The Evolution of Pan-EU HTA: A new era of harmonized clinical assessments in Europe

Health Technology Assessment (HTA) is the process of evaluating the value, effectiveness, costs, and broader impact of healthcare interventions, including medicines and medical devices. Through the harmonization efforts in the EU, the HTA process is set to become more streamlined, offering a new pathway for timely access to innovative therapies across Europe. However, challenges remain, and manufacturers must be prepared to meet the evolving demands of the European market.

Since 1995, when the European Medicines Agency (EMA) was granted the responsibility of evaluating the safety and efficacy of pharmaceuticals through a centralized drug approval process, the European Union (EU) has been a key player in streamlining HTAsacross its 27 member states. The implementation of Regulation (EU) 2021/2282, adopted on December 15, 2021, marks a significant step toward harmonizing the clinical assessment of drugs and medical devices in Europe. This new regulation aims to replace the redundant evaluations currently carried out by multiple national HTA bodies—such as G-BA in Germany or HAS in France—by creating a more unified and efficient process.

The primary goal behind this harmonization is to reduce the multiple country-specific assessments and enhance the transparency and efficiency of a single market for health technologies. At the core of this transformation is the introduction of the Joint Clinical Assessment (JCA), a process that will allow pharmaceutical manufacturers to submit clinical evidence only once, at the European level. However, while this change aims to streamline the process, individual member states will still maintain sovereignty over health services, resource allocation, reimbursement, and pricing decisions.^1,2,3

Framework for Joint HTA Cooperation^1,2,3

• Joint Clinical Assessments (JCAs): Coordinated assessments focusing on the clinical effectiveness of health technologies.
• Joint Scientific Consultations (JSCs): Guidance provided for product developers early in the development phase.
• Identification of Emerging Health Technologies: Efforts to monitor and identify upcoming innovations in health technology.
• Common Procedures and Methodologies: Standardized methods across the EU to ensure uniformity in assessments.

Key Principles of the HTA Regulation^1,2,3

• Focus on Clinical Domains: The assessments will strictly cover clinical outcomes, excluding economic assessments like pricing and reimbursement.
• EU HTA Bodies in Charge: National HTA bodies are still responsible for interpreting the added value of technologies for their specific health systems.
• High Standards: Emphasis on quality, timeliness, and transparency of the assessments.
• Integration in National Processes: Results from joint work will be integrated into national decision-making.
• Independent Expert Input: The process includes contributions from external experts.
• Stakeholder Engagement: The regulation encourages inclusiveness, allowing input from various stakeholders.
• Phased Implementation: The regulation will be gradually rolled out.

Benefits and Challenges of a Harmonized System^1,2,3

• The shift is expected to benefit countries lacking in-depth expertise or infrastructure for conducting their own Health Technology Assessments (HTAs).
• Manufacturers are likely to see reduced redundant evaluations, streamlining the market access process.
• New challenges arise, particularly in how manufacturers approach market access in Europe.
• The Joint Clinical Assessment (JCA) will systematically assess the clinical evidence of new drugs compared to the standard of care.
• The relative effectiveness of these drugs and their relevance to patients will be crucial in market access.
• The Member State Coordination Group on Health Technology Assessment will oversee the JCA process.
• The JCA will align closely with the European Medicines Agency (EMA) marketing authorization procedures to ensure timely assessments.
• The JCA process will support member states’ decision-making at the time of drug launch.
• While the JCA focuses on clinical evidence at the European level, national value judgments remain separate.
• Decisions on a drug’s medical added benefit will continue to be made at the national level.

The Role of Early Engagement

One of the key opportunities within this new system is the ability for manufacturers to engage early with assessors through Joint Scientific Consultations (JSCs). These consultations offer an important chance for companies to gather input from the EMA and national HTA bodies, providing guidance on clinical trial design, evidence requirements, and more. Early engagement could greatly improve a manufacturer’s strategic planning and increase the likelihood of successful market access.

Implementation of the JCA: A Step-by-Step Approach^1,2,3

Starting in 2025, the JCA will be applied to oncology drugs and advanced therapy medicinal products (ATMPs). By 2030, all drugs registered through the centralized procedure will be subject to a JCA. It is important to note that while the JCA will take into account the specific needs of different member states, additional evidence may still be required to meet national criteria.

Medical devices

• Class IIIb/IV devices, may be subject to the JCA process in future.
• Device manufacturers may be called upon to submit comparative clinical data based on defined criteria, such as unmet clinical needs or the involvement of artificial intelligence.

For both drugs and medical devices, early preparation and understanding of evidence requirements will be crucial for success.

Understanding Pan-EU: What It Means for Pharmaceutical Companies

The new EU-HTA regulation represents a profound shift in the way pharmaceutical companies will need to navigate market access. While the JCA aims to reduce redundant HTA activities, it may also result in increased demands for clinical evidence, particularly as patient relevance and the relative effectiveness of drugs become more significant.

There are also concerns that this stepwise approach—where the JCA occurs at the EU level and national assessments follow—could potentially delay market access. For example, the requirement for country-specific comparator therapies may create a need for additional evidence generation, complicating the process.

Preparing for the Future

• Pharmaceutical and medical device companies must remain vigilant as the JCA and other HTA processes continue to evolve
• Close attention to guidance documents, early engagement with assessors, and a strong understanding of both European and national requirements will be essential for navigating this new regulatory landscape

As the EU moves toward a more harmonized system, the future of drug approval and market access is poised to change dramatically. For companies, the key will be adapting to these changes early to ensure timely and successful access to the European market.

References:

1. Recommendations for production process of Relative Effectiveness Assessments after Joint Action 3 May 2021. This document is part of the project / joint action ‘724130 / EUnetHTA JA3’ which has received funding from the European Union’s Health Programme (20142020)
2. Recommendations for Early Dialogues Based on the Experience of EUnetHTA Joint Action 3. Diemen (The Netherlands): EUnetHTA; 2021. Available from: https://www.eunethta.eu
3. Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU

 Authors:

Asif Syed, PhD.
Senior Scientific Writer II
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Dhanya Mukundan, MDS (Oral Medicine and Radiology)
Expert Scientific Writer, Enago Life Sciences
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Raghuraj Puthige, PhD.
Function Head, Medical Communications – Enago Life Sciences
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