Patient experience data (PED) are data providing information about patients’ experiences with a disease or condition, including experiences with disease symptoms, impacts, and burden, as well as treatment burden and preferences. LUNGevity Foundation has long advocated for the inclusion of PED in clinical trials across the drug development process, including to inform the safety and/or efficacy of new therapies. We have previously commented on several of the US Food and Drug Administration (FDA) draft guidance documents in their Patient Focused Drug Development (PFDD) series. We were therefore encouraged to read the European Medicines Agency (EMA) Reflection Paper on Patient Experience Data published in September 2025 and appreciated the opportunity to provide feedback from the patient advocacy perspective.
After review, LUNGevity provided EMA with several comments, the main themes of which focused on additional clarity for the acceptability of methods for achieving a specific goal, promoting alignment across regulatory agencies, and encouraging the use of PED.
Acceptability of Methods for Achieving a Specific Goal
Patient experience data, as noted in the reflection paper, can be leveraged for a variety of purposes, and the necessary methodological rigor with which the data were gathered, and the necessary robustness of the data itself, will depend on the intended use. LUNGevity noted in several comments the need for additional clarity or granularity on the acceptability of particular PED generation methods for specific intended uses. For example, additional guidelines and methodology for use and interpretation of spontaneously generated online PED (e.g., patterns in side effects mentioned on social media platforms) were requested, as the EMA noted use of these data, but LUNGevity is concerned for possible biases and misrepresentation of patients’ experience with these types of passive data.
Additionally, several comments were directed towards the EMA’s mention of using PED from devices and wearables (e.g., smart watches or fitness trackers). Further clarity is needed on what would constitute PED from these devices, and LUNGevity noted the unique burdens that can be imposed on patients by requiring these devices that should be considered.
Lastly, the paper noted factors to consider when determining the appropriate data collection methods and types of PED to generate. However, one factor not included was how the data would be leveraged, i.e., to support an application, develop an endpoint, or to generally provide context. The level of rigor in methods and data collection will likely be very different depending on its intended use, which therefore should be considered.
Promoting Alignment with Other Regulatory Agencies
Most lung cancer clinical trials, as in other oncology and disease areas, include participants from multiple countries. For many of these trials, the goal is to submit the trial data to multiple regulatory agencies for approvals. Therefore, when establishing recommendations for clinical trial design and conduct, alignment across regulatory agencies is critical, including for PED.
Alignment can promote consistency in regulatory expectations for sponsors, enhance efficiency in global drug development, and minimize duplication of efforts. LUNGevity noted several opportunities for the EMA to better coordinate with other regulatory agencies, including the FDA, by using similar definitions and standards for reviewing PED. When agencies aren’t aligned, drug developers may have to run multiple studies just to meet the different requirements. Stronger alignment would help avoid duplication.
Encouraging Use of PED
LUNGevity urged the EMA to avoid language that diminishes the value of PED. Referring to PED as subjective risks sending the message that patient voices are secondary. Instead, regulators should use language that affirms the credibility of the methods built over decades and signals a real commitment to treating PED as essential evidence.
Additionally, several caveats to PED studies included in the paper, such as selection biases for patient preference studies, are not unique to collecting PED. Therefore, LUNGevity encouraged the EMA in their reflection paper to offer solutions for challenges unique to PED or acknowledge these challenges are not exclusive to PED in an effort to avoid inadvertently discouraging its use.
Lastly, the heterogeneity in PED noted in the reflection paper risks unintentionally downplaying its evidentiary value. The variation in patient experience should be recognized as a core strength of PED, not a limitation. When PED are collected using rigorous, transparent, and well-defined methodologies, they can be sufficiently robust to inform regulatory decision-making, even when individual experiences vary. Removing unnecessary negative commentary and focusing on presenting solutions and rigorous methodologies encourages broader PED use.