The status of European HTA regulation

The key objectives of the European HTA Regulation on Health Technology Assessment (EU HTAR), which covers both medicinal products and medical devices, include accelerating Europe-wide access to innovative medicinal products and medical devices, consolidating European cooperation in Health Technology Assessment (HTA), and reducing the burden on pharmaceutical companies. ¹

The EU HTAR was adopted in December 2021 and entered into force in early January 2022. The implementation of the EU HTAR is divided into several phases:

In preparation, essential structural, procedural and methodological foundations were laid down in implementing acts and guidelines from 2022 to 2024.

Starting in January 2025, Joint Scientific Consultations (JSCs) and Joint Scientific Assessments (JCAs) will be introduced and conducted. JCAs will be implemented gradually, initially focusing on procedures for oncology drugs and advanced therapy medicinal products (ATMPs). Starting in 2028, the focus will expand to include all orphan drugs, and starting in 2030, all newly authorized medicinal products will be included. Applications for Joint Scientific Consultations (JSCs) can be submitted in two application periods starting in February 2025.

The European JCA procedures only include the joint clinical assessment; the assessment of the additional benefit (appraisal) continues to be carried out at the national level.

The introduction of the European HTA procedure for medical devices and in vitro diagnostics (IVDs) will be delayed from 2026.

The EU HTA governance structure is shown in Figure 1. The overall process is coordinated by the coordination group established by the Member States and its four associated subgroups: one subgroup each was established for the development of methodological and procedural guidance, for the conduct of the JCA, for the conduct of the JSC, and for the identification of emerging health technologies ('Horizon scanning').

The Federal Joint Committee (G-BA), together with the Spanish agency Agencia Española de Medicamentos y Productos Sanitarios (AEMPS), is responsible for leading the JSC subgroup. IQWiG, together with the Portuguese agency Autoridade Nacional do Medicamento e Produtos de Saúde (INFARMED), is responsible for leading the methodology subgroup. The European Commission provides administrative and technical support. An IT platform with differentiated access areas provides the basis for collaboration. The network of interest groups and experts ('Stakeholder Network') supports the implementation of the EU HTAR. Its members include patient associations, associations of health technology developers, organizations of healthcare professionals, and other non-governmental organizations from the healthcare sector.

Important sources of information on the status of implementation of the Regulation are the website of the European Commission ² , the work programme of the Coordination Group ³ and the Implementation Rolling Plan ⁴ .

Details of the JSC process are laid down in several implementing acts, as well as in guidance documents on the process, the selection of health technologies, and the selection of the responsible (co-)assessors. In addition, the Coordination Group and the responsible subgroup have completed a number of document templates, which are available as templates for the required submissions and the outcome document.

A maximum of 10 JSC procedures are planned for 2025 (5 to 7 for pharmaceuticals and 1 to 3 for medical devices). Two application periods are available (February 3 to March 3 and June 2 to 30). Incoming applications will be subject to a step-by-step selection process by the JSC subgroup:

Examination of the admissibility criteria according to the EU HTAR, such as the state of development of the health technology or the orientation towards an envisaged JCA procedure;

Review of selection criteria according to the EU HTAR, such as unmet medical need, first product in a new product category, potential impact on patients, public health or healthcare systems, etc.;

Coordination with the current work program.

A significant increase in advisory capacity is planned for the coming years. Furthermore, the introduction of a fee mechanism is planned for 2028 as part of the evaluation of the EU HTAR.

Substantive questions addressed within the JSC may include the PICO scheme (patient population, intervention, comparator, study endpoints), exchanges on Post Licensing Evidence Generation (PLEG) as part of early consultation, or questions related to health economic assessment. The latter topic area represents voluntary cooperation within the framework of the regulation, and related questions will only be answered by Member States that routinely conduct a health economic assessment at their national level. The process is shown in Figure 2.

To carry out the JCA procedures, several relevant implementing acts and guidelines have been developed, including on the process flow, the selection of (co-)assessors, evidence processing, study endpoints, statistical issues such as multiplicity, subgroup formation, post hoc analyses, the validity of clinical study designs, and the handling of templates, etc. Of particular importance here is the so-called 'scoping', the determination of the scope of the assessment. According to Article 8, paragraph 6 of the EU HTAR ^1, the scope of the assessment must be inclusive and meet the needs of the Member States with regard to the parameters as well as the information, data, analyses, and other evidence to be submitted. These requirements are operationalized using the PICO scheme. This is shown schematically in Figure 3.

The initial process involves a survey of the Member States' requirements regarding the scope of the assessment, based on the documents submitted by the pharmaceutical company (PMU) and supported by a PICO proposal from the assessors. The PICOs submitted by the Member States are then consolidated in a complex process and communicated to the PMU. The data in the PMU's dossier must be prepared according to the consolidated PICO schemes. At EU level, the results for all required PICO schemes are subjected to scientific evaluation. The subsequent national processes are based on the data and analyses presented in the European dossier that are relevant to their needs and can be supplemented by specific requirements at the national level.

The determination of the assessment scope was simulated in three published practice procedures of the JCA subgroup. This resulted in 7 to 13 required PICO schemes for the selected indication areas. ⁵

The first regulation amending the Medicinal Products Benefit Assessment Regulation entered into force on 8 March 2025. ⁶ Reference is made to the EU HTAR in several places:

Paragraph 7, paragraph 1 specifies the EU dossier, the JCA report (if available), and other information on the IT platform as important bases for national benefit assessment.

Section 7, Paragraph 4 specifies the possibility that the JCA report may not yet be available at the start of the AMNOG procedure. A JCA report available after the relevant date for dossier submission at the national level shall be submitted for hearing by the Federal Joint Committee (G-BA).

Paragraph 9, paragraph 3 contains requirements for the G-BA regarding the publication of the EU dossier and for the pU regarding the transmission of the EU dossier if it is not yet publicly available at European level at the time of publication of the benefit assessment.

In summary, it should be emphasized that the adoption of all guidelines and document templates has been successful, in accordance with the work program. The first JCA and JSC procedures have already begun. The capacities for the procedures are being provided by the Member States, and the current procedures have already been adopted by various European agencies in (co-)assessor roles. The basic functionality of the IT platform is in place, and funding is being provided on the basis of the recently signed framework contract (FWC). ⁷

Dr. Stephanie Said studied pharmacy at the Friedrich Schiller University Jena and received her doctorate from the Institute of Pharmacology at the Free University of Berlin. She has been working for the Federal Joint Committee (G-BA) in Berlin since 2016. From the beginning, she has been involved in European projects, initially as project manager and project leader for EUnetHTA JA3 for early consultations, later as Head of EUnetHTA 21 Joint Scientific Consultations Secretariat, and currently as Chair of the HTA Subgroup on Joint Scientific Consultations.

¹ 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. https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32021R2282 [Accessed: May 28, 2025]

² https://health.ec.europa.eu/health-technology-assessment_en [Accessed: 28 May 2025]

³ https://health.ec.europa.eu/document/download/9f1d2ac8-3515-4be1-9a29-db3d9ffb5201_en?filename=hta_hatcg_awp_en.pdf [Accessed: 28 May 2025]

⁴ https://health.ec.europa.eu/document/download/397b2a2e-1793-48fd-b9f5-7b8f0b05c7dd_en [Accessed: 28 May 2025]

⁵ https://health.ec.europa.eu/publications/pico-exercises_en [Accessed: 28 May 2025]

⁶ https://www.recht.bund.de/bgbl/1/2025/75/VO.html [Accessed: 28 May 2025]

⁷ https://hadea.ec.europa.eu/news/theory-practice-implementing-eu-health-technology-assessment-regulation-2025-01-22_en#:~:text=On%2021%20January%202025%2C%20HaDEA%20signed%20a%20new,consortium%20of%20HTA%20bodies%20representing%2021%20EU%20countries [Accessed: 28 May 2025]