Navigating the New EU HTA Landscape: A Practical Guide for Health Technology Developers

The new EU Health Technology Assessment (HTA) regulation is now in effect, significantly impacting how health technologies gain market access. A core component is the Joint Clinical Assessment (JCA), bringing both challenges and opportunities. This post breaks down the JCA process, focusing on the crucial PICO scope determination, and offers guidance for Health Technology Developers (HTDs).

Understanding the JCA Process:

The JCA is triggered when a manufacturer submits a marketing authorization application for a new oncology or advanced therapy medicinal product (ATMP) to the European Medicines Agency (EMA). The process revolves around defining and addressing specific research questions using the PICO framework (Population, Intervention, Comparator, Outcome).



1. EMA Submission Triggers JCA: The process begins with the submission of a marketing authorization application to the EMA.
2. Scoping Process Begins: The crucial "scoping process" is initiated, where the PICO questions are formulated.
3. Member State Input is Key: National Competent Authorities (NCAs) and Health Technology Assessment (HTA) bodies from each EU Member State contribute their perspectives on relevant PICO elements. This step highlights the diversity of viewpoints across Europe.
4. Consolidation into a Unified Scope: A JCA Coordinating Group (often within or appointed by the HTA Network) consolidates the Member State input, aiming to create a unified and manageable set of PICOs. This consolidation process presents a significant challenge.
5. PICO Scope Publication (Expected): While still evolving, it's anticipated that the final PICO scope for a given product will be publicly available, likely through a dedicated platform. This transparency is vital for manufacturers.

Deciphering the PICO Scope:

Finding information about the PICO scope requires proactive engagement:

• Monitor EMA Submissions: Tracking new oncology and ATMP applications to the EMA is the first step.
• Follow EU HTA Network/HTA Bodies: Staying up-to-date with the EU HTA Network and individual HTA bodies is essential.
• Check for Official Publications: Look for announcements from the European Commission or related agencies.
• Engage with Stakeholders: Connecting with HTA experts, patient groups, and industry associations can provide valuable insights.
• Anticipate Future Guidance: As the JCA process matures, expect more detailed guidance on PICO scope determination and access.

Navigating the PICO Challenge:

The consolidation process can result in a substantial number of PICOs (potentially 10-30 or more for oncology products), creating the "PICO anxiety." However, this doesn't translate to needing as many separate clinical trials.

• Strategic Trial Design: Smart trial design, incorporating multiple arms, subgroup analyses, and multiple endpoints, allows data collection relevant to several PICOs within a single trial.
• Evidence Synthesis: Techniques like network meta-analysis can combine data from various studies to address all PICOs, even if trials don't perfectly align.
• Prioritization: Focus on the most critical PICOs likely to influence market access decisions.
• Flexibility is Paramount: The JCA process is evolving, requiring HTDs to be adaptable in their evidence generation strategies.

Key Recommendations for HTDs:

• Educate and Engage: Ensure all stakeholders understand the new EU HTA process.
• Start Early: Integrate PICO predictions into Phase 3 planning (and revisit 18 months before submission).
• Maintain Focus on National Procedures: Remember that national procedures remain crucial for funding decisions, even after the JCA.