Reflections from two EUPATI discussions on real-world evidence, patient engagement, and market access

Over the past few weeks, I had the opportunity to contribute to two EUPATI discussions approaching patient involvement in healthcare from different directions.

At the EUPATI Annual General Meeting in Brussels, we explored patient-led approaches to generating and using real-world data. During an EUPATI Switzerland training session, the conversation focused on patient engagement in market access and health technology assessment (HTA).

At first glance, these might appear to be separate conversations: one about generating evidence and the other about informing decisions. Yet together, they raise a more fundamental question:

If patients are increasingly contributing to the evidence we generate, how do we ensure that evidence actually influences the decisions we make?

From patient experience to decision-relevant evidence

Patients and their organizations are no longer simply contributors of personal experiences to research and healthcare discussions. Increasingly, they are partners in identifying evidence gaps, defining meaningful outcomes, supporting registries, collecting patient-reported information, and contributing to research design.

This evolution is particularly important in rare diseases, where conventional evidence generation may be constrained by small populations, heterogeneous disease trajectories, and limited longitudinal information.

However, an important distinction remains. Patient experience is not automatically real-world evidence, and collecting patient-generated data does not, by itself, establish its suitability for regulatory, HTA, or reimbursement decisions.

The value of evidence depends on the question it is intended to answer, the methods used to generate it, and its relevance to the decision-making context.

Initiatives such as IMI PREFER have helped advance the methodological foundations for incorporating patient preferences into medicines development and decision-making. Similarly, advances in real-world evidence methods have expanded opportunities to understand outcomes beyond controlled clinical settings.

However, methodological progress alone does not guarantee that patient perspectives will be meaningfully incorporated into healthcare decisions.

Generating evidence that matters to patients and generating evidence that matters to decision-makers should not be two separate exercises.

Where patient engagement meets market access

This brings us to the second EUPATI conversation.

Market access is often perceived as a technical process involving clinical evidence, comparative effectiveness, economic evaluation, pricing, and reimbursement negotiations. Patients may be invited to contribute their perspectives, but the relationship between those contributions and the evidence requirements underpinning access decisions is not always clear.

Consider the questions that frequently emerge during HTA.

How does a treatment affect everyday functioning beyond clinical endpoints? What outcomes are most meaningful to patients and caregivers? How does the disease affect families, employment, and quality of life? What happens to treatment outcomes outside the controlled conditions of a clinical trial?

These are not peripheral questions. They can shape our understanding of unmet need, relevant outcomes, uncertainty, and ultimately the value of a health technology.

The challenge is that patient involvement and evidence generation are often organized as parallel activities.

Patient organizations may collect information without knowing whether it will address a future assessment question. Manufacturers may develop evidence plans without sufficiently integrating patient priorities. HTA bodies may seek patient input when important decisions about study design and evidence generation have already been made.

The result is a familiar disconnect: patient perspectives are acknowledged, but their potential contribution to the evidence base is not fully realized.

Thinking beyond a single decision

One of the most important opportunities lies in rethinking when and how evidence is generated and assessed.

The value of a technology is not fully understood at the moment of its first assessment. Evidence evolves, clinical practice changes, and uncertainties may become clearer as treatments are used in broader populations.

This is central to the concept of lifecycle HTA: assessment and evidence generation should be considered as interconnected activities across the life of a technology, rather than as a single exercise around market entry.

From this perspective, patient-generated data and real-world evidence can play different roles at different moments.

Early in development, patient perspectives can help identify meaningful outcomes and inform study design. Around market entry, they can contribute to understanding unmet need, treatment burden, and the relevance of available evidence. After adoption, appropriately designed real-world studies may help address residual uncertainties about outcomes, effectiveness, and patient experience.

Not every uncertainty can be resolved through patient-generated data, and not every assessment requires formal reassessment. However, when new evidence could materially change a decision, there should be a clearer connection between the questions left unanswered and the evidence subsequently generated.

Here, patient engagement becomes more than participation in a process. It becomes part of an ongoing evidence strategy.

From participation to shared accountability

What would it take to make this connection more systematic?

First, patients and their organizations should be involved early enough to help define which questions matter, rather than being asked to comment only on evidence already generated.

Second, evidence-generation plans should be developed with a clear understanding of their intended use. Patient relevance, methodological credibility, and decision-maker requirements must be considered together.

Third, responsibilities should be made explicit. Manufacturers, researchers, patient organizations, regulators, and HTA bodies have different roles, but each can contribute to establishing a more coherent pathway from evidence needs to evidence generation and use.

Finally, engagement should include feedback to participants. When patients contribute their time, experiences, or data, they should be able to understand how their contributions were considered, what limitations were identified, and whether they influenced the resulting evidence or decisions.

This does not mean that every patient contribution must change a reimbursement recommendation. Meaningful engagement cannot be measured solely by whether a particular decision changes.

It does mean that participation should have a transparent purpose, a credible pathway to influence, and an accountable process for communicating outcomes.

Connecting the two conversations

What stayed with me from these EUPATI discussions was not simply the growing capacity of patient communities to contribute to evidence generation or the increasing recognition of patient involvement in HTA and market access.

It was the opportunity to connect these two developments more deliberately.

We have made progress in developing methods to capture patient experiences, preferences, and real-world outcomes. We have also made progress in creating opportunities for patients to contribute to healthcare assessments.

The next challenge is to ensure that these advances reinforce one another.

For patient organizations, this means understanding where their evidence can make a difference. For industry and researchers, it means integrating patient priorities into evidence strategies from the outset. For decision-makers, it means communicating more clearly what evidence is needed, how it will be evaluated, and where patient contributions can influence the process.

Ultimately, the ambition should not be to generate more patient data or create more opportunities for consultation.

It should be to build a more coherent relationship between what matters to patients, the evidence we generate, and the decisions that shape access to healthcare.

That is where patient engagement begins to translate into meaningful influence.