The International Council for Harmonisation (ICH) entitled “ICH E22 General considerations for patient preference studies” has released a draft scientific guideline open for public consultation on the EMA website until12 April 2026.

The aim of this harmonised guideline is to provide general considerations and scientific principles about the use, design, conduct, analysis, and submission of Patient Preference Studies (PPS), in order to inform drug development, regulatory submission and evaluation, drug approvals and maintenance of such approvals.

These studies are a valuable tool for capturing patient perspectives on disease outcomes, including but not limited to Patient Reported Outcome Measures (PROMs), and the effects of drugs and other health interventions, as well as for assessing their relative desirability or acceptability.

PPS can generate insights on aspects considered by patients when making decisions about drugs These insights can be useful for informing the design of clinical studies, drug development, pre- and post-marketing phases.

To read more about the guideline and provide your input follow this link: ICH E22 General considerations for patient preference studies – Scientific guideline | European Medicines Agency (EMA).

At the same time, the EMA has released a Reflection paper on patient experience data. Through this reflection paper, the EMA promotes a systematic inclusion of data that reflect patient’s lived experience, without mediation by healthcare professionals, through medicine development and in marketing authorisation applications. Stakeholders were invited to submit comments on the reflection paper by 31 January 2026

In collaboration with other consortia and networks like HemaFAIR and TEDDY, Fondazione Gianni Benzi has participated in the public consultation aimed at finalising the document through the considerations from the relevant stakeholders. We highlighted critical issues, particularly in relation to paediatrics and rare diseases, including haemoglobinopathies. In particular, we emphasised that the fact that Patient Experience Data (PED) data often relies on a combination of child- and caregiver-reported outcomes, should be explicitly mentioned. We also emphasised that validated, age-appropriate tools for paediatric patient-reported outcomes, including versions adapted for children, adolescents and young people, are needed. Furthermore, although the importance of PED is clearly articulated in the document, it would benefit from an explicit reference to chronic rare diseases, such as haemoglobinopathies, where patient experience plays a central role throughout the entire disease course.