(1)

The development of health technologies is a key driver of economic growth and innovation in the Union. It forms part of an overall market for healthcare expenditure that accounts for 10% of EU gross domestic product. Health technologies encompass medicinal products, medical devices and medical procedures, as well as measures for disease prevention, diagnosis or treatment.

(1)

The development of health technologies is key to achieving the high level of health protection that health policies must ensure, for the benefit of all citizens. Health technologies are an innovative sector of the economy which form part of an overall market for healthcare expenditure that accounts for 10% of EU gross domestic product. Health technologies encompass medicinal products, medical devices and medical procedures, as well as measures for disease prevention, diagnosis or treatment.

(1a)

Expenditure on medicines stood at 1,41% of GDP in 2014 and accounted for 17,1% of overall health expenditure, of which it is a major component. Health expenditure in the Union amounts to 10% of GDP, i.e., EUR 1 300 000 million per annum, EUR 220 000 million of which is pharmaceutical expenditure and EUR 110 000 million expenditure on medical devices.

(1b)

The Council conclusions of 16 June 2016 and the European Parliament resolution of 2 March 2017 on EU options for improving access to medicines  (2) highlighted that there are many barriers to access to medicine and innovative technologies in the Union, with the main barriers being the lack of new treatments for certain diseases and the high price of medicines, which in many cases do not have added therapeutic value.

(1c)

Marketing authorisations for medicinal products are granted by the European Medicines Agency on the basis of the principles of safety and efficacy. Normally the national health technology assessment agencies assess comparative effectiveness, because marketing authorisations are not accompanied by a comparative effectiveness study.

(2)

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

(2)

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

(2a)

As the World Health Organisation (WHO) stated at the 67th World Health Assembly in May 2014, HTA has to be a tool in support of universal health coverage.

(2b)

HTA should be instrumental in promoting innovation which offers the best outcomes for patients and society as a whole and is a necessary tool for ensuring the proper application and use of health technologies.

(3)

HTA covers both clinical and non-clinical aspects of a health technology. The EU 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 technology, the examination of the technical characteristics of the technology under assessment, its relative safety, and its relative clinical effectiveness. The five non-clinical assessment domains concern cost and economic evaluation of a technology, its ethical, organisational, social, and legal aspects. The clinical domains are therefore more suited to joint assessment at EU-level on their scientific evidence base, while the assessment of non-clinical domains tends to be more closely related to national and regional contexts and approaches.

(3)

HTA covers both clinical and non-clinical aspects of a health technology. The EU 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 (which form the ‘HTA Core model’) four are clinical and five are non-clinical. The four clinical domains of assessment concern the identification of a health problem and current technology, the examination of the technical characteristics of the technology under assessment, its relative safety, and its relative clinical effectiveness. The five non-clinical assessment domains concern cost and economic evaluation of a technology, its ethical, organisational, social, and legal aspects. The clinical domains are therefore more suited to joint assessment at EU-level on their scientific evidence base, while the assessment of non-clinical domains tends to be more closely related to national and regional contexts and approaches.

(3a)

Health professionals, patients and health institutions need to know whether or not a new health technology represents an improvement on existing health technologies, in terms of benefits and risks. Joint clinical assessments therefore aim to identify the added therapeutic value of new or existing health technologies in comparison with other new or existing health technologies, by undertaking a comparative assessment based on comparative trials against the current best proven intervention (‘standard treatment’) or against the current most common treatment where no such standard treatment exists.

(4)

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 to stimulate innovation that delivers better outcomes for patients.

(4)

HTA is an important tool for promoting high-quality innovation, steering research towards addressing the unmet diagnostic, therapeutic or procedural needs of healthcare systems as well as steering clinical and social priorities. HTA can also improve scientific evidence used to inform clinical decision-making, efficiency in use of resources, the sustainability of health systems, patient access to these health technologies, and the competitiveness of the sector through greater predictability and more efficient research. Member States use the outcome of HTA to augment the scientific evidence that informs decisions to introduce health technologies into their systems, i.e. , to inform decisions on how to allocate resources. HTA can therefore assist Member States in creating and maintaining sustainable healthcare systems and to stimulate innovation that delivers better outcomes for patients.

(4a)

Cooperation in the field of HTA can also play a role throughout the health technology cycle: in the early developmental stage through ‘horizon scanning’ in order to pinpoint technologies that will have a major impact; in the early dialogue and scientific advisory stages; in better study design to ensure greater research efficiency; and in the core stages of the overall assessment, once the technology is already established. Finally, HTA can help in decision-making on divestment in cases where a technology becomes obsolete and unsuitable compared to better alternative options that are available. Greater collaboration between Member States in the field of HTA should also help improve and harmonise standards of care as well as diagnostic and new-born screening practices across the Union.

(4b)

Cooperation in the field of HTA can extend beyond pharmaceutical products and medical devices. It can also cover areas such as diagnostics used to supplement treatment, surgical procedures, prevention, screening and health promotion programmes, information and communications technology (ICT) tools, health-care organisation plans and integrated care processes. Different demands are involved in assessing different technologies, depending on their specific features, meaning that a cohesive approach which can cater for these different technologies is needed in the field of HTA. Moreover, in specific areas such as treatments for rare diseases, paediatric medicines, precision medicine and advanced therapies, the added value of cooperation at Union level is likely to be even greater.

(5)

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 duplications and variations in outcomes that increase the financial and administrative burdens that act as a barrier to the free movement of the health technologies concerned and the smooth functioning of the internal market.

(5)

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 a duplication of requests for data that could increase the financial and administrative burdens that act as a barrier to the free movement of the health technologies concerned and the smooth functioning of the internal market. In some justified cases where the specificities of the national and regional healthcare systems and priorities need to be taken into account, a complementary assessment on certain aspects might be necessary. However, assessments that are not relevant for decisions in certain Member States could delay the implementation of innovative technologies and thus access of patients to beneficial innovative treatments.

(6)

While Member States have carried out some joint assessments within the framework of the EU co-funded joint actions, the production of output has been inefficient, relying on project-based cooperation in the absence of a sustainable model of cooperation. Use of the results of the joint actions, including their joint clinical assessments, at Member State-level has remained low, 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 .

(6)

Member States have carried out some joint assessments within the framework of the EU co-funded joint actions. Those assessments were carried out in three stages, under Article 15 of Directive 2011/24/EU of the European Parliament and of the Council  (3), and through three joint actions, each with specific objectives and a specific budget: EUnetHTA 1, 2010 to 2012 (EUR 6 million); EUnetHTA 2, 2012 to 2015 (EUR 9,5 million); and EUnetHTA 3, launched in June 2016 with an end date of 2020 (EUR 20 million). Given the timescales for those actions and in the interests of continuity, this Regulation establishes a more sustainable way of ensuring the continuation of the joint assessments. The main outcomes of the cooperation to date include the ‘HTA Core Model’ assessment model, which provides a framework for HTA reports; a database for sharing projects that are planned, ongoing or recently published by individual agencies (POP database); a data- and knowledge base for the storage of information and the stage reached in the assessment of promising technologies, or on the request for supplementary studies arising from the HTA; and a set of methodological guides and support tools for HTA agencies, including guidelines for adapting reports from one country to another .

(6a)

However, within the joint actions, the production of output has been inefficient and, in the absence of a sustainable model of cooperation, relying on project-based cooperation. Use of the results of the joint actions, including their joint clinical assessments, at Member State-level has remained low, 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.

(7)

The Council in its Conclusions of December 2014 (4) acknowledged the key role of health technology assessment and called on the Commission to continue to support cooperation in a sustainable manner.

(7)

In its Conclusions of December 2014 on innovation for the benefit of patients  (5), the Council acknowledged the key role of health technology assessment as a health policy tool to support evidence-based, sustainable and equitable choices in health care and health technologies for the benefit of patients. 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 . In addition, in its Conclusions of December 2015 on personalised medicine for patients, the Council invited Member States and the Commission to strengthen HTA methodologies applicable to personalised medicine, and the Council Conclusions of June 2016 on strengthening the balance in the pharmaceutical systems in the European Union and its Member States provided further evidence that Member States see clear added value in cooperation on HTA. The joint report of October 2016 of the Commission's DG for Economic and Financial Affairs and the Economic Policy Committee further called for enhanced European cooperation on HTA.

(8)

The European Parliament, in its resolution of 2 March 2017 on EU options for improving access to medicines, (6) called on the Commission to propose legislation on a European system for health technology assessment as soon as possible and to harmonise transparent health technology assessment criteria in order to assess the added therapeutic value of medicines .

(8)

The European Parliament, in its resolution of 2 March 2017 (7) on EU options for improving access to medicines, called on the Commission to propose legislation on a European system for health technology assessment as soon as possible and to harmonise transparent health technology assessment 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 order to ensure a better functioning of the internal market and contribute to a high level of human health protection it is appropriate to approximate the rules on carrying out clinical assessments at national level and clinical assessments of certain health technologies at Union level, and which also support the continuation of voluntary cooperation between Member States on certain aspects of HTA.

(10)

In order to ensure a better functioning of the internal market and contribute to a high level of human health protection it is appropriate to approximate the rules on carrying out clinical assessments at national level and clinical assessments of certain health technologies at Union level, and which also support the continuation of voluntary cooperation between Member States on certain aspects of HTA. That approximation should guarantee the highest quality standards and be aligned to best available practice. It should not stimulate a convergence towards the lowest common denominator nor force HTA bodies with more expertise and higher standards to accept lower requirements. It should rather lead to an improvement of the HTA capacity and quality at the national and regional level.

(11)

In accordance with Article 168(7) of the Treaty on the Functioning of the European Union (TFEU), the Member States remain responsible for the organisation and delivery of their healthcare. As such, it is appropriate to limit the scope of Union rules to those aspects of HTA that relate to the clinical assessment of a health technology, and in particular, to ensure that the assessment conclusions are confined to findings relating to the comparative effectiveness of a health technology . The outcome of such assessments should not therefore affect the discretion of Member States in relation to subsequent decisions on pricing and reimbursement of health technologies, including the fixing of criteria for such pricing and reimbursement which may depend on both clinical and non-clinical considerations, and which remain solely a matter of national competence.

(11)

In accordance with Article 168(7) of the Treaty on the Functioning of the European Union (TFEU), the Member States remain responsible for the organisation and delivery of their healthcare. As such, it is appropriate to limit the scope of Union rules to those aspects of HTA that relate to the clinical assessment of a health technology. The joint clinical assessment provided for by this Regulation constitutes a scientific analysis of the relative effects of health technology on efficacy, safety and effectiveness, commonly referred to as clinical outcomes, that is evaluated in relation to the comparative indicators currently deemed appropriate and chosen groups or subgroups of patients, taking into account the HTA Core Model criteria. It will include consideration of the degree of certainty on the relative outcomes, based on the available evidence . The outcome of such joint clinical assessments should not therefore affect the discretion of Member States in relation to subsequent decisions on pricing and reimbursement of health technologies, including the fixing of criteria for such pricing and reimbursement which may depend on both clinical and non-clinical considerations, and which remain solely a matter of national competence. The assessment conducted by each Member State as part of its national appraisal therefore falls outside the scope of this Regulation.

(12)

In order to ensure a wide application of harmonised rules on clinical aspects of HTA and enable pooling of expertise and resources across HTA bodies, it is appropriate to require joint clinical assessments to be carried out for all medicinal products undergoing the central marketing authorisation procedure provided for under Regulation (EC) No 726/2004 of the European Parliament and of the Council, (8) which incorporate a new active substance, and where those medicinal products are subsequently authorised for a new therapeutic indication. Joint clinical assessments should also be carried out on certain medical devices within the meaning of 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 have provided their opinions or views. A selection of medical devices for joint clinical assessment should be made based on specific criteria .

(12)

In order to ensure a wide application of harmonised rules and to foster collaboration among Member States on clinical aspects of HTA and enable pooling of expertise and resources across HTA bodies, thereby reducing waste and ineffectiveness in healthcare , it is appropriate to require joint clinical assessments to be carried out for all medicinal products undergoing the central marketing authorisation procedure provided for under Regulation (EC) No 726/2004 of the European Parliament and of the Council (10), which incorporate a new active substance, and where those medicinal products are subsequently authorised for a new therapeutic indication. Joint clinical assessments should also be carried out on certain medical devices within the meaning of Regulation (EU) 2017/745 of the European Parliament and of the Council (11), given the need for greater clinical evidence concerning all of those new health technologies .

(13)

In order to ensure that joint clinical assessments carried out on health technologies remain accurate and relevant, it is appropriate to establish conditions for the updating of assessments, in particular where additional data available subsequent to the initial assessment has the potential to increase the accuracy of the assessment.

(13)

In order to ensure that joint clinical assessments carried out on health technologies remain accurate, relevant, of high quality and based on the best scientific evidence available at any given time , it is appropriate to establish a flexible, regulated procedure for the updating of assessments, in particular when new evidence or additional data becomes available subsequent to the initial assessment and such new evidence or additional data may augment the scientific evidence and thus increase the quality of the assessment.

(14)

A coordination group composed of representatives from Member States' health technology assessment authorities and bodies should be established with responsibility for overseeing the carrying out of joint clinical assessments and other joint work.

(14)

A coordination group composed of representatives from Member States' health technology assessment authorities and bodies should be established with responsibility and proven expertise for overseeing the carrying out of joint clinical assessments and other joint work within the scope of this Regulation.

(15)

In order to ensure a Member-State led approach to joint clinical assessments and scientific consultations, Member States should designate national HTA authorities and bodies which inform decision-making as members of the Coordination Group. The designated authorities and bodies should ensure an appropriately high level of representation in the Coordination Group and technical expertise in its sub-groups, taking into account the need to provide expertise on the HTA of medicinal products and medical devices.

(15)

In order to ensure a Member-State led approach to joint clinical assessments and scientific consultations, Member States should designate national or regional HTA authorities and bodies which inform decision-making to conduct such assessments , as members of the Coordination Group. The designated authorities and bodies should ensure an appropriately high level of representation in the Coordination Group and technical expertise in its sub-groups, taking into account the possibility of providing expertise on the HTA of medicinal products and medical devices. The organisational structure should respect the distinctive mandates of the sub-groups conducting the joint clinical assessments and the joint scientific consultations. Any conflict of interest should be avoided.

(15a)

Transparency and public awareness of the process is essential. All clinical data being evaluated should have therefore the highest level of transparency and public awareness in order to gain confidence in the system. In case there is confidential data for commercial reasons, the confidentiality needs to be clearly defined and justified and the confidential data well delimitated and protected.

(16)

In order that the harmonised procedures fulfil their internal market objective, Member States should be required to take full account of the results of joint clinical assessments and not repeat those assessments . Compliance with this obligation does not prevent Member States from carrying out non-clinical assessments on the same health technology, or from drawing conclusions on the added value of the technologies concerned as part of national appraisal processes which may consider clinical as well as non-clinical data and criteria. It also does not prevent Member States from forming their own recommendations or decisions on pricing or reimbursement.

(16)

In order that the harmonised procedures fulfil their internal market objective and reach their aim of improving innovation and the quality of clinical evidence , Member States should take account of the results of joint clinical assessments and not repeat them . According to national needs, Member States should have the right to complement the joint clinical assessments with additional clinical evidence and analyses to account for differences in comparators or the national specific treatment setting. Such complementary clinical assessments should be duly justified and proportionate and should be notified to the Commission and the Coordination Group. In addition , compliance with this obligation does not prevent Member States from carrying out non-clinical assessments on the same health technology, or from drawing conclusions on the clinical added value of the technologies concerned as part of national appraisal processes which may consider clinical as well as the non-clinical data and criteria specific to the Member State concerned, at national and/or regional level . It also does not prevent Member States from forming their own recommendations or decisions on pricing or reimbursement.

(16a)

In order for the clinical assessment to be used for the purposes of the national reimbursement decision, it should ideally concern the population for which the drug would be reimbursed in a given Member State.

(17)

The time-frame for joint clinical assessments for medicinal products should, in as far as possible, be fixed by reference to the time-frame applicable to the completion of the central marketing authorisation procedure provided for under Regulation (EC) No 726/2004. Such coordination should ensure clinical assessments can effectively facilitate market access and contribute to the timely availability of innovative technologies for patients. As a rule, the process should be completed by the time of the publication of the Commission decision granting marketing authorisation.

(17a)

The joint scientific consultation, when addressing orphan medicinal products, has to ensure that any new approach should not result in unnecessary delays for the orphan medicinal products assessment compared to the current situation and taking into account the pragmatic approach undergone through the EUnetHTA.

(18)

The establishment of a time-frame for the joint clinical assessments for medical devices should take into account the highly decentralised market access pathway for medical devices and the availability of appropriate evidence data required to carry out a joint clinical assessment. As the required evidence may only become available after a medical device has been placed on the market and in order to allow for the selection of medical devices for joint clinical assessment at an appropriate time, it should be possible for assessments of such devices to take place following market launch of medical devices .

(18)

The establishment of a time-frame for the joint clinical assessments for health technologies should take into account the time-frames set out in Regulation (EC) No 726/2004 of the European Parliament and of the Council  (12) for completing the centralised procedure for authorising medicines and the CE conformity marking for medical devices provided for in Regulation (EU) 2017/745 of the European Parliament and of the Council  (*1) and the CE conformity marking for in vitro diagnostic medical devices provided for in Regulation (EU) 2017/746 of the European Parliament and of the Council  (*2). In any event, those assessments must take into account the availability of appropriate scientific evidence and supporting data in the quantity required to carry out a joint clinical assessment, and should take place in a time-frame as close as possible to their marketing authorisation, in the case of medicines, and, in any case, without unjustified and unnecessary delay.

(19)

In all cases the joint work carried out under this Regulation, in particular the joint clinical assessments, should produce high quality and timely results, and not delay or interfere with the CE marking of medical devices or market access of health technologies. This work should be separate and distinct from regulatory assessments of the safety, quality, efficacy or performance of health technologies carried out pursuant to other Union legislation and have no bearing on decisions taken in accordance with other Union legislation.

(19)

In any event the joint work carried out under this Regulation, in particular the joint clinical assessments, should produce high quality and timely results, without delaying or interfering with the CE marking of medical devices.

(19a)

HTA work covered under this Regulation should be separate and distinct from regulatory assessments of the safety and efficacy of health technologies carried out pursuant to other Union legislative acts and should have no bearing on other aspects falling outside the scope of this Regulation adopted in accordance with other Union legislative acts.

(19b)

In the case of orphan medicinal products, the joint report should not re-assess the criteria of the orphan designation. However, assessors and co-assessors should have full access to the data used by the authorities responsible for granting the marketing authorisation of a medicinal product, as well as the possibility of using or generating additional relevant data for the purpose of assessing a medicinal product in the context of a joint clinical assessment.

(19c)

Regulation (EU) 2017/745 concerning medical devices and Regulation (EU) 2017/746 concerning in vitro diagnostic medical devices provide for the authorisation of such devices on the basis of the principles of transparency and safety and not on efficacy. However, the gradual increase in the supply of medical devices to address clinical conditions has heralded a paradigm shift towards a new model in which the market is highly fragmented, innovation is chiefly incremental and clinical evidence is lacking, which means that closer cooperation and more frequent exchanges of information between assessment bodies are needed. It is therefore necessary to move towards a centralised authorisation system that assesses devices on the basis of safety, efficacy and quality. It is also one of the areas in which Member States are calling for greater collaboration via a future European HTA. Currently 20 Member States, together with Norway, have HTA systems for medical devices in place and 12 Member States, together with Norway, have established guidelines and are engaging in initial dialogues. EUnetHTA has been conducting high-quality evaluations of the relative efficacy of medical devices based on a methodology that can be taken as a benchmark for this Regulation.

(20)

In order to facilitate effective participation by health technology developers in joint clinical assessments, such developers should, in appropriate cases, be afforded an opportunity to engage in joint scientific consultations with the Coordination Group to obtain guidance on the evidence and data that is likely to be required for the purposes of clinical assessment . Given the preliminary nature of the consultation, any guidance offered should not bind either the health technology developers or HTA authorities and bodies.

(20)

Health technology developers can conduct joint scientific consultations with the Coordination Group or working groups set up for this purpose and composed of professionals from national or regional assessment bodies to obtain guidance on the clinical needs of research and the optimal design of studies to obtain the best possible evidence and maximise research efficiency . Given the preliminary nature of the consultation, any guidance offered should not bind either the health technology developers or HTA authorities and bodies.

(20a)

Joint scientific consultations should concern the clinical study design, the determination of best comparators based on the best medical practice in the interest of patients. The consultation process should be transparent.

(21)

Joint clinical assessments and joint scientific consultations necessitate the sharing of confidential information between health technology developers and HTA authorities and bodies. In order to ensure the protection of such information, information provided to the Coordination Group in the framework of assessments and consultations should only be disclosed to a third party after a confidentiality agreement has been concluded. In addition, it is necessary for any information made public about the results of joint scientific consultations to be presented in an anonymised format with the redaction of any information of a commercially sensitive nature.

(21)

Joint scientific consultations could necessitate the sharing of commercially confidential information between health technology developers and HTA authorities and bodies. In order to ensure the protection of such information, information provided to the Coordination Group in the framework of consultations should only be disclosed to a third party after a confidentiality agreement has been concluded. In addition, it is necessary for any information made public about the results of joint scientific consultations to be presented in an anonymised format with the redaction of any information of a commercially sensitive nature.

(21a)

Joint clinical assessments necessitate all available clinical data and publicly available scientific evidence from health technology developers. The clinical data employed, the studies, the methodology and the clinical results used should be made public. The highest possible level of public openness in scientific data and assessments will allow progress to be made in biomedical research and ensure the highest possible level of confidence in the system. Where commercially sensitive data is shared, the confidentiality of such data should be protected by presenting it in an anonymised format with the redaction of reports before publication, preserving the public interest.

(21b)

According to the European Ombudsman, where information in a document has implications for the health of individuals (such as information on the efficacy of a medicine), the public interest in disclosure of that information will generally defeat any claim of commercial sensitivity. Public health should always prevail over commercial interests.

(22)

In order to ensure the efficient use of available resources, it is appropriate to provide for "horizon scanning", to allow the early identification of emerging health technologies that are likely to have the most impact on patients, public health and healthcare systems. Such scanning should facilitate the prioritisation of technologies that are to be selected for joint clinical assessment.

(22)

In order to ensure the efficient use of available resources, it is appropriate to provide for "horizon scanning", to allow the early identification of emerging health technologies that are likely to have the most impact on patients, public health and healthcare systems, as well as to steer research strategically . Such scanning should facilitate the prioritisation of technologies that are to be selected by the Coordination Group for joint clinical assessment.

(23)

The Union should continue to support voluntary cooperation on HTA between Member States in areas such as in the development and implementation of vaccination programmes, and capacity building of national HTA systems. Such voluntary cooperation should also facilitate synergies with initiatives under the digital single market strategy in relevant digital and data-driven areas of health and care with a view to the provision of additional real world evidence relevant for HTA.

(23)

The Union should continue to support voluntary cooperation on HTA between Member States in other areas such as in the development and implementation of vaccination programmes, and capacity building of national HTA systems.

(24)

In order to ensure the inclusiveness and transparency of the joint work, the Coordination Group should engage and consult widely with interested parties and stakeholders. However , in order to preserve the integrity of the joint work, rules should be developed to ensure the independence and impartiality of the joint work and ensure that such consultation does not give rise to any conflicts of interest.

(24)

In order to preserve the objectivity, transparency and quality of the joint work, rules should be developed to ensure the independence, public openness and impartiality of the joint work and ensure that such consultation does not give rise to any conflicts of interest.

(24a)

Dialogue between the Coordination Group and patient organisations, consumer organisations, health non-governmental organisations, health experts and professionals should be ensured, especially through a stakeholder network, with a guarantee of the independence, transparency and impartiality of the decisions taken.

(24b)

In order to ensure efficient decision-making and facilitate access to medicines, an appropriated cooperation between decision-makers at key stages of the medicines’ life-cycle is important.

(25)

In order to ensure a uniform approach to the joint work provided for in this Regulation, implementing powers should be conferred on the Commission to establish a common procedural and methodological framework for clinical assessments, procedures for joint clinical assessments and procedures for joint scientific consultations. Where appropriate, distinct rules should be developed for medicinal products and medical devices. In the development of such rules, the Commission should take into account the results of the work already undertaken in the EUnetHTA Joint Actions. It should also take into account initiatives on HTA funded through the Horizon 2020 research programme, as well as regional initiatives on HTA such as the Beneluxa and Valletta Declaration initiatives. Those powers should be exercised in accordance with Regulation (EU) No 182/2011 of the European Parliament and of the Council. (13)

(25)

In order to ensure a uniform approach to the joint work provided for in this Regulation, the Coordination Group, composed of national and/or regional authorities and bodies responsible for health technology assessment, with proven capacity, independence and impartiality , should draw up the methodology for ensuring high quality of work as a whole. The Commission should endorse, by means of implementing acts, that methodology and a common procedural framework for joint clinical assessments and joint scientific consultations. Where appropriate, and in justified cases , distinct rules should be developed for medicinal products and medical devices. In the development of such rules, the results of the work already undertaken in the EUnetHTA Joint Actions, and in particular the methodological guidelines and evidence submission templates , initiatives on HTA funded through the Horizon 2020 research programme, as well as regional initiatives on HTA such as the Beneluxa and Valletta Declaration initiatives should be taken into account . Those powers should be exercised in accordance with Regulation (EU) No 182/2011 of the European Parliament and of the Council (14).

(25a)

The methodological framework, in accordance with the Declaration of Helsinki, should guarantee high quality and high clinical evidence by choosing the most appropriate benchmarks. It should be based on high standards of quality, the best available scientific evidence, stemming primarily from double-blind randomised clinical trials, meta-analysis and systematic reviews; and should take into account clinical criteria that are useful, relevant, tangible, concrete and tailored to suit the given clinical situation, with preference given to end points. The documentation to be provided by the applicant should relate to the most up-to-date and public data.

(25b)

Any specificities in the methodology, such as for vaccines, should be justified and adapted to very specific circumstances, should have the same scientific rigour and the same scientific standards, and should never be to the detriment of the quality of health technologies or clinical evidence.

(25c)

The Commission should provide administrative support for the joint work of the Coordination Group, which, after consultation with the stakeholders, should submit the final report on this work.

(26)

In order to ensure that this Regulation is fully operational and to adapt it to technical and scientific development, the power to adopt acts in accordance with Article 290 of the Treaty on the Functioning of the European Union should be delegated to the Commission in respect of the contents of documents to be submitted, reports, and summary reports of clinical assessments, the contents of documents for requests, and reports of joint scientific consultations, and the rules for selecting stakeholders. It is of particular importance that the Commission carries out appropriate consultations during its preparatory work, including at expert level, and that those consultations be conducted in accordance with the principles laid down in the Interinstitutional Agreement on Better Law-Making of 13 April 2016.  (15) In particular, to ensure equal participation in the preparation of delegated acts, the European Parliament and the Council should receive all documents at the same time as Member States' experts, and their experts systematically should be granted access to meetings of Commission expert groups dealing with the preparation of delegated acts.

(26)

The Commission should adopt implementing acts on procedural rules for the joint clinical assessments, joint scientific consultations, and for selecting stakeholders.

(27)

In order to ensure that sufficient resources are available for the joint work provided for under this Regulation, the Union should provide funding for the joint work and voluntary cooperation, and for the support framework to support these activities. The funding should cover the costs of producing joint clinical assessment and joint scientific consultation reports . Member States should also have the possibility to second national experts to the Commission in order to support the secretariat of the Coordination Group.

(27)

In order to ensure that sufficient resources are available for the joint work and stable administrative support provided for under this Regulation, the Union should ensure stable and permanent public funding under the Multiannual Financial Framework for the joint work and voluntary cooperation, as well as for the support framework to support these activities. Member States should also have the possibility to second national experts to the Commission in order to support the secretariat of the Coordination Group. The Commission should establish a system of charges for health technology developers requesting both joint scientific consultations and joint clinical assessments for research on unmet medical needs. Under no event can those fees be used to fund the joint work provided for in this Regulation.

(28)

In order to facilitate the joint work and the exchange of information between Member States on HTA, provision should be made for the establishment of an IT platform that contains appropriate databases and secure channels for communication. The Commission should also ensure a link between the IT platform and other data infrastructures relevant for the purposes of HTA such as registries of real world data.

(28)

In order to facilitate the joint work and the exchange of information between Member States on HTA, provision should be made for the establishment of an IT platform that contains appropriate databases and secure channels for communication, as well as all information on the procedure, methodology, training and interests of assessors of and participants in the stakeholder network, and the reports and results of the joint work, which should be made public . The Commission should also ensure a link between the IT platform and other data infrastructures relevant for the purposes of HTA such as registries of real world data.

(28a)

Cooperation should be based on the principle of good governance, which encompasses transparency, objectivity, independent experience and fair procedures. Trust is a precondition for successful cooperation and can only be achieved if all stakeholders make genuine commitments and if there is access to high-quality experience, capacity-building and the highest quality of execution.

(28b)

Since there is currently no commonly agreed definition of what constitutes high-quality innovation or added therapeutic value, the Union should adopt definitions of these terms with the agreement or consensus of all parties.

(30)

During the transitional period, participation in joint clinical assessments and joint scientific consultations should not be mandatory for Member States. This should not affect the obligation of Member States to apply harmonised rules to clinical assessments carried out at a national level. During the transitional period, Member States not participating in the joint work may at any time decide to participate. In order to ensure a stable and smooth organisation of the joint work and the functioning of the internal market, Members States which are already participating should not be allowed to withdraw from the framework for joint work.

(30)

During the transitional period, participation in joint clinical assessments and joint scientific consultations should not be mandatory for Member States. Moreover , during the transitional period, Member States not participating in the joint work may at any time decide to participate. In order to ensure a stable and smooth organisation of the joint work and the functioning of the internal market, Members States which are already participating should not be allowed to withdraw from the framework for joint work. Clinical assessments which have started in Member States before the application of this Regulation should be continued, unless Member States decide to stop them.

(31)

In order to ensure that the support framework continues to be as efficient and cost-effective as possible , the Commission should report on the implementation of the provisions on the scope of the joint clinical assessments and on the functioning of the support framework no later than two years after the end of the transitional period. The report may in particular consider whether there is a need to move this support framework to a Union agency and introduce a fee-paying mechanism through which health technology developers would also contribute to the financing of the joint work.

(31)

After the transitional period and before the harmonised system for HTA established under this Regulation becomes mandatory , the Commission should submit an impact assessment report on the whole of the procedure that has been introduced. That impact assessment report should evaluate, among other criteria, the progress made in relation to patients access to new health technologies and the functioning of the internal market, the impact on the quality of innovation and on the sustainability of health systems, as well as the appropriateness of the scope of the joint clinical assessments and the functioning of the support framework.

(32)

The Commission should carry out an evaluation of this Regulation. Pursuant to paragraph 22 of the Interinstitutional Agreement on Better Law-Making of 13 April 2016, that evaluation should be based on the five criteria of efficiency, effectiveness, relevance, coherence and EU added value and should be supported by a monitoring programme.

(32)

The Commission should carry out an evaluation of this Regulation. Pursuant to paragraph 22 of the Interinstitutional Agreement on Better Law-Making of 13 April 2016, that evaluation should be based on the five criteria of efficiency, effectiveness, relevance, coherence and EU added value and should be supported by a monitoring programme. The results of that evaluation should also be communicated to the European Parliament and Council.

(34)

Since the objectives of this Regulation, namely to approximate the rules of the Member States on carrying out clinical assessments at national level and establish a framework of mandatory joint clinical assessments of certain health technologies at Union level , cannot be sufficiently achieved by the Member States but can rather, by reason of their scale and effects, be better achieved at Union-level, the Union may adopt measures, in accordance with the principle of subsidiarity as set out in Article 5 of the Treaty on the European Union. In accordance with the principle of proportionality, as set out in that Article, this Regulation does not go beyond what is necessary in order to achieve that objective,

(34)

Since the objectives of this Regulation, namely to approximate the rules of the Member States on carrying out clinical assessments of the health technologies falling under the scope of this Regulation , cannot be sufficiently achieved by the Member States alone but can rather, by reason of their scale and effects, be better achieved at Union-level, the Union may adopt measures, in accordance with the principle of subsidiarity as set out in Article 5 of the Treaty on the European Union. In accordance with the principle of proportionality, as set out in that Article, this Regulation does not go beyond what is necessary in order to achieve that objective,

(a)

a support framework and procedures for cooperation on health technology assessment at Union level;

(a)

a support framework and procedures for cooperation on the clinical assessment of health technology at Union level;

(b)

common rules for the clinical assessment of health technologies.

(b)

common methodologies for the clinical assessment of health technologies.

(ba)

‘in vitro diagnostic medical device’ means an in vitro diagnostic medical device as defined in Regulation (EU) 2017/746;

(bb)

‘assessment of a medical device’ means the assessment of a method composed of more than one medical device or a method composed of a medical device and a defined care chain of other treatments;

(e)

‘clinical assessment’ means a compilation and evaluation of the available scientific evidence on a health technology in comparison with one or more other health technologies based on the following clinical domains of health technology assessment : the description of the health problem addressed by the health technology and the current use of other health technologies addressing that health problem, the description and technical characterisation of the health technology, the relative clinical effectiveness, and the relative safety of the health technology;

(e)

‘ joint clinical assessment’ means the systematic collection of scientific information and its comparative evaluation and a synthesis of these procedures, the comparison of the health technology in question with one or more other health technologies or existing procedures, constituting a benchmark for a particular clinical indication and , based on the best available clinical scientific evidence and on patient relevant clinical criteria, taking into account the following clinical domains: the description of the health problem addressed by the health technology and the current use of other health technologies or procedures addressing that health problem, the description and technical characterisation of the health technology, the relative clinical effectiveness, and the relative safety of the health technology;

(ga)

‘appraisal’ means drawing conclusions on the added value of the technologies concerned as part of national appraisal processes which may consider clinical as well as non-clinical data and criteria in the national care context.

(gb)

‘patient-relevant health outcomes’ means data that captures or predicts mortality, morbidity, health-related quality of life and adverse events.

(c)

ensure cooperation with relevant Union level bodies to facilitate additional evidence generation necessary for its work;

(c)

cooperate with relevant Union-level bodies to facilitate additional evidence generation necessary for its work;

(d)

ensure appropriate involvement of stakeholders in its work;

(d)

ensure appropriate consultation of relevant stakeholders and experts when pursuing its work. Such consultations shall be documented, including publicly available declarations of interest from the stakeholders consulted and shall be incorporated in the final joint assessment report ;

(c)

consult the Commission on the draft annual work programme and take into account its opinion .

(c)

consult the Commission and the stakeholder network, at annual meetings under Article 26 , on the draft annual work programme and take into account their comments .

(aa)

other medicinal products not subject to the authorisation procedure provided for in Regulation (EC) No 726/2004 where the health technology developer has opted for the centralised authorisation procedure, provided that the medicinal products in question constitute a major technical, scientific or therapeutic innovation, or their authorisation is in the interest of public health;

(b)

medical devices classified as class IIb and III pursuant to Article 51 of Regulation (EU) 2017/745 for which the relevant expert panels have provided a scientific opinion in the framework of the clinical evaluation consultation procedure pursuant to Article 54 of that Regulation;

(b)

medical devices classified as class IIb and III pursuant to Article 51 of Regulation (EU) 2017/745 for which the relevant expert panels have provided a scientific opinion in the framework of the clinical evaluation consultation procedure pursuant to Article 54 of that Regulation and considered to be a significant innovation and with potential significant impact on public health or health care systems ;

(c)

in vitro diagnostic medical devices classified as class D pursuant to Article 47 of Regulation (EU) 2017/746 (16) for which the relevant expert panels have provided their views in the framework of the procedure pursuant to Article 48(6) of that Regulation.

(c)

in vitro diagnostic medical devices classified as class D pursuant to Article 47 of Regulation (EU) 2017/746 (17) for which the relevant expert panels have provided their views in the framework of the procedure pursuant to Article 48(6) of that Regulation and considered to be a significant innovation and with potential significant impact on public health or health care systems .

(ea)

the need for greater clinical evidence;

(eb)

at the request of the health technology developer;

(a)

an analysis of the relative effects of the health technology being assessed on the patient-relevant health outcomes chosen for the assessment;

(a)

an analysis of the relative effectiveness and safety of the health technology being assessed in terms of the clinical end-points relevant to the clinical entity and patient group chosen for the assessment, including mortality, morbidity and quality of life, and compared to one or more comparator treatments to be determined by the Coordination Group ;

(b)

the degree of certainty on the relative effects based on the available evidence.

(b)

the degree of certainty on the relative effects based on the best available clinical evidence and compared to the best standard therapies . The assessment shall be based on the clinical end-points established in accordance with international standards of evidence-based medicine, in particular with regard to improving the state of health, shortening the duration of the disease, prolonging survival, reducing side effects or improving the quality of life. Reference shall also be made to subgroup-specific differences.

(a)

not carry out a clinical assessment or an equivalent assessment process on a health technology included in the List of Assessed Health Technologies or for which a joint clinical assessment has been initiated ;

(a)

use the joint clinical assessment reports in their health technology assessments at Member State level ;

(b)

apply joint clinical assessment reports, in their health technology assessments at Member State level.

(b)

not duplicate the joint clinical assessment at Member State level.

(b)

the initial joint clinical assessment report specified the need for an update once additional evidence for further assessment is available.

(b)

the initial joint clinical assessment report specified the need for an update once additional evidence for further assessment is available within the deadline set in that report ;

(ba)

at the request of a Member State or a health technology developer that considers that there is new clinical evidence;

(bb)

five years after the assessment, significant new clinical evidence exist, or earlier when new evidence or clinical data emerges.

(a)

submissions of information, data and evidence by health technology developers;

(c)

determining the detailed procedural steps and their timing, and the overall duration of joint clinical assessments ;

(c)

determining the detailed procedural steps and their timing;

(f)

cooperation with the notified bodies and expert panels on the preparation and update of joint clinical assessments of medical devices.

(f)

cooperation with the bodies and expert panels.

(fa)

Union clinical research priorities ;

(a)

submissions of requests from health technology developers and their involvement in the preparation of joint scientific consultation reports ;

(a)

submissions of requests from health technology developers;

(d)

the consultation of patients, clinical experts and other relevant stakeholders;

(d)

the submission of comments by patients, health professionals, patient associations, social partners, non-governmental organisations , clinical experts and other relevant stakeholders;

(a)

the contents of :

(a)

the procedure for :

(iiia)

stakeholder involvement for the purpose of this section, including rules on conflict of interest. Declarations of interest shall be made publicly available for all stakeholders and experts consulted. Stakeholders and experts with a conflict of interest shall not participate in the process.

(b)

the rules for determining the stakeholders to be consulted for the purpose of this Section.

(b)

patient organisations;

(b)

patient and consumer organisations and health professionals at its annual meeting ;

(da)

clinical assessments of medicinal products and medical devices carried out by Member States;

(db)

measures relating to compassionate use in clinical practice in order to improve the evidence basis and to create a register for this purpose;

(dc)

the development of best medical practice guides based on scientific evidence;

(dd)

disinvestment in obsolete technologies;

(de)

the tightening of the rules on clinical evidence generation and its monitoring.

(b)

clinical assessments of medicinal products and medical devices carried out by Member States.

(i)

ensuring that health technology authorities and bodies carry out clinical assessments in an independent and transparent manner, free from conflicts of interest;

(i)

ensuring that the members of the Coordination Group carry out clinical assessments in an independent and transparent manner, free from conflicts of interest, in accordance with Article 3(6) and (7) ;

(ii)

the mechanisms for the interaction between health technology bodies and health technology developers during clinical assessments;

(ii)

the mechanisms for the interaction between health technology bodies and health technology developers during clinical assessments, subject to the provisions of the previous articles ;

(iii)

the consultation of patients, clinical experts, and other stakeholders in clinical assessments.

(iii)

comments of patients, health professionals, consumer organisations , clinical experts, and other stakeholders in clinical assessments and the duly justified replies, subject to the provisions of the previous articles ;

(iiia)

addressing potential conflicts of interest;

(iiib)

ensuring that the assessment of medical devices is able to take place at the appropriate point in time after market launch, allowing for the use of clinical effectiveness data, including real world data. The appropriate time point shall be identified in cooperation with relevant stakeholders.