Preparing For The Implementation Of The EU Health Technology Assessment Regulation

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The EU Health Technology Assessment (HTA) Regulation (Regulation (EU) 2021/2282), which entered into force in January 2022, will start to apply from January 2025 for oncology medicines...
European Union Food, Drugs, Healthcare, Life Sciences
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The EU Health Technology Assessment (HTA) Regulation (Regulation (EU) 2021/2282), which entered into force in January 2022, will start to apply from January 2025 for oncology medicines, advanced therapy medicinal products and medical devices (the HTA Regulation will apply to orphan medicines from 13 January 2028, and all centrally authorised medicines from 13 January 2030).

The HTA Regulation aims to accelerate access to innovative health technologies. Currently, new technologies undergo national-level assessments to evaluate their costs effectiveness, and have to meet national pricing and reimbursement (P&R) requirements to determine if they will be made available on the national healthcare system. This is a costly process, both in economic, and time and resource terms.

The HTA Regulation seeks to increase cooperation across Europe, to ensure the efficient use of resources and strengthen the quality of HTAs. At the heart of the HTA Regulation lies the introduction of EU-level joint clinical assessments (JCAs), which are collaborative assessments, including resources and expertise from different EU Member States. With relevant data only needing to be submitted once within the EU, JCAs will minimize duplication and alleviate the workload for companies and authorities. Under the HTA Regulation, HTA bodies in the Member States are expected to consider the JCA reports in their decision-making processes, but the ultimate decision on whether a particular product will be available and reimbursed on the national healthcare system will remain the competence of the national authority.

Ahead of its introduction, several measures are being introduced, as set out in the Implementation Rolling Plan published by the Commission. These include a number of implementing acts, IT projects and EU support such as capacity building, training and awareness raising. As part of this, in the past few weeks, the Commission has published information on the evaluation of parameters used in JCA dossiers and a draft Implementing Regulation on JCAs.

Implementing Regulation on JCAs

On 5 March, the Commission published a draft Implementing Regulation that sets out procedural rules for the interaction during, exchange of information on, and participation in, the preparation and update of JCAs of medicinal products, as well as key template documents. This sets out the procedural steps and timelines of the new EU HTA procedure.

The Implementing Regulation addresses several requirements set out in the HTA Regulation. Specifically, it provides detailed procedural rules relating to:

  • Cooperation with the European Medicines Agency during the preparation and update of JCAs of medicinal products
  • The interaction between stakeholders during JCA and updates
  • The selection and consultation of stakeholders during JCAs
  • The format and templates for health technology developers (HTDs)

On the final point, the draft Implementing Regulation provides rules on the dossier and further data to be provided by HTDs. It includes annexes including a template for the dossier of the JCA of a medicinal product; a template for the JCA report; and a template for the JCA summary report.

The draft Implementing Regulation was open for public consultation until 2 April. On 26 April, the draft received the positive opinion of the Member States during the meeting of the HTA "Comitology" Committee. It is expected to be adopted shortly.

However, a number of industry bodies have expressed concern over the workability of the draft Implementing Regulation and believe that the draft rules "will create an unworkable framework for JCAs and consequently lead to duplication of work". For example, the draft procedures do not involve the companies whose products are subject to JCA, except in exceptional cases. Further, the industry bodies have noted that "the timelines are too short to allow companies to provide quality input regarding evidence, analysis and comments that is required of them throughout the JCA". The industry bodies set out some suggested solutions to these concerns, and request these are taken into account to ensure the procedure meets its intended aims.

Evaluation of parameters used in JCA dossiers

Meanwhile, the Practical Guideline for Quantitative Evidence Synthesis: Direct and Indirect Comparisons and the Methodological Guideline for Quantitative Evidence Synthesis: Direct and Indirect Comparisons were published by the Commission on 25 March.

The Guidelines are addressed to HTA assessors, who are appointed by the Member State Coordination Group on Health Technology Assessment (HTA CG) to conduct the JCA, consult stakeholders, and prepare a JCA report.

JCAs will allow the use of results from multiple studies, which will be combined through evidence synthesis. This should enable HTDs to obtain more robust estimates of the treatment effect of the health technology under development (with direct comparators), as well as relative treatment effects for interventions not studied in the same trial (with indirect comparators). However, to assess the relative effectiveness of a new intervention compared to existing ones in the context of multiple sources of evidence, appropriate methods for evidence synthesis should be used.

The Methodological Guideline seeks to describe the methods currently available for direct and indirect treatment comparisons, including underlying assumptions, strengths, and weaknesses. The Practical Guideline gives additional, more-detailed advice for use in practice. For example:

  • When assessing the HTD's dossier, HTA assessors must ensure that HTDs have used appropriate comparators, and that the estimates are obtained by pooling relative treatment effects for each treatment, rather than the absolute effect of a particular treatment in a trial.
  • They must also assess whether trials are sufficiently similar to be combined, and what interferences may be made based on the methods for direct and indirect comparisons and the data used.

As noted above, the ultimate decision on whether a particular product will be reimbursed on the national healthcare system in each Member State will remain the competence of the relevant national authority. In line with this, the Guideline notes that EU Member States may decide on the validity of direct or indirect treatment comparisons based on the JCA report.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

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