ICH releases draft E22 guideline on patient preference studies for drug development and regulatory assessment

The International Council for Harmonisation (ICH) has released the draft E22 guideline, General Considerations for Patient Preference Studies, for public consultation. The guideline sets out harmonized principles for how patient preference studies (PPS) can inform drug development, regulatory submissions, and benefit–risk assessments. 

Endorsed on November 19, 2025, the draft E22 guideline defines PPS as research that: 

“Aim to assess the relative desirability or acceptability of actual or potential health interventions, or their characteristics and outcomes.”  

According to ICH, PPS are designed to provide structured insight into the “characteristics, also referred to as attributes, that are considered by patients when making decisions about drugs,” and how patients weigh trade-offs among benefits, risks, and other treatment attributes across various stages of drug development. 

The guideline makes clear that PPS are intended to complement, not replace, clinical evidence. It states that “the information provided by PPS does not replace the information provided by efficacy and safety studies,” but that preference data “may be considered together with the efficacy and safety information in the benefit–risk assessment of drugs and related regulatory decisions.” This positioning reflects growing regulatory interest in systematically incorporating patient perspectives alongside traditional clinical data. 

E22 focuses on stated-preference methods, which use hypothetical scenarios to elicit how patients make trade-offs, and clarifies that “revealed-preference methods are outside the scope of this guideline.” It also draws clear distinctions between patient preferences and those of other stakeholders, noting that “caregiver preferences are different from, and not a replacement for, patient preferences,” and that healthcare professional preference studies should not be used as substitutes for PPS. 

The guideline places strong emphasis on scientific rigor and ethics, stating that “principles applicable to other types of studies involving human subjects, such as ethical conduct, compliance with the protocol, and protection of personal data, are applicable to PPS as well.” It highlights the importance of early planning, clear alignment between research objectives and study questions, and the use of multidisciplinary expertise in the design, conduct, and analysis of patient preference studies.  

Patient involvement is highlighted as a critical component of high-quality PPS. The guideline states that: 

“Patient input is valuable throughout drug development, including in the development of PPS,”  

supporting activities such as attribute selection, protocol development, and interpretation of findings. ICH also stresses the importance of robust preparatory work, including literature reviews and patient interviews, to ensure that studies capture issues that are relevant and comprehensive. 

Methodological expectations span study design, sampling, attribute development, and instrument design. The guideline cautions that: 

“A mismatch between the PPS sample and the target patient population can limit the generalizability and applicability of the PPS findings,”  

and emphasizes that attributes and their levels should align as closely as possible with clinical endpoints. It also warns that “extrapolation of PPS data beyond the levels included in the study is generally not recommended,” particularly when PPS are used to inform benefit–risk assessment. 

Data quality, pretesting, and piloting are presented as essential safeguards against bias and misinterpretation. ICH recommends built-in quality checks to detect illogical or inconsistent responses and highlights the need for instruments that support consistent interpretation by participants. Analysis plans should be pre-specified and transparent, with sensitivity analyses used to assess robustness. 

For regulatory submissions, PPS should be documented in Common Technical Document (CTD) Modules 2 and 5, with full reports included in Module 5.3.5.4 and key uses described in the Product Development Rationale and, where relevant, in benefit–risk conclusions. According to the E22 Expert Working Group work plan, public consultation is expected to conclude in May 2026, with Step 4 adoption targeted for December 2026. Once finalized, the guideline aims to support more consistent, transparent, and scientifically robust use of PPS across global regulatory systems.