EMA now needs patient experience data from R&D to access

While patients are valuable contributors across the medicines development lifecycle, patient experience data (PED) are still not systematically included at all stages. The recent publication of the European Medicines Agency (EMA) Reflection Paper on PED is one of the clearest regulatory signals yet that the patient voice must be embedded far more systematically across the entire medicine lifecycle. For our industry, the message is unambiguous: PED can no longer sit on the periphery of development. They must be designed, collected, and integrated with the same rigor as clinical and safety data.

Why PED must now be embedded across the lifecycle

Medicines are designed for patients. Incorporating PED across the lifecycle ensures development plans, study designs, and benefit–risk considerations remain truly patient-relevant. Patients add clear value in the medicines development lifecycle by drawing on lived experience to provide a real understanding of how health conditions and treatments impact their lives. Collecting and using robust PED helps ensure decisions are aligned with patients’ expectations, preferences, values, and priorities.

The EMA describes PED as “data that directly reflect the experience of a patient or carer, without input or interpretation by a healthcare professional, third party or device.” These data can be quantitative (e.g. patient-reported outcomes [PROs], patient preference studies, structured surveys); qualitative evidence (e.g. interviews, focus groups, open-text feedback); data from structured patient engagement activities; or real-world data collected in clinical care or registries.

Patients are our primary stakeholders. Patient-centric data must be our central concern. How do we reflect this in our approach to drug development?

The Reflection Paper makes clear that PED should be systematically integrated across all stages of the medicine lifecycle – from the earliest stages of research, through clinical development, regulatory assessment, HTA evaluation, and post-marketing activities.

The practical challenge for medicines development teams

Although the regulatory direction is clear, many teams still face real barriers when trying to operationalize PED that are robust, methodologically sound, and usable across both regulatory and HTA pathways:

• PED are often collected too late, preventing them from influencing design or endpoints
• Uncertainty exists around what constitutes “fit-for-purpose” PED in regulatory and HTA contexts
• Quality varies – data may be anecdotal, unvalidated, or insufficiently documented for use in submissions
• Misunderstandings persist between patient engagement activities (insight-gathering) and formal research (evidence-generating)
• Consent, governance, traceability, and reproducibility are not always fully considered

For pharma, this presents a pivotal question:

How do we generate PED that are high-quality, ethical, regulator-ready, and HTA-relevant, without creating new burdens and silos?

Why this matters now

Moving forward, PED will influence:

• clinical trial design (early scientific advice)
• benefit–risk assessments
• relative effectiveness (through EU Joint Clinical Assessments) and reimbursement
• label claims and product information
• post-marketing safety and risk minimization
• treatment pathway decisions and guideline development

As HTA coordination strengthens under the new EU HTA Regulation, the role of PED will expand significantly. HTA bodies will increasingly rely on patient-generated insights to determine not only whether a treatment delivers clinical benefit, but also whether it demonstrates real-world relevance and value from the patient’s perspective.

A milestone moment

The EMA Reflection Paper represents a milestone toward greater and more consistent use of PED across medicines development, regulatory submissions, and HTA evaluations. It reflects a growing expectation that patients’ lived experience should carry equal weight with clinical and safety data when decisions are made about the development, approval, access, and use of medicines.

For teams across R&D, regulatory, HEOR, market access, patient engagement, and medical affairs, this creates an imperative, and an opportunity, to rethink how patient experience is captured and used.

While we await further clarification about specific PED-related regulatory and reimbursement requirements, how should we evolve our approach to evidence generation?

Read the EMA PED Reflection Paper here. If you’d like to explore how to strengthen your PED approach in line with emerging regulatory and HTA expectations, click here to talk to us. we’d be happy to share best practices and discuss your needs.

 “The EMA’s Reflection Paper reinforces what we’ve long known: patient experience data isn’t an add-on – it’s essential evidence. To meet these expectations, teams need to bring the patient voice into development much earlier and with far more structure and rigor. When PED are built in from the start, they strengthen benefit–risk evaluations, drive smarter and more inclusive trial designs, and ensure endpoints truly reflect what matters to patients. And because PED now informs both regulatory and HTA decisions, early, high-quality PED are becoming critical to demonstrating real-world relevance and value across the entire development pathway.”

– Alex Morton, Global Head, Patient Engagement & Clinical Trial Optimization

“The EMA’s Reflection Paper emphasizes the value PED bring across all stages of the medicine lifecycle. Open discussion with HTAs and regulatory bodies at early stages ensures understanding of, and alignment with, the evidence needs of downstream decision-makers. Integrating the generation of robust and appropriately validated PED into development programs and regulatory submissions is more likely to inform decisions, not only during assessment of marketing authorization applications, but also in post-marketing settings.”

– Krupa O’Neill, Client Services Director, Value and Evidence

“By integrating data-driven site optimization, targeted recruitment and retention strategies, and bespoke patient-facing engagement, as delivered through MEDiSTRAVA’s clinical-trial-optimization framework, sponsors can not only accelerate trials, but also embed true patient-centricity. This creates fertile ground for capturing [PED] by demonstrating real-world experiences, preferences and quality-of-life impacts that matter most to patients, and ensures trials deliver evidence that resonates beyond only study endpoints.”

–  Anthony Haywood, VP Clinical Trials Optimization