A consultation focused on Innovations in Clinical Effectiveness Trial Design will review the evidence required by various stakeholders, such as regulators, payers, patients, healthcare providers, and physicians, and incorporate it into the design of a prospective study. Examples of innovations in clinical effectiveness include Efficacy-to-Effectiveness (E2E), Efficacy-and-Effectiveness-Too (EE2), and N-of-1 trial designs. 

 

The consultation may include: 

• working with the investigator to design a study that includes both efficacy and effectiveness endpoints, 
• providing advice on suitable populations to include in the trial, 
• creating a statistical analysis plan, and 
• identifying the role of the Data Safety Monitoring Board.