The International Council for Harmonisation (ICH) has published a draft scientific guideline, “ICH E22 General considerations for patient preference studies”, with a public consultation open on the European Medicines Agency (EMA) webpage for ICH E22 until 12 April 2026. More information on ICH’s work with European regulators is also available via the EMA page on the International Council for Harmonisation (ICH).
Patient preference studies (PPS) are increasingly used to bring the patient voice into medicine development and evaluation. The draft ICH E22 guideline sets out general considerations and scientific principles for the use, design, conduct, analysis and submission of PPS, with the aim of supporting decision-making across the medicine lifecycle, including development, regulatory submissions, approvals and the maintenance of those approvals.
By focusing on what matters most to people living with a condition, PPS can complement other sources of evidence about outcomes and treatment impact, including (but not limited to) patient-reported outcome measures (PROMs). In practice, these studies can help clarify which benefits, risks and trade-offs are most acceptable to patients, and can inform choices in clinical study design and evidence generation in both pre- and post-marketing phases.
To read the draft and provide your input, visit: ICH E22 General considerations for patient preference studies – Scientific guideline | European Medicines Agency (EMA).
In parallel, the EMA has also consulted on its Reflection paper on patient experience data, which encourages a more systematic inclusion of data reflecting patients’ lived experience, without mediation by healthcare professionals, across medicine development and in marketing authorisation applications. The reflection paper consultation invited comments up to 31 January 2026.
Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus (Fondazione Gianni Benzi) has participated in the public consultation aimed at finalising the reflection paper, working alongside other consortia and networks including HemaFAIR and TEDDY. In its contribution, the Foundation highlighted considerations particularly relevant to paediatrics and rare diseases, including haemoglobinopathies. This included the need to explicitly acknowledge that patient experience data may rely on a combination of child- and caregiver-reported outcomes, and that validated, age-appropriate tools for paediatric patient-reported outcomes (including versions adapted for children, adolescents and young people) are needed.
The Foundation also noted that, while the importance of patient experience data is clearly articulated in the reflection paper, it would benefit from an explicit reference to chronic rare diseases, such as haemoglobinopathies, where patient experience can be relevant throughout the full disease course.
