European groups weigh in on ICH E22 guideline on patient preference studies
European groups have raised questions about how patient preference studies (PPS) can be adapted from one region to another under the International Council on Harmonization (ICH) E22 guideline. Similar to their counterparts in the US, one major pharma group in the EU expressed the need for caregiver input for these studies. This feedback was in responses submitted by industry groups and manufacturers to the European Medicines Agency (EMA) during its call for comments on the guideline.
The ICH E22 guideline, released in November 2025, includes harmonized considerations regarding the use, design, analysis, and submission of PPS to inform drug development. The guidance recommends that the PPS should be included in modules 2 and 5 of the Common Technical Document (CTD) (RELATED: ICH announces new topics, adopts harmonized template for clinical trial protocols, Regulatory Focus 26 November 2025).
The comments were somewhat aligned, although they differed from the US industry's feedback on the guidelines. (RELATED: Pharmaceutical industry urges ICH to expand E22 guidelines to cover caregiver input, Regulatory Focus 10 April 2026).
Guidelines should address caregiver input
The European Federation of Pharmaceutical Industries and Associations (EFPIA), like their counterparts in the US, stated that the guidelines should be expanded to include input from caregivers.
EFPIA wrote that “the vast majority of the principles, methods, and considerations outlined in E22 are equally applicable to caregiver preference studies. It would add significant value to the guidance to add a brief paragraph or note acknowledging that in cases when patients cannot self-report preferences (e.g. young children, severe neurodegenerative conditions), it is important and appropriate to involve caregivers and stating that these general principles apply when collecting caregiver preferences.”
Questions on acceptance of PPS data across regions
Several comments had questions regarding the transfer of PPS data between regions.
The guidance states that “in some circumstances, PPS conducted in other region(s) may be useful and may inform regulatory drug assessment and related decisions in the local region (i.e., the region not studied in the PPS). This has the potential to conserve resources and decrease burden for the patient community. The degree of applicability of PPS results from other region(s) to the local region should be evaluated. Applicants should justify why a PPS conducted outside of the local region is informative to the local region.”
The European Alliance for Vision Research and Ophthalmology, also known as EU Eye, recommended that the guidelines provide greater clarity regarding the definition of "local regions." The group noted that it is unclear whether the guidance refers to an ICH region or another type of region.
“The distinction between a local region when compared to a region must be made clear,” said EU Eye. “If the term region refers to the ICH regions, the need for robust justification for using studies from one region to another (under Global application) should also apply for using studies from one MS [Member State] of EU to another MS as although both MSs belong to the same ICH region, there are documented differences between EU member states.”
The Lymphoma Coalition concurred.
“The concept of global applicability is introduced, but lacks practical guidance on how to assess transferability of PPS results across regions. The text mentions culture and healthcare similarity, but this is not enough,” said the group. “Preferences can be shaped by access constraints, legislation, cultural context, reimbursement rules, health literacy, trust in the system, social protections, travel burden, and local standards of care.
A PPS conducted in one setting may not travel well if patient choices are shaped by what is realistically available or affordable,” it added.
Guideline should incorporate modern digital platforms
There were also calls to update the guidelines to include modern digital research platforms, such as electronic data capture systems and decentralized trial platforms.
The Association of Clinical Research Organizations (ACRO) wrote that “while the guideline appropriately focuses on methodological considerations, it could acknowledge the growing role of interoperable digital infrastructure in supporting PPS conduct, improving data quality, and facilitating regulatory transparency. Recognizing the role of modern digital research platforms would help ensure the guideline remains applicable as clinical research continues to evolve.”
Patients should be involved in writing protocols
Thermo Fisher Scientific wrote that the guideline should be revised to incorporate the patient viewpoint in writing protocols.
“We agree that patient input is incredibly valuable throughout a PPS. However, protocols are often long and have technical information that is mandatory and not necessarily patient or lay friendly. We suggest patient advisors/partners input into key areas of the protocol to use their time effectively, particularly any patient facing materials, but we would advise against recommending patient advisors review the full protocol, unless they particularly wish to.”
Better distinction needed on qualitative and quantitative studies
EFPIA said the guideline should make a better distinction between qualitative and quantitative patient preference studies.
“The current description of qualitative and quantitative patient preference studies (PPS) could be clarified to better reflect their distinct purposes and methodologies,” said EFPIA. “As written, the text primarily contrasts ‘non-numerical’ and ‘numerical’ approaches, which may not fully convey the depth and role of each type of study.
“Revising this section to emphasize these conceptual differences will improve clarity and ensure alignment with established guidance and best practices in benefit–risk assessment,” the group added.
More information needed on PPS informing regulatory decisions
Thermo Fisher Scientific suggested the guidance expand on how PPS data will be used to inform regulatory decision-making.
“The document provides limited clarity on how PPS evidence is expected to inform specific regulatory decision contexts - for example what are in scope regulatory decision points (regulatory approval, label restrictions etc.), and how evidence needs differ in early development versus regulatory evaluation,” said Thermo Fisher. “Benefit-risk is mentioned, but the document does not provide decision relevant examples, for example stating that preference evidence may support approval in a population subgroup where benefit-risk is preference sensitive, or e.g. a PPS could be used to inform which dose receives regulatory approval.”
Leo Pharma says guideline should distinguish between patients
Leo Pharma suggested that the guideline distinguish between patients who are experienced with treatment versus those who are treatment naïve.
The company wrote that “preferences often evolve over the course of a chronic disease. Patients who are "treatment-experienced" or refractory to multiple therapies often have significantly different risk-tolerance profiles compared to treatment-naïve patients. Accounting for this heterogeneity in the PPS study design and reporting is critical for interpreting PPS results correctly.”
