Update on ICH Guidelines E21 & E22

The ICH is currently working on two new Efficacy Guidelines: E21 on Inclusion of Pregnant and Breastfeeding Individuals in Clinical Trials and E22 on General Considerations for Patient Preference Studies. Following the 2023 announcement, work on the two guidelines has now reached a further stage.

Draft ICH E21 Guideline on Inclusion of Pregnant and Breastfeeding Individuals in Clinical Trials

Clinical trials often exclude pregnant and breastfeeding individuals, and those who become pregnant during a trial are frequently withdrawn. Consequently, information on the benefits and risks of medicinal product use during pregnancy and breastfeeding is, at best, sparse in the product labelling, and treatment decisions must be made in the absence of this information. 

The new ICH guideline therefore aims to provide recommendations for the appropriate inclusion and/or retention of pregnant and/or breastfeeding individuals in clinical trials, facilitating the generation of robust clinical data to inform evidence-based decisions on the safe and effective use of medicinal products by these individuals. The guideline covers pre- and post-marketing clinical trials of investigational medicinal products (IMPs) for conditions affecting the general population and/or indications specific to pregnant or breastfeeding individuals. The EMA recently published the draft E21 guideline on their website. Comments must be submitted by 15 September 2025.

Concept Paper for ICH E22 Guideline on General Considerations for Patient Preference Studies

The new ICH guideline intends to provide general considerations for patient preference studies (PPS) to inform the development of drugs and related decisions for medical products. The guideline aims to optimize the use of PPS information in pharmaceutical product development, enabling regulatory authorities and the pharmaceutical industry to benefit from harmonized PPS approaches during drug development, including submission to different regulatory agencies. PPS include any qualitative or quantitative assessment of the relative desirability or acceptability to patients of aspects that differ among alternative health interventions. They can contribute to the body of evidence and supplement clinical trial data when assessing benefits and risks. Among other things, PPS can help with the characterization of medical needs, the selection of endpoints, as well as identifying subgroups with different preferences. Incorporating patient perspectives has become more systematic throughout drug development, helping to align decisions more closely with patients’ values and needs, and in the benefit-risk assessment.

More information is available at the ICH's Efficacy Guidelines website.