The European Union is in the midst of a transformative shift toward a harmonized Health Technology Assessment (HTA) system under the Joint Clinical Assessment (JCA) process, enacted by the Health Technology Assessment Regulation (HTAR). Starting January 12, 2025, with oncology drugs and advanced therapy medicinal products (ATMPs), the JCA will gradually expand—covering orphan drugs by 2028 and all EMA-authorized medicines by 2030. In this changing environment, early integration of real-world patient insights is a strategic best practice, particularly for rare disease therapies where patient experiences reveal critical unmet needs and unique challenges.
Early Trends in the JCA Process
Recent industry reports underscore how quickly the JCA is taking shape. For example, as noted in a Pharmaceutical Technology article from April 22, 2025, two therapies are already moving through this new system:
- Tovorafenib by Ipsen, aimed at pediatric low-grade glioma, is under review by Ireland’s National Centre for Pharmacoeconomics (NCPE) and Germany’s IQWiG.
- Lifileucel (Amtagvi) by Iovance Biotherapeutics, designed for melanoma, is being evaluated by France’s HAS with insights from Poland’s AOTMiT.
These early milestones, aligned with EMA marketing authorizations, reflect a clear drive towards faster, more consistent decision-making. They also underline a rising expectation among regulators: evidence must be rooted in real-world impact. With rare diseases affecting around 30 million Europeans, capturing patient input early becomes essential, especially for ATMPs that often contend with limited data from small patient populations.
The PICO Framework—Enhanced by Patient Insights
At the heart of the JCA is the PICO framework (Population, Intervention, Comparator, Outcomes), which aims to create a clear, consistent basis for HTA evaluations:
- Population: Detailed understanding of a patient’s journey is essential. For rare diseases, this means acknowledging challenges like lengthy diagnostic processes and variable responses.
- Intervention: It is no longer enough to rely solely on traditional clinical endpoints. Robust Patient-Reported Outcome Measures (PROMs) provide crucial insights into improvements in quality of life.
- Comparator: Real-world patient feedback helps in establishing what constitutes a meaningful improvement compared with existing treatments.
- Outcomes: Regulatory bodies value endpoints that capture tangible benefits in daily living, complementing the standard measures of effectiveness and safety.
Industry experience suggests that embedding patient insights early not only enriches the clinical dossier but also builds trust with regulators and payers by illuminating the real impact of therapies.
A Phased Approach to Patient Engagement
Experts recommend that patient engagement should be strategically integrated throughout the product development lifecycle to align with JCA expectations. A typical roadmap might include:
- Pre-Clinical Development (18–36 Months Before Phase III): Engaging with patient advocacy groups (PAGs) early can uncover key unmet needs and help define study endpoints that truly reflect patient challenges.
- Clinical Trial Design (Before Phase III Initiation): Methods such as qualitative interviews and digital surveys can be used to embed PROMs and ensure that study protocols resonate with patient priorities.
- Pre-JCA Submission (6–12 Months Prior): Consolidating patient preference data and developing real-world case studies, in collaboration with PAGs, strengthens the clinical dossier during the crucial submission window following scoping (typically 100 days post-scoping as per JCA timelines).
- Post-Market Authorization (Ongoing for HTA Reassessment): Establishing patient registries and continuous feedback loops ensures that real-world evidence stays current—a key element for ongoing pricing and reimbursement discussions.
This lifecycle strategy, which benefits from early planning to meet the rigorous and tight timelines of the JCA, positions patient insights as a vital asset for navigating the assessment process.
Navigating Challenges and Seizing Opportunities
Although the JCA aims to streamline access across the EU, challenges remain. Smaller markets may see earlier launches, yet larger markets like France and Germany—where decision timelines average between 311 to 320 days—could face delays due to existing bureaucratic processes. There is also concern over how ATMPs and rare disease therapies will be handled, given their inherent evidence limitations. Moreover, research by the European Patients’ Forum (EPF) highlights gaps in patient engagement across member states, with smaller patient groups often lacking the necessary resources to meaningfully participate. EPF’s recommendations, which range from standardized frameworks to timely feedback on inputs, are viewed by industry observers as key measures to enhance overall inclusivity.
On the flip side, the JCA’s commitment to transparency—through shared PICO frameworks and stakeholder summaries—offers the potential for faster, more informed pricing and reimbursement decisions. Striking the right balance between protecting sensitive data and facilitating transparent communication will be vital for maintaining trust across the board.
Building a Patient-Centric HTA Ecosystem
The early JCA assessments of therapies like Tovorafenib and Lifileucel signal a turning point in the EU HTA landscape. For rare disease therapies, integrating robust patient data from the very start is more than a procedural step—it’s an investment in transforming patient outcomes and ensuring equitable market access.
References:
- Pharmaceutical Technology, “EU HTA Joint Clinical Assessments: Navigating Potential Challenges Ahead of 2025,” April 22, 2025.
- European Commission, Joint Clinical Assessments webpage (latest updates, April/May 2025).
- European Patients’ Forum (EPF), “10 Key Recommendations for Enhancing Joint Clinical Assessments,” 2024.
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.