Making Patient Partnership a Reality in EU JCA

Introduction: As the EU’s Joint Clinical Assessment (JCA) system rapidly evolves, patient involvement is no longer a “nice-to-have”—it’s a regulatory imperative and a strategic advantage. With orphan medicines entering the JCA process in 2028, organisations must move beyond token engagement and embed authentic patient partnership into every stage of development and assessment.

Setting the Stage: Patient Engagement as a Regulatory Imperative

As the JCA process expands to include orphan medicines in January 2028, the focus for all stakeholders must shift from procedural compliance to authentic, early engagement with rare disease communities. Regulation (EU) 2021/2282 mandates structured patient input at multiple stages—scoping, draft, and final reports—delivered through the secure HTA IT platform and synchronised with EMA timelines. For rare diseases, where each patient’s experience can be pivotal, late-stage engagement is no longer sufficient.

Building Genuine Partnership: Practical Steps

Start Even Earlier and Inclusively

Engage from the Outset: Begin involving patients at least 24 months before Phase III—ideally during Phase II—to ensure their insights shape trial design, endpoint selection, and study comparators. This early engagement is especially vital for rare diseases.
Broaden Representation: Proactively identify and invite a diverse range of patient advocates, including those from smaller or underrepresented groups, to participate in co-design workshops rather than just feedback sessions.
Lower Participation Barriers: Offer flexible meeting options, fair compensation, and provide materials in plain language and multiple languages, following best practices set by ABPI and EU guidance.
Empower and Support: Provide basic training on HTA and JCA processes so patient advocates can contribute confidently and effectively.
Shape JCA Success: Early, inclusive involvement enables patient insights to directly inform comparator choices and PICO (Population, Intervention, Comparator, Outcomes) definitions—foundational to robust and relevant JCA evaluations.

Embed Patient Perspective Across Functions

All functions—Medical Affairs, HEOR, Market Access, Regulatory Affairs—should be trained to think and act from the patient’s viewpoint, moving beyond one-off webinars to experiential “listening labs” and ongoing mentorship programs.
Role-play exercises and mentorship pairings help staff internalise empathy and translate patient stories into actionable endpoints.
Leverage Real-World Evidence (RWE) to ensure patient-defined outcomes are embedded in regulatory filings, especially for rare diseases where traditional data may be limited.

Measure and Celebrate Progress

Track metrics such as:
The speed at which patient diary entries appear on the HTA platform.
The number of patient-defined endpoints included in scoping reports.
Celebrate milestones—such as when a patient’s quote reshapes a protocol—in internal communications, and host regular “Patient Insight Roundtables” to keep lived experience at the forefront.

Regulatory Framing and Implementation Hurdles

Regulation (EU) 2021/2282 establishes mandatory patient input at key stages, a requirement reinforced by European Commission guidance from April and May 2025. However, standardisation across member states remains inconsistent, and the European Patients’ Forum (EPF) highlights resource constraints and feedback timelines as persistent barriers. Addressing these challenges is essential for equitable patient engagement across the EU. Best practices suggest leveraging digital tools and structured templates to streamline patient contributions, enabling more standardised and comparable evaluations.

The Value Proposition: Why Early Engagement Matters

Early and meaningful patient engagement not only meets regulatory requirements, but also de-risks development, improves market access, and builds trust with payers and regulators. Real-World Evidence (RWE) is increasingly recognised as a key component of JCA evaluations, with digital patient engagement tools and registries helping to capture patient-defined outcomes. Patient input is now systematically integrated into JCA processes, especially for rare diseases, where traditional clinical evidence may be limited.

Key Takeaways for Stakeholders

Engage Even Earlier: Involve patient groups from Phase II onward, ensuring patient-defined outcomes shape trial design and comparators.
Invest in Support: Provide resources, training, and practical support to enable diverse patient participation.
Prioritize Transparency: Use plain-language communication and feedback loops to keep patients informed and motivated.
Leverage Real-World Evidence: Incorporate patient registries and digital tools to inform both initial and ongoing assessments.

Looking Ahead

By January 2028, dossiers built on early, authentic engagement—where organisations are fluent in patient stories—will not just comply with JCA rules; they will set the standard for rare-disease assessment in Europe. The question is not whether you can afford to start now—it’s whether you can afford not to.

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About the Author

Rob Wyer is the founder and managing director of swii.ch, a consultancy that advances healthcare communication, patient advocacy, and regulatory strategy in the rare disease space. With extensive experience in digital-first patient engagement and EU HTA readiness, the Rob offers practical guidance to help clients navigate today’s dynamic healthcare environment.

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Making Patient Partnership a Reality in EU Joint Clinical Assessment (JCA)