Botanical drug clinical trial: Common issues and future options

Area of Science:

• Pharmacology
• Ethnobotany
• Clinical Research

Background:

• Botanical drugs have a long history of human use, yet fewer gain regulatory approval compared to synthetic drugs.
• Understanding trends in botanical drug clinical trials is essential to bridge the gap between traditional use and modern drug approval.

Purpose of the Study:

• To analyze botanical drug clinical trials registered on ClinicalTrials.gov from 2016-2019.
• To identify trends in trial phases, participant numbers, and study designs.
• To provide recommendations for future botanical drug clinical trial design and data collection.

Main Methods:

• Data extraction from ClinicalTrials.gov for botanical drug trials registered between 2016 and 2019.
• Analysis of trial phases (Phase I, II, II/III), participant numbers per arm, and study designs.
• Review of trial characteristics to identify patterns and potential areas for improvement.

Main Results:

• A total of 195 botanical drug clinical trials were registered.
• 81 trials were Phase II or Phase II/III, with 95% having ≤100 participants per arm, suggesting observational designs.
• 14% of trials were Phase I, which may be redundant for botanicals with extensive human use history.

Conclusions:

• Current botanical drug trials, particularly Phase II/III, often have limited participant numbers, potentially impacting outcome subjectivity.
• Phase I trials may not always be necessary for botanical drugs with a substantial history of human use.
• Standardized data collection and criteria are vital for leveraging traditional knowledge as robust evidence for regulatory approval.