EU Adopts Regulation on Pan-European Health Technology Assessment

Published February 18, 2022

Executive Summary

• A new pan-European Health Technology Assessment (HTA) regulation that will redefine the processes for the evaluation of new medicines in the EU entered into force in January 2022
• Although it will not become fully active until 2025, the new regulation will require EU member states to give “due consideration” to joint clinical assessment (JCA) reports and provide feedback to the European Commission
• These new JCAs will only focus on the clinical domain of HTAs and thus they will not set a framework for economic assessments; however, voluntary cooperation between member states on other aspects of HTA will remain possible
• Member countries will remain free to conduct complementary clinical and non-clinical analyses to support these assessments
• Next steps that will be taken during the three-year implementation phase will be critical to determine additional details of the new regulation and reveal its true impact

The New Health Technology Regulation Focuses on the Clinical Aspects of HTA

Pan-European HTA will enter into force in January 2022 and finally become active in 2025. This comes almost four years after the European Commission’s original proposal, and at a time where urgency for European HTA processes is growing, this will support timely patient access to treatments at prices that reflect the value of that treatment while managing uncertainty associated with limited data on long term benefits of treatment at the time of launch.

The new regulation came into effect last month, but member states will be allowed three years to adapt their national legislation and help interested parties prepare for the new process. The regulation paves the way for mandatory joint clinical assessment (JCA) as well as joint scientific consultation (JSC), and horizon scanning, focusing on the relative clinical effectiveness and relative clinical safety of a new health technology as compared with existing technologies.

Health Technology Regulation Benefits

The European Commission notes that JCA reports will provide a new framework for health experts, providers, manufacturers and patients that will help address unmet medical needs and facilitate access to innovative medicines and some high-risk medical devices. Patients and clinicians will highly benefit from JCA reports that are of high scientific quality, transparent and accessible to the public.

In addition, the new JCA regulation will support Member States in taking more evidence-based and timely decisions on patient access to new medicines and medical devices, as well as allowing them to pool their HTA resources and expertise.

Finally, manufacturers in the pharmaceutical and medical device sectors will benefit from JCAs by having more clarity and predictability concerning the clinical evidence requirements for HTA. In addition, they could also profit from efficiency gains when submitting clinical data for HTA assessments, as the new JCA regulation has the potential to entail a single EU-level submission of clinical evidence to support HTA reports rather than multiple parallel submissions to each national system.

New Health Technology Assessment Process

As part of the new European JCA process, member states will be required to report to the Commission and give “due consideration” to JCA assessments when performing their own national HTA of drugs that have undergone pan-European evaluation. The JCA report will be attached as an annex to national HTA reports, and member states will have to provide information on how they used the JCA in their national processes.
 
Furthermore, while JCAs will have a narrow focus on clinical domains, EU member states will remain free to engage in additional voluntary cooperation on economic aspects of HTA due to growth of broader cross-border collaborations in recent years. In an FAQ on the new regulation, the European Commission notes that member states will be able to complement JCAs with additional clinical analyses (e.g., national epidemiological data, information on the national healthcare context) and non-clinical analyses (e.g., budget impact or cost-effectiveness assessments). Member states will also retain sole responsibility for drawing conclusions regarding the value of a new health technology and making pricing and reimbursement decisions.

Within two years of the application of the new regulation, member states will be expected to report to the Commission regarding the consideration of joint work in their national HTA processes, as well as the workload of the Coordination Group and whether they have considered the developed methodological guidance.

Next Steps to Implement the Health Technology Regulation

As a first step towards the implementation of the new regulation, in the first half of 2022, the Commission will set up the Coordination Group composed of representatives designated by member states. The group is tentatively scheduled to hold its first meeting in June 2022. Around the same time, a conference will be held to inform stakeholder organizations about the new regulation.

The actual methodology guiding assessments will be developed though meetings of country representatives over the next few years. Alignment around priorities for each clinical assessment will be critical at this stage. The representatives will need to resolve key differences such as acceptability and value of surrogate endpoints and evidence beyond RCTs, as well as perception of clinically meaningful thresholds. 

Future-facing Considerations for Price and Market Access

The new EU HTA regulation is expected to have a major impact on the future of EU HTA submissions. All pharmaceutical manufacturers, especially for products launching from 2025 onwards, should ensure that clinical development programs align with the new JCA requirements, as the outcomes of this assessment will be key to inform the clinical value and potential pricing and reimbursement throughout EU member states.

It will be critical to track next steps and updates from the European Commission during this three-year transition phase, as well as ensuring early engagement with the Joint Scientific Consultation Group, to confirm alignment on all clinical aspects of the development of new drugs, such as the appropriate pivotal trial design, endpoint selection, and adequate clinical comparator.

Some challenges related to this new regulation can be anticipated. For example, significant differences exist today across member states regarding HTA value frameworks, such as differing requirements for clinical trial endpoints, emphasis on different drivers beyond efficacy and safety measures (e.g., QoL) as well as differing approaches to the measurement of these, which may be hard to integrate within the new assessments. Additionally, some European countries assess the clinical and economic aspects of HTA in an intertwined manner (e.g., GBR), and it may be challenging to separate these two aspects in the new JCA reports. Thus, manufacturers should consider these expected challenges and be prepared to effectively mitigate and manage these.

Additionally, pharmaceutical manufacturers should anticipate the need for complementary submission requests for national-level HTA assessments, potentially including additional clinical as well as non-clinical analyses (e.g., cost-effectiveness evaluations) to support the assessment process.

To conclude, the new pan-European regulation provides a framework for clinical assessments that is expected to facilitate transparency and access to new and innovative treatments. Time will tell what the real impact of the new evaluation will be for the future of health technology assessments in Europe, as well as its potential implications for speed and quality of access to pharmaceuticals globally.

By: Salomé Monreal Louly