Explanatory Memorandum to COM(2018)51 - Health technology assessment - Main contents
Please note
This page contains a limited version of this dossier in the EU Monitor.
| dossier | COM(2018)51 - Health technology assessment. |
|---|---|
| source | COM(2018)51  |
| date | 31-01-2018 |
1. CONTEXT OF THE PROPOSAL
Health technology assessment (HTA) is a multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased and robust manner. Its aim is to inform the formulation of safe and effective health policies that are patient focused and seek to achieve best value. The term "health technology” is to be understood in a broad sense comprising medicinal products, a medical device or medical and surgical procedures as well as measures for disease prevention, diagnosis or treatment used in healthcare.
HTA is thus an evidence-based process that independently and objectively assesses a new or existing technology and compares it with other health technologies and / or the current standard of care. HTA is primarily used to inform decision-making in Member States by providing a scientific evidence base for decisions on the pricing and reimbursement of health technologies. HTA can cover different aspects (domains) ranging from clinical domains (e.g. safety, clinical effectiveness) to non-clinical domains (e.g. economic, ethical, organisational). This proposal focuses on clinical assessments which are typically based on global evidence (e.g. worldwide clinical trials in the case of medicinal products and multi-national clinical trials for medical devices) compared with non-clinical assessments which include domains that are often more sensitive to national / regional contexts.
At EU level, cooperation on HTA has been ongoing since the 1980s. To support cooperation between HTA bodies, the European Union has made substantial investments. Two Joint Actions (EUnetHTA JA) have been carried out together with a number of projects. A third Joint Action (EUnetHTA Joint Action 3) was launched in June 2016 and runs until 2020 with a total budget of EUR 20 million. Participation in the Joint Actions has been very high, including participation from all EU Member States. The third Joint Action focuses on developing common assessment methodologies, piloting and producing joint clinical assessments and full HTA reports, and on developing and maintaining common ICT tools. In addition, following the adoption of the Cross-Border Healthcare Directive (Directive 2011/24/EU), the HTA Network was established in 2013 to provide strategic and political guidance to the scientific and technical cooperation at Union-level.
·Reasons for and objectives of the proposal
Despite the achievements of the current EU cooperation, a number of problems have been identified, which cannot be sufficiently addressed by continued project-based voluntary cooperation on HTA.
Contents
- Problem 1. Impeded and distorted market access
- Problem 2. Duplication of work for national HTA bodies
- Problem 3. Unsustainability of HTA cooperation
- Chapter II - Joint work on health technology assessment at Union-level
- Section 1 - Joint clinical assessments
- Scope
- Progressive implementation
- Preparation of joint clinical assessment reports
- Use of joint clinical assessment reports by Member States
- Section 2 - Joint scientific consultations
- Section 3 - Emerging health technologies
- Section 4 - Voluntary cooperation
- Chapter III - Rules for clinical assessments
- Chapter IV - Support framework
- Chapter V - Final provisions
The different national processes and methodologies of national and regional HTA bodies mean that health technology developers who want to introduce a health technology in multiple Member States are confronted with various data and evidence requests. This in turn contributes to impeded and distorted market access, leading to a lack of business predictability, higher costs, and, in the long run, negative effects on innovation. Differences in national processes and methodologies also lead to differences in how evidence is considered in assessments, which can contribute to delays and inequalities in availabilities of innovative health technologies for patients.
Clinical assessments of the same technologies are being conducted in parallel or within a similar time-frame by HTA bodies in different Member States, resulting in duplication of work and inefficient use of resources. In addition, the current low levels of use at national level of the joint clinical assessments produced through the Union-level cooperation result in duplication and incur additional work and costs. The duplication may be associated with different outcomes/conclusions, which negatively affect business predictability and contribute to delays and inequalities in availabilities to patients of the most innovative health technologies.
The current Union-level cooperation on HTA is project-based. This means its funding is short-term, needs to be secured and renegotiated in every financial cycle, and there is no guarantee for the continuation of activities in the long-term. During the initiation and closing of each such large-scale project, substantial time and resources are spent on organisational issues leading to disruption in the output of the scientific cooperation.
With regard to the problems identified, the objectives of the current proposal are the following:
General objectives:
–Ensure a better functioning of the internal market;
–Contribute to a high level of human health protection.
Specific objectives:
–Improve the availability of innovative health technologies for EU patients;
–Ensure efficient use of resources and strengthen the quality of HTA across the EU;
–Improve business predictability.
Operational objectives:
–Promote convergence in HTA tools, procedures and methodologies;
–Reduce duplication of efforts for HTA bodies and industry;
–Ensure the use of joint outputs in Member States;
–Ensure the long-term sustainability of EU HTA cooperation.
·Consistency with existing policy provisions in the policy area
Currently the Union-level HTA cooperation is facilitated through Article 15 of the Cross-Border Healthcare Directive (Directive 2011/24/EU). This Directive provides for the establishment of a network of Member States' HTA authorities and bodies to facilitate cooperation and the exchange of scientific information among Member States. The objectives of the HTA Network are, inter alia, to support cooperation between national HTA bodies, support the provision and exchange between Member States of information on the relative efficacy of health technologies and to avoid duplication of assessments. The network thus provides a strategic steer to the scientific cooperation carried out under it and through the EU-funded initiatives described above (i.e. the Joint Actions).
This proposal incorporates the provisions of Article 15 of Directive 2011/24/EU and builds upon these through reinforced cooperation at Union-level. The proposal thus deletes Article 15 of that Directive. The definition of health technology used in Directive 2011/24/EU is also used in this proposal, ensuring a consistent approach across the two texts.
·Consistency with other Union policies
The proposal is in line with the EU's overarching objectives, including a smooth functioning of the internal market, sustainable health systems, and an ambitious research and innovation agenda. In addition to coherence with these EU policy objectives, the proposal is also consistent with and complementary to existing EU legislation related to medicinal products and medical devices 1 . For example, while the regulatory process and the HTA process will remain well separated as they have different purposes, there are opportunities to create synergies, through mutual information-sharing and better alignment of the timing of procedures between the proposed joint clinical assessments and the centralised marketing authorisation for medicinal products 2 . Synergies are also expected between joint clinical assessments of medical devices and some of the provisions in the new EU Regulations for medical devices and in vitro diagnostics (e.g. strengthened rules on clinical evaluation and clinical investigation; EU-level expert panels for high-risk medical devices).
Given the recent entry into force of the new Regulations on medical devices, their on-going implementation, and the impact of this on medical device authorities and manufacturers, a coherent approach has been taken to ensure that the implementation of the provisions in this proposal will be phased in, to avoid overlapping timelines and to ensure that the implementation of both sets of legislation meet their respective objectives without creating uncertainty or undue administrative burden in the sector.
Moreover, the joint scientific consultations provided for by this proposal, through which advice can be given to health technology developers in the development phase of a technology, will contribute to the objectives of related EU legislation on clinical trials to ensure that the evidence generated in clinical studies is robust and benefits patients and public health.
In addition, the proposal will provide useful input to and synergies with the EU Digital Single Market agenda by encouraging innovation and research of high-tech health technologies, facilitate information sharing on registries of real world evidence, and by supporting the development of a Union-level IT infrastructure supporting EU cooperation on HTA.
2. LEGAL BASIS, SUBSIDIARITY AND PROPORTIONALITY
·Legal basis
The proposal is based on Article 114 of the Treaty on the Functioning of the European Union (TFEU).
Article 114 TFEU allows for the adoption of measures for the approximation of the provisions laid down by law, regulation or administrative action in the Member States, provided they are necessary for the establishment or functioning of the internal market, whilst at the same time ensuring a high level of public health protection. Article 114 TFEU provides an appropriate legal basis given the objectives of the proposal, namely to remove some of the existing divergences in the internal market for health technologies caused by procedural and methodological differences in clinical assessments carried out in Member States along with the considerable duplication of such assessments across the EU.
In line with Article 114(3) (TFEU), a high level of human health protection has been considered in the preparation of the proposal which is expected to improve the availability of innovative health technologies for EU patients.
·Subsidiarity (for non-exclusive competence)
The diversity and multitude of approaches to clinical assessments across the Member States means that, due to their scale and effect, only action at Union-level can eliminate the obstacles described. Without action at EU-level it is unlikely that national rules on how HTAs are carried out would be further aligned and thus the current fragmentation of the internal market would persist.
While the on-going cooperation, namely the Joint Actions and the HTA Network, has illustrated benefits of EU cooperation, in terms of establishing the professional network, the tools and methodologies for cooperation and piloting joint assessments, this cooperation model has not contributed to the removal of the fragmentation of national systems and the duplication of efforts.
The impact assessment report accompanying this proposal identified a distinction between clinical assessments where there is considerable scope for alignment in the Member States' procedures for carrying out such assessments, assessment methods and the types of data requested and non-clinical assessments which focus more on domains (e.g. economic, organisational, ethical) which are linked more to national contexts, and which are closer to the final decisions on pricing and reimbursement which remain strictly in the hands of Member States. By thus focusing on clinical assessments, the proposal targets the domains of HTA where EU added value is considered to be strongest.
The aims of this initiative cannot therefore be achieved sufficiently without strengthened HTA cooperation at EU-level.
·Proportionality
The proposal constitutes a proportionate and necessary response to addressing the problems described in section 1. In particular, the proposed requirement of not repeating at national level joint clinical assessments performed at Union-level and the nature of the joint clinical assessment, which will be limited to the assessment of the evidence, is necessary to reduce duplication and avoid discrepancies. At the same time, the proposal will avoid any interference with Member States competences in decisions concerning access to the health technologies assessed at Union-level. By focusing the joint work on clinical aspects of HTA, where EU cooperation can bring both quality and efficiency gains, the proposal does not go beyond what is necessary. The assessment of more context-specific HTA domains (e.g. economic, organisational, ethical) and decision-making on pricing and reimbursement remain at Member State-level.
By reducing the current duplication and fragmentation, the proposal will optimise resources in Member States and also reduce the administrative burden for health technology developers currently undergoing assessments on the same health technology in multiple national systems.
The proposal is proportionate in that it limits the scope of joint work to specific types of medicinal products and medical devices and allows flexibility concerning the timing of joint clinical assessments for medical devices. This takes into account the differences between medicinal product and medical device sectors and their market access pathways. By focusing on the type of health technologies where current duplication of work among HTA bodies is most prominent and the benefit of joint assessment is strongest the proposal brings clear added value.
The proportionality of the proposal is also well reflected in the approach adopted for medical devices. The proposal does not introduce any new requirements on health technology developers when these are not already set out in national legislation. On the other hand, the proposal will ensure that when HTA is performed, the methodologies and procedures applied are more predictable across the EU and when subject to joint clinical assessment such assessments are not repeated, avoiding duplication and discrepancies.
Finally, the proposal respects the principle of proportionality by allowing sufficient time for both Member States and industry to adapt to the new EU system through a phase-in approach for the number of assessments carried out at Union-level and a transitional period for Member State participation.
·Choice of the instrument
The proposal takes the form of a new Regulation. This type of instrument is considered to be the most suitable considering that a key element of the proposal is the establishment of procedures and structures for cooperation on joint work at Union-level. While inevitably such a transition to a Union-wide approach will require some adjustments to national rules, for example, as regards allowing for the use of joint clinical assessments at national level as part of the overall HTA, that transition does not result in a need for significant implementing measures establishing those procedures and structures at national level.
In addition, the majority of detailed national rules on how HTA is actually carried out are contained in the administrative provisions of Member States’ HTA bodies rather than in national legislation. This suggests that a suitable adaptation period before the date of application of a Regulation would be a more adequate and proportionate approach than the transposition needed for a Directive, in ensuring use of joint clinical assessments and common rules at national level.
3. RESULTS OF EX-POST EVALUATIONS, STAKEHOLDER CONSULTATIONS AND IMPACT ASSESSMENTS
·Stakeholder consultations
Extensive consultation with stakeholders took place in the preparation of this proposal. In order to reach all interested stakeholders and to ensure a high quality and balanced input, a combination of consultation methods was used:
·Besides the feedback received in response to the publication of the Inception Impact Assessment, the Commission held a broad online public consultation between October 2016 and January 2017. In addition, position statements from different interest groups were received by email;
·Bilateral meetings with interested stakeholder representatives were organised throughout the preparation phase to allow in-depth discussion on specific topics and the expression of non-organised interests; 3
·Consultation of experts was carried out through the existing cooperation mechanisms, EUnetHTA Joint Action 3 and the HTA Network. Presentations at external events were used to reach out to stakeholders, to explain the main elements of the initiative, to invite them to participate in the public consultation, and listen to their views and opinions.
A large majority of stakeholders emphasised that EU cooperation beyond 2020 is needed to ensure a constant exchange of information and knowledge between HTA institutions in Europe, to increase synergies between Member States, to streamline HTA methodologies, to increase transparency and evidence-based decision-making, as well as to ensure business predictability. The possibility to access a larger number of HTA reports with less duplication of work and better allocation of resources by HTA bodies was highlighted.
While all representatives of public administrations are in favour of continuing EU cooperation on HTA beyond 2020, some indicated a preference for voluntary cooperation while others supported a system with mandatory elements (i.e. a legal framework for EU cooperation on HTA to streamline interoperability of HTA national systems). Most contributors highlighted that in case of a mandatory system, use of joint work should be limited to clinical and technical matters, whereas assessment of non-clinical domains (e.g. economic, legal, ethical) should be carried out individually or jointly by interested Member States/HTA bodies on a voluntary basis. The idea of progressive implementation was also raised.
Citizens, patients and consumers representatives, as well as healthcare providers and academia were extremely positive, with most of them in favour of a collaboration covering both the clinical and economic parts of assessments. They underlined the need for involving patients and healthcare professionals in the HTA process, the need for transparency (e.g. making the summaries of HTA reports publicly available, including criteria and rationale for evaluation), and the need to ensure the independence of HTA bodies from industry and other interests.
As regards health technology developers, the pharmaceutical industry and their trade associations supported the harmonisation of clinical assessments at the time of the launch of their health technologies. It was stressed that the economic part of assessments should remain the responsibility of Member States. Medical device manufacturers and their trade associations reiterated the importance of taking into account the particularities of their sector and the need for a Member State-driven approach. It was underlined that HTA should focus on products that are innovative and address high unmet patient needs in disease areas where appropriate clinical and economic evidence has been or can be generated (e.g. transformative in vitro diagnostics and medical devices).
·Collection and use of expertise
In addition to the extensive stakeholder consultation described in previous sections, the following three external studies were conducted to support assessing the impacts of this initiative:
–Mapping of HTA National Organisations, Programmes and Processes in EU and Norway. 2017, Contract No. 17010402/2016/734820
–Mapping of HTA Methodologies in EU and Norway. 2017, DG SANTE Contract No. 17010402/2016/736040
–Study on Impact Analysis of Policy Options for Strengthened EU Cooperation on HTA. 2017. Sogeti, Austrian Public Health Institute, London School of Economics. (CHAFEA/2016/Health/16)
The Commission has also used and benefitted from, the vast pool of expertise available in the HTA Network and EUnetHTA Joint Action 3.
·Impact assessment
The impact assessment report 4 provides an in-depth analysis of four policy options: No Joint Actions after 2020 (policy option 1, baseline scenario); Project-based cooperation on HTA activities (policy option 2); Permanent cooperation on common tools, procedures and early dialogues (policy option 3); and Permanent cooperation on common tools, procedures, early dialogues and joint clinical assessments (policy option 4). Based on this analysis, the impact assessment report presents a preferred policy option, which has provided the basis for the contents of this proposal (see section 8 of Impact Assessment). This preferred option builds primarily on policy option 4, but also integrates elements of policy option 2 as well as some adjustments (e.g. transitional arrangements for Member States and progressive implementation of the product scope for joint clinical assessments).
As described in more detail in the impact assessment report, the preferred option is considered to provide for the best combination of effectiveness and efficiency in reaching the policy objectives, while also respecting the principles of subsidiarity and proportionality. It allows for the best possible achievement of the internal market objectives by promoting convergence in procedures and methodologies, reducing duplication (e.g. of clinical assessments) and therefore the risk of divergent outcomes, thus contributing to improving the availability of innovative health technologies for patients. Moreover, it provides Member States with a sustainable framework, allowing them to pool expertise, reinforce evidence-based decision-making, and supporting them in their efforts to ensure the sustainability of national health systems. The preferred option is also cost efficient in the sense that the costs are significantly outweighed by savings for Member States and industry, as a result of pooling of resources, avoiding duplication and improving business predictability.
The Regulatory Scrutiny Board gave its initial opinion on the impact assessment report on 27 October 2017 and asked for resubmission of a revised version. On 4 December 2017, the Regulatory Scrutiny Board delivered its second opinion (positive with reservations), indicating a number of points where further changes to the report were needed. The necessary changes have been made in the final version of the report. In particular, the final version of the report provides further clarifications on the proportionality of the preferred option and describes in more detail the rationale for and implications of mandatory use of the joint work. Further details are also provided on how views expressed by Member States have been taken into account in the preferred option. Finally, the report describes in more detail how possible risks and implementation challenges are addressed by the preferred option.
·Regulatory fitness and simplification
The proposal is relevant for small and medium-sized enterprises (SMEs), which are particularly prominent in the medical device sector. However, no specific provisions are foreseen for micro-enterprises, as these are not expected to play a major role in bringing new health technologies to the market. The proposal is expected to benefit SMEs by reducing the current administrative burden and compliance costs linked to submissions of multiple dossiers to meet different national HTA requirements. In particular, joint clinical assessments and joint scientific consultations foreseen by the proposal would increase business predictability for industry. This is particularly relevant for SMEs, as they tend to have a smaller product portfolios and more limited dedicated resources and capacities for HTA. Of note, the proposal does not provide for fees for joint clinical assessments or joint scientific consultations. Improved business predictability due to joint work on HTA across the EU is expected to positively impact on the competitiveness of the EU health technology sector.
The IT infrastructure foreseen by the proposal relies on standard IT tools (e.g. for databases, document exchange, internet-based publication), building on tools that have already been developed by the EUnetHTA Joint Actions.
·Fundamental rights
The proposal has limited consequences for the protection of fundamental rights. Where personal data is processed to fulfil the provisions of the proposal this will be done in line with the relevant Union legislation on personal data protection. The proposal contributes to achieving a high level of human health protection and is thus consistent with the Charter of Fundamental Rights in this regard.
4. BUDGETARY IMPLICATIONS
The implementation of this proposal has no impact on the current Multiannual Financial Framework 2014-2020 as the current cooperation on HTA is financed by the Public Health Programme. The financial impact on the EU budget post-2020 will be part of the Commission's proposals for the next Multiannual Financial Framework.
The budgetary implications are mainly related to the support framework provided for in this proposal, namely a central secretariat hosted by the European Commission, which will provide:
–administrative support (e.g. organisation of meetings, travel arrangements etc.) to the Coordination Group and its sub-groups including HTA experts nominated by the Member States' authorities who will carry out the joint work (e.g. joint clinical assessments, joint scientific consultations, studies on emerging health technologies, and provide expertise for the development and update of common rules and methodologies);
–scientific support (e.g. advice for the meetings of the coordination group and sub-groups, preparation of the documentation, manage procedures for involving stakeholders, ensure quality management including scientific scrutiny of reports and support the implementation of the joint work etc.);
–IT support (e.g. establish, host, and maintain an IT platform, including databases/repositories of joint and national HTA reports, secure communication etc.).
The proposal foresees remuneration in the form of special allowance to Member States' HTA bodies carrying out the joint work as assessors and co-assessors and travel expenses for Member State experts contributing to the activities of the coordination group and its sub-groups.
In-kind contribution from the Member States is foreseen in the form of seconded national experts 5 to the central secretariat and by national experts who will participate in the meetings and contribute to the activities of the Coordination Group and relevant sub-groups (e.g. on joint clinical assessments and joint scientific consultations).
5. OTHER ELEMENTS
·Implementation plans and monitoring, evaluation and reporting arrangements
The proposal provides for regular Commission monitoring and reporting on the implementation of the proposed Regulation starting, at the latest, one year after its date of application. To facilitate monitoring and reporting, Members States would be required to provide the Commission with the information necessary for the monitoring programme which will also benefit from the annual reports of the Coordination Group which will summarise the outputs of the joint work. The Commission will also carry out a formal evaluation of the Regulation and report on the conclusions of this evaluation.
An obligation is also placed on the Commission to report specifically on the implementation of the scope of the joint clinical assessments and support framework no later than five years after the date of application. This will allow the Commission to consider whether the proposed Regulation ensures that the most innovative health technologies are being assessed, taking into account technological developments in the sector. The report will also allow for an assessment of whether the support framework provided by the Commission continues to provide the most efficient and cost-effective governance mechanism for the joint work.
The proposal would place on the Commission an obligation to verify the joint clinical assessment reports prior to their publication. This will allow the Commission to ensure that the reports have been prepared in accordance with the requirements proposed and help to build trust in the system. The Commission will also monitor both the implementation of the common rules and the use of the joint work at Member State-level. In order to facilitate this task and also allow for the exchange of information between Member States, the proposal provides for specific reporting obligations on the Member States where they use joint clinical assessment reports at Member State-level and where they carry out clinical assessments based on the common rules.
In addition, monitoring and evaluation of the specific objectives will use several means of data collection, including a number of quantitative indicators to assess effectiveness, as outlined in section 9 of the Impact Assessment Report, evaluation of the wider impacts will also require a number of qualitative tools such as desk research, surveys, focus groups and Delphi surveys.
·Detailed explanation of the specific provisions of the proposal
The proposal consists of five chapters comprising a total of 36 articles.
Chapter I – General provisions
This chapter outlines the subject matter of the proposal and defines the key terms used in the proposed Regulation. To ensure consistency with other Union legislation, the definitions of medicinal product, medical device, and health technology in the proposal are aligned with those applied in Directive 2001/83/EC, Regulation (EU) No 2017/745, and Directive 2011/24/EU respectively. The Member State Coordination Group on Health Technology Assessment (the Coordination Group) is formally established in Article 3 along with its composition, roles and responsibilities to oversee the joint work referred to in Chapter II.
The Coordination Group will be Member State-led and manage the overall governance of the joint work. The Group will meet regularly to provide guidance and steer the cooperation. Under the authority of the Coordination Group, a number of sub-groups consisting of experts nominated by Member States will carry out the joint work foreseen in this proposal. For example, for the joint clinical assessments, Member States' HTA bodies acting as assessor and co-assessor will carry out the clinical assessment, prepare a draft report and consult relevant stakeholders. The Coordination Group will thereafter approve the joint reports which will then be published by the Commission and included in a list of health technologies having undergone joint clinical assessments.
This joint work is based on the annual work programme of the Coordination Group which is outlined in Article 4 of the proposal. The annual work programme provides clarity on the planned work of the Group and allows health technology developers to foresee any expected involvement they may have in the joint work for the year ahead.
This chapter establishes the four pillars of the future cooperation between Member States at Union-level (the joint work) namely, joint clinical assessments, joint scientific consultations, the identification of emerging health technologies, and voluntary cooperation. The work will be Member State-led through the Coordination Group.
The joint clinical assessments will be one of the main proponents of the future joint work and, following the end of the transitional period, participation in the assessments and use of the joint clinical assessment reports at Member State-level will be mandatory. As described below, there will be a progressive phase-in approach to the annual number of joint clinical assessments carried out during the transitional period.
Joint clinical assessments are limited to:
·medicinal products undergoing the central marketing authorisation procedure, new active substances and existing products for which the marketing authorisation is extended to a new therapeutic indication; and
·certain classes of medical devices and in vitro diagnostic medical devices for which the relevant expert panels established in accordance with Regulations (EU) 2017/745 and 2017/746 have given their opinions or views and which have been selected by the Coordination Group set up under this Regulation based on the following criteria:
·unmet medical need;
·potential impact on patients, public health, or healthcare systems (e.g. burden of disease, budget impact, transformative technology);
·significant cross-border dimension;
·Union-wide added value (e.g. relevance to a large number of Member States);
·the resources available to it.
This relatively limited scope and the selection criteria reflect the need to take a proportionate approach concerning the type and amount of health technologies assessed at Union level. By focusing on the most innovative technologies and selecting those with the most Union-wide and public health impact, the EU added value of the assessments will be maximised.
The timing of the procedure for joint clinical assessments for medicinal products will be coordinated with that of the central marketing authorisation procedure (i.e. the joint clinical assessment report will be available at the time of or shortly after the final Commission Decision granting marketing authorisation), ensuring its timeliness for supporting Member States decision-making at the time of market launch.
Taking into account the more decentralised market access pathway for medical devices, the timing of the joint clinical assessment will not necessarily be aligned with the timing of the conformity assessment i.e. it will not always be at the time of market launch. Instead, the Coordination Group will consider the most appropriate time point for a joint clinical assessment in line with the abovementioned selection criteria.
The identified scope and phase-in approach take into account the current level of duplication among Member States' HTA bodies, the EU added value of a joint approach, and stakeholder views and concerns.
The proposal provides for progressive implementation of the amount of joint clinical assessments during the transitional period. This means that the number of joint clinical assessments will increase gradually during the first three years after the date of application, taking into account specific selection criteria (same as those used permanently for medical devices, described above). The Coordination Group will select the health technologies based on these criteria and include them in the annual work programme. Following the end of the transitional period, all medicinal products falling within the scope and granted marketing authorisation in a given year will be assessed, while a selection of medical devices falling within the scope will undergo assessment.
The joint clinical assessments will cover the four assessment domains described in the definition of clinical assessment in Chapter I. A step-by-step procedure on how the joint clinical assessment reports will be prepared is outlined in this section. Member States, through their HTA authorities and bodies, will be in the lead, selecting the assessor authority or body which will draft the report providing support and comments throughout the drafting process, and approving the final reports. The selection of the assessors and co-assessors will be a particularly important step in ensuring the quality of the report and the independence of the drafting process and this selection will thus be made based on specific procedural rules to be developed in tertiary legislation. The health technology developer whose health technology is the subject of the report, as well as patients, clinical experts and other stakeholders will also be given opportunities to provide input in order to ensure a thorough, independent and transparent assessment process. Once verified by the Commission, the final reports will be published and then used by the Member States.
The detailed procedural rules for each step in the process will be further developed in tertiary legislation while the common rules and documentation developed in tertiary legislation for clinical assessments at Member State-level will also be used for joint clinical assessments, ensuring a consistent approach across national and Union-level clinical assessments. The development of the tertiary legislation will take as a basis the work on common procedures, methodologies and documents already being developed in the EUnetHTA Joint Action 3.
The proposal does not oblige Member States to carry out a HTA on health technologies which are the subject of joint clinical assessments. However, where Member States do carry out HTAs on such health technologies, there is a requirement for mandatory use of the joint clinical assessment report and no repetition of the clinical assessment in Member States' overall HTA processes. This means that Member States will continue to carry out non-clinical assessments i.e. on the non-clinical HTA domains (e.g. economic, organisational, ethical) and will draw conclusions on the overall added value of the assessed health technology based on the joint clinical assessment report and their own non-clinical assessment.
The proposal provides for the possibility for health technology developers to make a request to the Coordination Group for a joint scientific consultation. The joint scientific consultations, commonly referred to as early dialogues, allow a developer in the development phase of a health technology to seek the advice of HTA authorities and bodies on the data and evidence likely to be required as part of a potential future joint clinical assessment. The Coordination Group will carry out an annual number of joint scientific consultations based on its annual work programme, taking into account the resources available to it.
The preparation of joint scientific consultation reports will mirror the approach taken for joint clinical assessments as described above. The main difference will be that the joint scientific consultation reports approved by the Coordination Group will be addressed to the health technology developer, will not be published, nor will they bind the developer or the Member States at the time of (joint) clinical assessment. To ensure transparency, information on the consultations will be included in the Coordination Group's annual reports.
The joint work would also encompass an annual study to be carried out under the responsibility of the Coordination Group on the identification of emerging health technologies. This exercise, commonly referred to as horizon scanning, will act as a key input for the annual work programmes, helping to ensure that the health technologies expected to have a major impact on patients, public health or healthcare systems are identified at an early stage in their development and are included in the joint work of the Coordination Group. The proposal requires the Coordination Group to fully consult with all relevant interest groups during this exercise.
Under this section, the proposal provides for the possibility for Member States to continue to cooperate on a voluntary basis at Union-level. This voluntary cooperation would allow for HTA on health technologies other than medicinal products or medical devices, non-clinical assessments, collaborative assessments on medical devices i.e. on medical devices not selected for joint clinical assessment, and cooperation on the provision of additional evidence which can facilitate HTA.
Voluntary cooperation should take advantage of the outputs from research on HTA, such as methods for the use of real world evidence to reduce the uncertainty on effectiveness, the evaluation of innovative technologies (e.g. eHealth, personalised medicine) and the assessment of non-clinical domains (e.g. the impact of medical devices on the organisation of care).
This cooperation will benefit from the support framework set up under this proposal while participation in it and use of the results would be fully voluntary.
This Chapter lays down common rules for carrying out clinical assessments at Member State-level which will then be developed in detail in tertiary legislation. These rules will ensure a harmonised approach to clinical assessment across EU Member States. In the development of the rules, use will be made of the tools already developed under the EUnetHTA Joint Actions as a base and the common rules will also be used for the joint clinical assessments at EU-level. An important part of these rules will be to ensure that clinical assessments, whether carried out at EU or Member State level, are done in an independent and transparent manner, free from conflicts of interest.
This chapter sets out the support framework which will support the joint work at EU-level. It provides for its funding and support from the Commission acting as its secretariat and providing its IT infrastructure. A stakeholder network is also established under this chapter along with reporting and monitoring obligations placed on the Commission.
The Commission will support the work of the Coordination Group and the sub-groups, in particular by providing scientific, administrative and IT support (as described in detail in the section on budgetary implications).
This chapter outlines the timeline for the implementation of the Regulation. Following the entry into force, a three-year period before the date of application is proposed which will allow for the development and adoption of all tertiary legislation (the implementing and delegated acts) provided for in the proposal as well as the preparatory steps necessary for the joint work. Following the date of application, a further three-year transitional period is envisaged to allow for a phase-in approach in terms of the work undertaken and to allow Member States to fully adapt to the new system. During this transitional period, Member States would have the option to delay their participation in the joint work on joint clinical assessments and joint scientific consultations. Under such circumstances they would not be obliged to use the output of this joint work at Member State-level but would be obliged to use the common rules for their own clinical assessments. Member States will not be able to delay their participation partially i.e. for only one category of health technology or for only one part of the joint work.
The proposal also includes a safeguard clause allowing clinical assessments to be carried out at national level using means other than the common rules, on grounds related to the need to protect public health specific to the Member State wishing to invoke the clause. Such measures would need to be justified and notified to the Commission for an assessment of the justifications presented.