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. 2023 Jan;57(1):49-56.
doi: 10.1007/s43441-022-00438-5. Epub 2022 Aug 12.

Protocol Design and Performance Benchmarks by Phase and by Oncology and Rare Disease Subgroups

Kenneth Getz  1 , Zachary Smith  2 , Marcy Kravet  3
Affiliations

Protocol Design and Performance Benchmarks by Phase and by Oncology and Rare Disease Subgroups

Kenneth Getz et al. Ther Innov Regul Sci. 2023 Jan.

Abstract

Background: Benchmark data characterizing protocol design practices and performance informs clinical trial design decisions and serves as important baseline measures for assessing protocol design behaviors and their impact during and post-pandemic.

Methods: Tufts CSDD, in collaboration with a working group of 20 major and mid-sized pharmaceutical companies and CROs, gathered phase I-III data from protocols completed just prior to the start of the global pandemic.

Results: Data for 187 protocols were analyzed to derive benchmarks overall and for two primary subgroups: oncology vs. non-oncology protocols and rare disease vs. non-rare disease protocols. The results show a continuing upward trend across all protocol design variables. Phase II and III protocols average more endpoints, eligibility criteria, protocol pages; investigative sites; countries and datapoints collected. Oncology and rare disease protocols' enrolled-to-completion rates are much lower, involve a much higher average number of countries and investigative sites, require more planned patient visits and generate considerably more clinical research data. As such, oncology and rare disease clinical trial cycle times are longer-most notably at time periods occurring after study startup and prior to database lock-due to intense patient recruitment and retention challenges.

Conclusions: The results of this study present valuable design insights and comparative baseline measures. The implications of these results and the expected impact of decentralized clinical trials on protocol design practices and performance is discussed.

Keywords: Clinical trial performance benchmarks; Oncology protocols; Protocol complexity; Protocol design; Protocol scope; Rare disease protocols.

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Conflict of interest statement

Kenneth Getz, Tufts CSDD and Zachary Smith, Tufts CSDD have nothing to disclose. Marcy Kravet, EMD Serono, declares that she is an employee and has financial holdings in the company.

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