European Union ushers in new era in health technology assessment as new regulation comes into force

As of January 12, 2025, the European Union's Health Technology Assessment Regulation (EU HTAR) became applicable, heralding a significant advancement in the collaboration and coordination of HTA processes across EU Member States.

After months of preparation and speculation, the European Medicines Agency (EMA) and other stakeholders have marked the implementation of the EU Health Technology Assessment Regulation (EU HTAR) 2021/2282, effective January 12, 2025. This pivotal regulation establishes a harmonized framework for joint clinical assessments (JCAs) of new medicines and selected high-risk medical devices, aiming to standardize evaluations across Member States and accelerate patient access to innovative treatments.

The EU HTAR aligns EU-level clinical assessments with national systems for value assessments, pricing, and reimbursement decisions. It underscores the importance of collaboration between regulators and HTA bodies, building on the cooperation developed under the European Network for Health Technology Assessment (EUnetHTA). The EMA is integral to implementation of the legislation, focusing on three key roles:

• Facilitating JCAs: EMA will collaborate with the Health Technology Assessment Coordination Group (HTACG) to evaluate the clinical effectiveness and safety of new health technologies, utilizing data from EMA’s regulatory reviews.
• Engaging in joint scientific consultations (JSCs): EMA and HTACG will provide developers with scientific advice, ensuring evidence generation aligns with regulatory and HTA needs.
• Exchanging information: EMA will share insights on upcoming applications and emerging technologies to support strategic horizon scanning.

As HTA bodies within Member States grapple with the new rules, a cautious approach is being taken with staggered timings for JCAs. The new rules initially apply to medicines for cancer and all advanced therapy medicinal products (ATMPs), expanding to orphan medicines in 2028 and all centrally authorized medicines by 2030. High-risk medical devices will be assessed under HTAR starting in 2026.

In 2025, the HTACG, comprising representatives from Member States' HTA authorities and bodies, plans to conduct 17 JCAs for cancer medicines and 8 for ATMPs. However, the actual number of JCAs will depend on the formal submissions received by EMA. Additionally, 5–7 JSCs for medicinal products and 1–3 for medical devices are anticipated this year.

The first JSC request period is set to open in February 2025, providing developers with the opportunity to engage in parallel consultations with EMA. Despite this progress, pharmaceutical industry groups have expressed significant concerns over the limited availability of JSCs in 2025, warning that “future JCAs will either be delayed due to perceived evidence gaps or that national HTA procedures will be protracted.”

While JCA do not replace the current reimbursement processes or pricing negotiations of Member States, national authorities have been required to adapt their HTA processes to align with the regulation's requirements, as highlighted in our recent webinar. These adjustments are designed to ensure that evidence generation meets both EU and national standards, preventing delays in decision-making and maintaining consistency with the harmonized EU framework. To assist stakeholders in navigating the newly established rules, procedures, and guidance, the EMA has published a raft of guidance documents to aid this transition.

Cautious optimism for the new regulation

For months, experts have been discussing the promise and associated challenges of the EU HTAR. As the regulation officially came into effect, it sparked widespread commentary, with stakeholders reflecting on both its potential to harmonize HTA processes across Europe and the practical hurdles that lie ahead.

In a press release, EMA’s Executive Director, Emer Cooke, welcomed the legislation, stating: “Facilitating the path to accessibility of new medicines for patients is a priority, and the framework for cooperation and the exchange of information between regulators and HTA bodies provided by the new rules will help galvanize our efforts.”

Writing in the Journal of Comparative Effectiveness Research, Sreeram Ramagopalan emphasized the complexity of implementation, noting:

“The success of the EU HTA regulation hinges on balancing the rigorous scientific standards of JCA with practical implementation. Manufacturers must prepare to navigate these demands by enhancing organizational analytical capabilities while proactively participating in discussions that shape future processes. Achieving this balance is vital to fulfilling the regulation's goal of harmonizing HTA across Europe without introducing unsustainable burden.”

Across LinkedIn, experts have marked this milestone. Independent market access expert Neil Grubert described it as “the biggest change ever in international HTA,” reflecting on the arduous journey from the European Commission’s original proposals to its realization today. He added, “We are now embarking on a voyage of discovery of how joint HTA will affect agencies and health technology developers in practice.” Grubert also posed a critical question: “Will the upheaval prove worthwhile?” – underscoring both the potential and uncertainties surrounding the regulation's implementation.

Iga Lipska (Health Policy Institute & Pomeranian Hospitals, Poland) celebrated the occasion as a “BIG DAY for #HTA at European level”. She hoped that JCAs will bring a “new (and hopefully better) reality.”

For Finn Boerlum Kristensen, renowned as a pioneer for bringing the new regulation to fruition, EU HTAR fulfils a vision first proposed 20 years ago:

“The EU Regulation on health technology assessment (HTAR) applies in 27+ countries from today. It is the realisation of an explicit vision for collaboration proposed in 2005 by European HTA bodies led by the Danish Health Authority to the EU public health programme. EUnetHTA was established to create an effective and sustainable network for HTA across Europe in 2006.”

The Heads of HTA Agencies Group (HAG), an independent group of 32 European healthcare agencies, added: "We are proud to support this achievement and remain deeply committed to fostering collaboration among Member States and the European Commission...This is just the beginning. We eagerly anticipate the progressive implementation of the regulation through to its full application in 2030."