Using Internally Developed Risk Models to Assess Heterogeneity in Treatment Effects in Clinical Trials
Clinical Trials Wednesday, February 26th, 2014CIRCOUTCOMES: January 14, 2014
Background—Recent proposals suggest that risk-stratified analyses of clinical trials be routinely performed to better enable tailoring of treatment decisions to individuals. Trial data can be stratified using externally developed risk models (eg, Framingham risk score), but such models are not always available. We sought to determine whether internally developed risk models, developed directly on trial data, introduce bias compared with external models.
Methods and Results—We simulated a large patient population with known risk factors and outcomes. Clinical trials were then simulated by repeatedly drawing from the patient population assuming a specified relative treatment effect in the experimental arm, which either did or did not vary according to a subject’s baseline risk. Read more
