Happy New Regulation - The beginning of 2025 fires the starting gun for the new Health Technology Assessment Regulation

The economic aspect should not be underestimated either, as healthcare expenditure amounted to approx. 11 % of gross domestic product in Austria and approx. 10.4 % of gross domestic product in the EU in 2023.

However, developers of healthcare technologies often face the challenge of submitting identical information, data, analyses and other evidence to multiple Member States at different times for the clinical evaluation of their healthcare technology. The administrative, time and cost burden is enormous, sometimes resulting in barriers to market access. Moreover, the varying outcomes of these assessments can create uncertainties in business planning.

Against this backdrop, the EU Commission has created an instrument with Regulation 2021/2282 (HTAR) to standardise the assessment of health technologies and improve the availability of innovative health technologies across the EU. The HTAR has been in force since 11 February 2022 and – together with four associated implementing regulations – is now applicable since 12 January 2025.

The assessment of health technologies

The HTAR defines a health technology assessment (HTA) as a scientific evidence-based process that allows competent authorities to determine the relative effectiveness of new or existing health technologies. The HTA focuses specifically on the added value of a health technology compared to other new or existing health technologies. Information on a specific health technology, its functioning and its impact on a health system is collected, organised, analysed and evaluated in a systematic and transparent manner. This includes clinical and non-clinical aspects in equal measure so that, for example, the effectiveness and safety, but also economic, legal, social, ethical and organisational aspects of health technologies can be properly assessed. In the HTAR, the term healthcare technology refers to medicinal products, medical devices, in-vitro diagnostic medical devices and medical procedures, as well as measures for disease prevention, diagnosis or treatment.

HTAs therefore serve as a basis for health policy decisions in the Member States when allocating funds and may inform decisions on reimbursement eligibility. As reimbursement eligibility is often directly linked to demand from healthcare facilities and, therefore, to product sales, this is highly relevant for developers and manufacturers. In turn, healthcare professionals may use HTAs to inform their therapy decisions.

In summary, HTAs aim to provide a comprehensive basis on which informed decisions can be made for a fair, efficient and high-quality healthcare system.

The HTAR as a framework for HTA procedures in the EU

• Before the HTAR
  

  HTA procedures have so far primarily taken place primarily at the national level. The methods and assessment criteria of HTA procedures and the requirements for the scientific evidence to be provided varied among Member States. Although only a few Member States had a legal obligation to use HTAs in limited cases, the practical significance of HTAs has been considerable and increasing.
  
  At the European level, on the other hand, HTAs have so far only been used selectively in the EUnetHTA network, based on voluntary project-based cooperation of Member States. In recent years, calls to strengthen EU cooperation and pooling of expertise in the field of HTA have become louder.
  
  By creating a common legal framework for HTA procedures at the EU level, the HTRA is now responding to these calls.
  
  

• What's new at the HTAR and what are the benefits?
  

  The HTAR's aim is to assess health technologies quickly and to a high standard, and to avoid duplication of work and effort. To this effect, the procedure for HTAs will be standardised. A mandatory joint clinical assessment) of healthcare technologies such as medicinal products, medical devices and in-vitro diagnostics is intended to create a comparable basis for decision-making across the EU, e.g. for reimbursement eligibility.
  
  The HTAR also promises advantages for developers. This is because under it, most documents for a clinical assessment will only have to be submitted once at the EU level instead of several times in various Member States. This should significantly reduce the workload and create planning security. Smaller companies, in particular, should be able to focus on innovation activities, which in turn should benefit patients in the longer term.
  
  The sustainable enhancement of EU-wide cooperation in the field of HTA should also ensure the efficient functioning of the internal market for these health technologies and guarantee a high level of health protection for patients and users.
  
  These objectives are essentially to be achieved through the following regulatory content:
  
  
  • establishment of a Coordination Group of national HTA authorities and a network of stakeholders;
    
    
  •  implementation of JCA and joint scientific consultations (JSC); and
    
    
  • offering scope for voluntary cooperation in adjacent areas.
    
    

• The Coordination Group
  

  The central actor is the Member State Coordination Group on HTA, established in accordance with Art 3 HTAR. As the name suggests, the group comprises members delegated by the Member States and is responsible for various tasks, including establishing and coordinating national subgroups, identifying emerging health technologies (horizon scanning), drawing up strategic and methodological guidelines for national subgroups, specifying procedural steps and deadlines for JCAs and JSCs, involving interest groups, and promoting cooperation between the Member States and the exchange of scientific information.
  
  

• Joint clinical assessments
  

  The key element of the HTAR is the establishment of JCAs (Art 7 to 15 HTAR). However, the assessment at the EU level is limited to the description of the health problem and the technology, as well as the purely factual examination of the technical and clinical properties of the technology (particularly efficacy and safety). A value judgment on the overall clinical added benefit is not permitted. The assessment of all non-clinical aspects and the conclusions drawn from this, such as pricing and eligibility for reimbursement, is still left to the Member States.
  
  The scope of application of the JCA is also limited. On the one hand, only medicinal products authorised in the central authorisation procedure are covered according to a staggered deadline model (Art 7 para 1 lit a, b and para 2 HTAR). New oncological medicinal products and advanced therapy medicinal products (ATMPs) made the start on 12 January 2025, followed by orphan drugs from 13 January 2028 and all other medicinal products from 13 January 2030.
  
  A selection of medical devices and in-vitro diagnostics are also subject to JCA since 12 January 2025 (Art 7 para 1 lit c and d HTAR). In short, only particularly high-risk, novel and/or promising Class III implantable medical devices and Class IIb active devices within the meaning of Regulation 2017/745 on medical devices (MDR) and certain Class D in-vitro diagnostic medical devices within the meaning of Regulation 2017/746 on in-vitro diagnostic medical devices (IVDR) are covered.
  
  The JCA report produced at the end of the EU-HTA procedure is not binding. National authorities are only required to consider the report appropriately as part of the national HTA (Art 13 para 1 lit a HTAR). The HTAR does not explicitly interfere with the core competencies of Member States, such as pricing and reimbursement. Instead, it generally respects the autonomy of Member States in decision-making regarding the use of health technologies, as well as the management and delivery of health services and medical care. Accordingly, Member States may diverge from the EU-HTA recommendations if necessary.
  
  In addition to the non-clinical assessment, which in any case is not covered by the HTAR, Member States are also free to carry out additional clinical analyses required for the general national HTA process. In this context, Member States may also request further evidence from the developer or manufacturer.
  
  

• Joint scientific consultations
  

  For health technologies that are likely to be the subject of a JCA, and where the clinical studies and clinical trials are still in the planning phase, developers or manufacturers can apply for a JSC with the Coordination Group (Art 16 HTAR). The discussions focus on study designs with regard to comparators, interventions, health-related endpoints and patient populations (Art 16 para 1 HTAR).
  
  JSCs can take place in parallel with scientific consultations with the European Medicines Agency within the meaning of Regulation (EC) No 726/2004. The same applies to scientific consultations with the expert committees under the MDR.
  
  

• Voluntary cooperation
  

  The HTAR also establishes a legal basis for the organisational and financial promotion of voluntary cooperation between the Member States and the exchange of scientific information between them in the HTA area. This primarily concerns (i) non-clinical assessments of health technologies, (ii) joint assessments of medical devices and in-vitro diagnostic medical devices that are not already subject to EU-HTA under Art 7 HTAR, and (iii) HTAs that are neither medicinal products nor medical devices or in-vitro diagnostic medical devices (Art 23 HTAR).
  
  

Realisation and implementation

• Implementing regulations for the HTAR
  

  To date, the EU has issued four implementing regulations for the HTAR (Implementation Regulations) setting out the rules for JCA, JSC, handling conflicts of interest and exchange of information with the European Medicines Agency:
  
  
  • Implementing Regulation (EU) 2024/1381 of 24 May 2024 sets out procedural rules for interaction, information exchange and participation in the preparation and update of JCA of medicinal products for human use at the Union level and provides templates for those JCA.
    
    
  • Implementing Regulation (EU) 2024/2699 of 18 October 2024 sets out procedural rules for the cooperation between (i) the Coordination Group and the European Commission, which acts as the Coordination Group's secretariat, and (ii) the European Medicines Agency for the exchange of information as regards the JCA of medicinal products and medical devices and in-vitro diagnostic medical devices, and as regards the JSC on medicinal products and medical devices.
    
    
  • Implementing Regulation (EU) 2024/2745 of 28 October 2024 sets out rules for the management of conflicts of interest of those involved in JCA and JSC with the aim of ensuring that HTA assessments are conducted independently, impartially and transparently, free from conflicts of interest.
    
    
  • Implementing Regulation (EU) 2024/3169 of 18 December 2024 sets out procedural rules for JSC on medicinal products for human use at the Union level as regards (i) the submission of requests from health technology developers, (ii) the selection and consultation of the parties involved in the JSC, and (iii) cooperation with the European Medicines Agency.

The EU Commission has yet to accept the fifth Implementing Regulation setting out procedural rules for JSC on medical devices and in-vitro diagnostic medical devices.

• Applicability and national implementation
  

  The HTAR and the related Implementation Regulations have been directly applicable since 12 January 2025. From this day forth, JCAs are mandatory under European law. As noted, new oncological medicinal products and ATMPs are the first to undergo assessment, followed by orphan drugs in 2028 and all other medicinal products in 2030. While the HTAR initially applies in 2025 to 2027 only to therapies mainly used in the inpatient sector, its obligations are also relevant for the outpatient sector.
  
  However, the HTAR leaves open which national institutions are responsible for the application of HTA assessments and how they embed the European clinical assessments of health technologies in the national HTA processes. This is a matter for the respective national legislator.
  
  In Austria, there is currently no legal regulation on how a national HTA authority is defined and who the contact persons for EU-HTAs are. It is unclear which processes should take place and how. It remains to be seen, how the Austrian legislator will address these issues.
  
  

Conclusion

In summary, the HTAR aims to improve access to innovative health technologies, make better use of healthcare resources and strengthen cooperation between Member States in the field of HTA. All of this contributes to a high-quality healthcare system with a high level of patient and user confidence.

However, despite its many advantages and good intentions, the HTAR also has certain weaknesses. In many cases, it still leaves the final decision on health technologies to individual Member States. A more resource-efficient approach for developers and manufacturers would be to mandate the clinical assessment of selected health technologies at the EU level. Nevertheless, this assessment is limited to the clinical dimension and purely factual analyses. The evaluation of additional benefits remains at the national level, allowing national authorities to determine the outcomes of the assessment, such as application, pricing and eligibility for reimbursement. While the Coordination Group reports are not binding but recommendations only, this does not mean they are irrelevant. They will likely serve as a basis for decisions by the national authorities. On the one hand, they must be considered appropriately within the framework of the national HTA (Art 13 HTAR). On the other hand, some argumentation will be required to override or fundamentally question an assessment of the Coordination Group at the national level, as the national decision-makers are likely to be the same as those involved in the Coordination Group.

Whether the HTAR will indeed provide bureaucratic relief for developers or will instead become another hurdle for manufacturers of medical devices and in-vitro diagnostics after the MDR and IVDR remains to be seen. The national legislators will also play a decisive role when implementing the HTAR into their respective national assessment systems.