Computing individual and collective ethical utility for optimally planning phase III trials.

Area of Science:

• Clinical Trials
• Biostatistics
• Medical Ethics

Background:

• Standard clinical trial sample size determination relies on power calculations (80-90%).
• This approach may not fully encompass the ethical considerations for both enrolled and non-enrolled patient populations.
• A need exists for a more comprehensive ethical framework in sample size estimation.

Purpose of the Study:

• To propose a quantitative evaluation of individual and collective ethics for sample size determination.
• To introduce the Global Ethical Utility (GEU) model for phase III trials.
• To advocate for the adoption of GEU in clinical trial planning.

Main Methods:

• Developed the Global Ethical Utility (GEU) model, summing individual and collective ethical utilities.
• GEU considers treatment efficacy, quality of life, adverse effects, study duration, population size, and experimental power.
• Applied the GEU model to determine sample sizes that maximize ethical utility.

Main Results:

• The GEU model allows for sample size determination that maximizes ethical considerations.
• Ethically optimal sample sizes can result in power values outside the traditional 80-90% range.
• Factors like high efficacy and quality of life improvement may favor larger sample sizes and higher power.

Conclusions:

• The GEU model offers a robust tool for optimizing sample size in clinical trials.
• Adoption of GEU can lead to more ethically sound trial designs.
• The model provides flexibility to adjust sample size based on a nuanced ethical evaluation.