Refining the Parallels Between EMA Expansion and JCA Implementation: Quantitative Insights and Recommendations

The implementation of Joint Clinical Assessment (JCA) under the EU HTA Regulation represents a significant step toward harmonized health technology assessment (HTA) in Europe. It shares many parallels with the establishment and expansion of the European Medicines Agency (EMA), while both aim to harmonize decision-making across the EU—EMA for regulatory approvals and JCA for HTA clinical assessments (Ref Table 1). Historical lessons from EMA—particularly the unequal distribution of rapporteur and co-rapporteur roles—suggest that similar challenges may arise in JCA, affecting not only the allocation of assessors/co-assessors but also participation in key preparatory steps, such as the PICO survey. Below we share some potential parallel scenarios with JCA as was seen in the past with EMA and also lay out some recommendations to enable balanced JCA implementation.

Table 1 Key Parallels Between EMA and JCA Implementation

1. Potential centralization of assessments with unequal participation

When EMA established a centralized drug approval system 30 years ago, and expanded in 2004 to 10 additional Member States (MS), certain member states became dominant players in the assessment process. Over the past decade (2013-2022), the vast majority of rapporteur and co-rapporteur roles were assigned to:

• Germany – 230 cases (8%)
• United Kingdom (until Brexit) – 220 cases (8%)
• France – 210 cases (7%)
• Netherlands – 200 cases (7%)
• Sweden – 190 cases (7%)
• Italy and Spain (6% each)

Together, these five countries handled ~40% of all EMA assessments, concentrating expertise and decision-making power (Ref. Table 2).

Table 2 Distribution of Rapporteur Co rapporteurs EMA 2013-2023¹

Smaller member states, in contrast, had minimal participation:

• Bulgaria, Cyprus, Luxembourg, and Malta – Only 10 assignments over a decade (<1% each)

What this means for JCA:

• Although the guidance on the appointment of assessors and co-assessors calls for geographic diversity, countries with strong HTA infrastructure (Germany, France, Netherlands, Sweden) are likely to dominate assessor roles for JCA.
• Smaller or less experienced HTA bodies (e.g., Bulgaria, Malta, Cyprus) will struggle to secure assessor/co-assessor roles, limiting their influence.
• Without structured burden-sharing, the same countries that dominate EMA assessments may also lead most JCA evaluations.

2. Limited contribution of smaller Member States (MS) to the PICO survey

The PICO survey (which determines the Population, Intervention, Comparator, and Outcomes to be assessed in JCA) is a critical step in defining the scope of assessments. Ideally, all MS contribute to ensure JCA reflects national needs. However, there is a high risk that smaller Member States will have minimal input, as seen in EMA assessment participation trends.

Evidence from EMA Rapporteur Trends

• In EMA assessments, smaller countries contributed <5% of total assessments.
• The same pattern is likely to occur in the PICO process, where larger HTAs will drive the definition of comparators and endpoints.
• This can lead to lower probability of any potential application of JCA report outcomes by national/local payer in smaller member states.

Potential Consequences

• JCA-defined comparators may not align with national treatment practices in smaller member states.
• HTA bodies from smaller countries may continue conducting their own assessments, or may have difficulty in using JCA assessment, undermining JCA’s goal of harmonization.
• Delays in reimbursement may occur if national payers do not consider JCA-recommended comparators and outcomes relevant.

3. Risk of fragmentation or lack of consistency across indications/approvals in JCA adoption

Although EMA’s centralized approval process is legally binding, its impact on pricing and reimbursement remains decentralized, causing variations across MS.

Today, only ~43% of centrally approved innovative medicines are available across EU countries, indicating that more than half of these medicines are not accessible to patients in certain countries². This is largely because national HTA bodies have different focus (assessment of drug benefit/risk profile vs. value for money and comparative effectiveness), methodologies, comparators, and endpoints than those used in regulatory decision-making. For JCA, this means:

• Since JCA is not legally binding, some Member States may continue conducting their own HTA assessments of the relative effectiveness
• Large HTA bodies could prioritize their national assessments over JCA results, leading to fragmentation.
• A lack of perceived neutrality in JCA outcomes (if dominated by a few HTA agencies) could reduce trust in the process.

Recommendations for a more balanced JCA implementation

To prevent historically strong HTA bodies from dominating both the assessor roles and PICO process, lessons from EMA suggest the following considerations:

For JCA Assessor/Co-Assessor roles:

• Quota system for assessors: No single country should handle more than 15% of JCAs, ensuring fair distribution.
• Mandatory rotation for assessor/co-assessor roles: This would prevent over-concentration of decision-making in a small number of countries.
• Capacity-building initiatives for smaller HTA bodies:
  • Funding and training programs should be established to improve expertise in underrepresented member states.
  • This would help avoid situations where <5% of JCAs are handled by certain regions, as happened under EMA.

For the PICO survey process:

• Minimum contribution requirement: Each country should provide input on at least a predefined percentage of JCA assessments.
• Structured engagement with smaller HTA bodies: Create regional working groups to ensure smaller markets’ needs are incorporated.
• Comparator validation process:  When available use EU RWE data (e.g. DARWIN network) to ensure the comparators chosen are used/applicable to all countries

Conclusion

The EMA rapporteur and co-rapporteur system provides a clear precedent for how JCA assessors will likely be distributed. Without intervention, a small number of large HTA agencies may dominate JCA assessments and the PICO survey process, leading to:

• Limited involvement of smaller member states
• Potential resistance to JCA report recommendation by countries that feel underrepresented
• JCA-defined comparators and endpoints that do not match the needs of all EU markets

By learning from EMA’s experience, policymakers and pharmaceutical companies/biotechs can take proactive measures to ensure a fair, representative, and efficient JCA process, ensuring that both assessments and the PICO definition phase reflect the diversity of healthcare systems across Europe and proactive involvement of all member states in shaping assessments.

To learn more from our experts on navigating the JCA, contact us.

References

¹EFPIA Patients W.A.I.T. Indicator 2023 Survey https://efpia.eu/media/vtapbere/efpia-patient-wait-indicator-2024.pdf

²https://www.ema.europa.eu/en/about-us/annual-reports-work-programmes1