On May 18, 2023, Dr. Patrizia Cavazzoni, Acting Director of the Center for Drug Evaluation and Research (CDER) addressed the top issues CDER has faced since Dr. Cavazzoni began her role in the summer of 2021. Dr. Cavazzoni also detailed CDER’s priorities and goals for the upcoming year.
CDER’s past work primarily focused on tackling three issues: the availability of medicines, safety with respect to substance use and misuse and supply chain integrity, and process enhancement and modernization.
On the issue of drug shortages and availability, Dr. Cavazzoni stated that CDER acted to further availability while minimizing risk to patients through several critical programs. By way of example, CDER piloted a new research program for biosimilars to study how biosimilars can be developed and approved more efficiently to get more biosimilars to market. To make drugs more accessible to more patients, CDER made updates for prescription to non-prescription switches, including the approval of non-prescription Narcan. To balance risk, CDER implemented a new requirement for accelerated approvals designed to ensure that post-market confirmatory trials are completed in a timely manner through the Consolidated Appropriations Act, 2023 Section 3120 Modernizing AA.
Ultimately, while CDER’s authority is limited with respect to its ability to affect the supply chain in the face of drug shortages, CDER has worked with companies and sponsors on a voluntary basis to prevent and mitigate drug shortages. Dr. Cavazzoni praised the willingness of these entities to cooperate with CDER on such important issues.
Regarding drug safety, including the issues of substance use and misuse and supply chain integrity, CDER developed and implemented the FDA Overdose Prevention Framework. Additionally, CDER’s “Opioid Policy Refresh” resulted in a new guidance, safety label changes for stimulants, and advances in safe disposal methods for unused opioids, including mail-back envelope and in-home disposal. CDER also acted to restrict the unlawful import of Xylazine, which is a veterinary drug used to treat large animals that, when combined with opioids, can cause infections serious enough to require amputation. Finally, with respect to nitrosamine impurities, CDER published a Federal Register Notice requesting comments from the public to advance collaborative efforts with industry. CDER hopes its collaborative efforts can help avoid duplicative testing for nitrosamine formation in similar drugs on the market.
Finally, with respect to process enhancement and modernization, CDER achieved reauthorization of the Prescription Drug User Fee Act (PDUFA VI) for fiscal years 2023–2027. With PDUFA VI, CDER implemented multiple pilot programs, including split real-time application review, rare disease endpoint advancement, and advancing real-world evidence. According to Dr. Cavazzoni, PDUFA VI also brings multiple important changes on review of post-marketing requirements, including new processes, timelines, and performance goals to ensure timely availability of public safety and efficacy information. CDER’s recently issued decentralized clinical trial guidance clarifies roles and responsibilities of sponsor and investigators and provides recommendations on design, digital technology use, and obtaining informed consent. CDER is hopeful that its new guidance will advance its goal of achieving greater diversity in clinical trial populations. CDER also modernized its advisory committee processes to ensure access to world-class experts’ advice on scientific, technical, and policy matters.
Looking forward, CDER will continue to focus on advancing these goals and others. Specifically, CDER will continue to study Real World Data/Evidence (RWD/RWE), particularly with an eye toward accelerating RWD/RWE for use in post-market requirements. CDER will also work on quantitative medicine under the Accelerating Rare disease Cures (ARC) program and expand the use of modeling in drug development through a multidisciplinary approach. CDER is focused on encouraging clinical trial innovation, and Dr. Cavazzoni expressed a desire for CDER to “turbo charge” that space. On the issue of drug supply chain disruption, CDER plans to spend time analyzing what role CDER should play and how it can involve others to alleviate the issue. Finally, CDER is focused on workforce retention, hiring, and fostering a hybrid work environment in the fiercely competitive labor market to ensure that it can attract the highest quality candidates to continue their important work.
Reprinted with permission of FDLI.
