An evidence based approach to individualising treatment

Area of Science:

• Clinical epidemiology
• Evidence-based medicine
• Medical statistics

Background:

• Clinical trial entry criteria are often misapplied to define patient applicability of results.
• Assessing treatment applicability requires a nuanced approach beyond simple inclusion/exclusion criteria.

Purpose of the Study:

• To propose a method for determining which patient groups can appropriately benefit from clinical trial findings.
• To differentiate between treatment benefit and harm for individual patient risk stratification.

Main Methods:

• Utilize relative risks from meta-analyses of randomized trials.
• Employ multivariate risk equations from cohort studies to estimate individual patient risk.
• Assess net benefit by comparing individual risk with treatment-related relative risks.

Main Results:

• Net treatment benefit is typically greatest for patients at the highest risk of disease.
• Treatment benefits often increase with patient risk, while harms remain relatively constant.
• Requires validation of assumptions regarding fixed adverse effects and constant relative risk reduction.

Conclusions:

• Individualized assessment of treatment benefit and harm is crucial for applying clinical trial results.
• Risk stratification using validated equations enhances the appropriate use of trial data.
• Careful consideration of underlying assumptions is necessary before drawing firm conclusions.