavored nicotine products, oversight of independent testing labs, and programs to limit food contamination outbreaks. The commissioner has emphasized the importance of promoting vaccination rates, despite continued opposition from vaccine deniers. And in another speech, he outlined plans to implement nutrition labeling standards and menu disclosures, which Gottlieb considers key to improving public health (4).
FDA also faces numerous deadlines for implementing key provisions of the 21st Century Cures legislation, including policies to support regenerative medicine and to speed the development of new cancer therapies, personalized medicines, and gene therapies. At the other end of the spectrum is a press for more efficient oversight and approval of over-the-counter medicines through a new user-fee program. And transparency remains a thorny issue, as FDA supports wider disclosure of data and analyses related to regulatory, safety, and enforcement decisions. Yet proposals for release of complete response letters remain on hold, as industry seeks to keep confidential certain regulatory decisions.
Combating the opioid epidemic is a prime challenge. While FDA supports the development of safer and more effective pain therapies and treatments for substance abuse and is re-examining the safety of abuse-resistant products, physicians are pushing back on proposals to limit opioid prescribing. FDA has gained added funds to expand its capacity to block imports of unlawful controlled substances at the border, and new legislation may strengthen its authority to require small-dose packaging of opioids and to remove from the market approved pain medicines found to raise safety issues.
Gottlieb plans to remain a visible advocate for lower drug prices, anticipating a continued roll-out of incremental changes to create a more competitive prescription drug market place. He has loudly criticized drug reimbursement and coverage policies for limiting consumer access to drugs, including a “rigged payment scheme” that blocks biosimilars, and suggested that FDA may be more flexible in setting standards for demonstrating product interchangeability (5).
The commissioner’s concerns about the high cost of medicines reflect his own experience as a physician and seeing ill patients “struggling very hard at the worst moments in their lives” to try to afford drugs that are “absolutely indicated for their disease.” He wants to be sure “that in my time here at FDA, I do something to address that.”
1. FDA, “Statement from FDA Commissioner Scott Gottlieb, MD, on Administration’s Request for New FDA Funding to Promote Innovation and Broaden Patient Access through Competition,” FDA.gov, Feb. 13, 2018.
2. FDA, Department of Health and Human Services Fiscal Year 2019, Food and Drug Administration, Justification of Estimates for Appropriations Committee.
3. FDA, Agency Announcement, Solicitation Number: FDABAA-18-00123N, FBO.gov, Feb. 14, 2018,
4. S. Gottlieb, MD, “Reducing the Burden of Chronic Diseases,” FDA.gov, March 29, 2018.
5. S. Gottlieb, MD, “Capturing the Benefits of Competition for Patients,” FDA.gov, March 7, 2018.
Vol. 31, No. 5
When referring to this article, please cite it as J. Wechsler, "Gottlieb Tackles Tough Issues in First Year at FDA" BioPharm International 31 (5) 2018.