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Advancing innovative clinical trials to efficiently deliver medicines to patients
Complex innovative designs in clinical trials have the potential to increase efficiency and lower the cost of drug development, improving patient access to therapies. This article highlights designs and approaches based on a meeting linked to an ongoing FDA pilot program in the field.
Departments of Oncology and of Biostatistics, Bioinformatics, and Biomathematics, Lombardi Comprehensive Cancer Center and Innovation Center for Biomedical Informatics, Georgetown University Medical Center, Washington, DC, USA.
The US 21st Century Cures Act and the US Prescription Drug User Fee Act (PDUFA) VI include provisions to advance the use of complex innovative trial designs (CIDs). In particular, the FDA CID Pilot Meeting Program aims to foster discussions and education on the use and value of CID in drug development programs1. In 2020, the Drug Information Association (DIA) Adaptive Design Scientific Working Group and Bayesian Scientific Working Group partnered with the FDA, Biotechnology Innovation Organization (BIO) and Pharmaceutical Research and Manufacturers of America (PhRMA) to organize the conference “Advancing Complex Innovative Clinical Trial Designs to Efficiently Deliver Medicines to Patients”, which was attended by participants from the USA, Europe and Japan, including patient advocates, statisticians, clinicians and clinical trialists, regulatory affairs experts, and other professionals from industry, academia and government. Here, we highlight selected innovative designs and approaches presented at the meeting, with an emphasis on those accepted into the CID program. The meeting agenda and a detailed summary are provided in the Supplementary information.
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Nature Reviews Drug Discovery21, 543-544 (2022)
doi: https://doi.org/10.1038/d41573-022-00109-y
Acknowledgements
The authors acknowledge the indispensable work of organizing committee members S. Benedetti (DIA), D. Friend (BIO) and S. Ramon (BIO). F.N. (chair), K.P., F.C., A.X. and P.S. are current or former (A.X.) members of the DIA-BSWG leadership team. R.A.B. (chair), Z.A., C.M., R.T. and Y.L. are members of the DIA-IDSWG leadership team.
Disclaimer
The opinions expressed in this article are the authors’ own and do not represent opinions or policies of national health authorities.
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Ghadessi, M. et al. A roadmap to using historical controls in clinical trials—by Drug Information Association Adaptive Design Scientific Working Group (DIA-ADSWG). Orphanet J. Rare Dis.15, 69 (2020).
Antonijevic, Z. et al. Patient benefits from innovative designs in rare diseases, in Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives (ed. Huml, R. A.) 147–160 (Springer, 2021).
R.A.B. consults for AstraZeneca and Boehringer-Ingelheim, and is an uncompensated part-time employee of Onco-Mind LLC. F.N. and K.P. are employees of Eli Lilly and Company. P.S. is an employee of Novartis. F.C. and A.X. are employees of Amgen. Z.A. is an employee of Abond CRO. Y.L. is an employee of Nektar Therapeutics. C.M. is an employee of Johnson and Johnson Vision. R.T. is an employee of Servier Pharmaceuticals. M.A. is an employee of PhRMA.
