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

Area of Science:

• Clinical trial management
• Pharmaceutical research
• Biostatistics

Background:

• Protocol design practices and performance data are crucial for informed clinical trial decisions.
• Establishing baseline measures is essential for assessing protocol design impacts, especially during and after global health events.

Purpose of the Study:

• To establish benchmarks for clinical trial protocol design and performance.
• To analyze trends in protocol complexity and identify differences between oncology/rare disease and non-oncology/non-rare disease trials.

Main Methods:

• Analysis of 187 Phase I-III clinical trial protocols completed before the COVID-19 pandemic.
• Data gathered in collaboration with 20 pharmaceutical companies and CROs.
• Benchmarking conducted overall and for oncology vs. non-oncology and rare disease vs. non-rare disease subgroups.

Main Results:

• A consistent increase in protocol design variables observed across all trials.
• Phase II and III protocols show higher averages in endpoints, eligibility criteria, protocol pages, sites, countries, and data points.
• Oncology and rare disease trials exhibit lower enrollment-to-completion rates, increased international site involvement, more patient visits, and greater data generation, leading to extended cycle times.

Conclusions:

• The study provides valuable design insights and comparative benchmarks for clinical trial protocols.
• Results highlight the complexities and challenges in oncology and rare disease trial designs.
• The implications for protocol design practices and the potential impact of decentralized clinical trials are discussed.