European Medicines Agency seeks stakeholders' views on its reflection paper on patient experience data

Medicine regulators and other decision-makers in healthcare systems increasingly value the patient's perspective on treatment outcomes/preferences in their considerations. Medicines are developed and approved based on studies that assess the measurable health outcomes or endpoints established in regulatory guidance. These endpoints, however, may not always reflect all outcomes valued by patients. For example, a patient with cancer may favour better quality of life over an extended survival period if they experience serious side effects. Such information may be captured in patient experience data (PED), a subset of 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.* PED include patient-reported outcomes (PROs), patient preference studies (PPS) and data from patient engagement activities. Examples may include how a patient rates the effect of a medicine on the medical condition, suspected side effects reported directly by patients, trade-offs between benefits and risks of a treatment, and patient preference in risk minimisation measures. These so-called ‘lived experiences’, which can be reflected in quantitative or qualitative PED, can complement other more established types of data. A multi-stakeholder discussion in late 2022 resulted in agreement on the need to progress in this area.1 Regulatory submissions received by EMA often do not include PED and developers have identified a number of challenges to overcome to collect and provide them, including a lack of regulatory guidance specific for EU PED. To address this, a key deliverable of the multi-stakeholder discussion was the development of a reflection paper delineating a general EU framework and principles to generate, collect and analyse PED for use in medicines development and regulation. This reflection paper has now been published on EMA's website for public consultation. Patient organizations, medicine developers, academics and other stakeholders interested in using data reflecting patients' lived experience are being encouraged to take part in the public consultation.2 Stakeholders' contributions will help shape EMA's approach to PED in the EU in the coming years. The reflection paper is intended to provide relevant information to medicine developers and applicants for marketing authorisation of medicines, patient groups, researchers, regulators and other decision-makers. It aims to encourage a more systematic consideration of including PED in medicine development programmes and regulatory submissions. It also presents general principles on the use of PED across the lifecycle of medicinal products (i.e., during pre-authorization, regulatory evaluation and post-authorisation), briefly touching also upon the roles that PED can play in clinical practice and in economic evaluations informing access and reimbursement decisions. To facilitate alignment between stakeholders, the reflection paper dedicates two sections to describing types and sources of PED. Separate sections are presented on PROs, PPSs and data from patient engagement activities. The section on PED sources covers all settings where PED can be obtained, including clinical trials, real-world data (which includes data from safety surveillance systems) and other sources yet to be fully validated and utilized (such as mobile health technologies and social media data). The experience lived by patients can include health and functional status, disease symptoms, disease course, treatment preferences, quality of life (QoL), factors impacting treatment adherence and treatment outcomes and side effects of medicines, and such data can be collected and submitted by different stakeholders (patient, carer, advocacy group member, researcher, developer such as a marketing authorization holder and healthcare professional). For information to be considered PED, the submission must confirm that data directly reflect the patient's experience and have not been subjected to third party interpretation. PED can be collected using quantitative methods (e.g., quantitative surveys exploring relevant clinical outcomes or minimum relevant thresholds for patients; instruments for health-related QoL or other patient-reported outcome measures [PROMs]), qualitative methods (e.g., interviews, focus groups or qualitative surveys that reflect the wider perspective of patients' experience) or mixed methods that integrate both quantitative and qualitative approaches.3 Although regulatory experience with PED is still limited, EMA anticipates that medicine developers can consider PED to complement other types of data obtained during medicines development through to post-marketing, since PED can contribute to the totality of evidence on a medicine's benefits and risks. However, for PED to inform or support regulatory benefit–risk assessment and decisions, the data collected must be of high quality and the resulting evidence should be generated using robust and validated methodologies.4 It is important to note that the scope of the reflection paper does not include detailed methodological guidance, which will be pursued via collaboration with the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH),5 and EMA's reflection paper must be seen as complementary to efforts being pursued towards global development of PED.5, 6 Until regulatory experience allows for more detailed guidance in the EU, EMA offers multiple platforms for patient groups, industry and other stakeholders to engage with regulators and discuss the best approach to generate, collect and analyse such PED. These platforms include EMA's Innovation Task Force (ITF), academia briefing meetings, scientific advice (SA) and qualification of novel methodologies (QoNM), all of which offer developers and researchers the opportunity for targeted discussion on their specific development plans or PED methodologies. The ITF briefing meetings provide developers with a forum for early dialogue with EMA on innovative medicines, digital devices and novel methods. Similarly, for developments by academic and not-for-profit entities, EMA offers academia briefing meetings where integration of PED in their development programmes from early stages can be addressed. Once developments become more concrete and related to a specific medicinal product or novel method to be applied in a concrete context of use, developers are encouraged to discuss with EMA via SA procedures or qualification of novel methodologies,7 respectively. SA is a formal procedure that involves sharing with EMA a summary of the company's development plan and a number of meetings with applicants, where discussions should be targeted to the specific medicine development plan. Such discussions can cover what type of PED would be most relevant to generate and how it can be generated and collected. Early engagement should be sought when the key questions for the development plan have been identified. Since PED collected during clinical trials can serve for later decisions by Health Technology Assessment bodies (HTAs)/payers, EMA also offers joint SA with HTAs to facilitate alignment with downstream decision-makers. This aims to encourage collection of PED that is relevant for regulators and for postlaunch evidence generation, relative effectiveness assessments and economic evaluations. Collection methods and analyses of PED must be fit-for-purpose and produce reliable data. Some methodologies have been developed for collecting and analysing PED, but more are needed. EMA's qualification procedure for novel methodologies offers a route to assess and endorse innovative methods for collecting and using PED. If accepted, a qualification opinion is issued by EMA, confirming that the proposed method is suitable for use in a defined context of regulatory evidence generation. To address questions and challenges raised by stakeholders, and work towards more systematic implementation of PED, the reflection paper dedicates a final section to important considerations when generating, collecting and using PED. Without intending to offer methodological guidance per se, the paper briefly addresses aspects like data quality, representativeness, study design and further operational aspects relevant to obtain input from patients, such as participant burden, language and training. Transparency on which PED are submitted by companies as part of applications for marketing authorization, as well as on how PED are evaluated by regulators, has been requested by all stakeholders. Also, the grounds for acceptance or rejection of evidence to assess the benefit/risk balance of a medicine are deemed important by all stakeholders. The final section reminds stakeholders that if applicants wish to include PED in regulatory documents, such as the EU product information, to support the conditions of use of the medicine, it is recommended that they consult applicable guidelines and seek SA on their proposals. This is because, ultimately, inclusion of PED in the EU product information will depend on the scientific assessment by EMA's scientific committees responsible for the evaluation of human medicines and safety monitoring/risk minimisation (the CHMP or Committee for Medicinal Products for Human Use, and the PRAC or Pharmacovigilance and Risk Assessment Committee, respectively). Stakeholders' input into the public consultation (until 31 January 2026) will complement discussions held by EMA with all stakeholders so far. Obtaining broader input into EMA's initial framework for PED in the EU will hopefully allow for fully integrating patients' lived experiences into the evaluation of medicines as an important and complementary contribution, for the benefit of patients and healthcare systems alike. R.G-Q, J.G-B and B.S. contributed to the drafting of the patient experience data reflection paper. R.G.-Q. drafted the manuscript. J.G-B., S.S., U.W-L., M.G. and B.S. revised the manuscript and provided overall direction. We thank Melanie Carr, Peter Arlett and Jarno Jansen for the review of the manuscript, as well as all the experts involved in the drafting group of the reflection paper. The authors declare no competing interests. The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the European Medicines Agency, or one of its committees or working parties, or national competent authorities. Data sharing not applicable to this article as no datasets were generated or analysed during the current study.