nput from patients, as users of medicines, can inform medicines development, enhance regulatory decision making, and result in more patient-relevant outcomes. Clinical trial data for example typically constitute snapshots in time. Patient experience data collection helps to paint a clearer picture of how patients and families feel and function as a result of a clinical trial or lived experience with a disease. In a recent draft reflection paper, the European Medicines Agency (EMA) acknowledges the usefulness of such Patient Experience Data (PED) gained directly from patients. The paper is now open for public comment until January 31, 2026. It is now time to read and react.

Such PED may include sharing their experiences of symptoms, natural history of their disease, impact on quality of life, and unmet needs, as well as which outcomes are important to them including preferences for consideration in future treatments. PED can be obtained, for example, through collecting patient-reported outcome measures (PROMs), specific patient preference studies (PPS), or gathering data from other direct patient engagement activities.

The EMA reflection paper has been developed in response to stakeholders asking for EMA and CHMP guidance on PED at a Multi-stakeholder workshop: Patient experience data in medicines development and regulatory decision-making convened by EMA in September 2022. The paper also references the findings of IMI PREFER, the public-private partnership project established to assess when and how it is best to perform and include patient preference study results in decision making at various stages of the medical product lifecycle. PREFER completed its work in May 2022 by delivering PREFER Recommendations and a Qualification Opinion from EMA. The results have been comprehensively considered in the reflection paper as currently drafted as one of three key methods to gather PED.

The reflection paper was produced by a multidisciplinary group of experts from the EU regulatory network, including representatives from patient and consumer organizations. It is intended for medicine developers, patient groups, researchers, and other decision-makers.

The EMA reflection paper content outlines the European regulatory approach to PED. It focuses on general principles to include patient experience data in medicine development programs and regulatory submissions in the European Union (EU). The reflection paper provides examples of methods that could be considered in the context of PED gathering (see Table 1).

Table 1. Examples of methods applicable to PED for which scientific advice and qualification procedures are available

It encourages medicine developers to gather and include data reflecting patients’ real-life perspectives and preferences throughout the lifecycle of medicines development (i.e., during pre-authorization, benefit-risk evaluation, and post-authorization), and describes general principles on how to generate, collect, and analyze these 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.

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 utilized, including mobile health technologies and social media data.

In addition to this paper, the EMA has also taken the step to increase transparency on the use of PED in the Benefit Risk assessment of the Committee for Medicinal Products for Human Use: A new section has been included in the D80-210 Overview assessment report template, and these aspects will now also be visible in the European Public Assessment Report published after approval of new medicines. To date, this level of transparency is not available; the updated template is expected to improve the situation. With respect to PED being reflected in the EU product information, the observation so far is also that only a relatively small proportion of the PED is currently included in drug labeling.

Why is this guidance on PED important?

The EMA has a clear view on the importance of providing guidance on PED, as pointed out by Juan Garcia Burgos, Head of Public Engagement at EMA:

“The agency has long recognized the importance of incorporating patient views, reported outcomes, and preferences into medicines regulation. Patient experience data and their perspectives on benefits and risks provide medicines regulators with valuable insights into what matters most to the patients.

“We would like to receive more applications for marketing authorization including PED, and for these data to be embedded in early medicines’ development plans. To support this, we offer a number of early dialogue platforms for companies and other stakeholders seeking guidance on PED. Stakeholders can discuss their plans with regulators early on through mechanisms such as scientific advice or qualification of novel methodologies. This can help developers steer their plans towards meeting regulatory requirements and address potential challenges early on, while bringing the voice of patients as a complement to traditional endpoints.”

The paper is also highly welcomed by patient support organizations:

Maria Dutarte, executive director, European Patients Academy on Therapeutic Innovation (EUPATI), points this out very clearly: “EUPATI welcomes this reflection paper on patient experience data, as it recognizes the importance of patients’ expectations, preferences, and values and provides guidance on how these can be systematically included in the different stages of medicines development. The paper underlines the foundation EUPATI is built on: that patient voice is of great value and is critical to informing healthcare decisions. We also acknowledge that the proposed approach implies important training and capacity-building needs, among patients and all other stakeholders. EUPATI is committed, together with its partners, to support the collective upskilling around collecting and analyzing PED.”

While this draft EMA reflection paper is considered a general EU framework or principles—not a methodological guidance—it is intended to complement the ongoing work on ICH E22 guidance General Considerations for Patient Preference Studies.

All stakeholders anticipate that these publications, taken together, will foster the use of PED in drug development and allow a greater impact of the patient voice in drug development and healthcare decision making—a shared aspiration of many.

The PREFER Network in particular welcomes the reference to the EMA’s Qualification opinion for patient preference research obtained by IMI PREFER: “The draft paper acknowledges that patient preference studies (PPS) can, among other things, help with characterising medical need, selecting endpoints and estimating meaningful effect size, as well as identifying subgroups with different preferences. This will help to further establish and systematically integrate patient preferences in drug development.”

How can you get involved in fostering the use of patient experience data?

As an interested party, respond to the PED draft reflection paper’s call for comment to support the development of a systematic patient voice. 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.

As a developer of new therapies, collect and document PED to develop therapies that matter, share PED in your briefing books and regulatory dossiers when interacting with regulatory authorities, and add it to the product label where relevant.

As a patient expert or patient organization, contribute to PED; collect PED, conduct systematic PED such as PPS, and share it with healthcare decision makers to support the premise that the patient voice must be heard loud and clear.

As a researcher on outcomes and digital endpoints or health technology, seeking regulatory alignment early, and publishing your case studies and examples, will help to foster the practical use of the research and its impact on healthcare decision-making.

As a researcher on patient preference studies, seeking regulatory alignment early and publication of your results is encouraged to foster development of this research area and its impact on healthcare decision making. DIA’s peer-reviewed journal Therapeutic Innovation and Regulatory Science (TIRS) is calling for papers for a special collection entitled Patient Preferences to Inform Decision Making in Medical Product Development. Patient Preference Research is a topic that is generally suitable for TIRS, whether within or outside the Collection.

If you are interested in contributing to the PREFER EN network, contact the authors through Publications@DIAglobal.org.