EMA has published a draft reflection paper on patient experience data for public consultation. These are data directly reflecting patients’ experience or preferences on treatments or outcomes, without any interpretation by a clinician or anyone else.
Patients’ perspectives on medicines and their benefits and risks are of great value to EMA. Patient experience data provide medicines regulators and other decision-makers in healthcare systems with valuable insights into what matters most to patients, beyond other well-established scientific outcomes. An example of the value of these data are cancer medicines where patients may, in some cases, prioritise quality of life over traditional clinical endpoints like overall survival. As such, patients’ lived experiences, reflected in quantitative or qualitative patient experience data, can play a complementary role to other types of data currently collected, notably through clinical trials, and further inform regulator’s decision-making on medicines.
The reflection paper is intended for medicine developers, patient groups, researchers and other decision-makers. It encourages medicines’ developers to gather and include data reflecting patients’ real-life perspectives and preferences throughout the lifecycle of medicines (i.e. during pre-authorisation, benefit-risk evaluation and post-authorisation), and describes general principles on how to generate, collect and analyse this data. To support developers in this process, the Agency offers early interaction platforms, such as scientific advice and qualification of novel methodologies, to discuss specific development plans and proposals for regulatory submissions.
Patient experience data can be provided through Patient Reported Outcomes (PROs), Patient Preference Studies (PPSs), as well as other forms of information obtained via patient engagement activities. The paper also highlights the sources where these data can be obtained, including clinical trials, real-world data such as from safety surveillance systems and other sources yet to be fully validated and utilised, including mobile health technologies and social media data.
While detailed methodological guidance is outside the scope of the paper, EMA is working with the International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) to harmonise existing methodological guidelines globally. In this context, the reflection paper provides a complementary framework to ICH work and focuses on general principles to include patient experience data in medicine development programmes and regulatory submissions in the European Union (EU).
The reflection paper has been produced by a multi-disciplinary group of experts from the EU regulatory network, including representatives from patient and consumer organisations. Feedback from stakeholders on this consultation will help shape the EU approach to patient experience data, with the goal ultimately to further improve evidence generation and optimise future medicine development towards outcomes that matter most to patients.
This draft reflection paper is open for public consultation until 31 January 2026. Comments should be provided using the template for submission of comments and sent to PED_RP@ema.europa.eu.
Notes
The reflection paper responds to a request from stakeholders asking for clarity on the value that the Agency sees in these type of data, reflected in a workshop held in 2022:
For more information on the work done by ICH on patient experience data, please see: