EMA Looks to Include Patient Perspectives in Drug Regulation

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The European Medicines Agency (EMA) announced on Sept. 29, 2025 that it has published a draft reflection paper on the use of patient experience data in the regulation of drugs (1,2). The reflection paper was written to encourage drug developers to gather and include data on real-life experiences of patients throughout a drug’s lifecycle so that regulators can use that insight in regulatory decisions. The draft reflection paper is open for public consultation until Jan. 31, 2026.

What can the patient perspective provide?

According to EMA, patient perspectives are of “great value” and include data such as cancer patients choosing quality of life over clinical endpoints. These data can play a “complementary role” to other scientific data during the regulatory review process. These types of data can be provided through Patient Reported Outcomes (PROs), Patient Preference Studies, and other patient engagement activities.

“Recent years have seen efforts by regulators internationally to steer medicine development toward programs that not only meet the requirements for quality, safety and efficacy of individual products, but also incorporate the broader perspectives of patients and carers. This is because patients may value different aspects of their disease and available treatments than medicine developers, including the type of relevant outcome measures to be assessed (e.g., quality of life), populations or stages of disease to be studied, or risk tolerability. An optimal patient-relevant medicine development program incorporates patients’ perspectives and documents their experience. Such patient experience data are directly collected from patients or carers experienced in managing the disease and capture their needs and preferences,” the paper states.

What guidelines does the paper propose?

The reflection paper provides drug developers with principles on the generation, collection, and analyzation of these data. The paper also provides sources for where the data can be obtained, such as clinical trials and real-world data from safety surveillance systems. Also included in the paper are discussions on training and capacity building, challenges related to using PROs, study design, transparency issues, and considerations for systemic implantation of these data.

The paper does not include detailed methodological guidance; however, EMA is working with the International Council for Harmonisation (ICH) to harmonize existing global methodological guidelines. The EMA reflection paper is a complement to the ICH work and focuses on development programs and regulatory submissions in the European Union. EMA is supporting developers by offering early interaction platforms to discuss development plans and proposals for regulatory submissions.

What is the importance of real-world evidence?

Real-world evidence, such as the patient experience, is gathered after a drug goes on the market. Data are used to determine the continued safety of the product. In the September 2025 episode of Ask the Expert, Siegfried Schmitt, vice president, Technical, at Parexel, and Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, discussed the role real-world evidence plays in drug development and manufacturing (3).

“Real-world evidence is part of the pharmacovigilance activities, and these are obviously mandatory for all marketed medicines,” explains Schmitt. “Real-world evidence collation wasn't really required in the past, but it is now. And, in my opinion, it's very likely to stay, given the benefits that real-world evidence delivers. And so, therefore, the real-world evidence for the product must be gathered for as long as you keep the product on the market.”

Schniepp goes on to explain that data about a product must be collected for the product’s lifetime, because of the potential for unknown side effects once used by patients. “You get indications or side effects, [when] you are in the clinical trials. But then once you open it up to the greater population, and everybody can take the medicine, you get more side effects, because it's going to affect everyone differently.”

The entire episode can be found at PharmTech.com.

References

1. EMA. A Path to Better Include Patients’ Perspectives in the Regulation of Medicines. Sept. 29, 2025. https://www.ema.europa.eu/en/news/path-better-include-patients-perspectives-regulation-medicines
2. EMA. Reflection Paper on Patient Experience Data. Sept. 18, 2025. https://www.ema.europa.eu/en/documents/scientific-guideline/reflection-paper-patient-experience-data_en.pdf
3. Schniepp, S.; Schmitt, S.; Haigney, S. Ask the Expert: Questions about Real-World Evidence and Supply Chain Security. PharmTech.com. Sept. 25, 2025. https://www.pharmtech.com/view/ask-the-expert-questions-about-real-world-evidence-and-supply-chain-security