EUROPEAN COMMISSION Brussels, 12.5.2022 SWD(2022) 200 final COMMISSION STAFF WORKING DOCUMENT Accompanying the document REPORT FROM THE COMMISSION TO THE EUROPEAN PARLIAMENT AND THE COUNCIL on the operation of Directive 2011/24/EU on the application of patients' rights in cross-border healthcare {COM(2022) 210 final}



Table of contents 1.Introduction 2.What was the expected outcome of the intervention? Description of the intervention and its objectives The Directive’s Intervention Logic Legal Framework Points of comparison 3.How has the situation evolved over the evaluation period? Implementation in the Member States Operation of the Directive 2015-2020 4.Evaluation findings 4.1.To what extent was the intervention successful and why? Effectiveness 4.1.1. How effective was the Directive in the area of patients’ rights? 4.1.2. How effective was the Directive to encourage cross-border cooperation in the EU? Efficiency Coherence 4.2.How did the EU intervention make a difference?

4.3.Is the intervention still relevant? 5.What are the conclusions and lessons learned? Annex I. Procedural information Annex II. Methodology and analytical models used Annex III. Evaluation matrix and, where relevant, details on answers to the evaluation questions (by criterion) Annex IV. Overview of benefits and costs Annex V. Stakeholders consultation – synopsis report Annex VI. European Reference Networks Annex VII. Information on healthcare under the CBHC Directive and the Regulations on the Coordination of Social Security Systems



Glossary Term or acronym Meaning or definition Competent Member State Member State under whose social security system the patient concerned is insured at the time of the cross-border treatment. Cross-border healthcare Cross-border healthcare refers to medical treatment outside the patient’s country of residence, whether or not insured under the social security legislation of another Member State. The treatment is considered to be cross-border when received in any EU/EEA Member State or (but only in case of the application of the Regulations) in Switzerland and the UK. CBHC Directive Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare. European Economic Area (EEA) The European Economic Area includes the Member States of the European Union and three countries of the European Free Trade Association: Iceland, Liechtenstein and Norway. European Health Insurance Card (EHIC) Free card, issued by the national health insurance institution that allows the patient to receive medically necessary, state-provided healthcare during a temporary stay in another EU/EEA country, Switzerland or the UK, under the same conditions and at the same cost (free of charge in some countries) as the persons insured in that country. European Reference Networks (ERNs) European Reference Networks are virtual networks involving healthcare providers across Europe, aiming to facilitate discussion on complex or rare diseases and conditions that require highly specialised treatment, and concentrated knowledge and resources (https://ec.europa.eu/health/european-reference-networks/overview_en) Follow-up care Healthcare that may be required in the home country as a result of cross-border treatment or medical intervention with the purpose of providing aftercare or surveillance to ensure a good recovery. Home country The country where the patients reside and are entitled to sickness benefits, regardless of whether or not they are insured under the social security system of that country. Insured person Person or family member of a person who is subject or has been subject to the social security legislation of an EU/EEA Member State or (but only in case of the application of the Regulations) of Switzerland or the UK. Member State of affiliation Member State which under the Regulations is competent to grant to the insured person a prior authorisation to receive appropriate treatment outside the Member State of residence and issue the S2 form. This will normally be the country under whose social security system the patient is insured. Member State of treatment Member State on whose territory the cross-border treatment is actually provided or in the case of telemedicine the Member State where the healthcare provider is established. National Contact Points (NCPs) Under Directive 2011/24/EU, all EU/EEA Member States are obliged to designate one or more National Contact Points which provide patients with information on all aspects of cross-border healthcare. Planned healthcare Healthcare provided during a temporary stay abroad of which the explicit purpose was to receive it there. Prior authorisation Authorisation that patients need to receive from their national health insurance institution/health insurer in advance of their travel abroad in order to be guaranteed reimbursement for cross-border healthcare. Regulations on the coordination of social security systems Regulation (EC) No 883/2004 of the European Parliament and of the Council of 29 April 2004 on the coordination of social security systems and Regulation (EC) No 987/2009 of the European Parliament and of the Council of 16 September 2009 laying down the procedure for implementing Regulation (EC) No 883/2004 on the coordination of social security systems. Reimbursement Repayment to the patient by the national health insurance institution/health insurer, on certain conditions, of the costs incurred for cross-border healthcare services. Necessary (unplanned) healthcare Healthcare received by a patient in an EU/EEA country, Switzerland or the UK, which becomes necessary on medical grounds during a temporary stay in that State for work, study or leisure (i.e. without the initial purpose of the patient's travel being to receive treatment there) and that, taking into account the length of the stay, cannot wait until the patient returns home. This includes treatments provided in conjunction with chronic or existing illnesses. See explanatory note from the European Commission: https://ec.europa.eu/social/BlobServlet?docId=6481&langId=en Acronyms and abbreviations AEBR Association of European Border Regions ANEC European consumer voice in standardisation CBA Cost-Benefit Analysis CBHC Cross-Border Healthcare CPMS Clinical Patient Management System DG Directorate-General DG SANTE Directorate-General for Health & Food Safety ECA European Court of Auditors EDF European Disability Forum EEA European Economic Area EFTA European Free Trade Association EHDS European Health Data Space EHIC European Health Insurance Card ePAGs European Patient Advocacy Groups EPF European Patients’ Forum EPHA European Public Health Alliance EQ Evaluation Question EQM Evaluation Questions Matrix ERICA European Rare disease research Coordination and support Action ERN European Reference Network EU European Union EUR Euro EXPH Expert Panel on Effective Ways of Investing in Health IT Information Technology JC Judgement Criteria / Criterion LGBTIQ Lesbian, gay, bisexual, transgender, intersex, and queer/questioning MS Member State(s) NCP National Contact Point OECD Organisation for Economic Co-operation and Development PGEU Pharmaceutical Group of the European Union SWD Staff Working Document TFEU Treaty on the Functioning of the European Union ToR Terms of Reference ZOAST Zones Organisées d’Accès aux Soins Transfrontaliers 1.Introduction Access to high quality health services is a priority for EU citizens. Directive 2011/24/EU on the application on patients’ rights on cross-border healthcare (the CBHC Directive or the Directive) aims to ensure patients with safe and high quality healthcare services in a Member State other than the Member State of affiliation. It also provides a legal framework for reinforcing voluntary cooperation between EU countries in healthcare. While the Regulations on the coordination of social security systems have enshrined the right to receive healthcare in another Member State for various categories of insured persons since the beginning of the European integration, the Directive is the first piece of EU legislation in the area of health services. At the same time, the organization and financing of health systems remain the responsibility of the Member States. Thanks to the application of the Directive, around 300,000 patients a year take advantage of the systems put in place under this Directive to receive (partial) reimbursement for the healthcare costs incurred in another EU country. The first 24 European Reference Networks (ERNs) bring together highly specialised medical experts from 900 healthcare units located in 313 hospitals offering their expert advice on the diagnosis and treatment of thousands of patients with rare or low prevalence complex diseases. The Commission encourages cross-border cooperation in healthcare, particularly in border regions, where EU funding has supported an estimated 400 cross-border initiatives to cooperate in healthcare. European cooperation in health technology assessment and digital health has evolved significantly to meet new needs in healthcare across the EU.

The purpose of this evaluation is to assess how well the CBHC Directive has performed, a decade after its adoption in 2011 and eight years since its transposition deadline in 2013. Its objective is to check whether the Directive is still relevant and “fit for purpose” to meet current and future patient needs. It also identifies where the Directive was not applied effectively and draws conclusions based on the evidence gathered for future policy decisions on the Directive. This report assesses two key parts of the CBHC Directive using the evaluation criteria of effectiveness, efficiency, relevance, coherence and its EU added value. First, the CBHC Directive’s provisions (Articles 1-9) covering the responsibilities of Member States with regard to cross-border healthcare, including the National Contact Points (NCPs). Secondly, this evaluation covers the provisions governing the recognition of prescriptions issued in another EU country (Article 11 and Implementing Directive 2012/52/EU), the mutual assistance and European cooperation between regions (Article 10) and, in the area of rare and low prevalence complex diseases, the setting up of the ERNs (Article 12 including the Commission Delegated and Implementing Decisions). The evaluation covers the CBHC Directive’s application in the EU-27 and EEA EFTA States Norway, Iceland, Liechtenstein from the period 2015, the time by which most EU countries had transposed the Directive into national law, until the end of 2020. The CBHC Directive’s provisions on eHealth (Article 14) have been evaluated separately as part of the work on the Commission’s proposal for a Regulation on the European Health Data Space (EHDS). Following the adoption and entry into force of the Regulation on cooperation in health technology assessment in 2021, the CBHC Directive’s relevant provision (Article 15) was repealed and therefore not part of this evaluation. This evaluation largely draws on the evaluation study conducted by an external contractor, the replies received to the Commission’s public consultation, which ran from May to July 2021, as well as several events organised by the Commission to gather feedback on the CBHC Directive. An important limitation was that little research has been conducted on the Directive and that there is insufficient comparative research across countries on the impact of the Directive. The engagement of some stakeholders was also low: the researchers found it difficult to contact and engage the stakeholders during the course of the Covid-19 pandemic. In addition, there was evidence of stakeholder fatigue, as there were many concurrent activities in the area of healthcare. As not all sources of evidence are equally robust, consideration was given as to when and how the evidence was collected and whether there was any bias or uncertainty in it. The contractors used triangulation of data from the different data collection as a method to arrive at robust and evidence-based results that could be confirmed by more than one source. More information on the methodology and process to carry out this evaluation is available in Annex II and can be found on the Commission’s dedicated webpage for the evaluation of the Directive. This evaluation is part of the Commission’s regulatory fitness and performance programme (REFIT). It refers to the opinion of the Fit for Future platform adopted on 10 December 2021. 2.What was the expected outcome of the intervention? Description of the intervention and its objectives EU legislation to facilitate cross-border healthcare in the European labour market has been in place since 1958. Currently, Regulation No 883/2004 on the coordination of social security systemsensures the reimbursement for healthcare that becomes necessary on medical grounds during an insured person’s temporary stay in another EU country using the European health insurance card (EHIC). It also covers planned healthcare subject to prior approval by the EU country where the citizen is insured. In 1998, the Court of Justice of the EU (the Court of Justice) clarified principles for cross-border healthcare reimbursement through its rulings in two cases. Over the next decade, there were further Court rulings on the principles on the freedom to provide health services based on Article 56 of the TFEU. The case law has made clear that the TFEU provisions on free movement apply to the reimbursement of health services regardless of how they are organised and financed at national level. To provide legal certainty of the 10-year case law on the freedom to provide health services in the EU and to give effective application of the citizens’ rights in the EU internal market, the Commission proposed a Directive on the application on patients’ rights in cross-border healthcare in 2008. The CBHC Directive was adopted on 9 March 2011 and entered into force on 24 April 2011. It was due to be transposed by the Member States by 25 October 2013. It is important to bear in mind that the CBHC Directive came into being at a time of economic recession with huge impacts on healthcare budgets in the EU. The CBHC Directive’s main aim was therefore to provide sufficient legal certainty for citizens wishing to use cross-border healthcare. It does this by setting out patients’ rights, including the rules on reimbursement for cross-border healthcare costs in accordance with the Court of Justice’s rulings, to ensure that EU citizens could use those rights in practice. The legal provisions put in place complemented the existing legal framework for cross-border healthcare provided by Regulation (EC) No 883/2004 by removing certain obstacles to cross-border healthcare and by aiming to give citizens in certain instances the right of choice to healthcare services in the internal market. While both these EU instruments are about cross-border healthcare, their approaches differ: the Directive centres around patients’ rights and encouraging cross-border cooperation in healthcare whereas the focus of the Regulation is coordinating social security systems. The Directive was not intended to replace the rights to cross-border healthcare under the Regulations nor to deprive patients of the more beneficial rights guaranteed therein when the conditions are met. The second objective of the CBHC Directive was to make sure that the necessary requirements for high quality and safe cross-border healthcare were respected throughout the EU. The Commission’s impact assessment accompanying the legal proposal recognised that while citizens prefer healthcare to be available close to where they live and work, there are certain situations where the citizen may wish to be treated abroad. This is the case for highly specialised care requiring resources or expertise that is not available in every EU country such as for rare diseases, as well as where the nearest appropriate healthcare provider may be across the border or where the local services are unable to provide the appropriate healthcare yet there is capacity in another EU country. Some citizens may wish to be treated abroad to be close to their family, who may live in another EU country, or to seek a better quality or a cheaper procedure in another Member State. At the same time, patient flows between Member States were expected to remain limited. The CBHC Directive also intended to promote EU cooperation between health systems in several areas where economies of scale of coordinated action between Member States could bring significant benefits to national health systems. EU countries were encouraged to provide mutual assistance and to cooperate in border regions to improve access to healthcare for patients and using resources more efficiently. In the area of rare and low prevalence complex diseases, the aim of the ERNs was to provide healthcare to patients who have conditions requiring a particular concentration of resources and to act as focal points for medical training and research, information dissemination and evaluation. A European network exchanging information to assess health technology could provide important evidence-based strategic advice to policy-makers and, lastly, in the area of eHealth, where different and incompatible formats and standards hampered the provision of eHealth services, a network of national authorities could work together to improve the interoperability of systems. The Directive’s Intervention Logic The Commission’s impact assessment accompanying the legal proposal did not develop an intervention logic model to describe how the CBHC Directive was expected to work at the outset. The Commission, with the support of an external contractor, therefore developed an intervention model retrospectively by analysing available document sources. This analytical work shows the different steps involved in the implementation of the Directive and highlights the expected cause and effect relationship regarding patient rights and rare diseases. The intervention logic in Figure 1 excludes health technology and eHealth for the purposes of this evaluation.





Figure 1: Intervention Logic of Articles 1-13 of the Directive

Source: Study on enhancing implementation of the Cross-Border Healthcare Directive 2011/24/EU to ensure patient rights in the EU Legal Framework The CBHC Directive’s framework for ensuring patients’ rights in cross-border healthcare applies to all health services provided by health professionals to patients, excluding organ transplants, public vaccination programmes and long-term care the purpose of which is to support people in need of assistance in carrying out routine, everyday tasks. It ensures that all citizens who are insured in a Member State are eligible to receive treatment in another Member State and be reimbursed for it from the social security system of their own Member State. It sets out the responsibilities of the Member State of treatment and the Member State of affiliation. It requires Member States to have complaint procedures in place and sets out the rights of patients, such as the right to information and the right to follow-up care back home. It establishes the NCPs for cross-border healthcare to provide information to patients and it covers the administrative procedures for patients seeking healthcare in another EU country. Moreover, the Directive encourages voluntary cooperation on healthcare between Member States. Its provisions ensure the recognition of prescriptions for medicinal products and medical devices issued in other countries under the conditions established therein. It puts in place the legal framework for the creation of the ERNs in the area of rare and low prevalence complex diseases and promotes cooperation in the areas of health technology assessment and eHealth. It also requires Member States to provide mutual assistance as is necessary for the implementation of the Directive and encourages cooperation in the provision of cross-border healthcare at regional and local level, particularly in neighbouring countries and in border regions. The CBHC Directive co-exists with the Regulations on the coordination of social security systems. Points of comparison This evaluation uses the Commission’s first evaluative study on the Directive of 2015 as the point of reference as most EU countries had transposed the Directive’s provisions by then. Also since 2015, Member States provided the Commission with data to monitor the Directive’s application in the EU. 3.How has the situation evolved over the evaluation period? Implementation in the Member States The CBHC Directive came into force on 24 April 2011 and was due to be transposed into national law in the Member States by 25 October 2013. However, transposition was not completed until 2015 following infringements proceedings against 26 Member States (and 21 Member States in the case of Implementing Directive 2012/52/EU). More than five hundred national measures transposing the Directive were notified to the Commission. The large number of national laws involved was partly due to the fact the Directive regulated a number of issues which fell under regional competences and were subject matter of separate pieces of national legislation. The Commission’s conformity checks focused on the application of the Directive in four key areas with the greatest potential to act as barriers to patients seeking cross-border healthcare: the systems of reimbursement, the use of prior authorisation, the administrative requirements and charging incoming patients. Infringement proceedings started against three Member States – Finland, Austria and the Netherlands – for a breach of the Directive in relation to the reimbursement of cross-border healthcare costs. The Directive’s provisions have been subject to four preliminary rulings of the Court of Justice that further clarified its rules on reimbursement of cross-border healthcare costs, also in light of the TFEU and the Charter of Fundamental Rights of the EU, as well as on recognition of prescriptions issued in other Member States. Operation of the Directive 2015-2020 The Commission triennial reports describe how the operation of the CBHC Directive has progressed since 2015. The Commission reported in 2015 and 2018 on cross-border patient flows, the financial dimension of patient mobility, the limitations on the application of the rules for reimbursement and the application of prior authorisation and the functioning of the ERNs and the NCPs. The Commission’s third report, due by October 2021, was postponed until April 2022 so as to include the key findings of the Commission’s evaluation of the Directive as described in this staff working document. In summary, the Commission reports conclude that the Directive has improved the legal certainty for cross-border patients as well for domestic patients over their rights, however there remain important shortcomings in the application of the Directive in the EU as described in the evaluation findings in Section 4 below. In terms of patient flows, in the countries that provided data for all reference years 2016-2020, the number of prior authorisation requests received and granted peaked in 2018 followed by a modest decline in 2019 and a larger decrease in 2020 (in view of Covid-19). Similarly, in countries for which there is complete data, the number of requests for reimbursement received and granted saw a steady increase until 2018 and a slight decline in 2019 and 2020. The total numbers of prior authorisation and reimbursement requests received by all the countries are illustrated in Figures 2 and 3. Figure 2: Number of prior authorisation requests received and granted for all countries that provided data, 2016-2020 The totals excluding the UK are as follows: in reference year 2016: 6,009 prior authorisation (PA) requests received and 3,822 granted; in 2017: 5,471 PA requests received and 3,727 granted; in 2018: 6,301 received, 4,447 granted; in 2019: 5,352 received, 3,291 granted; and in 2020: 5,218 received, 3,542 granted.

Source: Questionnaires on Directive 2011/24/EU reporting on patient mobility Figure 3: Number of requests for reimbursement without prior authorisation received and granted for all countries that provided data, 2016-2020 The totals excluding the UK are as follows: in reference year 2016: 238,680 reimbursement requests received and 197,152 granted; in 2017: 233,508 reimbursement requests received and 193,803 granted; in 2018: 269,006 received, 225,186 granted; in 2019: 280,594 received, 236,891 granted; and in 2020: 188,013 received and 153,960 granted. Source: Questionnaires on Directive 2011/24/EU reporting on patient mobility The most prominent patient flows have remained stable over the years and the majority take place between neighbouring countries and North European citizens using healthcare during short-term stays in the South. In 2019, the largest flows were from Ireland to the United Kingdom, France to Germany, France to Spain and from Norway and Sweden to Spain. In addition, the Commission reports that the period 2018-2020 saw significant progress to enhance cross-border cooperation in healthcare with the adoption of EU implementing and delegated acts, paving the way for the creation of the ERNs in 2017 followed in 2021 by the adoption of a new EU Regulation on health technology assessment with the subsequent repeal of Article 15 from the Directive. In 2017, the ERNs were established on the basis of Article 12 of the Directive, Commission Implementing Decision 2014/287/EU and Commission Delegated Decision 2014/286/EU. Currently there are 24 ERNs on rare or low prevalence complex diseases (see Annex VI). By 2021, the ERNs were bringing together more than 900 highly-specialised healthcare units from over 300 hospitals in 26 EU countries and Norway. As of January 2022, the existing ERNs have been joined by 620 new members, bringing the total number of members to almost 1,500 and covering all the EU Member States and Norway. The members of the existing ERNs currently treat more than 1.7 million patients with rare or low prevalence complex diseases. The Commission launched a continuous monitoring system of the ERNs based on the set of 18 agreed key performance indicators in 2020. The analysis of the collected data is on-going. Other EU institutions and bodies have drawn up reports on the implementation of the CBHC Directive. In 2018/2019, the European Court of Auditors (ECA) carried out an audit on the Commission’s work overseeing the Directive’s implementation and EU actions in cross-border healthcare. Its special report concluded that, while EU actions had enhanced cooperation between Member States, the impact on patients was rather limited. The ECA recommends improving information provided by the NCPs to patients and calls for more EU actions on eHealth and rare diseases. Both the Council and the European Parliament supported the ECA’s recommendations. The European Parliament called for simpler administrative procedures and improving access to healthcare to patients. In 2020, the European Committee of the Regions recommended more support for the authorities to comply with the Directive’s obligations and recognised the value of prior notification of reimbursement costs and the use of prior authorisation to protect national health systems. The Fit for Future Platform Opinion recognizes the same obstacles to the Directive and makes eight concrete suggestions how to make it easier for European patients to understand their care options and access the best possible treatment and care. The CBHC Directive has had an unexpected positive impact on domestic healthcare systems according to evidence from the literature review. It has acted as a driver for the development and greater transparency of patients’ rights as well as leading to the introduction or adaptation of mandatory professional liability insurance and the implementation of quality indicators and standards. Examples include the adoption of explicit benefit packages in Malta and Finland; reimbursement of telemedicine services in Belgium; the introduction of mandatory professional liability insurance in Malta and Poland (and expected in Estonia); and the adaptation of this liability insurance in Germany. Some efforts to lower waiting times for healthcare in Poland and Malta may also be attributed to the influence of the Directive. In addition, other studies show that the Directive encouraged Member States (such as Austria, Belgium, Finland, Hungary, Latvia, Luxembourg, Malta, Norway, Poland, Spain) to be more transparent about patients' rights in general and that existing national rules on, for example, informed consent, privacy and access to medical records apply to cross-border patients in the same way as for domestic patients. Another change that can be attributed to the Directive is related to private clinics in Belgium, which will be made subject to quality standards that previously only applied to hospitals as from 2022. There are also examples of countries (as mentioned above) which have introduced explicit statements of what is included in a patient’s benefit basket, and those which have introduced or adapted mandatory liability insurance for professionals. Secondly, there is evidence that medical tourism agencies, for example in Ireland and Poland, have emerged since the CBHC Directive was adopted. However, there is a lack of data on the actual extent of usage and reimbursement of these services, both within and outside the framework of the Directive.

With regard to external factors, due to the temporary restrictions on the free movement of people in response to the Covid-19 pandemic, patient mobility under the Directive dropped significantly from 283,719 applications for reimbursement without prior authorisation received in 2019 to 191,080 in 2020. The Commission has published all the above reports and external studies on the implementation of the CBHC Directive on its website. 4.Evaluation findings 4.1.To what extent was the intervention successful and why? Overall the evaluation found that the Directive has been successful in delivering its objectives to a certain extent. It has brought additional legal clarity on the rights of patients to use healthcare services anywhere in the EU and it has enshrined important patient rights, such as equal treatment of EU and domestic patients. It has achieved a more consistent approach at EU level to reimbursement of cross-border healthcare costs for EU citizens. It has acted as a driver for patient rights in general, increasing transparency on treatment prices and bringing about changes in various national health systems to the benefit of patients. EU citizens are making use of their rights and the reimbursement mechanisms provided by the Directive, although their numbers remain small. The general public has largely benefited from the Directive’s provisions regulating the recognition of prescriptions. The Directive has made a significant contribution in the area of rare diseases with the creation of the ERNs to support the diagnosis and treatment of rare disease patients. It has also played a crucial role to deepen European cooperation between health systems in the area of health technology assessment, leading to the adoption of a separate Regulation in 2021, and also in eHealth, leading to the proposal for a Regulation on the EHDS. Effectiveness 4.1.1.How effective was the Directive in the area of patients’ rights? The CBHC Directive has facilitated access to safe and high quality cross-border healthcare to some extent by clarifying the responsibilities of the Member States with regard to cross-border healthcare and the rules on reimbursement of costs of such healthcare. To enable citizens to make an informed choice regarding healthcare abroad, the Directive obliges Member States to ensure that citizens receive the relevant information and sets out other important responsibilities with regard to cross-border healthcare. Among the responsibilities of the Member States’ of treatment are the obligations to ensure that patients from other Member States are treated equally with domestic patients regarding access to treatment, that standards and guidelines on quality and safety laid down by that Member State and EU legislation are ensured, as well as that the healthcare providers apply the same scale of fees for services for patients from other Member States as for domestic patients or that they charge a price calculated according to objective, non-discriminatory criteria if there is no comparable price for domestic patients. The Member State of affiliation is required, among other things, to ensure the same medical follow-up as would have been available if the healthcare at issue had been provided on its territory. Unequal access to health services in the Member State of treatment has not been reported as encountered by patients. The same is true regarding the different quality of healthcare in the Member State of treatment for domestic and cross-border patients. Nor has the Commission identified that healthcare providers are setting, or are allowed to set, discriminatory prices for patients from other Member States. This evidences certain success of the CBHC Directive in ensuring patients’ rights. However, the CBHC Directive is perceived as less effective in creating legal certainty and clarity over the patients’ rights to cross-border healthcare based on the replies of the majority of public consultation respondents, particularly patients and patient organisations. Conversely, 34% of healthcare authorities, providers and insurers consider the Directive has achieved clarity and certainty to a limited extent. Insufficient and unclear information on cross-border healthcare, multiple interpretations of the relevant rules and uncertainty over the level of reimbursement were among the reasons cited. The gaps in information for patients about their rights alongside with financial burdens are considered as the most important problems resulting from cross-border healthcare. By codifying the case law, the CBHC Directive has ensured a more consistent approach at EU level to reimbursement of cross-border healthcare costs for EU citizens compared to the situation where Member States were to comply directly with the TFEU provisions, as interpreted by the Court of Justice. At the same time, recognising the organisation and delivery of healthcare as a national competence, the Directive’s provisions are formulated in a way that leaves sufficient room for manoeuvre for Member States to limit certain patients’ rights on overriding reasons of general interest, if the principle of proportionality is respected. However, the conformity checks undertaken by the Commission show that this resulted in quite a divergent implementation of the Directive across EU countries and each restrictive national measure has to be assessed on a case-by-case basis. This also essentially means that patients’ rights to cross-border healthcare cost reimbursement might differ in each Member State and full clarity about the content of these rights could only be ensured on a national level. EU citizens are using the CBHC Directive to seek healthcare across borders as a result of the Directive removing some obstacles to cross-border healthcare. Patient mobility data from 2015-2020 shows that reimbursement cases for treatment in another EU country under the Directive steadily increased until 2018 and then declined in 2019 and 2020. Patient mobility under the Directive remains low with around 300,000 requests for the reimbursement of cross-border healthcare. However, taking into account the data robustness and that several countries fail to report patient flow data, the Directive has to some extent met the expectations of the Commission’s impact assessment accompanying the legal proposal, which anticipated an estimated 390,000-780,000 patients annually to benefit from the preferred option. Moreover, while the Directive’s impact on patient flows is rather limited, the promotion of cross-border healthcare is not the objective of the Directive. At the time of the adoption of the Directive, it was expected that patient flows would remain limited. Stakeholders indicated that most patients prefer to receive healthcare close to home and that going abroad is very difficult and costly. Even if they do go abroad, in most instances they prefer to do so under the Regulations on the coordination of social security systems or any existing parallel schemes as there might be no or less out-of-pocket expenses for the patient to bear. Information to patients on cross-border healthcare Limited access to information for patients about their rights was highlighted by public consultation respondents as a key reason why the CBHC Directive do not meet patients’ needs adequately. According to a 2021 Eurobarometer survey, EU citizens still do not feel well informed about their healthcare rights, indicating that many are not able to make an informed choice about cross-border healthcare: 25% of EU citizens surveyed felt “well-informed” about what healthcare they have the right to receive and to be reimbursed for in another EU country compared to 17% in 2015; however 72% felt “not well informed” compared to 78% in 2015. Survey findings by country shows a higher awareness of rights in Northern/Western Europe over respondents in Southern/Eastern Europe. The NCPs for cross-border healthcare play a key role in enhancing patients’ opportunities to seek healthcare abroad. By 2016, all Member States had established a NCP for cross-border healthcare to provide clear and accessible information to citizens about cross-border healthcare in accordance with Article 6 of the Directive. Through the creation of the NCPs, the Directive has effectively contributed to the gradual improvement of patients’ awareness of their rights. However, awareness remains rather low with just over half of the public consultation respondents aware that NCPs existed. Those that found information on the NCPs websites ranked it higher for clarity and quality rather than completeness. Overall, information on cross-border healthcare has improved between 2015 and 2021 according to an analysis of NCPs websites in 2018 and in 2021. A Commission study in 2018 considered the information adequate. Nonetheless, many stakeholders, particularly patient organisations, highlighted information gaps, which hinder the effectiveness of the Directive to facilitate access to healthcare in another country. The European Patients’ Forum states in its position paper that clear information about patients’ entitlements, even within the domestic healthcare system, was a gap. When it comes to receiving treatment abroad, financial implications of the choices available are quite important to know in advance. The Fit For Future Platform also highlights how the NCPs are generally not known as information point for healthcare abroad and there is a lack of awareness at regional level. Information on healthcare providers has increased considerably when comparing the results of 2021 with those of 2015 and 2018, although there are significant differences between countries. Gaps were identified particularly in relation to the provision of contact details and the provision of search tools to help patients find specific healthcare providers. Particularly important for patients is information on whether to seek healthcare under the CBHC Directive or the Regulations on the coordination of social security systems, and to know whether prior authorisation is a pre-requisite for reimbursement of medical costs. A 2021 Commission study found that out of the 22 Member States and EEA EFTA countries having prior authorisation system in place, only 17 NCPs websites provide a specific list of treatments subject to prior authorisation in their country. Other countries, both in their implementing legislation and on the NCPs websites, only refer to general criteria of healthcare that could be made subject to prior authorisation in accordance with Article 8(2) of the Directive thereby undermining legal certainty for patients about the exact content of their rights to healthcare abroad. Moreover, less than half the NCPs were found to provide information on the distinction between the Directive and the Regulations, according to an analysis of NCP websites. The ECA noted that this issue affected patients’ choice for reimbursement of healthcare. While for 20 out of 30 NCPs information on reimbursement costs improved, information on pricing of treatments is generally lacking. This is due to a lack of information at national level.

Only 10 out of 31 websites provided options for people with decreased sensory functions. The European Disability Forum (EDF) report stated that patients with disabilities seeking cross-border healthcare face difficulties finding information on their rights under the CBHC Directive, due to the lack of options for people with decreased sensory functions and due to the amount of information being limited. To improve the information provision to patients, the Commission developed and published a multi-lingual toolbox for NCPs with the support of the EPF. This includes guiding principles for the practice of NCPs under the CBHC Directive (see Box 1) and a Manual for Patients. While sixteen NCPs have published the toolbox on their websites, respondents to a survey of patients’ organisations, healthcare insurers and healthcare providers showed that they are not very familiar with the toolbox. Box 1: Guiding Principles for the Practice of NCPs General Principles: ·Principle of visibility ·Principle of accessibility ·Principle of transparency ·Principle of inclusion Information provision in accordance with Directive 2011/24/EU: ·Principle of duality ·Principle of information to outgoing patients in accordance with the Directive ·Principle of information to incoming patients in accordance with the Directive Cooperation: ·Principle of information to healthcare providers ·Principle of cooperation The CBHC Directive has contributed to some extent to enhance transparency and comparability of healthcare (regarding safety, quality, costs and waiting times) across the EU, according to interviewees. In many cases, it has acted as a driver for Member States to make information on patients’ rights and quality of care more transparent and to adapt professional liability standards for health professionals. However, this has not been systematic across all Member States. Moreover, according to an analysis of NCPs websites, there are persisting gaps in the provision of information regarding safety and quality standards, costs, waiting times etc. Only 9 NCPs websites provide information on national laws regarding patient safety. The Directive requires that NCPs consult with key stakeholders. However, a 2021 Commission funded study showed that, in total, only 16, 19 and 21 of the NCPs had such consultations arrangements (informal or formal) with patient organisations, insurers and healthcare providers respectively. Moreover, where they are in place, the NCPs are in contact with these stakeholders on an irregular or rare basis. Obstacles to access healthcare abroad Citizens continue to face important obstacles hindering the effectiveness of the CBHC Directive to meet its main objectives. The public consultation responses identified the five biggest barriers (out of 20 listed) as paying for healthcare costs upfront, extensive use of prior authorisation, complex administrative procedures and uncertainty over reimbursement and information to patients. Paying for healthcare costs upfront represented the biggest barrier to healthcare abroad according to the public consultation respondents (117 out of 169 respondents) and interviewees. While the Directive provides a mechanism for citizens to seek (partial) reimbursement from their health insurer, patients must pay upfront for their treatment abroad and face additional costs for travel and accommodation as usually these are often not reimbursed in Member States (nor is this required under the Directive). Because patients are reimbursed at the same level of costs as if treated where they are insured, patient organisations in particular take the view that the Directive increases inequalities in healthcare, as there is a discrepancy in tariffs for healthcare services between EU countries. Patients from countries with lower tariffs for healthcare have to pay a large difference out of their own pocket if treated in a country with higher tariffs/prices and seek reimbursement under the Directive, thereby undermining the Directive’s aim to facilitate access to healthcare EU-wide. Paying costs upfront also impacts those unable to afford to pay in advance for healthcare abroad. The issue of access becomes particularly acute for patients with rare and low complex diseases whose only option may be to receive care and life-saving treatment via a specialized centre in a different country. In this context, it is important to recall the objectives of the Directive to facilitate cross-border healthcare: first, it complements the Regulations on the coordination of social security systems. In cases such as for rare disease patients where highly specialised and cost-intensive care is necessary, the Regulations offer a more favourable route to treatment in another country as the social security bodies are responsible for the reimbursement of the treatment costs and in most instances, the patient will not need to pay costs upfront. Secondly, under the Directive, patients pay costs upfront as it is the only viable way to empower the citizen to choose a healthcare provider, whether public or private, in another EU country without prior approval outside the circumstances coordinated under the Regulations for the coordination of social security systems while also giving the patient the right to reimbursement of (some) costs by their health insurer. The extensive use of prior authorisation to access cross-border healthcare and associated application processes prevents the realisation of the Directive’s full potential and hampers its effectiveness. In line with case law of the Court of Justice, making reimbursement of costs for cross-border healthcare subject to prior authorisation is a restriction to the free movement of services. Therefore, as a general rule, the Member States should not require prior authorisation. Indeed, the highest added value of the Directive’s route for cross-border healthcare reimbursement compared with the Regulations is the reduction of administrative burden for patients in that they can choose, for example to have outpatient treatment or a day surgery in another country without the need for prior authorisation and be reimbursed for the costs according to the domestic tariff where they are insured. The Directive only allows Member States to use a system of prior authorisation for the categories of healthcare and under specific conditions set out therein, including with due respect to the principle of proportionality. However, currently, only seven EU Member States and one EEA EFTA State have no prior authorisation system in place, giving patients freedom to choose a healthcare provider abroad and reducing administrative burden. Other countries have introduced a prior authorisation system mainly for cross-border healthcare that involves overnight hospital accommodation and/or for major non-hospital healthcare. The Member States’ lists of healthcare subject to prior authorisation also differ significantly in the extent to which the healthcare is further specified and in the number of the separate items on the lists. At the same time, a steady very low number of prior authorisation requests, as well as a relatively low number of reimbursement requests for healthcare that is not subject to prior authorisation, having marginal impacts on national healthcare budgets, do not generally point to a need of extensive prior authorisation systems under the Directive. Complex administrative procedures for prior authorisation for cross-border healthcare and reimbursement are hampering the effectiveness of the Directive to facilitate the access to cross-border healthcare to the benefits of patients. The Directive requires that administrative procedures relating to prior authorisation requests and reimbursement must be based on objective, non-discriminatory criteria that are necessary and proportionate. Yet 66 out of 169 respondents to the public consultation, from most stakeholder groups, particularly patients and citizens, selected administrative procedures to cross-border healthcare as a key barrier. In 2018, the Commission in its triennial report identified administrative procedures as a key area with potential to act as barriers to healthcare and the European Parliament report refers to “unduly burdensome and/or restrictive” systems implemented at national level. Recent research into national administrative procedures for cross-border healthcare confirm that some national requirements may pose disproportionate barriers to patients seeking healthcare in other EU countries. Box 2: Examples of potentially disproportionate administrative requirements for cross-border healthcare Requirements for prior authorisation: ·In three countries, patients have to submit, together with the application, documentation issued directly by the foreign healthcare provider, either confirming information on the healthcare to be received abroad, or on the healthcare provider and/or its availability to provide the requested service. ·In one country, patients have to submit the calculation of expected costs for the planned healthcare abroad prepared by the foreign healthcare provider which will provide healthcare. ·In nine countries, the patient seems to be required to provide in the application information concerning the availability of the healthcare and/or the waiting time for the service in the country of affiliation. For example, in one country, it is required to provide a certificate from at least two hospitals confirming that the proposed healthcare cannot be provided in that country within a medically acceptable period. ·In one country, it is required to provide a document issued by the NCP in the country of treatment showing that the healthcare provider does not give rise to serious and specific concerns relating to the observance of the standards and guidelines regarding the quality of medical assistance and safety of patients, including provisions regarding supervision. ·In seven countries, the practitioner whose referral is necessary for prior authorisation must be a national/contracted doctor, whilst in other twelve countries, the sources consulted do not seem to provide any indication on the country affiliation of the practitioner, thus the legal certainty is not ensured for patients. ·In two countries, official/certified translations of supporting documents have to be provided together with the application. Requirements for reimbursement: ·In one country, a certificate from the treating doctor regarding the legal status of the institution of treatment must also accompany the request. In addition, all documentation shall be legally issued and certified by the consulate and officially translated. ·In one country, the reimbursement request should include an evaluation of the national treating physician on the effectiveness of the treatment abroad. ·In two countries, patients are required to inform whether they have applied for reimbursement at a private insurance company or whether they have travel insurance with medical coverage. ·In one country, flight tickets must be submitted as a proof of travel abroad to substantiate reimbursement applications. ·In seven countries, patients might be required to provide official translation of documents. Uncertainty about the amount that the health insurer will reimburse for healthcare abroad was among the biggest barriers to cross-border healthcare identified by respondents to the public consultation. While the system of voluntary prior notification is believed to be a useful way of reducing the financial risk for patients, providing them with an estimation of the amount to be reimbursed after being treated abroad, only eight countries apply this system. The uncertainty about the reimbursable amount is due to several factors, such as the disparities in Member States’ healthcare baskets and their transparency, complicated systems on assumption of costs for healthcare in certain Member States, lack of transparency of the public tariff applied for the purposes of reimbursement, varied medical coding systems etc. The uncertainty about the prices charged by healthcare providers in other EU countries was another barrier mentioned, but this was ranked in the eleventh place. The Fit For Future Platform recalls that, in general, information on the applicable fees is not readily available and urges healthcare providers and hospitals to supply patients with cost estimates. Problems with the continuity of care after treatment abroad was pointed out by 84 out of the 181 respondents (46%) in public consultation, mostly concerning patients, citizens and HCPs, with 54 respondents describing problems, which mainly concerned unrecognised medical prescriptions/referrals, lack of effective transfer of medical records between treating doctors and difficulties in accessing follow-up treatment. Patients continue to face challenges in continuity of care, according to 46% of public consultation respondents, often arising from differences in health systems between their country of treatment and of affiliation. Administrative and language issues also play a role as does a lack of effective data-sharing. While 46% of respondents said that healthcare providers transferred medical records or a patient summary to the healthcare provider back home to a great or to some extent, 41% said this was done to a limited extent or not at all. An organisation representing health professionals noted that continuity of care raised issues of professional liability as health professionals from different countries are responsible for the treatment and the aftercare and healthcare is to be provided in accordance with the legislation, standards and guidelines of different countries.

Recognition of prescriptions The CBHC Directive provides for the mutual recognition of prescriptions in the EU to make it easier for patients to receive a prescribed medicinal product or medical device in a Member State different from where the prescription originated. Implementing Directive 2012/52/EU gives effect to the principle of mutual recognition of medical prescriptions and lays down measures for uniform implementation in the EU by setting out a non-exhaustive list of contents to be included in cross-border medical prescriptions that should enable health professionals to verify the authenticity of prescriptions issued in other Member States. National authorities interviewed noted that the mutual recognition of prescriptions is an example of where the Directive has worked to decrease barriers for citizens. The Pharmaceutical Group of the European Union (PGEU) highlighted persisting issues with regards to the mutual recognition of medical prescriptions despite the issuing of rules and/or guidance for the recognition of foreign prescriptions at European level and at national level in several countries. Patients continue to experience issues in relation to the verification of prescriptions in another EU country. Four in ten public consultation respondents (38%) said that they were aware of problems with pharmacists in another EU country not recognising prescriptions and three in ten said they were not aware of problems (another 31% did not provide an opinion on this). Issues commonly identified by respondents included pharmacists refusing prescriptions provided by a doctor in another EU country; a pharmacist not being able to verify whether the prescription was issued by a doctor legally entitled to do this in another EU country; or a pharmacist who could not understand the language of the prescription. To a lesser extent, respondents reported the inability of the pharmacists to understand the doctor’s handwriting or the failure to provide for a substitute medicine to that prescribed in the home country, and “other” situations such as the lack of a standardised format of prescriptions across countries, the variation of packages and dosages across Member States, the presence of different medical product names and the different legislative obligations regarding who can issue prescriptions. Patients may also face challenges in having prescriptions prescribed as part of cross-border treatment recognised by their home country. In an open question related to problems that patients may face when seeking follow-up care at home, the unrecognised medical prescriptions from abroad was one of the most frequent issues mentioned by the public consultation respondents. They are sometimes presented with prescriptions written in a language they do not understand and are often unable to contact the prescriber. The PGEU contribution notes that many patients might not be aware of their rights under the CBHC Directive and the need to inform prescribers about their intention to present any prescriptions for medicines or medical devices to a pharmacist in another country, allowing the prescribing healthcare provider to issue the prescription in line with the guidelines for cross-border use. The public consultation results revealed that six in ten respondents were aware of the possibility of having their prescriptions recognised by a pharmacist in another EU country, whereas a third (31%) were unaware of that possibility. However, citizens were significantly less aware of this, with only 38% being aware, compared to those representing organisations working at the EU/international (79%) or national (66%) level. The issues identified by the PGEU are the same as those highlighted in the Commission’s impact assessment accompanying the legal proposal, the academic papers reviewed and the targeted survey of pharmacists conducted in 2021. This indicates that, despite a reduction in the rate of non-dispensation of prescriptions, the CBHC Directive and Implementing Directive 2012/52/EU did not completely resolve the issues (language, verification and authenticity problems) that continue to hamper the recognition of prescriptions in Member States. According to the PGEU and several national authorities, the ongoing initiatives to develop the interoperability of systems facilitating the cross-border provision of electronic healthcare services, including the exchange of ePrescriptions, has the potential to strongly improve the recognition of prescriptions across the EU and overcome the barriers. 4.1.2.How effective was the Directive to encourage cross-border cooperation in the EU? The CBHC Directive provides the legal framework for voluntary cooperation in healthcare between Member States and regions as well in the areas of ERNs, rare diseases, health technology assessment and eHealth (the latter two are outside the scope of this evaluation). Cross-border cooperation between regions and neighbouring countries The CBHC Directive requires the Member States to provide mutual assistance and to facilitate cooperation in cross-border healthcare between neighbouring countries and border regions. The Commission is to encourage such cooperation. Well-functioning healthcare in cross-border regions contributes to the well-being of populations and is essential from the perspective of economic, social and territorial development and the sustainability of these regions. A specific objective of the Directive is to facilitate healthcare for citizens living in border regions where adequate healthcare services may be lacking and the nearest healthcare provider is across the border. Multiple layers of cooperation and mechanisms exist to address the structural need for healthcare in border regions and between neighbouring countries, which pre-date the Directive. An assessment of the Directive in the EU region Meuse–Rhine found that the Directive does not necessarily address the unique demographics, characteristics and needs of patients seeking healthcare across the borders of Belgium, the Netherlands and Germany. The use of the Directive’s mechanism for cross-border healthcare is hampered by prior authorisation and reimbursement procedures as well as differences in health systems and healthcare baskets. On the other hand, the Directive explicitly encourages Member States, particularly between neighbouring countries, to conclude agreements between themselves with more favourable and flexible conditions adapted to the healthcare needs in border regions. The ZOAST framework agreement along the French-Belgian border represents a benchmark for good practice of healthcare cooperation in the EU by providing conditions more favourable than those in the Directive. In this way, ZOAST overcomes the obstacles of prior authorisation and upfront payment as well as reducing costs in healthcare. The Committee of the Regions “Regional Hubs” found that few “regional hubs” were monitoring patient flows as did the Association of European Border Regions’ (AEBR) study on patient mobility in four case studies carried out in border regions. It also found that few regions monitor patient flows and therefore the extent of cross-border healthcare in border regions is unknown. However, the AEBR study concludes that the Directive provides an additional framework to share information and cooperate in healthcare at regional level. This view is borne out by the public consultation respondents where six in ten believe that the exchanges of information and good practices promoted by the Directive have at least somewhat supported cross-border healthcare cooperation in between neighbouring countries. The ECA report highlighted positively how the Commission has encouraged and financially supported cooperation in cross-border healthcare between neighbouring countries and border regions by means of studies, projects and partnerships as provided by the Directive. A 2018 Commission study provided a picture of 423 EU-funded projects supporting regional cooperation in healthcare and implemented over the period 2007-2017, the large majority of which took place between countries with similar welfare state traditions to share knowledge in treatment and diagnosis of patients. However, there is no evidence to support to what extent the Directive can be credited with the promotion of regional cooperation in healthcare by the Member States and the Commission. European Reference Networks This evaluation provides a preliminary assessment of how the ERNs are meeting their objectives with the support of the Commission and the Member States as required by the CBHC Directive. An in-depth performance assessment and evaluation of all ERNs and their members in line with the requirements of Article 14 of Commission Implementing Decision 2014/287/EU will start in 2022. The Directive aims to ensure the right of EU citizens, including people living with a rare disease, to have access to treatment in another EU country. Article 12 sets out that the Commission shall support Member States in the development of the ERNs to pool knowledge and expertise in the area of rare diseases. The ERNs are required to work towards a number of aims to improve prevention, diagnosis, treatment, care and research in this area. Besides the Commission’s financial support for the ERNs as described in more detail below, the Commission supported the ERNs system by preparing their establishment and then ensuring secretariat support and policy input for the work of the ERN Board of the Member States and the ERN Coordinators Group. The Commission (DG SANTE) also supported the work of the ERN working groups on specific topics. They include the working group on ERN knowledge generation and dissemination, a working group on the ERN monitoring, a working group on legal, ethical and stakeholder issues, the ERN IT advisory group, working group on the ERN integration in national healthcare systems and working group on ERN research (including a task force on patient registries). Research activities of the ERNs are also supported via several projects under the EU research and innovation funding programmes. The evaluation findings show that the CBHC Directive has been effective in meeting its objectives in the area of cooperation in rare and low prevalence complex diseases, bearing in mind that it has been less than five years since the 24 ERNs were set up in 2017. The rare disease patient community claims that the ERNs “are the greatest achievements that the rare disease community as a whole has ever accomplished” (while also highlighting shortcomings in the Directive’s objective to ensure access to treatment for rare diseases patients with regard to information, prior-authorisation and reimbursement). The public consultation showed that respondents who were aware of the ERNs were quite positive about the extent to which the ERNs helped healthcare providers provide diagnosis and treatment options for patients with rare and low prevalence complex diseases, and contributed to the delivery of and access to high-quality healthcare by patients. There has been a continuous growth of the ERNs patient population, with 1.7 million patients being treated by the ERNs members. There is increasing involvement from patient organisations and patient representatives participating in the ERN activities and being represented in the governance structure of all ERNs. Many ERNs have also developed action plans to foster an operational collaboration with different patient associations European Patient Advocacy Groups (ePAGs) have been developed by EURORDIS for each ERN disease group so patient organisations are able to participate in the ERN decision making. The ERNs have been effective in providing healthcare providers with access to a large pool of expertise and knowledge to help rare disease patients with diagnosis and treatment options. 85% of respondents to a targeted survey of the ERN representatives carried out for this evaluation agreed that ERNs effectively contributed to the exchange of knowledge and best practices in rare diseases (43% strongly agree and 42% agree). 87% of survey respondents agreed that the Directive has been effective in supporting the diagnosis and treatment of patients, including through the virtual consultation panels. Similarly, the majority of respondents to the public consultation who were aware of the ERNs (mostly NGOs but also public authorities, businesses, EU citizens) believe that the ERNs help health professionals provide diagnosis and treatment options for patients with rare and low prevalence complex diseases to at least some extent (6% completely, 21% to a great extent and 48% to some extent). Interviewees also pointed out that the effectiveness of the ERNs varied between the ERNs and that, in the initial years of their existence, the networks had mainly focused on setting up the ERNs and therefore had less time available to treat and diagnose patients. For the time being, the impact of the ERNs’ collaboration is likely to be more important in improving diagnosis and treatment of rare diseases patients by knowledge generation, the development and sharing of best practices and guidelines with practitioners within and beyond the ERNs, and in advancing research on rare diseases than in providing diagnosis and treatment to individual patients through the Clinical Patient Management System (CPMS). This is partly due to the fact that only the most complex cases should be submitted to the CPMS and the lack of clear national pathways for patient referral to the ERNs (to be developed under national law). Nor is the ERN process often fully understood by the patients and health professionals. Both the ERNs and NCPs stakeholders consulted noted the lack of readily available information for patients and doctors on the ERNs. To address this shortcoming some stakeholders suggested that NCPs could in the future play a more proactive role in assisting patients and health professionals beyond just making the standard information about the ERNs available on their websites. The CPMS, supported and financed by the Commission, is increasingly used by health professionals in the ERNs for the diagnosis and treatment of patients with rare and low prevalence complex diseases through virtual expert panels and it is seen as a positive development by stakeholders. However, only 2,100 patients’ cases have been dealt with through the CPMS so far. This relatively low number is due to the combination of factors, including the fact that the CPMS currently handles only the most complex and very rare patient cases, which require the consultation of experts from different specialisations in different EU countries. Another issue affecting the effectiveness of the clinical collaboration through the ERNs is the fact that healthcare providers are often not reimbursed for the time that their health professionals dedicate to providing their medical advice on foreign patient cases through their participation on the virtual panels. Clinicians highlighted the lack of clarity regarding resourcing responsibilities for ERNs. They noted that whereas payment schemes for physical cross-border patient referrals were well established, no reimbursement system exists for virtual consultations via the CPMS. Although the Directive’s rules on reimbursement do apply to cross-border telemedicine, its mechanism for cross-border healthcare cost reimbursement entails that patients pay for healthcare directly to the foreign healthcare provider and then are being reimbursed up to the public tariff in their Member State of affiliation, bearing the price difference. Thus, this mechanism does not represent a viable solution for reimbursing for the CPMS consultations with a panel of medical specialists from different countries.

Lastly, some issues with the CPMS related to the system itself were identified as limiting its use and effectiveness. 20% of the respondents who were aware of the ERNs saw the non-interoperable IT facilities as an issue. The CPMS is quite burdensome with regard to the amount of information that needs to be entered onto its database for each patient and the time required to set up and use the CPMS virtual panels. A modification of the CPMS is already underway to improve its user-friendliness and to provide a mobile version of the tool in addition to the existing desktop version. Financing and sustainability of ERNs Article 12 of the CBHC Directive requires the Commission to support Member States in the development of ERNs between healthcare providers and centres of expertise. The Commission has supported the ERNs’ operations with funding from different spending programmes (primarily the Third Health Programme and the Connecting Europe Facility (CEF) and the EU Research and Innovation (R&I) programme Horizon 2020 for research activities . Over the period of 2016-2019 the overall funding contribution provided by the Commission to the ERNs amounts to just under EUR 31 million under the Third Health Programme and around EUR 12 million under the CEF. The Commission’s support includes grants for ERN coordination and management activities, grants for establishment of ERN patient registries, grants for ERN IT related activities (such as CPMS Helpdesk services, data collection for ERN monitoring exercise or maintenance of ERN registries), funding for an independent assessment body to conduct the assessment of ERNs at the time of their establishment and of new members wishing to join existing ERNs, support for the ERN clinical guidelines programme, support for the ERN professional mobility programme or support for development of ERN integrated quality improvement system (AMEQUIS). The ERNs are also important beneficiaries of the research projects such as the European Joint Programme Rare Diseases, SOLVE-RD or ERICA funded under Horizon 2020 . The consultation highlighted the importance of continuous financing to ensure sustainability of the ERNs and therefore its future effectiveness. A Commission survey of the ERN coordinators showed that sustainability of financing is one of the main challenges facing the ERNs. Currently the grant application, management and reporting under the EU Health Programme entail a high administrative workload for the ERNs. The ECA recommended that the Commission should “work towards a simpler structure for any future EU funding to the European Reference Networks and reduce their administrative burden”. The Commission intends to address this recommendation by streamlining the existing funding sources for ERNs under the Health Programme with the launch of new direct grants for ERNs under the new EU4Health Programme. In the context of ERN funding with regard to other activities such as drug development, knowledge building, collaboration with the private sector (pharmaceutical and medical devices industry) has been considered, for example, in the area of the ERNs’ research and clinical trials on rare and low prevalence complex diseases (while taking appropriate transparency and conflict of interest management measures). Recognising the importance of the role of industry to improve the knowledge of rare conditions and to develop diagnostics tools and therapies, the Board of Member States for ERNs adopted “Statement on ERNs and industries” in 2019 that provides general principles for this future collaboration. Integration into national healthcare systems The CBHC Directive requires Member States to facilitate the development of the ERNs. All interviewees representing the ERNs stressed the importance of integrating the ERNs into the national healthcare systems to increase their visibility and, more importantly, to ensure appropriate referral and reimbursement mechanisms for rare and low prevalence complex disease patients. The Board of Member States for ERNs addressed this key issue in 2019 with a statement encouraging Member States to facilitate the integration of the ERNs into national healthcare systems. However, feedback from the consultation (17% of public consultation respondents) clearly shows that the general principles set out by the Board of Member States for ERNs have not yet been fully implemented in the Member States. An additional reported barrier is the lack of awareness or knowledge on how to access the ERNs among healthcare providers which are outside the networks. The Commission intends to further support Member States in their efforts to better integrate the ERNs into their national healthcare systems by launching in 2022 a Joint Action on ERN integration under the EU4Health Programme. ERNs’ role in knowledge generation and information sharing on rare and low prevalence complex diseases Beyond the general support for the ERNs’ coordination and management activities (including training programs or conferences organised within the respective ERNs), the Commission supported the ERNs in the area of knowledge generation and sharing through various specific projects including the ERN clinical guidelines programme, the ERN professional mobility programme or EU high-level ERN conferences. The ERNs have organised workshops and webinars to increase capacity building, and surveys to identify educational gaps in medical knowledge on patients with rare and low prevalence complex diseases. The ERNs have also invested in enhancing highly specialised knowledge about rare diseases through various dissemination activities (websites, newsletters, scientific journals, conferences etc.). The majority of stakeholders and respondents to the public consultation agree that the ERNs have been effective in developing knowledge sharing activities to support health professionals, in enhancing professional training and mobility of expertise in this area and in facilitating exchange of knowledge and best practices. According to interviewees, effective knowledge sharing is one of the areas where the objectives of the networks are being best achieved. The level of awareness provides an important indicator to assess the extent to which the use of the ERNs and the knowledge sharing has been effective. In relation to this, the public consultation results showed that a majority of respondents were aware of the ERNs and the possibilities to seek diagnosis and treatment of rare diseases in another EU country with prior approval from their healthcare insurer (11% completely, 16% to a great extent and 25% to some extent). However, both the ERNs and NCPs stakeholders consulted also noted the lack of coordination between the NCPs and the ERNs resulting in a lack of clear information for patients on how to access the ERNs services and on how to get from the national system to the ERNs. They noted that doctors are often not aware of the existence of the ERNs and are not always willing to bring patients into these networks. The ERNs’ role in research on rare and low prevalence complex diseases With regard to the impact of the ERN collaboration on the research, knowledge sharing and networking activities within the ERNs have resulted in increased collaboration between experts and directly contribute to the research on rare and low prevalence complex diseases. The interviewees considered that collaboration in various cross-ERN expert groups has been quite fruitful and pointed out that many important research initiatives have emerged from the ERNs, such as the EU-funded ERICA project, which is a “Coordination and Support Action” for coordinating clinical research efforts of the 24 ERNs. ERNs are also important beneficiaries of the research projects such as the large European Joint Programme on Rare Diseases (EJP RD, co-fund with Member States research funding agencies) and the Solve-RD project closely cooperating with several ERNs. It is important to keep in mind that many other EU-funded research projects are relevant and involve ERN clinicians and researchers from specific ERNs (for example ImmunAID for ERN RITA), thus also enhancing the research potential of the ERN community as a whole. Other EU-funded projects involving ERN clinicians and researchers are: Conect4Children (C4C), Screen4Care (both funded under the IMI joint undertaking thus with public (H2020) and private funding) or Rare2030, led by the patients organisation EURORDIS. In addition, with the financial support from the Commission, all 24 ERNs have established or they are in the process of setting up or consolidating ERN patient registries in order to create a critical mass of patients’ data. These are essential building blocks for advanced research in the area of rare and low prevalence diseases. Furthermore, the ERNs have facilitated large clinical studies to improve understanding of diseases and develop new drugs by gathering a large pool of patient data. Patients participated in 732 clinical trials within the ERNs. 162 new clinical practice guidelines were drafted by ERNs as well as the development of 143 clinical decision making tools. Moreover, 405 observational studies were conducted within the ERNs. When asked to assess the contribution of the ERNs in several areas, six in ten respondents to the public consultation believed that the ERNs had helped to exploit innovation in medical science and health technologies completely (9%), to a great extent (15%) or to some extent (36%). The same proportion agreed that the ERNs had helped to collect, analyse and make available health data completely (6%), to a great extent (16%) or to some extent (38%). Overall, a majority (79%) of respondents who were aware of the ERNs believed to at least some extent that the ERNs helped to generate knowledge and contribute to research on rare and low prevalence complex diseases in the EU (9% completely, 23% to a great extent and 47% to some extent). Cooperation in Rare Diseases Article 13 of the CBHC Directive has strengthened existing European cooperation in rare diseases supported by the Commission. Between 2010 and 2015, a series of activities were carried out to support the development of Orphanet as a European portal for information of rare diseases and orphan drugs, to enhance the visibility and recognition of rare diseases, the development and dissemination of knowledge and support improvements in access to quality services and care. As of 2015 this was followed by further support to Member States to promote implementation of recommendations on policy, information and data, and to implement the European rare diseases codification system, as well as to Orphanet. Between 2010 and 2021, financial support for these efforts totalled nearly EUR 12.5 million via funds provided through subsequent European health programmes. The registration of rare diseases across Europe is supported by the European Platform on Rare Disease Registration (EU RD Platform), set up in 2019 by the European Commission’s Joint Research Centre. The EU RD Platform facilitates information exchange on rare disease patients and supports registries at national, regional and local level as well as the ERNs to collect and share information. The EU RD Platform makes rare diseases patient data searchable and findable -something that so far was not possible- and is available for patients as well as for the community of researchers, healthcare professionals and policymakers. While the Platform's infrastructure is openly accessible and training is being provided for people working in existing registries or willing to create new registries, it also sets EU-wide standards for data collection and exchange.

The pillars of the EU RD Platform are 1) the European Directory of Registries, 2) the Central Metadata Repository and 3) the Pseudonymisation Tool, which makes sure patient data is held under a pseudonym, thus not traceable back to the individual but permitting the cross-border or cross-registries data exchange. The EU RD Platform collaborates closely with the ERNs supporting the creation of ERN registries and aims at linking patients’ clinical data with other health-related data like genomic data and biobanks. Interviewees representing the ERNs, the ERN Board of Member States and researchers in the field of rare diseases, noted that ERNs are an appropriate tool that fit well with initiatives such as the Orphanet database, the European Joint Programme on Rare Diseases. With support from the Commission and Member States, this is creating a rare diseases research eco-system in Europe, bringing together researchers and practitioners. The programme has generated important ties with health professionals who are and who are not involved in the ERNs. With regard to Orphanet particularly, stakeholders working in the field highlighted the synergies with the ERNs which helped develop, for example, the ORPHAcodes. Another area of good synergies with the ERNs mentioned in the interviews is the EHDS, for which the networks will be a building block. ERNs and Orphanet thus constitute a unique European framework dedicated to rare diseases with key complementary roles. ERNs have the clinical and scientific expertise on rare diseases and Orphanet has the expertise on databasing and standardisation. Through the objectives outlined in Article 12 (on ERNs) the CBHC Directive takes into account this complementary role and supports the existing framework on rare diseases not only by promoting these tools under Article 13, but also by reinforcing their role through shared objectives. Efficiency The evaluation on the efficiency of the CBHC Directive focuses on the actual costs and benefits associated with the implementation of the Directive as well the administrative burden linked to it. Cost/benefits on Member States The additional costs arising from cross-border treatment under the CBHC Directive have had a very minor impact on national healthcare budgets. Based on data received from Member States as well as data from Eurostat, the Commission’s 2022 report estimates that the share of the amount reimbursed under the Directive on the total government expenditure on healthcare corresponded to 0.01%. This estimate is in line with the ECA estimate and also corresponds to feedback received from stakeholders. They also noted the Directive’s very modest impact on public health funding. National health insurers and health ministry representatives interviewed for the evaluation confirmed that the costs of treatment are low as the Directive provides that patients are reimbursed up to the same level of costs that would have been assumed by the Member State of affiliation if they had been treated domestically and therefore the financial burden is negligible. Member States’ reimbursements to cross-border patients grew between 2016 and 2019, from EUR 67 million to EUR 92.1 million, while remaining low in absolute terms . It is important to note that, as pointed out in the Commission’s patient mobility data trend report 2018-2020, the countries which provided data on reimbursements differ between the reference years. In the countries for which there is complete data for all reference years 2016-2020 , the total amount of reimbursement peaked in 2019 (EUR 41.0 million), with a substantial drop in 2020 (EUR 29.2 million). The exact magnitude of the costs arising from the implementation of the Directive, for example, processing prior authorisation and reimbursement requests is not known as Member States countries do not collect this cost data. Moreover, while resources apportioned for cross-border authorisation and reimbursement cannot be easily calculated as health insurance staff are usually also processing domestic reimbursements, no stakeholder reported that the Directive has created excessive or disproportionate costs for public authorities or health insurers. In terms of benefits for health systems, the CBHC Directive has not yet delivered efficiency gains for national health systems as the volume of cross-border healthcare cases facilitated remains negligible in most Member States with an estimated 6 out of 10,000 people across the EU receiving reimbursement for cross-border healthcare treatments under the Directive. Even when allowing for variances in the use of the Directive across Member States, the benefits remain very minor. However, qualitatively, the Directive has enabled comparisons between health systems and increased transparency. Nor is there evidence that the CBHC Directive provided direct benefits for health systems during the Covid-19 crisis. For example, while several regions in Germany provided life-saving assistance for Covid-19 patients to alleviate the burdens of overstretched hospitals across borders, these acts of European solidarity are attributable to existing regional cooperation. However, responding whether the Directive could tackle the backlog of postponed treatments arising from the pandemic, out of 184 public consultation respondents, 28% considered the Directive could help to a large extent and 20% to some extent. Costs/benefits to the patient The total quantitative benefits for patients using the Directive’s reimbursement mechanism is marginal due to the low cross-border patient mobility flow of around 300,000 reimbursements per year. Less than 1% of citizens on a waiting list benefit from faster treatments, however there is no data available on the reduction of waiting times overall. The Regulations on the coordination of social security systems are generally more favourable route to healthcare abroad as the patients do not normally bear the costs of treatment and are being reimbursed based on the level of the tariff in the Member State of treatment. For example, in the reference year 2020, Member States and EFTA States issued 23,400 Portable Documents (PD) S2 for planned cross-border healthcare and received around 1.9 million claims to be settled/reimbursed for necessary cross-border healthcare based on the EHIC. However, in terms of qualitative benefits, patients with rare and low prevalence complex diseases have benefitted from the Directive, particularly through the intermediate outcomes of better diagnosis and understanding of treatment options available by health professionals participating in the ERNs. For physical treatment in a healthcare facility abroad, the Regulations’ route represents a better option in most instances, as explained above, because where the Member State of treatment provides benefits-in-kind to its insured persons, cross-border patients also have access to such benefits under the same conditions. There is no quantitative data available on the use of the Directive by different patient groups. However, qualitative evidence shows that several patient groups benefit from the Directive’s rules on cross-border healthcare cost reimbursement. These include patients who need an outpatient treatment that is quicker to access or cheaper abroad; patients who travel frequently to other EU countries for work or family purposes; patients from smaller countries or for whom the closest facility is in another Member State; tourists who need necessary treatments which they cannot access under the Regulations on the coordination of social security systems; pensioners who live part of the year abroad, but do not qualify as residents of that country; patients who are expats who wish to be treated in their country of origin. For certain groups of patients the costs of using the Directive can be substantial. This financial barrier is hindering the effectiveness of the Directive as described above. The patient pays the costs of treatment directly to the healthcare provider and then requests reimbursement back home. If the public tariff (or a price calculated under Article 4(4) first paragraph of the Directive) is higher in the country of treatment than where the patients are insured, they cover the difference out-of-pocket. Moreover, the Directive is without prejudice to national legislation, which allows healthcare providers to set their own – private – prices, if they do not discriminate against patients from other EU countries. Cross-border patients under the Directive are also charged private prices in some Member States where public/contracted healthcare providers providing benefits-in-kind in the public system can also act in private capacity. Thus, the difference that the patient has to cover out-of-pocket can in certain instances be even higher than the difference between the public tariffs in the Member States of treatment and affiliation. In addition, in the vast majority of Member States, patients bear costs for travelling and accommodation abroad, or extra costs, which persons with disabilities might incur, as the Directive does not oblige the Member States to reimburse such costs, even where prior authorisation is issued for healthcare abroad. Furthermore, as highlighted above in Section 4.1.1, burdensome administrative procedures in many countries add to the costs for patients. Processing times are slow and varied ranging from three weeks up to six months for prior authorisation applications and from one month up to six months for reimbursement requests. In several countries, processing times are not laid down in the national legislation. Patients might also bear indirect costs related to the administrative procedures, such as postage costs, where applications cannot be submitted by electronic means (as illustrated in Box 2, in certain instances certified translation is required). In addition, patients might also be required to provide translation of the medical records, for the purposes of accessing treatment abroad or receiving follow-up treatment back home. Nor are the costs of the Directive borne proportionately by different patient groups. The costs can be particularly significant for those from lower income countries, for patients with lower socio-economic status or patients requiring access to more expensive highly specialised treatment as is particularly the case for patients with rare and low prevalence complex diseases.

Data gathered for the cost/benefit analysis are insufficient to estimate the costs across different stakeholder groups and thus prevent an assessment on whether the costs of the Directive are proportionate to the associated benefits for these patient groups. On the other hand, as mentioned above, the Directive’s rules on reimbursement of cross-border healthcare costs supplement the relevant rules under the Regulations on the coordination of social security systems. Thus, in particular in those instances where the Regulations do not apply or where cross-border patients failed to ascertain and pursue their rights under the Regulations (for example, where the healthcare provider did not consider the treatment as “necessary care” under Article 19 of Regulation (EC) No 883/2004) the Directive’s rules allow them to receive reimbursement of at least part of cross-border healthcare costs (compared to zero reimbursement under the Regulations). With regard to the ERNs, a quantitative assessment of the cost effectiveness of the ERNs is challenging as only data on the EU level funding is available while there are no estimates for the total funding from the ERN coordinating centres and hospital hosting the ERNs. Similarly, data on the funding from private donors, patient organised campaigns and Member States is not available and not all costs incurred by the ERNs are taken into account. As a result, it was not possible to quantitatively assess the extent to which the costs of the ERNs and their tools were justified and proportionate given the objectives achieved and benefits obtained. To support the ERNs’ operations, the Commission has provided funding through different funding mechanisms. These amount to just under EUR 31 million under the Third Public Health Programme and EUR 12.4 million under the Connecting Europe. The co-funding provided by the ERN coordinating centers is estimated at EUR 12.4 million (40% co-funding of the grants provided). This brings the estimated total cost of the ERNs to EUR 48.2 million. However this estimate does not take into account all costs incurred by the ERNs. Nor does it take into account research activities of the ERN members. With regard to the objectives achieved, the ERNs have improved the care of patients with rare and low prevalence complex diseases across the EU through diagnosis and treatment (diagnosing patients and ensuring access to and delivering high-quality healthcare). The members of the ERNs treat 1.7 million patients with rare and low prevalence complex diseases, including almost 600,000 new patients referred to the ERN healthcare providers with a diagnosis of diseases/conditions that fall within the scope of expertise of the ERNs. The ERNs have also contributed to research and innovation by generating knowledge, exploiting innovation in medical science and health technologies, and collecting, analysing and making available health data. The ERNs have also provided support to healthcare systems by developing quality and safety benchmarks and helping EU countries with an insufficient number of patients with a particular medical condition, or lacking technology or expertise, to provide highly specialised services of high quality. 52% of respondents to the public consultation believe that the ERNs have helped to make cost-effective use of resources within EU-wide networks to reduce the burden and fill gaps at national level to at least some extent; while 19% reported that the ERNs have contributed on this matter to a limited extent or not at all. The relatively high cost at the development and initial implementation stage of the ERNs, especially in the IT organisational infrastructure, were to be expected at this early stage of the ERN project. This is supported by the findings from the public consultation whereby 81% of respondents believed the costs of the ERNs and their IT tools to be justified and proportionate to at least some extent, given the objectives achieved and benefits obtained. However, the absence of a mechanism for reimbursement of healthcare providers’ costs incurred as part of the ERNs services was also highlighted as an issue potentially affecting the cost-effectiveness of the ERNs. In addition, the ERNs provide the framework allowing rare and low prevalence complex disease patients to receive diagnosis and treatment without necessarily physically transporting the patient to another Member State. Therefore, and as highlighted during the EU Health Programme Conference in 2019, the ERNs are more cost effective as they save the patient or, as the case might be, the health insurer the expense of travelling abroad for a diagnosis (including related costs of accommodation, cost of family members moving with the patient etc.). This assessment of the cost-effectiveness of the ERN model was supported to at least some extent, by 75% of the ERN members consulted as part of the ERN targeted survey. By shortening the time of diagnosis and improvements in treatment through virtual consultations, knowledge sharing or development of clinical guidelines, the ERNs produce cost-savings in the long run. This is particularly significant as patients with rare diseases tend to remain undiagnosed (for many years) or to be misdiagnosed resulting in a higher number of hospital visits and accompanying costs per rare diseases patient compared to the general patient population . Interviewees noted however that given the relatively low uptake of the virtual consultations in the first years since the establishment of the ERNs, specific data on cost-savings were limited so far. In addition, research takes time and given the ‘young age’ of ERNs, their research potential is not yet fully deployed. Coherence The evaluation of coherence involves assessing whether or not different actions related to the CBHC Directive work well together. It helps highlighting areas where there are complementarities or synergies, which improve overall performance; or sheds light on issues that are contradictory or cause inefficiencies. In the evaluation, the complementarities or overlaps between different provisions of the Directive (internal coherence), as well as the alignment with other inter-related EU policies and initiatives (external) has been assessed. Internal coherence The evaluation has not revealed any specific issues relating to the internal coherence of the CBHC Directive. The feedback from different stakeholder groups suggested that the Directive is well structured and that its objective to facilitate the access to safe and high quality cross-border healthcare has largely translated into legal provisions. However, there are some issues with regard to the application of the provisions of the Directive across the EU, most importantly, around the level of reimbursement of cross-border healthcare costs, the prior authorisation systems, administrative procedures for cross-border healthcare and reimbursement of telemedicine. In addition, as mentioned above, in recognition of national competences, the Directive’s provisions allow the Member States to introduce certain limitations based on overriding reasons of general interest, if the principle of proportionality is respected. As a result, each restrictive national measure has to be assessed on a case-by-case basis. Level of reimbursement of cross-border healthcare costs: Member S