The EU’s Health Technology Assessment Regulation: Implementation one year on

STANDALONE: The EU’s Health Technology Assessment Regulation: Implementation one year on

DOWNLOADPDF | 1.19 Mb

Introduction 

The EU’s Health Technology Assessment Regulation (EU-HTAR), which came into effect in January 2025, marked the first permanent partnership between the European Medicines Agency (EMA) and national health technology assessment (HTA) bodies. The cornerstone of this potentially transformative shift was joint clinical assessments (JCAs). JCAs provide a single assessment of the clinical effectiveness and added value of new health technologies, replacing the need for separate health technology assessments (HTAs) in each Member State.

This article explores how EU-HTAR is being used and implemented, the impact on national HTA bodies, health technology developers (HTDs) and patients, and how the HTA Regulation implementation can be improved.

Overview of the EU-HTAR

Most EU countries have a HTA system informing health policy decisions.^[1] While this allowed Member States to evaluate the effectiveness and value of health technologies, it left developers carrying the burden of completing multiple HTAs, each with their own divergent data requests. This led to unpredictable timelines and increased costs. The EU-HTAR aims to reduce the burden of multiple submissions, remove barriers to innovation and accelerate access to the market across EU Member States.^[2]

A key change under EU-HTAR was the introduction of JCAs – a single assessment of the clinical effectiveness and added value of new health technologies conducted at an EU level. The aim was to provide a single ‘scientific compilation and the description of a comparative analysis of the available clinical evidence on a health technology in comparison with one or more other health technologies or existing procedures’.^[2] Predicted benefits included the pooling of HTA resources and expertise to support evidence-based and timely decisions on patient access for Member States, greater clarity and predictability concerning clinical evidence requirements for HTDs, and greater transparency and access to innovative medicines for patients and clinicians.^[3]

The framework for JCAs is structured around population, intervention, comparator and outcomes (PICO) components as defined by the EMA, national HTA bodies and industry stakeholders.^[4] This ensures assessments are tailored to the needs of the target population and facilitates submission of a single, comprehensive, evidence-based evaluation of the product’s value. However, Member States may still request additional analyses to meet their specific requirements.

Initially, the framework applied to all new therapeutic medicines and oncology drugs.^[3] On 17 October 2025, the EC adopted an implementing regulation establishing the rules for JCAs of medical devices and in vitro diagnostic medical devices.^[5]

Progress so far

In its Annual Work Programme for 2025, the HTA Coordination Group estimated it would initiate 25 JCAs in 2025: 17 for cancer drugs with a new active substance and eight for advanced therapy medicinal products (ATMPs).^[6]

In April 2025, the first JCAs for paediatric low-grade glioma (LGG) and melanoma treatments were announced.^[7] There are currently nine ongoing JCAs for treatments targeting conditions including bladder cancer, extensive-stage small cell lung cancer (ES-SCLC) and synovial sarcoma.^[8] At the time of writing, no JCAs have been completed. This appears to put the Annual Work Programme significantly behind schedule.

The Programme also estimated that five to seven joint scientific consultations (JSCs) for medicinal products would be initiated in 2025 and also one to three JSCs for medical devices.^[6] The aim of the JSCs is to streamline preparation for JCA and enhance the quality of clinical studies. The HTA Coordination Group scheduled seven JSCs in 2025, with the first announced as complete in October 2025.^[5]

The impact on national HTA bodies

While the long-term aim of EU-HTAR is to streamline processes, the Regulation is initially creating an additional burden for national HTA bodies who are faced with new, complex procedures, while continuing their usual work.^[9]

Despite this, the reaction to the principle of EU-HTAR has been broadly positive.^[10] The long-term gains, including the opportunity to pool resources and expertise, are thought to outweigh the short-term costs. As the use of EU HTAs becomes more common place, it is hoped that the ability to work more collaboratively will lower the administrative burden on national healthcare systems, making the process more cost-effective, increasing efficiency and freeing up resources for other healthcare activities. If achieved, these benefits will be particularly beneficial for smaller, less resourced countries.^[9]

The PICO framework also provides a rigorous, scientifically-sound evaluation of new technologies, which should support evidence-based decision-making by policymakers and healthcare providers.^[11] However, stakeholders have emphasised the need to limit the number of final PICOs to avoid creating a ‘many-headed monster’.^[10] Yet this PICO framework is not meant to be a one-size-fits-all approach, but rather a backbone for harmonised, consistent and robust evidence to be leveraged by national jurisdictions.

The impact on developers

For developers, EU-HTAR requires planning to start much earlier in the drug development cycle and generation of a large volume of evidence, including the accommodation of language-specific requirements, within a compressed timeframe.

A letter of intent is submitted at the point of EMA submission for regulatory approval. A PICO-scoping exercise by the EU consortium, which is likely to include contributions from each country, then finalises the scope of the JCA. Developers have just 100 days to complete the dossier preparation and submission phase once the finalised scope is available. These accelerated timelines require proactive planning, clear documentation and the ability to quickly adapt to evolving requests.

There have also been concerns about the availability of JSCs and the striking gap between the proposed number of JSCs compared to JCAs. JSCs are designed to offer recommendations to HTDs on their development plans for a medicinal product or medical device, ensuring evidence meets requirements for a subsequent JCA. However, the industry has said that the number of scheduled JSCs for 2025 did not reflect the realities of product development.^[12] Early advice is critical to ensure developers can adapt clinical trial designs and compile quality dossiers. If misunderstandings are not resolved early, there is a higher risk of delays. This threatens the key aims of EU-HTAR: to reduce developer burden and speed up access to innovative treatments for patients.

Developers are expected to strategise the PICO-related evidence generation, in line with the value story of the product in development. It is expected that integrated quantitative evidence generation will become an even greater part of the critical path for drug development. This includes the early identification and use of external data (including real-world data (RWD)) and the use of proper modelling and simulation tools (for example, to transport evidence across countries, bridge efficacy to effectiveness or ascertain surrogacy, to name a few).

Evidence generation for ATMPs is typically more complex and parsimonious (for example, involving innovative designs, single-arm trials and registry-based trials). While there is still uncertainty raised by patients’ associations and medical societies about how JCA PICO templates and evaluation processes will adjust to ATMP development, it is expected that the role of real-world evidence (RWE) and modelling will be more important.^[13]

The impact on patients

A core aim of EU-HTAR was to address unmet medical needs and facilitate equitable access to innovative health technologies.^[3] The outlined six-month timeframe following EC approval of the marketing authorisation application (MAA) should expedite the adoption of new technologies and allow patients to benefit from innovative treatments more quickly. Greater transparency in procedures and assessments, and increased patient engagement, have also been identified as key benefits of the changes, increasing trust among all stakeholders.^[10]

EU-HTAR explicitly calls for patient and clinician involvement in both JSCs and JCAs.^[3] Patient organisations are also represented on the HTA Stakeholder Network, which supports the implementation of EU-HTAR.^[14] This involvement should mean assessments become more aligned with patient needs, as patients can provide insights into their own experiences, as well as the experiences, needs and perspectives of the patient groups they represent. Patient advocates have also expressed hope that the publication of JCAs will allow them to put more pressure on Member States that are slow to make new treatments available.^[9]

However, although patients and clinicians can be involved in the scoping process and can provide feedback on the draft report, they are not directly involved in the assessment phase of JCAs.^[15] This has led to calls from some patient organisation leaders for patient experts and organisations to be involved more consistently throughout the entire process.^[16] Concerns have also been raised that EU-HTAR could have an impact on national timelines of reimbursement, resulting in delayed access and undermining one of the primary purposes of the regulation.^[10]

What comes next?

In 2026, selected high-risk medical devices will be covered by JCAs. The rules will be extended to orphan medicinal products in January 2028. All medicinal products will be covered by 2030.^[3] There are likely to be new challenges as EU-HTAR is expanded into new areas of health technology. Analysis of the appraisal of vaccines across the EU, for example, found significant variation across Member States and highlighted the urgent need to develop standardised and vaccine-specific methodologies and processes.^[17]

To be successful, EU-HTAR must be convenient for both HTA bodies and HTDs and increase, not delay, patient access to effective technologies.^[10] This relies on clear guidance and timelines from the EC, including guidance to increase homogeneity in the writing of JCAs. Member States must also work together to avoid duplication and minimise the individual workload.

For HTDs, early preparation, clear documentation and careful time management will be needed to maintain momentum and progress clinical development pipelines under EU-HTAR. Clear communication between stakeholders and flexibility in data analysis are crucial to ensure that complex submission packages are completed within strict timeframes. There must also be strict quality controls on translated outputs, especially when timelines are tight.

It is in the interests of all stakeholders to ensure JCAs are fit for purpose and inform evidence-based decision-making. As the scope of EU-HTAR expands, offering more JSC slots will help developers prepare more effectively to deliver the evidence required by assessors. This will be particularly important for small to medium-sized companies who may have less knowledge of the European market. The industry has expressed interest in a range of options to achieve increased JSCs, including a fee-paying system to cover additional resources.^[12]

The EC should also consider opportunities to involve patient experts and patient organisations earlier and more consistently in the JCA process, to gain greater insights into disease burden and the added value of new technologies.

Further opportunities

On a global level, non-EU countries have expressed positive views on EU-HTAR and a willingness to use JCAs for their own decision-making.^[18] Perceived benefits include efficiency, timeliness and access to knowledge. However, non-EU countries are less certain about the potential impact on patient access in their own nations. Highlighted challenges to establishing a similar collaboration outside the EU include differences in healthcare systems, inadequate resources and political resistance.

New technologies could offer opportunities to reduce the burden of HTAs for all stakeholders. Generative AI (GenAI) and large language models could be used to automate aspects of evidence synthesis, facilitate the processing and analysis of RWD and streamline health economic modelling, for example.^[19] However, there are unique challenges for AI technologies in HTA, including timelines becoming obsolete before completion, a lack of transparency and explainability, and the risk of biased or flawed datasets leading to inequitable outcomes.^[20] Awareness of the current limitations of AI is crucial to overcome these challenges, as is building cross-border data infrastructure to compare evidence and taking a patient-centric approach to AI which ensures equity and inclusiveness in training and testing data.

Conclusion

EU-HTAR offers the opportunity to address many of the challenges associated with the old nation-by-nation approach to HTAs. It aims to ensure healthcare resources are used more effectively to speed up time to market for developers and to improve access to innovative treatments for patients.

While attitudes to the shift are largely positive, there remain real concerns about the practicalities of implementation. Clear communication, support from the EC and the ability of HTA bodies and HTDs to adapt to new ways of working will be crucial to overcome these challenges.

If implemented correctly, with clear benefits for all stakeholders, EU-HTAR could set a new global standard for harmonised, efficient and evidence-based assessment of new technologies, leading to more equitable access to health technologies.

References

^[1]  European Commission (EC) (2017) ‘Mapping of HTA national organisations, programmes and processes in EU and Norway’. (Accessed: February 2026).

^[2]  European Union (EU) (2021) ‘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’. (Accessed: February 2026).

^[3]  European Commission (EC) (2025) ‘Questions and Answers on the new Health Technology Assessment’. (Accessed: February 2026).

^[4]  Health Technology Coordination Group (2024) ‘Guidance on the scoping process’. (Accessed: February 2026).

^[5]  European Commission (EC) (2025) ‘Health Technology Assessment - Commission adopts rules for joint clinical assessments of medical devices and in vitro diagnostic medical devices’. (Accessed: February 2026).

^[6]  Health Technology Coordination Group (2024) ‘Annual Work Programme 2025’. (Accessed: February 2026).

^[7]  European Commission (EC) (2025) ‘Health Technology Assessment: First joint clinical assessments begin’. (Accessed: February 2026).

^[8]  European Commission (EC) (2025) ‘List of ongoing joint clinical assessments’. (Accessed: February 2026).

^[9]  EURACTIV (2025) ‘Europe’s new health assessment system could be a game-changer’. (Accessed: February 2026).

^[10]  Desmet T et al (2024) ‘Implementing the EU HTA regulation: Insights from semi-structured interviews on patient expectations, Belgian and European institutional perspectives, and industry outlooks’. Frontiers in Pharmacology. Vol 15. doi: 10.3389/fphar.2024.1369508

^[11]  European Commission (EC) (2021) ‘New HTA Regulation: key elements and next steps’. (Accessed: February 2026).

^[12]  EUCOPE (2024) ‘Pharmaceutical innovators are concerned over not enough advice meetings being offered in 2025 for discussing trial designs and evidence generation plans, with serious ramifications on the ability to undergo Joint Clinical Assessments’. (Accessed: February 2026).

^[13]  Alliance for Regenerative Medicine (2024) ‘Call to Action of Concerned Stakeholders on the Implementation of the EU HTA and Joint Clinical Assessment for ATMPs’. (Accessed: February 2026).

^[14]  European Commission (EC) (2025) ‘Health technology assessment stakeholder network’. (Accessed: February 2026).

^[15]  McCartney F et al (2024) ‘Training Patients to Be Effective Stakeholders in the EU HTA Process’. Value & Outcomes Spotlight. Vol. 10(5). (Accessed: February 2026).

^[16]  Cancerworld (2025) ‘Empowering Patients in Europe’s New Health Technology Assessment’. (Accessed: February 2026).

^[17]  Largeron N et al (2024) ‘Guiding Principles for Evaluating Vaccines in Joint Health Technology Assessment in the European Union: Preparing for the European Union’s Regulation on Health Technology Assessment for Vaccines’. Value in Health. Vol. 27(10). doi: 10.1016/j.jval.2024.06.011

^[18]  Garcia M et al (2024) ‘The Global Impact of EU HTA: Insights From 13 Non-EU Countries’. Value in Health. Vol. 27(22). doi: 10.1016/j.jval.2024.10.1559

^[19]  Fleurence R et al (2025) ‘Generative Artificial Intelligence for Health Technology Assessment: Opportunities, Challenges, and Policy Considerations: An ISPOR Working Group Report’. Value in Health. Vol 28(2). doi: 10.1016/j.jval.2024.10.3846

^[20]  Baptista Leite R (2025) ‘The Need for HTA to scale AI in Europe’. EU health technology assessment: Advent of a new era of collaboration conference (July 2025). (Accessed: February 2026).