Pragmatic Phase 3 Pharmaceutical Trials: Recommendations for the Design of Clinical Trials that Are More Informative for Patients, Clinicians, and Payers (September 2010)

This EGD provides recommendations to guide the implementation of pragmatic study designs for phase 3 pharmaceutical trials. Pragmatic Clinical Trials (PCTs) are designed to assist patients, clinicians, and payers in making informed decisions about alternative drug therapies.

The recommendations cover broad topic areas including: enhancing stakeholder engagement in study design, aspects of trial design, and other operational, analytical and ethical aspects of using pragmatic designs for regulatory approval trials. This guidance is not intended to replace guidance issued by the FDA or other agencies. The objective is to provide recommendations for incorporating pragmatism into Phase 3 clinical trials, while simultaneously meeting regulatory requirements of the FDA.

A major conclusion of this work is that any incremental steps towards improving the pragmatic nature of trial design in three major domains of trial design; improving the generalizability of the patient population, selecting active comparators and selecting consistently measured, clinically-relevant outcomes, can markedly improve the utility of information obtained from clinical studies designed for regulatory approval. Other features of pragmatic trial design, such as loosening tight controls on patient adherence with therapy may be better reserved for study in the post-regulatory environment.