EMA tells sponsors to incorporate patient experience across product lifecycle
The European Medicines Agency (EMA) has released a reflection paper regarding the use of patient experience data (PED) in drug development. In the document, the EMA states that PED should be incorporated into all phases of drug development, as it can significantly contribute to the overall evidence generated throughout the lifecycle of a medicine.The reflection paper outlines key principles regarding the use of PED throughout the product lifecycle. It is aimed at medicine developers, patient groups, and researchers, but it does not provide detailed methodological guidance.
The publication of the paper followed a multi-stakeholder workshop in 2022 that focused on incorporating PED throughout all stages of medicine development and regulatory decision-making. The workshop reached consensus on the “importance of including PED at all stages of medicine development and regulatory decision making.”
The paper discusses the EU’s regulatory approach to patient experience data, highlighting its use and significance throughout the lifecycle of medicines. It outlines various types of patient experience data, including patient-reported outcomes (PROs) and patient preference studies (PPS). The sources of this data are examined, including information gathered from clinical trials and real-world data, as well as safety surveillance systems. The paper also considers the systematic implementation of patient experience data and the need for global alignment in this area.
EMA stated that “while some types of PED (such as patient-reported outcomes, PROs) have already been accepted as efficacy endpoints in clinical trials, there is less experience with other PED types such as patient preferences or qualitative data from patient engagement activities.”
For example, in clinical trial design, PED can help in formulating trial questions that are most relevant to patients. It can also help refine the study design and objectives by selecting appropriate endpoints, including PRO instruments that reflect how patients feel and function.
The most common types of PED are PROs, which have typically been collected in clinical trials, particularly in Phase III studies, to support decision making in regulatory settings, health technology assessment (HTA) reimbursement decisions, and clinical care.
“Collection of PED within phase III studies is encouraged as it has a number of advantages over collecting them in early phase studies. Such advantages include the possibility of using confirmatory studies with sufficiently large samples and linking the observed safety and efficacy data with PROs and patient preferences, for example so that minimally clinically relevant changes for the PRO (from the patient perspective) can be defined and validated,” EMA wrote.
In real-world clinical care settings, the collection of PED is valuable for understanding patients' healthcare needs and preferences, said the paper. It also enhances knowledge of the benefit-risk profile of various treatments. For instance, PROs provide crucial information regarding safety signals since any adverse event reported by a patient qualifies as a PRO.
EMA indicated that the reflection paper is intended to support the efforts of the International Council for Harmonisation (ICH) in standardizing the incorporation of patient experience data into medicine development programs and regulatory submissions within the EU. In May 2024, ICH issued a concept paper on general Considerations for Patient Preference.
The US Food and Drug Administration (FDA) has been at the forefront of the initiative to gather and integrate PED into decision-making processes. In April 2023, FDA published its fourth and final guidance in a series of PFDD guidances meant to help sponsors collect and incorporate patient experience information that can factor into regulatory decision making. (RELATED: FDA issues last guidance in patient-focused drug development series, Regulatory Focus 5 April 2023)
The paper was developed by a multi-disciplinary group of experts from the EU regulatory network, including representatives from patient and consumer organizations.
EMA said that feedback from stakeholders will help shape the EU's approach to patient experience data. Ultimately, the goal is to improve evidence generation and optimize future medicine development to focus on outcomes that matter most to patients.
The draft reflection paper is open for public consultation until 31 January 2026. Comments should use this template and should be sent to [email protected].
EMA reflection paper
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