European HTA Regulation: How will it work in practice?

By Chloe Sheppard, Associate Consultant

Contact: [email protected]

A major milestone in the creation of pan-European joint health technology assessment (HTA) was reached in January this year with the final adoption of the long-awaited EU HTA Regulation. This means that, starting in January 2025, all advanced therapy medicinal products (ATMPs) and oncology drugs will be mandated to undergo a joint clinical assessment (JCA) at European level.

Currently, biopharma and other stakeholders have many unanswered questions around key aspects of the Regulation. Perhaps most critical are the unknowns on the JCA evaluation methodology (e.g. comparator selection, acceptability of clinical evidence), the potential new role of individual country agencies, and how exactly the joint HTA process will link to national processes to make the operation more efficient.

What’s the deal with the EU HTA Regulation?

The Regulation seeks to provide a permanent framework for sustainable cooperation on HTA in Europe. The framework will be governed by a Coordination Group including representatives from national HTA authorities in all Member States and will replace the voluntary EUnetHTA system. Over the next 36 months, there will be a phased implementation of the Regulation.

Four key activities will be conducted as part of the EU HTA Regulation:

  1. Joint clinical assessment (JCA) – mandatory assessments focusing on the most innovative health technologies
  2. Joint scientific consultation (JSC) – offering manufacturers advice on evidence requirements for the HTA submission
  3. Identification of the most innovative health technologies
  4. Voluntary cooperation on areas outside of the mandated scope

It will be mandatory for manufacturers to take part in JCAs, but it will not be mandatory for individual Member States to adopt the joint report. National authorities would be free to draw conclusions on the overall clinical added value of a health technology in the context of their specific healthcare system. However, they will not be able to request any information or evidence from the manufacturer that has already been submitted in the joint assessment. Member States will still have the right to conduct economic assessments and make pricing and reimbursement decisions on health technologies.

How will the EU HTA Regulation work in practice?

The aim of the Regulation is to harmonise and improve patient access to innovative therapies in Europe. The EUnetHTA21 Consortium, led by the Dutch Healthcare Institute (ZIN) and comprised of 21 national HTA agencies, will be responsible for developing the frameworks and methodologies that will be used for the JCAs.

Methodology for joint clinical assessments

EUnetHTA have stated that the lessons learned from the EUnetHTA Joint Action 3 will be taken into account in the JCA methodology under the new Regulation. In September 2021, EUnetHTA published a white paper summarising the learnings from the Joint Action 3 and outlined a “future model of cooperation”. However, this is largely focused on a framework for cooperation and outlines the stakeholders and processes required to make a joint HTA system a success. This framework has limited details on exactly how the JCA will consider key aspects of the clinical assessment such as endpoints, comparators, acceptability of evidence (e.g. indirect treatment comparisons, real world evidence). Could it be possible that ZIN, as the Secretariat of the Consortium, has a larger influence in the development of the methodology than other countries? If so, we may see elements of ZIN’s clinical assessment process come through more strongly in the JCA framework.

The EUnetHTA21 Consortium has published a project plan covering the deliverables that will be published throughout 2022 and 2023 covering topics including the scoping process, validity of clinical studies, comparators and comparisons and applicability of evidence. It will be important for industry to monitor and review the work of the Consortium as it is released.

Potential new role of national HTA agencies

For national HTA agencies it is possible that, as a result of the Regulation, their focus will be partly shifted to economic evaluations of new health technologies. However, for individual countries to have no further role in clinical assessments at all would require Member States to be fully aligned with the EU JCA methodology, as it will not be mandatory for countries to simply adopt the JCA.

The question then becomes: how aligned will all Member States be with the JCA methodology? And moreover, is it possible to develop a clinical assessment methodology which aligns with the perspectives of all Member States who have historically had differing ideas about value and the acceptability of evidence?

Consensus from Member States on key elements of the JCA methodology is critical, or it is possible that certain countries (most likely larger countries with very established and specific evaluation processes) will request additional clinical information from manufacturers which is relevant to their local requirements. In instances where the standard of care treatment for a particular condition varies between EU countries, or a clinical trial endpoint is accepted by one national HTA agency but rejected by another, without alignment then country HTA agencies may request additional evidence from the manufacturers.

Merging pan-European and country-level HTA processes – will efficiency increase?

One potential issue with individual countries requesting additional evidence on top of the JCA is efficiency. Assuming the additional evidence request from the national agency is made after they have reviewed the JCA, this could cause delays to the HTA process and subsequently impact the time to reach a final reimbursement decision.

However, in countries which do not currently perform formal, obligatory HTAs, such as Greece, the adoption of JCA reports by national authorities would in principle be smoother and more efficient than in those countries which have established formal HTA systems, as existing processes would not have to be accounted for. In these countries, there is the most potential for the EU HTA Regulation to increase efficiency by providing reports upon which Member States can make evidence-based decisions about reimbursement.

Next steps for manufacturers

For the Regulation to achieve its goal to improve patient access to innovative health technologies across Europe, cooperation will be required not only between national HTA agencies but also between manufacturers and HTA agencies. Manufacturers should seek to engage in the public consultations that will be held by EUnetHTA21 throughout this year to represent the industry perspective on the JCA methodology which, looking ahead, will become the foundation of pan-European joint HTA.

A sustainable ecosystem for collaboration on HTA is a worthwhile aspiration but it requires flexibility and alignment from all stakeholders, including national HTA agencies, manufacturers and EU policy makers. We hope that in the coming months the work of the EUnetHTA21 Consortium will provide a starting point for cooperation which will help Europe deliver on this important goal.

If you would like to understand more about how the EU HTA Regulation will impact your company’s assets and to tailor your PRMA strategy accordingly, please reach out to the P4A team at [email protected].



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