Patient Voice: Where do we need it and why in regulatory documents?

Description

Asking for patient involvement – How do we use patient voice, especially in Paediatric trials

The patient voice in clinical trials

What are EMA’s expectations?

Engaging patients in the development of medicines and their subsequent authorisation is not just good practice, it is critical for our modern healthcare systems. As the end users of medicines, with invaluable experience of living with a particular condition and its treatment, patients are key stakeholders for the European Medicines Agency (EMA). Patients’ perspectives are integral to EMA’s decision-making process and embedded in all stages of the medicine’s lifecycle. Capturing the patient’s voice early is vital to ensure that, once authorised, a medicine’s benefit is meaningful to the patient. In addition to the established involvement of patient representatives in EMA’s scientific activities, the Agency is exploring new ways to strengthen the patient voice in clinical trials. Examples include the Accelerating Clinical Trials in the European Union (ACT EU) initiative and ongoing work by EMA to improve the collection and use of patient experience data to achieve patient-centered medicine development and regulation in the EU.

Catriona Ester

From “FOR patients” to “WITH patients”: systematically involving patients in the development of lay summaries of clinical trial results.

Lay summaries of clinical trial results play an important role in transparency and patient‑centred communication, yet patient involvement in their development is often limited. This session describes how Boehringer Ingelheim systematically embedded patient input into the development of Phase II and III lay summaries. Patient involvement was integrated into existing medical writing workflows and enabled meaningful patient input without extending submission timelines or compromising regulatory compliance. Examples illustrate how patient feedback led to concrete changes in language, structure, and presentation, improving the clarity and relevance of the final lay summaries.

Julia A Hild

The Patient Voice in Regulatory Documents – Where Do We Need It and Why?

The path from patient to regulatory contributor is neither linear nor obvious. It begins, most often, with a diagnosis.
In 2017, our family stumbled upon diagnosis of Langerhans Cell Histiocytosis, which initiated a trajectory that would move through treatment completion in 2019, into structured engagement with the rare disease ecosystem, and ultimately toward the intersection of patient experience and regulatory science.
That trajectory included the EURORDIS Winter School on Scientific Innovation and Translational Research (2020), the founding of Patient Association “Bromologos”, a regional patient community organized around Phenylketonuria (2021).
Than came the launch of Rare and Share, a podcast on rare disease lived experience (2022), completion of the EUPATI Patient Expert Training Programme (2024).
One more personal goal was achieved with the establishment of Empowera Consulting as a strategic communications practice (June 2025), and appointment to the EUPATI Faculty Committee (October 2025).
The central argument of this presentation is straightforward: informed, empowered patients and caregivers, and particularly trained patient advocates, are reliable, structured partners in the drug development process, including in the design of clinical research and in the development of regulatory documents.
Only patients possess the lived experience of a condition, its symptomatic burden, its functional consequences, and the trade-offs that define meaningful outcomes for them. This “user experience,” when systematically captured, constitutes a legitimate and increasingly expected input into regulatory science.
Patient-Reported Outcome Measures (PROMs) are one formalized mechanism for this contribution.
Peer-to-peer groups, focus groups, and in-depth interviews extend the methodological toolkit, enabling the kind of qualitative depth that quantitative instruments alone cannot achieve.
Together, these approaches generate the patient-experience data that can and should inform trial design, endpoint selection, benefit-risk assessments, and the patient-perspective sections of regulatory submissions.
The question of how to identify and engage appropriately trained patient partners in this work has a concrete answer. EUPATI Connect, the European Patients’ Academy’s searchable platform of trained patient experts, provides sponsors, regulatory agencies, and medical writers with direct access to a pool of individuals who combine lived experience with structured scientific and regulatory literacy.
This presentation will map the conditions under which patient involvement adds the most value, identify the specific regulatory documents where the patient voice is most consequential, and propose a framework for engagement that is grounded in both personal experience and formal advocacy practice.

Ana Pataki

Patient involvement in Advisory boards: what is important for them?

Over recent years the importance of the “patient experience” in clinical trials has moved from a nice-to-have to a must-have. Here, we will talk about methods of gathering information on what is important to patients and more importantly, why it is crucial to do this for both patients and sponsors.

Sarah Milner and Art Gertel

 

Q&A and panel discussion

Lisa Chamberlain James