Introduction
Background
Objectives of new HTA Regulation
Beneficiaries
Involved parties
Expectations from EU member states
Next steps


Introduction

After many years of negotiations, the European Parliament adopted the Health Technology Assessment Regulation (the HTA Regulation)(1) on 15 December 2021.

This article outlines the background of the HTA Regulation, the objectives, the beneficiaries and the consequences for stakeholders. It also describes what is expected from EU member states and the next steps following the regulation coming into force.

Background

HTA
A "health technology assessment" (HTA) is a multidisciplinary process that summarises information about the medical, social, economic and ethical issues relating to the use of a health technology in a systematic, transparent, unbiased and robust manner. It includes clinical questions (for example, about comparing a new health technology to existing alternatives or assessing which patients might benefit) as well as economic questions, such as the costs entailed for the health system.

Thus, an HTA is a key tool for EU member states to use to ensure accessibility, quality and sustainability of healthcare. However, the HTA Regulation focuses on clinical aspects of HTA, such as a definition of the problem and the relative safety and clinical effectiveness of a health technology as compared with existing technologies.

EU HTA history
The HTA Regulation comes into force after more than 20 years of voluntary cooperation in this subject area. Following the adoption of the EU Cross-border Healthcare Directive,(2) a voluntary EU-wide network on HTA composed of national HTA bodies and agencies was established. This should provide strategic and political guidance to the scientific and technical HTA cooperation on an EU level.(3) This work, complemented by three consecutive joint actions on HTA, should enable the EU Commission and EU member states to build up knowledge on methodologies and information exchange regarding HTA. However, as several decades of project-based EU cooperation on HTA have shown, these issues cannot be sufficiently addressed by a voluntary approach.

Current status
Currently, and until the applicability of the HTA Regulation in 2025, a manufacturer, after obtaining the respective authorisation(4) for health technologies (medicinal products, medical devices, in vitro diagnostic medical devices and medical procedures, as well as measures for disease prevention, diagnosis or treatment), must apply to different HTA agencies in various EU member states before new health technologies are broadly accessible. Such agencies have varying procedures and standards, which may result in different outcomes. Ultimately, this may lead to varying or even limited access to health technologies within the European Union. This should now be addressed by the HTA Regulation.

Thus, market access for innovative technologies is impeded and distorted, which leads to a lack of business predictability, higher costs for the industry, delays in accessibility and negative effects on innovation. Moreover, the result is a duplication of work for national HTA bodies and the industry, inefficient use of resources and limited transparency for patients.

Objectives of new HTA Regulation

As a consequence of the problems arising due to the current status quo, the HTA Regulation aims to harmonise the various processes and intends to ensure the quality, safety and accessibility of health technologies and consequently healthcare within the European Union. Further, the objective is to strengthen EU-wide collaboration in HTA by pooling resources and high-level expertise on issues concerning health technologies. Of particular importance in this regard is the establishment of a coordination group pursuant to article 3 of the HTA Regulation.(5) Each EU member state appoints a member of the coordination group. Pursuant to article 3(4), this group will generally act by consensus. The coordination group will be responsible for overseeing the joint clinical assessments (JCAs) and other joint work carried out by designated national experts organised in subgroups dedicated to specific types of joint work (eg, a subgroup on JCAs and a subgroup on joint scientific consultations (JSCs)).

Ultimately, this should result in an improvement in the quality of health technology. The involvement of all EU member states shall ensure intensive discussion and high-quality outcomes. Further, health technology safety shall be ensured. Parts of the clinical evaluations will be made available to the public via an IT platform set up in accordance with article 30. This shall create transparency and the opportunity to check the assessment work done. Finally, accessibility shall be improved. By setting up joint valuations, the HTA Regulation is expected to provide faster market access and thus accessibility for patients.

Beneficiaries

Patients and clinicians will benefit from JCA reports of higher scientific quality that are more transparent and accessible to the public. Health experts, the industry and patients will have a new framework that helps to address unmet medical needs and facilitate access to innovative medicines and some high-risk medical devices. Thus, patients who suffer from serious diseases will benefit from accessibility through the HTA Regulation. The HTA Regulation could potentially help to overcome critical situations, especially when timing is important and patients potentially have to wait months to access new treatments or medicines.

EU member states will be able to pool their HTA resources and expertise. JCAs are, generally, expected to be timely scientific reports that EU member states will give due consideration to in their national HTA processes. It is also thought that they will be of increased quality. The HTA Regulation will therefore support EU member states in taking more evidence-based and timely decisions on patient access to new medicines and medical devices. Additionally, the HTA Regulation intends to reduce inequality within the European Union. EU member states that are not yet up to date with the latest medical standards will receive access to them more quickly.

The developers gain more clarity and predictability concerning the clinical evidence requirements for HTA. Additionally, they benefit from efficiency gains when submitting clinical evidence, as there will be only a single EU-level submission file for JCAs. The challenge in the past was to set up clinical trials that were accepted globally (or at least on a European level) by different regulatory authorities and also meet national specifics. Developers hope for and expect relief and the ability to save resources by eliminating the need for multiple assessments.

Involved parties

The core element of the HTA process is the JCA. Therefore, the coordination group and its subgroups are considered the decisive factor in the HTA Regulation being successful. Further, a stakeholder network and, of course, the developers are involved in the assessment process.

JCA
The JCA is based on dossiers requested from the respective developer. Prerequisites of the dossiers are set out in Annex I and Annex II. JCAs are performed initially by an assessor with the assistance of a co-assessor pursuant to article 11(1). Members of the designated subgroup of the coordination group provide their comments on the assessor's draft reports pursuant to article 11(3). Pursuant to article 11(4), the subgroup ensures that patients, clinical experts and other relevant experts are involved in the assessment process by being given the opportunity to provide input on the assessor's draft reports. The developer may subsequently have the opportunity to address technical or factual inaccuracies according to article 11(5) before the report is forwarded to the coordination group.

If endorsed by the coordination group, the endorsed reports are forwarded to the EU Commission which, pursuant to article 12(4), publishes the reports via the IT platform (article 30). The result of this process is a so-called "JCA report" pursuant to article 9(1). JCA reports will be accompanied by summary reports. Those JCA reports will not contain any value judgement or conclusions on the overall clinical added value of the assessed health technology and will be limited to a description of the scientific analysis of the relative effects of the health technology as assessed on the health outcomes against the chosen parameters as well as of the degree of certainty of the relative effects, taking into account the strengths and limitations of the available evidence.

Stakeholder network
The EU Commission establishes a stakeholder network pursuant to article 29(1), which will support the work of the coordination group and its subgroups upon request. Pursuant to article 29(5), the coordination group will meet with the stakeholder network at least once each year in order to update stakeholders on the joint work of the coordination group, including its main output, and to provide for an exchange of information.

All eligible stakeholder organisations, in particular patient associations, consumer organisations, non-governmental organisations in the field of health, health technology developers and health professionals may become members under the prerequisites as set out in article 29(2) and (3). The list of stakeholders included in the stakeholder network as well as other information will be made publicly available on the IT platform referred to in article 30.

Developers
The developers are initially responsible for submitting a dossier as requested as well as to provide any further information requested regarding the JCA.

However, with article 16, developers are given the opportunity to use JSCs. Such JSCs are the means by which the coordination group can carry out an information exchange with health technology developers on their development plans for a given health technology. Those consultations will facilitate evidence generation to meet the likely evidence requirements of a subsequent JCA on that health technology. It will include meetings with the health technology developer as well as result in an outcome document that outlines the scientific recommendation made. JSCs will, in particular, concern all relevant clinical study design aspects, or clinical investigation design aspects, including comparators, interventions, health outcomes and patient populations. Health technologies that are likely to be the subject of a JCA and where the clinical studies and clinical investigations are still in the planning stage will be eligible for JSCs. However, the respective outcome document will not give rise to any legal effects on EU member states, the coordination group or the health technology developer. JSCs will not prejudice the JCA, which may be carried out on the same health technology.

Expectations from EU member states

Article 13 stipulates that EU member states will give due consideration to the JCA report when carrying out a national HTA on the health technology concerned. They will also have to annex the published JCA report to the national HTA report. In addition, for each national assessment on a technology which has been subject to a JCA, information on how the JCA has been considered in the national process will have to be provided.

EU member states may complement the JCA with additional clinical analyses that may be needed in their national process (eg, analyses related to national disease epidemiology or the specific national healthcare context). They may also complement the JCA with non-clinical analyses (eg, on budget impact or cost-effectiveness). However, each EU member state remains responsible for drawing conclusions on the overall value of a new health technology for their healthcare system, pricing and reimbursement decisions.

Next steps

The HTA Regulation entered into force on 11 January 2022. However, the Regulation will not apply until 12 January 2025. This means that joint work on JCAs and JSCs will not be carried out before January 2025. The delayed application period aims to provide sufficient time to set up the organisational framework, and to establish the coordination group, its subgroups and the stakeholder network as well as time to adopt the implementing and delegated acts and methodological guidance documents. Further, EU member states have time to adjust their national HTA legislation and processes, if necessary, and stakeholders, in particular health technology developers, have time to familiarise and comply with the new set of requirements.

As a first implementation step, the EU Commission will set up the coordination group in Summer 2022. Around the same time, a conference is envisaged to inform a wide range of stakeholder organisations about the new HTA Regulation and its implementation.

For further information on this topic please contact Malte Scheel or Tobias Maier at Eversheds Sutherland LLP by telephone (+49 89 54565 0) or email ([email protected] or [email protected]). The Eversheds Sutherland LLP website can be accessed at www.eversheds-sutherland.com.

Endnotes

(1) Regulation on Health Technology Assessment (EU) 2021/2282.

(2) Directive 2011/24/EU on the application of patients' rights in cross-border healthcare.

(3) EUnetHTA.

(4) For example, a marketing authorisation in case of medicinal products.

(5) Articles without an indicated reference refer to the HTA Regulation.