Update on ICH Efficacy Guidelines E22 and E23

The ICH published the E22 Draft guideline on "General Considerations for Patient Preference Studies" for public consultation until 12 April 2026 (Deadline for comments in Europe).

Patient preference studies ask patients what matters most to them about drugs and other health interventions, such as effectiveness, side effects, safety outcomes, convenience and other factors. These studies help drug developers and regulatory authorities to understand and consider medical needs from the perspective of patients. The draft guideline covers general principles, information on different types of patient preference studies, and recommendations and practical considerations for designing and conducting these studies.  

Following the final ICH M14 Guideline on the use of RWD for Safety Assessment, the ICH published a concept paper for an new ICH E23 guideline on "Considerations for the Use of Real-World Evidence (RWE) to Inform Regulatory Decision Making with a focus on Effectiveness of Medicines". According to the concept paper, the new E23 guideline will aim to support the development and assessment of medicines throughout their entire lifecycle (from pre-approval to post-market studies) regardless of any particular disease and patient population.

Moreover, a concept paper for ICH M18, "Framework for Determining the Utility of Comparative Efficacy Studies in Biosimilar Development Programs", was also published on the ICH website. 

More information is available on the ICH website under Efficacy Guidelines.