EMA issues reflection paper encouraging systematic use of patient experience data in medicines development 

The European Medicines Agency (EMA) has released a new draft reflection paper encouraging developers and other stakeholders to plan, generate, and submit patient experience data (PED) across the medicine lifecycle, and to engage early with regulators on methods and use cases. 

Adopted by the Pharmacovigilance Risk Assessment Committee (PRAC) and Committee for Medicinal Products for Human Use (CHMP) for consultation on September 18, 2025, with public consultation open from September 29, 2025 to January 31, 2026, the paper sets out its scope and audience, including industry, regulators, adademic researchers, and patient groups. It defines patient experience data in the EU context as: 

“Data that directly reflect the experience of a patient or carer, without input or interpretation by a healthcare professional, third party or (artificial intelligence [AI]-based) device.”  

The EMA notes that “all PED submitted by applicants are reviewed and can be considered by medicines regulators for decision making,” while highlighting the need for high-quality, validated methods aligned with the EU Data Quality Framework. PED can capture how patients feel and function, their preferences and tolerance of trade-offs, adherence factors, and real-world experiences with outcomes and side effects.  

The EMA highlights the relevance of PED from the earliest stages of research through to post-authorization. They can be used to shape research questions, refine trial design and eligibility criteria, select patient-relevant endpoints, and improve feasibility and retention. Patient input may also inform benefit–risk assessments, product information, post-launch studies, health technology assessment (HTA) relative effectiveness assessments, and shared decision-making in practice. As the paper states: 

“EMA’s view is that PED should be systematically considered for informing medicines development from the earliest stages (including non-clinical stages) through to post marketing.” 

The reflection paper identifies three main categories of PED: 

• Patient-reported outcomes (PROs/PROMs): Direct reports from patients on symptoms, functioning, quality of life and side effects. They “enrich regulators’ understanding of a patient’s experience related to symptoms, adverse effects and overall satisfaction” and can strengthen approvals and labelling. The paper highlights the importance of validated instruments and notes challenges such as missing data, recall bias and participant burden. 

• Patient preference studies (PPS): Qualitative and quantitative approaches such as interviews or discrete choice experiments that assess how patients value benefits versus risks and the trade-offs they are willing to accept. EMA “considers it valuable to encourage the conduct of well-designed and reliable PPS,” referencing the IMI PREFER framework and noting that international guidance on PPS is expected to enter consultation in 2026. 

• Patient engagement activities: Surveys, written consultations, focus groups, EMA scientific advice and advisory groups, committee consultations, and public hearings that capture patient perspectives on disease burden, treatment priorities, and expectations. These inputs provide context to structured data and can inform “benefit–risk assessment, labeling, risk management plans or post-marketing surveillance.” 

A recurring theme in the paper is the importance of early engagement with regulators. Stakeholders are encouraged to use platforms such as the Innovation Task Force, EMA scientific advice, including joint scientific advice with HTA bodies, and the qualification of novel methodologies pathway. According to the document:  

“Medicine developers are encouraged to liaise early with regulators to seek scientific advice (SA) to discuss the best way to generate and collect PED,”  

with joint advice recommended “to ensure alignment with downstream decision-makers.” 

Beyond clinical trials, PED may be collected from non-interventional studies, registries, mobile health and wearable devices, or through safety reporting systems such as EudraVigilance. The paper also considers less conventional sources such as social media, while noting risks of bias and data quality limitations.  

This reflects wider EMA initiatives on real-world data, with Catherine Cohet noting at the GetReal Conference 2025 that “ideally, data quality should be checked using a data quality framework, and of course, preferably EMA’s.” 

The EMA outlines key challenges to implementation, including data completeness, representativeness, study design, validation of instruments, participant burden, training and capacity building, language accessibility, and the need for transparency in how PED are assessed. It also calls for global alignment, pointing to the ICH Reflection Paper on Patient-Focused Drug Development and the need for harmonized methodological guidance, particularly on PROs and PPS. 

The EMA concludes that PED can provide valuable insights to support development programs, marketing authorization applications, and post-authorization activities:  

“Stakeholders are therefore encouraged to embed PED across all stages of medicine development. This can be achieved by liaising early with EMA through scientific advice/qualification of novel methodologies, in order to enable case-by-case discussions on specific development plans and regulatory submissions.”