Introduction

On 15 December 2021, the European Union (EU) embarked upon an uncharted regulatory approach by adopting Regulation (EU) 2021/2282 on Health Technology Assessment (HTA). This regulatory regime aims to improve the availability of innovative health technologies (such as pharmaceuticals and medical devices associated with high risks) for EU patients.

It seeks to harmonize the national HTA processes across Member States by enhancing cooperation and avoiding duplication of efforts while ensuring a thorough evaluation of their added value before being adopted by national health systems.

The HTA process involves a systematic evaluation of the properties, effects, or the impact of a health technology in comparison to another technology. This comparative assessment considers evidence related to medical, economic, social, and ethical aspects associated with the use of an HTA. Specifically, aspects such as the health problem and currently used technologies, relative clinical effectiveness and safety, economic feasibility, and other ethical and social aspects, etc.

On its own, the HTA process is not a novel approach, but the novelty of the approach rests with the harmonized procedural alignment in the EU. It involves collaboration between the Member States through the establishment of a Coordination Group (HTACG), which will oversee joint work and the development of EU Methodological Guidelines.

The regulation officially entered into force on 11 January 2022. However, industry must comply with the requirements starting from 12 January 2025. This transition period allows stakeholders to prepare for the new requirements.

Regulatory Scope

The Regulation covers a broad range of health technologies, with a particular focus on pharmaceuticals and medical devices in the risk category class III.

However, it does provide for certain exemptions for existing technologies that have already undergone assessment and are currently in use – unless significant modifications or new indications arise that warrant re-evaluation.

It also provides Member States the authority to conduct national HTAs for technologies that fall outside the scope of the regulation, allowing for local adaptation and assessment of lower-risk or less critical technologies.

Impact on International Research Projects

As stakeholders gear up for full implementation by January 2025, understanding the potential impacts – both positive and negative – on international collaboration, data sharing, regulatory alignment, resource allocation, market access, innovation, and transitional processes is crucial. Let's explore the anticipated benefits and challenges.

• Enhanced Collaboration: Streamlined, harmonized, and standardized HTA criteria across the EU will facilitate smoother integration of multinational study results. Collaborating entities can align their research methodologies with the EU’s requirements, ensuring that findings are applicable across all member states. At the same time, international projects may face bureaucratic challenges in aligning with varying national interpretations of the EU framework and non-legally binding guidelines, potentially complicating collaboration and endangering the key principle of legal certainty and legitimate expectations.
• Data Sharing and Transparency: The regulation encourages comprehensive data provision, fostering more open and robust research practices. Joint Clinical Assessments (JCA) will require detailed and consistent data, improving the quality and reliability of research outputs. On the other hand, the need to provide comprehensive data may impose significant administrative and financial burdens on manufacturers and research institutions, particularly smaller entities. The transparency requirements viewed in light of intellectual property rights and the use of scientific data and know-how may yet require further developments. The European Commission has already identified issues related to conflicts of interest as a key issue by initiating a public consultation on 29 May 2024.
• Regulatory Alignment: International projects will need to adjust research protocols and documentation practices to meet EU HTA requirements. Non-EU researchers and companies must familiarize themselves with the guidelines, ensuring compliance and facilitating smoother market entry. However, different member states may again interpret and apply HTA guidelines differently, leading to inconsistencies and fragmentation within the EU market and impede trans-border projects.
• Resource Allocation: Meeting HTA requirements may demand additional resources, including administrative support and financial investment for data collection and reporting. Smaller businesses and research groups may still struggle with the demands, potentially hindering innovation and market entry.
• Market Access: Compliance with HTA regulations can expedite market entry for new health technologies across the EU. Focus on clinical effectiveness ensures that only high-quality technologies gain approval, enhancing reputation and marketability. Standardized HTA processes could nevertheless lead to increased complexity in navigating requirements, resulting in delays in approval and market entry.
• Impacts on Innovation: The Regulation’s focus on clinical effectiveness and added value ensures that only high-quality, impactful technologies are approved. However, rigorous requirements for demonstrating clinical effectiveness might deter innovation, particularly in areas with harder-to-quantify long-term benefits. Moreover, stringent data requirements could stifle creativity and risk-taking essential for breakthrough innovations.
• Transitional Challenges: The transition period until January 2025 allows stakeholders time to prepare for the new regulatory landscape and make necessary adjustments. However, experiences from past legislative actions show that the transition period may not be sufficient for all stakeholders to fully adapt, potentially disrupting ongoing projects and delaying new initiatives. Existing projects may need significant adjustments to comply, leading to additional time and resource investments.

Conclusion

While the Regulation offers several positive impacts, it also presents negative impacts and risks for which industry is left to find practical and pragmatic solutions. Balancing these impacts will be crucial for stakeholders navigating the new regulatory environment in international cross-border research projects.

As stakeholders embark on new research projects and international collaborations, it is imperative to understand the impacts of this regulatory scheme on the anticipated project. Strategic planning, a thorough understanding of the regulatory requirements, appropriate contractual arrangements, and proactive adjustments will be essential in successfully navigating this new landscape.

For detailed insights and tailored legal advice on how to safeguard your innovations and research projects, contact our regulatory experts.

Contact person: Bobby Arash