Presentation (CTI)

OBJECTIVES: To describe and evaluate pivotal clinical trials and regulatory approval characteristics in FDA approval packages for novel agents approved in 2024.
METHODS: We abstracted data on clinical trial and drug approval characteristics from FDA approval packages issued in 2024 by the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).
RESULTS: We included 54 drugs approved in 2024, including 6 gene or cell therapies. Of those, 31% were oncology drugs, 11% were endocrine/metabolic, 11% were hematology, 9% were cardiovascular, and 9% were dermatology. Most drugs received priority review (57%), and 19% received accelerated approval. Regarding regulatory designations, 61% received orphan drug designation, 41% received fast track designation, and 37% were given breakthrough designation. There were differences across therapeutic areas and types of drugs. Overall, 72% of the drugs were approved based on one pivotal clinical trial (mean: 1.5) and a mean of 569 participants (median: 193). Although the majority were controlled trials, 28% were based solely on single-arm trials. More than half (52%) included placebo-controlled trials, whereas only 19% included active-controlled trials. These findings will be compared with trends observed in 2022 and 2023.
CONCLUSIONS: With significant use of shortened drug development designations and review pathways, alongside few trials overall and over a quarter of single-arm trials, our evaluation highlights the limited evidence for new drugs, impacting the ability to make clear comparisons between treatments, and raises concerns about whether there may be potential for increased uncertainty in clinical trial evidence. Both of which can impact HTA evaluations and economic models, requiring more complex analyses to account for this uncertainty.