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New agency leadership is pressed to promote innovation while addressing safety and quality issues.
During the nearly two years that Scott Gottlieb headed FDA, the agency achieved record approvals of new drugs and generics, and advanced important innovations in clinical research methods and in quality drug manufacturing to enhance competition and innovation in the bio/pharmaceutical industry. At the same time, the commissioner navigated the political pitfalls of Washington to gain increased funding for the agency and support for more aggressive policies to promote public health. FDA issued multiple new rules and guidance documents and utilized important new authorities to expand staff expertise and agency capabilities.
Two critical public health issues drew considerable attention from Gottlieb. He championed steps to curb inappropriate prescribing of opioids while also encouraging the development of safer and more effective painkillers. Most recently, he promoted a comparative standard for approving new opioid painkillers to permit only treatments with clear additional benefits to come to market. And data showing sharp increases in teen use of nicotine products prompted a high-profile campaign to curb “vaping” and flavored cigarettes for the young, while maintaining access for adults looking to stop smoking.
Gottlieb expects FDA to continue these initiatives under Acting Commissioner Ned Sharpless and a strong leadership team at the agency. But it will be a challenge for any newcomer, even an experienced scientist and administrator such as Sharpless, to maintain support and implement the broad spectrum of critical agency programs outlined in FDA’s 2020 budget request to Congress. In addition to promoting medical product safety and innovation, top priorities include advancing medical countermeasures, ensuring a safe national blood supply, implementing drug compounding rules, and preventing and detecting contamination of the nation’s food supply.
As the Trump Administration and congressional leaders press for action to reduce drug prices, the FDA commissioner will have the task of explaining how expanded drug importing could open the door to unsafe products, particularly with the rise of lethal forms of fentanyl coming from overseas. Gottlieb was particularly adept at articulating how FDA can help bring down drug prices through more efficient approval of generic therapies and biosimilars that enhance competition. He railed at a farewell discussion sponsored by the Alliance for a Stronger FDA that innovators blocking access to supplies needed to test new generics for equivalence was “particularly pernicious.”
Gottlieb expects continued efforts at FDA to implement innovative methods for conducting clinical trials and documenting new drug efficacy and safety. One promising development, he predicted, is that increased agency access to electronic health records will bring about a “profound change” in using real-world data for determining new drug effectiveness and safety.
Another challenge for FDA leaders is to continue efforts to modernize good manufacturing practices (GMPs), particularly for APIs coming from overseas. The continuing wave of recalls and shortages of blood pressure medicines due to contamination with cancer-causing impurities illustrates ongoing difficulties in this area. Gottlieb provided strong support for industry investment in continuous manufacturing systems and modern drug production methods to better ensure product quality and to reduce recalls and shortages in the process, issues that have challenged the Center for Drug Evaluation and Research (CDER) for the past decade.
During his tenure at FDA, Gottlieb sought to increase direct contact between the commissioner and center directors so that agency leadership could better understand staff challenges and priorities. That reform was formally implemented as part of an agency reorganization plan finalized just before Gottlieb’s departure in early April 2019. In addition, FDA centers implemented a range of internal organizational changes during the past two years, particularly for the approval and oversight of medical devices and for cell and gene therapies regulated by the Center for Biologics Evaluation and Research (CBER). CDER continues to revise its Office of New Drugs, which recently formed a new Office of Therapeutic Biologics and Biosimilars and shifted the division of hematology out of Oncology Products. Still to come are broader changes in oversight of over-the-counter medicines and updates in FDA regulation of diagnostics.
Gottlieb was successful in expanding FDA resources and authority by building strong relationships with legislators, constituencies, and industry. He generated enthusiasm for many proposals through a barrage of official announcements and social media commentary (1). Many observers expected him to come to FDA as a deregulator, canceling regulations and requirements, he acknowledged in his parting comments. Instead he expanded FDA authority, particularly in the critical areas of opioid prescribing and tobacco regulation. He left FDA in good hands but facing further upheavals in federal healthcare policy and partisan infighting likely to stymie regulatory innovation and squeeze agency funding.
1. FDA, “Remarks by Dr. Scott Gottlieb to Final All Hands Meeting,” FDA.gov, April 4, 2019.
Volume 43, Number 5
When referring to this article, please cite it as J. Wechsler, "FDA Faces Challenges After Gottlieb," Pharmaceutical Technology 43 (5) 2019.