Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (Text with EEA relevance)

THE EUROPEAN PARLIAMENT AND THE COUNCIL OF THE EUROPEAN UNION,

Having regard to the Treaty on the Functioning of the European Union, and in particular Articles 114 and 168 thereof,

Having regard to the proposal from the European Commission,

After transmission of the draft legislative act to the national parliaments,

Having regard to the opinions of the European Economic and Social Committee (1),

After consulting the Committee of the Regions,

Acting in accordance with the ordinary legislative procedure (2),

Whereas:

(1) The development of health technologies is a key driver of economic growth and innovation in the Union and is key to achieving the high level of health protection that health policies need to ensure for the benefit of all. Health technologies constitute an innovative sector of the economy and form part of an overall market for healthcare expenditure that accounts for 10 % of Union gross domestic product. Health technologies encompass medicinal products, medical devices, in vitro diagnostic medical devices and medical procedures, as well as measures for disease prevention, diagnosis or treatment.

(2) Health technology assessment (HTA) is a scientific evidence-based process that allows competent authorities to determine the relative effectiveness of new or existing health technologies. HTA focuses specifically on the added value of a health technology in comparison with other new or existing health technologies.

(3) HTA is able to contribute to the promotion of innovation, which offers the best outcomes for patients and society as a whole, and is an important tool for ensuring proper application and use of health technologies.

(4) HTA can cover both clinical and non-clinical aspects of a health technology, depending on the healthcare system. The Union’s co-funded joint actions on HTA (EUnetHTA Joint Actions) have identified nine domains by reference to which health technologies are assessed. Of these nine domains, four are clinical and five are non-clinical. The four clinical domains of assessment concern the identification of a health problem and current health technology, the examination of the technical characteristics of the health technology under assessment, its relative safety, and its relative clinical effectiveness. The five non-clinical assessment domains concern cost and economic evaluation of a health technology, and its ethical, organisational, social and legal aspects.

(5) HTA can improve scientific evidence used to inform clinical decision-making and patient access to health technologies, including where a health technology becomes obsolete. The outcome of HTA is used to inform decisions concerning the allocation of budgetary resources in the field of health, for example in relation to establishing the pricing or reimbursement levels of health technologies. HTA can therefore assist Member States in creating and maintaining sustainable healthcare systems, and stimulate innovation that delivers better outcomes for patients.

(6) The carrying out of parallel assessments by multiple Member States and divergences between national laws, regulations and administrative provisions on the processes and methodologies of assessment can result in health technology developers being confronted with multiple and divergent requests for data. It can also lead to both duplication and variation in outcomes, resulting from the specific national healthcare context.

(7) While Member States have carried out some joint assessments within the framework of the EUnetHTA Joint Actions, the voluntary cooperation and production of output has been inefficient, relying on project-based cooperation in the absence of a sustainable model of cooperation. The use of the results of the EUnetHTA Joint Actions, including their joint clinical assessments, at Member State level has remained limited, meaning that the duplication of assessments on the same health technology by HTA authorities and bodies in different Member States within identical or similar timeframes has not been sufficiently addressed. On the other hand, the main outcomes of the EUnetHTA Joint Actions should be considered when implementing this Regulation, in particular scientific output such as methodological and guidance documents as well as information technology (IT) tools to store and exchange information.

(8) In its conclusions of 1 December 2014 on innovation for the benefit of patients (3), the Council acknowledged the key role that HTA has as a health policy tool to support evidence-based, sustainable and equitable choices in healthcare and health technologies for the benefit of patients. In those conclusions, the Council further called on the Commission to continue to support cooperation in a sustainable manner, and asked for joint work between Member States on HTA to be enhanced and for opportunities for cooperation on exchange of information between competent bodies to be explored. Furthermore, in its conclusions of 7 December 2015 on personalised medicine for patients (4), the Council invited Member States and the Commission to strengthen HTA methodologies applicable to personalised medicine, and the Council conclusions of 17 June 2016 on strengthening the balance in the pharmaceutical systems in the European Union and its Member States (5) provided further evidence that Member States see clear added value in cooperation on HTA. The joint report of October 2016 of the Commission’s Directorate-General for Economic and Financial Affairs and the Economic Policy Committee further called for enhanced European cooperation on HTA. Finally, in its conclusions of 15 June 2021 on Access to medicines and medical devices for a Stronger and Resilient EU (6), the Council invited Member States and the Commission to explore the possibility of establishing an EU Real-World data collection and evidence generation action plan, which will promote better collaboration between ongoing national and cross-border initiatives and which could contribute to reducing evidence gaps in HTA and payer decisions.

(9) The European Parliament, in its resolution of 2 March 2017 on EU options for improving access to medicines (7), called on the Commission to propose legislation on a European system for HTA as soon as possible and to harmonise transparent HTA criteria in order to assess the added therapeutic value and relative effectiveness of health technologies compared with the best available alternative, that takes into account the level of innovation and benefit for patients.

(10) In its communication of 28 October 2015 on Upgrading the Single Market: more opportunities for people and business, the Commission declared its intention to introduce an initiative on HTA to increase coordination in order to avoid multiple assessments of a product in different Member States and improve the functioning of the Single Market for health technologies.

(11) This Regulation aims to achieve a high level of protection of health for patients and users while ensuring the smooth functioning of the internal market as regards medicinal products, medical devices and in vitro diagnostic medical devices. At the same time, this Regulation establishes a framework to support Member State cooperation and the measures needed for clinical assessment of health technologies. Both objectives are being pursued simultaneously and, whilst inseparably linked, one is not secondary to the other. As regards Article 114 of the Treaty on the Functioning of the European Union (TFEU), this Regulation sets out the procedures and the rules for carrying out joint work and establishing a framework at Union level. As regards Article 168 TFEU, whilst aiming at providing a high level of health protection, this Regulation allows for cooperation between Member States on certain aspects of HTA.

(12) Joint work should be produced following the principle of good administrative practice, and it should aim to achieve the highest level of quality, transparency and independence.

(13) Health technology developers often face the difficulty of submitting the same information, data, analyses and other evidence to different Member States, and also at various points in time. The duplication of submissions and consideration of different timings for submission across Member States can constitute a significant administrative burden for health technology developers, in particular for smaller companies with limited resources, and might contribute to impeding and distorting market access, leading to a lack of business predictability, higher costs and, in the long run, negative effects on innovation. Thus, this Regulation should provide for a mechanism that ensures that any information, data, analyses and other evidence required for the joint clinical assessment should be submitted only once at Union level by the health technology developer.

(14) In accordance with Article 168(7) TFEU, the Member States are responsible for the definition of their health policy and for the organisation and delivery of health services and medical care. Those responsibilities include the management of health services and medical care and especially the allocation of the resources assigned to them. It is necessary therefore that Union action is limited to those aspects of HTA that relate to the joint clinical assessment of a health technology and to ensure in particular that there are no value judgements in joint clinical assessments in order to respect the responsibilities of Member States pursuant to Article 168(7) TFEU. In that regard, the joint clinical assessments provided for by this Regulation constitute a scientific analysis of the relative effects of the health technology as assessed on the health outcomes against the chosen parameters which are based on the assessment scope. The scientific analysis will further include consideration of the degree of certainty of the relative effects, taking into account the strengths and limitations of the available evidence. The outcome of joint clinical assessments should therefore not affect the discretion of Member States to carry out assessments on the clinical added value of the health technologies concerned or predetermine subsequent decisions on pricing and reimbursement of health technologies, including the fixing of criteria for such pricing and reimbursement decisions, which could depend on both clinical and non-clinical considerations individually, or together, and which remain solely a matter of national competence.

(15) Member States should be able to perform complementary clinical analyses, which are necessary for their overall national HTA process, on the health technologies for which a joint clinical assessment report is available. In particular, Member States should be able to perform complementary clinical analyses relating, inter alia, to patient groups, comparators or health outcomes other than those included in the joint clinical assessment report, or using a different methodology if that methodology would be required in the overall national HTA process of the Member State concerned. If additional information, data, analyses and other evidence is needed for complementary clinical analyses, Member States should be able to ask the health technology developers to submit the necessary information, data, analyses and other evidence. This Regulation should not restrict in any way Member States’ rights to perform non-clinical assessments on the same health technology prior to, during the preparation of, or after the publication of a joint clinical assessment report.

(16) In order to guarantee the highest quality of joint clinical assessments, ensure a wide acceptance and enable pooling of expertise and resources across national HTA authorities and bodies, it is appropriate to follow a stepwise approach, starting with a small number of jointly assessed medicinal products and only at a later stage requiring joint clinical assessments to be carried out for other medicinal products undergoing the centralised marketing authorisation procedure provided for under Regulation (EC) No 726/2004 of the European Parliament and of the Council (8) and where those medicinal products are subsequently authorised for a new therapeutic indication.

(17) Joint clinical assessments should also be carried out on certain medical devices as defined in Regulation (EU) 2017/745 of the European Parliament and of the Council (9) which are in the highest risk classes and for which the relevant expert panels referred to in Article 106(1) of that Regulation have provided their opinions or views, as well as on in vitro diagnostic medical devices classified as class D pursuant to Regulation (EU) 2017/746 of the European Parliament and of the Council (10).

(18) Taking into consideration the complexity of certain medical devices and in vitro diagnostic medical devices, and the expertise required to assess them, Member States should be able, where they see an added value, to undertake voluntary cooperation on HTA on medical devices classified as class IIb or III pursuant to Article 51 of Regulation (EU) 2017/745 and in vitro diagnostic medical devices classified as class D pursuant to Article 47 of Regulation (EU) 2017/746 which are software and which do not fall within the scope of joint clinical assessments under this Regulation.

(19) In order to ensure that joint clinical assessments carried out on health technologies remain accurate and relevant, of high quality and based on the best scientific evidence available at any given time, it is appropriate to establish conditions for updating those assessments, in particular where additional data that becomes available subsequent to the initial assessment has the potential to increase the accuracy and quality of the assessment.

(20) A Member State Coordination Group on Health Technology Assessment (the ‘Coordination Group’) composed of Member States’ representatives, in particular from HTA authorities and bodies, should be established with responsibility for overseeing the carrying out of joint clinical assessments and other joint work within the scope of this Regulation. In order to ensure a Member State-led approach to joint clinical assessments and joint scientific consultations, Member States should designate the members of the Coordination Group. Those members should be designated with the goal of ensuring a high level of competence in the Coordination Group. Members of the Coordination Group should designate HTA authorities and bodies to the subgroups, which provide adequate technical expertise for carrying out joint clinical assessments and joint scientific consultations, taking into account the need to provide expertise on the HTA of medicinal products, medical devices and in vitro diagnostic medical devices.

(21) To reflect the scientific nature of the cooperation and ensure that decisions taken by the Coordination Group meet the objectives of guaranteeing joint work of the highest scientific quality and impartiality, the Coordination Group should use its best endeavours to reach a consensus. If such a consensus cannot be reached, and in order to ensure a smooth decision-making mechanism in the Coordination Group, decisions of a technical and scientific nature should be taken on a simple majority basis where one vote is given to each Member State irrespective of the number of members of the Coordination Group from any given Member State. By way of exception, and given its different nature, decisions on the adoption of the annual work programme, the annual report and the strategic direction for the work of the subgroups should be taken on a qualified majority basis.

(22) The Commission should not take part in votes on joint clinical assessments or comment on the content of joint clinical assessment reports.

(23) The Coordination Group should ensure that the scientific joint work as well as the procedures and methodology for the preparation of joint clinical assessment reports and joint scientific consultation outcome documents guarantee the highest quality, are prepared in a timely manner, and reflect the state of the art of medical science at the time of their preparation.

(24) Methodologies for performing joint clinical assessments and joint scientific consultations should be adapted to include specificities of new health technologies for which some data may not be readily available. This may be the case for, inter alia, orphan medicinal products, vaccines and advanced therapy medicinal products.

(25) The assessment scope for joint clinical assessments should be inclusive and should reflect all Member States’ needs in terms of data and analyses to be submitted by the health technology developer.

(26) Where joint clinical assessments are used to prepare subsequent administrative decisions at Member State level, they constitute one of several preparatory steps in a multi-step procedure. Member States remain the sole entity responsible for national HTA processes, for the conclusions on the value of a health technology and for the decisions resulting from HTAs. Member States should be able to determine at which step of their HTA process, and by which authority or body, the joint clinical assessment reports should be considered.

(27) The Coordination Group should make all efforts to endorse the joint clinical assessment report by consensus. Where a consensus cannot be reached, and with a view to ensuring the finalisation of joint clinical assessment reports within the timeframe set, divergent scientific opinions should be included in those reports. To ensure the integrity of the system of joint clinical assessments and the aim for consensus, the inclusion of divergent scientific opinions should be limited to those opinions which are fully justified on scientific grounds, and therefore should be considered as an exceptional measure.

(28) Member States should remain responsible for drawing conclusions at national level on the clinical added value of a health technology, as such conclusions depend on the specific healthcare context in any given Member State, and on the relevance of individual analyses included in the joint clinical assessment report (for example, several comparators could be included in the joint clinical assessment report, of which only a selection is relevant to a given Member State). The joint clinical assessment report should include a description of the relative effects observed for the health outcomes analysed, including numerical results and confidence intervals, and an analysis of scientific uncertainty and strengths and limitations of the evidence (for example, internal and external validity). The joint clinical assessment report should be factual and should not contain any value judgement, ranking of health outcomes, conclusions on the overall benefit or clinical added value of the assessed health technology, any position on the target population in which the health technology should be used, or any position on the place the health technology should have in the therapeutic, diagnostic or preventive strategy.

(29) Transparency and public awareness of the process is essential. Where there is confidential data for commercial reasons, the reasons for confidentiality need to be clearly set out and justified and the confidential data well delimitated and protected.