European HTA process

Before placing an innovative medicinal product on the market in Europe, drug manufacturers must prove its quality, safety and harmlessness as part of the approval process. In addition, a so-called Health Technology Assessment (HTA) must generally be carried out for national authorities or institutions. These procedures represent a systematic, evidence-based evaluation of medical procedures and technologies with regard to their effects on healthcare and can serve as a decision-making tool for the inclusion or rejection of new procedures, technologies or measures in the standard medical care of the individual member states.

The benefit assessment procedure in Germany

In Germany, for example, the Federal Joint Committee (G-BA) assesses the benefit of reimbursable drugs with new active ingredients in accordance with Section 35a SGB V. This includes, in particular, the assessment of the additional benefit compared to an appropriate comparative therapy, the determination of the extent of the additional benefit and its therapeutic significance. On the basis of this early benefit assessment by the G-BA, the National Association of Statutory Health Insurance Funds (GKV-Spitzenverband) and the pharmaceutical companies agree on a reimbursement amount for the drug in question in accordance with Section 130b SGB V.

Aims of European HTA-Process

While central authorization procedures at European level enable the applicant to obtain authorization for a medicinal product in all member states of the European Economic Area (EU member states, Iceland and Norway) with a single application, this is not yet possible for HTA assessments.

With the aim of

the harmonization of methods,
the creation of transparency,
the avoidance of redundancies and
and faster access for patients to innovative therapies for the whole of Europe

the European Commission therefore initiated legislation that will come into force on January 11, 2022 with Regulation (EU) 2021/2282 on health technology assessment (hereinafter referred to as the EU HTA Regulation). This establishes a European process for the clinical evaluation of medicinal products and certain medical devices and the associated scientific advice. The assessment of advanced therapy medicinal products (ATMPs) and oncological medicinal products will begin as early as January 2025. Orphan drugs will follow in a staged procedure from 2028 and all other innovative medicinal products from 2030. However, the individual Member States remain responsible for drawing conclusions on the clinical added value of a health technology at national level, taking "due account" of the results of the EU HTA. This clause on the binding nature of the use of the results in national procedures was the subject of a long struggle between the political camps until the EU HTA Regulation was adopted.

What happened so far?

The implementation follows a timetable set by the European Commission, which includes the most important activities for the implementation of the EU HTA Regulation. The plan is subject to regular review in order to provide national authorities and stakeholders with up-to-date information.

As part of the establishment of the EU HTA process, a governance structure has now been created that forms the legal and de facto regulatory framework. The coordination group set up in March 2022 manages and monitors the work of the four subgroups, which are divided into the areas of Joint Clinical Assessment, Joint Scientific Consultations, Identification of emerging health technologies and Methodology (see Figure 1). Each Member State sends representatives to both the Coordination Group and the subgroups established in April 2023. The representatives must be free and independent of industry interests. Even if several representatives are delegated, each Member State has one vote. Germany's interests are represented by representatives from the Federal Ministry of Health (BMG), the G-BA and the Institute for Quality and Efficiency in Health Care (IQWiG). Germany's strong commitment is reflected, among other things, in the fact that two of the four subgroups are chaired by German representatives. Dr. Beate Wieseler (IQWiG) heads the Methodology subgroup and Dr. Stephanie Said (G-BA) the Joint Scientific Consultations group.

Unterstützt werden die Arbeiten von einem bei der Europäischen Kommission angesiedelten Sekretariat sowie dem im Juni 2023 etablierten Stakeholder-Netzwerk. Diesem gehören Patientenvertreter, Nichtregierungsorganisationen im Bereich Gesundheit, Herstellerverbände sowie Klinische Experten an. Zudem wurde eine IT-Infrastruktur eingerichtet, die unter anderem dem Einreichen der Daten durch die Hersteller sowie der Veröffentlichung der HTA-Berichte dienen wird.

At the same time, the EUnetHTA 21 consortium was commissioned in September 2021 to prepare the implementation of the EU HTA Regulation and thus the future binding joint HTA work. With the participation of the G-BA and IQWiG, the consortium of 13 HTA organizations developed various guidelines to create uniform standards for joint European benefit assessments. The work was completed in September 2023 as part of the two-year service contract. The results, which were critical in many respects, now serve as the basis for defining the EU HTA standards.

How is the EU HTA procedure organized?

From January 2025, an EU HTA procedure must be carried out for all new oncological medicinal products and ATMPs for which an application for marketing authorization is submitted in the European Union. This includes joint scientific advice and joint clinical evaluation. The joint clinical assessment is preceded by a scoping process. For this purpose, the individual requirements of the member states regarding patient population, intervention, comparator and outcome (PICO) are reported to the coordination group. The scope of the assessment is then determined on the basis of this information. In accordance with Article 10 of the EU HTA Regulation, the European Commission then informs the manufacturer of the scope of the assessment and invites it to submit a dossier. For medicinal products, the submission deadline is at least 45 days before the planned deadline for the opinion of the Committee for Medicinal Products for Human Use in accordance with Article 5(2) of Regulation (EC) No 726/2004.

Based on the submitted dossier and the defined scope of the assessment, an assessor, with the help of a co-assessor, prepares the draft EU HTA report and a summary of the joint clinical assessment. The HTA report must be approved by the coordination group no later than 30 days after adoption of the Commission's decision to grant authorization. Within a further 10 days, a procedural review is carried out by the European Commission and the results are published on the IT platform.

What is coming?

By January 2025, corresponding standards must be defined in implementing acts. A total of six implementing acts are planned:

on the joint clinical evaluation of medicinal products,
on the handling of conflicts of interest,
on cooperation and information exchange with the European Medicines Agency (EMA),
on the joint scientific consultation on medicinal products,
on the joint scientific consultation on medical devices and
on the joint clinical evaluation of medical devices.

Pharma Deutschland will continue to actively support the process, present the positions of our member companies and report on further progress.

See the German page with Stellungnahmen & Positionspapiere.