Following the trialogue negotiations, on June 22, 2021, the Council of the European Union and the European Parliament reached a provisional agreement on the European Commission’s proposal for a European health technology assessment regulation (HTA Regulation), which aims to harmonize the clinical benefit assessment of health technologies across the EU. The scope of the proposal encompasses both medicinal products and medical devices, including in vitro diagnostics. The final agreed text is not available at the time of writing.

What is the HTA Regulation about?

The proposed Regulation aims at assisting cross-border healthcare delivery and will amend Directive 2011/24 (the Cross-Border Healthcare Directive), which provides for establishment of a voluntary network of Member States’ HTA bodies to support cooperation and exchange of scientific information among Member States. At its core is the principle of a united framework for increased cooperation of EU Member states, industry, and patients on HTA.

The four pillars/main elements of the HTA Regulation, coordinated through the Member State Coordination Group, are (a) joint clinical assessments for innovative medicines with the greatest patient relevance across the EU; (b) joint scientific advisory consultations for early benefit assessments; (c) a joint horizon scanning of innovative technologies to identify the emerging health technologies with the greatest potential; and (d) voluntary collaborations in other areas.

The Regulation, first proposed in 2018, has been through protracted discussions and changes due to Member State concerns. HTA has been and is a national prerogative of each Member State, as it relates to how best to allocate national budgets. Accordingly, it was feared that a Regulation trying to align elements of the HTA procedure, such as the clinical assessment, would interfere with Member States’ independence to determine how to spend their national “health budgets.” Some Member States, such as France and Germany, had initially sought to block the Regulation under the principle of subsidiarity.

The main stumbling block in the Regulation’s adoption was the nature of the joint clinical assessment (JCA), namely whether it should be voluntary or mandatory. A mandatory JCA would help align the evidentiary evaluation of a medicine but on the other hand would be seen as limiting/infringing on the prerogative of each national authority to conduct its own methodologies. Conversely, a voluntary JCA could be meaningless, with countries able to choose to depart from its conclusions, which may result in duplicative clinical assessments. The compromise reached was that Member States must take JCAs into “due consideration” in their national assessments and may determine whether they consider relevant any part of the JCAs. Member States remain fully competent to reach the final decision on pricing and reimbursement of the products.

How has the agreement on the HTA Regulation been received?

The HTA Regulation is seen overall as a unique opportunity for greater alignment on clinical evidence requirements and has been broadly welcomed by the pharmaceutical industry and bodies such as European Federation of Pharmaceutical Industries and Associations and the European Confederation of Pharmaceutical Entrepreneurs. It is seen as providing consistency and transparency and being a positive development for European patients, who may have more equitable and prompt access to innovative treatments.

However, the medtech industry has had divergent views on the HTA Regulation and questions the value and relevance of this legislation to the medical technology sector, concerned that it may create additional bureaucracy. The divergence in the market access processes for medicines and medical devices is such that the specificities of medical technologies are not adequately addressed in the HTA Regulation but could be covered at the implementation stage to ensure better patient access and funding decisions. This can be aided by having medical technology-specific membership in the Coordination Group and by developing appropriate methodologies. However the obstacle remains that there is currently no common goal of the use of HTA in medical technologies, and this should be clarified as a priority.

Implementation and next steps

The Regulation is aimed to enter into force gradually, initially limited to oncology products for the first three years, then extended to orphan and advanced therapy medicinal products (ATMPs) and after an additional five to eight years extended to all other therapeutic areas. Adjustments are required to ensure that orphans and ATMPs are dealt with in a flexible way to address evidentiary uncertainty and data gaps, which are not adequately met by existing more traditional approaches to clinical assessment.

The subsequent implementation phase is expected to produce Implementing and Delegated Acts to fine tune the Regulation’s practicalities, such as the assessment methodology.

Although the JCAs is far from being perfect, it provides a basis to engage at European level in a more harmonized way. It is possible that the industry and other stakeholders will be consulted and are encouraged to engage with the trade association and other bodies to ensure they can provide constructive input.