Navigating the path towards successful implementation of the EU HTA Regulation: key takeaways from the 2023 Spring Convention of the European Access Academy.

BACKGROUND: The European Regulation on Health Technology Assessment (EU HTA R), effective since January 2022, aims to harmonize and improve the efficiency of common HTA across Member States (MS), with a phased implementation from January 2025. At "midterms" of the preparation phase for the implementation of the Regulation our aim was to identify and prioritize tangible action points to move forward. METHODS: During the 2023 Spring Convention of the European Access Academy (EAA), participants from different nationalities and stakeholder backgrounds discussed readiness and remaining challenges for the Regulation's implementation and identified and prioritized action points. For this purpose, participants were assigned to four working groups: (i) Health Policy Challenges, (ii) Stakeholder Readiness, (iii) Approach to Uncertainty and (iv) Challenges regarding Methodology. Top four action points for each working group were identified and subsequently ranked by all participants during the final plenary session. RESULTS: Overall "readiness" for the Regulation was perceived as neutral. Prioritized action points included the following: Health Policy, i.e. assess adjustability of MS laws and health policy processes; Stakeholders, i.e. capacity building; Uncertainty, i.e. implement HTA guidelines as living documents; Methodology, i.e. clarify the Population, Intervention, Comparator(s), Outcomes (PICO) identification process. CONCLUSIONS: At "midterms" of the preparation phase, the focus for the months to come is on executing the tangible action points identified at EAA's Spring Convention. All action points centre around three overarching themes: harmonization and standardization, capacity building and collaboration, uncertainty management and robust data. These themes will ultimately determine the success of the EU HTA R in the long run.

[Is there a need for more patient participation in Germany? : Analysis and outlook]. / Brauchen wir mehr Patientenvertretung in Deutschland? : Analyse und Ausblick.

The involvement of patients in German healthcare lags far behind the required level. Despite the subject status of patients in the healthcare system, possibilities for codecision and active participation are rare. This paper points out structural fields of patient involvement, how a profound quality of participation can be ensured, and how implementation can be designed.Aiming at a higher acceptance of medical care decisions for persons affected, the integration of personal experience and knowledge is reasonable and necessary for the design and development of the healthcare system. Patients must be offered an education imparting information and competences for their participation. Learning modules from different training providers should transmit professional expertise as well as general systematic and methodical skills. Because of the key role the patient occupies administrating their health data, educational services should also be offered in this area. Possibilities for patient participation are offered at all interfaces between healthcare facilities, services, and products. Furthermore, decisions on allocations by the statutory health insurance and higher federal authorities require the participation of patients. However, this involvement requires a preceding democratic legitimation.For efficient patient involvement in the German healthcare system, regulatory challenges must be overcome by all involved parties in the healthcare system. The time for implementation has come.

Praxis der Teledermatologie.

HINTERGRUND: Teledermatologische Anwendungen werden im deutschen Versorgungssystem in den nächsten Jahren erheblich an Bedeutung gewinnen. Das vorliegende Empfehlungspapier wurde als Expertenkonsens auf der Basis einer qualifizierten Literaturrecherche und eines strukturierten Entscheidungsprozesses der Autorengruppe entwickelt. ZIELSETZUNG: a) die IST-Analyse zum Einsatz der Telemedizin in der Dermatologie, b) die Bewertung der Evidenz ihres Nutzens und ihrer Sicherheit und, c) die Entwicklung von Verfahrensstandards für die ärztliche Praxis in den deutschsprachigen Ländern. Auf der Basis dieser Erkenntnisse soll durch einen Expertenkonsens eine Handlungsorientierung für den Einsatz der Teledermatologie gegeben werden. METHODEN: Dreistufiges Vorgehen: 1) Systematische Literaturrecherche in den internationalen medizinischen Onlinedatenbanken Pubmed und Embase, 2) Weitere, teils manuelle Recherchen, 3) Expertenkonsens mit einem systematischen Entscheidungsverfahren mit 21 Teilnehmern. ERGEBNISSE: In der strukturierten Literaturrecherche fanden sich 204 wissenschaftliche Originalarbeiten, in denen Anwendungen der Telemedizin bei Hautkrankheiten thematisiert wurden. Diese wurden systematisch aufgearbeitet, analysiert und bewertet. In der zweiten Stufe wurden in einer Handsuche zusätzliche relevante Schriften identifiziert und ebenfalls ausgewertet. Das Expertengremium entwickelte dann auf der Basis der externen Evidenz sowie der internen Diskussion Handlungsempfehlungen für die Praxis. Schlussfolgerung der wissenschaftlichen Studienlage ist, dass die telemedizinische Unterstützung der dermatologischen Behandlung und Prävention bei Einsatz leistungsfähiger Systeme, Kenntnis ihrer Anwendung sowie Beachtung der Indikationen und Kontrainidikationen einen erheblichen Mehrnutzen darstellt. SCHLUSSFOLGERUNGEN: Die Teledermatologie hat in den deutschsprachigen Ländern wie auch weltweit einen zunehmenden Stellenwert und bietet aufgrund des hohen Innovationsgrades eine Vorreiter- und Vorbildfunktion für weitere telemedizinische Anwendungen anderer Fachrichtungen. Eine qualitätsgesicherte teledermatologische Behandlung ist in den deutschsprachigen Ländern praktikabel und kann zu einem relevanten Mehrnutzen in der Versorgung führen. Ihr Einsatz ist immer dann in Erwägung zu ziehen, wenn relevante Zusatznutzen für die Patienten ohne relevante Nachteile für sie und für die Versorgenden zu erwarten sind. Für die teledermatologische Behandlung wurden mit dem vorliegenden Konsensuspapier praxisrelevante Maßgaben festgelegt. Etwaige situationsabhängige Limitationen in der Versorgung sind stets zu beachten.

An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators.

OBJECTIVES: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation's (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. METHODS: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. RESULTS: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. CONCLUSIONS: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an "inclusive civil society dialogue", as suggested by the European Commission's Pharmaceutical Strategy.

[Legal integration of artificial intelligence into internal medicine : Data protection, regulatory, reimbursement and liability questions]. / Rechtliche Einordnung von künstlicher Intelligenz in der Inneren Medizin : Von Datenschutz und Regulatorik, Erstattungs- und Haftungsfragen.

Artificial intelligence (AI) opens up new opportunities to improve medical care in internal medicine; however, legal uncertainties in the application of AI impede its integration into the daily practice of internal medicine. To clarify the situation this paper gives an overview of the legal aspects related to AI and shows which frameworks must be adhered to in order to exploit the benefits of AI without neglecting the rights and protection of patients. The paper first addresses data protection issues which arise when sensitive health data are processed by AI. This is followed by a discussion of the key regulatory requirements for the use of AI in internal medicine. As the establishment of AI in practice also depends on sufficient funding, legal issues of reimbursement are additionally examined. Finally, the specific features that need to be considered when using AI to avoid medical liability consequences are highlighted.

The role of stakeholder involvement in the evolving EU HTA process: Insights generated through the European Access Academy's multi-stakeholder pre-convention questionnaire.

Involvement of all relevant stakeholders will be of utmost importance for the success of the developing EU HTA harmonization process. A multi-step procedure was applied to develop a survey across stakeholders/collaborators within the EU HTA framework to assess their current level of involvement, determine their suggested future role, identify challenges to contribution, and highlight efficient ways to fulfilling their role. The 'key' stakeholder groups identified and covered by this research included: patients', clinicians', regulatory, and Health Technology Developer representatives. The survey was circulated to a wide expert audience including all relevant stakeholder groups in order to determine self-perception by the 'key' stakeholders regarding involvement in the HTA process (self-rating), and in a second, slightly modified version of the questionnaire, to determine the perception of 'key' stakeholder involvement by HTA bodies, payers, and policymakers (external rating). Predefined analyses were conducted on the submitted responses. Fifty-four responses were received (patients 9; clinicians: 8; regulators: 4; HTDs 14; HTA bodies: 7; Payers: 5; policymakers 3; others 4). The mean self-perceived involvement score was consistently lower for each of the 'key' stakeholder groups than the respective external ratings. Based on the qualitative insights generated in the survey, a RACI Chart (Responsible/Accountable/Consulted/Informed) was developed for each of the stakeholder groups to determine their roles and involvement in the current EU HTA process. Our findings suggest extensive effort and a distinct research agenda are required to ensure adequate involvement of the key stakeholder groups in the evolving EU HTA process.

Shaping a research agenda to ensure a successful European health technology assessment: insights generated during the inaugural convention of the European access academy.

OBJECTIVES: Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. METHODS: Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books identifying the work assignments were shared in advance. Two leads and one note taker per working group facilitated the process. All rankings were conducted on an ordinal Likert Response Scale scoring from 1 (low priority) to 7 (high priority). RESULTS: Identified key domains include for processes: i) address (resource-) challenge of multiple PICOs (Patient/ Intervention/ Comparator/ Outcomes), ii) time and capacity challenges, iii) integrating all involved stakeholders, iv) conflicts and aligning between different multi-national stakeholders, v) interaction with health technology developer; for uncertainty: i) early and inclusive collaboration, ii) agreement on feasibility of RCT and acceptance of uncertainty, iii) alignment on closing evidence gaps, iv) capacity gaps; for comparator choice: i) criteria for the choice of comparator in an increasingly fragmented treatment landscape, ii) reasonable number of comparators in PICOs, iii) shape Early Advice so that comparator fulfils both regulatory and HTA needs, iv) acceptability of Indirect Treatment Comparisons (ITC), v) ensure broad stakeholder involvement in comparator selection; for endpoint selection: i) approaching new endpoints; ii) patient preferences on endpoints; iii) position of HTA and other stakeholders; iv) long-term generation and secondary use of data; v) endpoint challenges in RCTs. CONCLUSIONS: The implementation of a joint European HTA assessment is a unique opportunity for a stronger European Health Union. We identified 19 domains related to the four key areas, processes, uncertainty, comparator choice and endpoint selection that urgently need to be addressed for this regulation to become a success.

Legal and social responsibility in health service chains.

Personal health settings establishing health service chains lead to new legal challenges. The safe harbor principle of doctor-patient relationships has to be extended for including multilateral relations and other parties by reconciling a broad variety of legal regulations with detailed contractual agreements. Beyond security and privacy, also liability, risk management and reimbursement have to be ruled.

[Integrated care from a legal point of view]. / Integrierte Versorgung aus juristischer Sicht.

Integrated care requires reinventing the statutory health insurance in a nutshell format. This paper outlines 13 essential criteria that a contract will have to meet for establishing the basis for integrated care.

[The binding quality of non-binding--the paradox of disease management guidelines]. / Das Verbindliche des Unverbindlichen--zum Paradox der Versorgungsleitlinien.

The recommendations on "Medical Standard and Guidelines--Economisation of Medical Care" issued by the German Society of Medical Law (DGMR) critically focus on the binding effects of guidelines in the statutory health insurance system. But even after the Modernisation of the Statutory Health Insurance System Act (GMG) has been passed, concerns remain.

[Reproductive medicine--the state of the law]. / Reproduktionsmedizin--Die "Rechtslage".

Reproductive Medicine is subject to a large number of legal regulations. Professional law, public law, civil law and criminal law hold several special regulations in store for this particular area. This article gives a brief overview of the regulations applicable to reproductive medicine. Special problems that demand legislative action are pointed out.

Legal and social implications of health telematics in the EU.

Health telematics involve the processing of data concerning health. The European Directive 95/46/EC constitutes a general prohibition of processing this special category of data. Exemptions to that principle are found in the data subject's consent, vital interests and professional obligation to secrecy. These exemptions, however, do not sufficiently secure legal grounds for health data protection in practice. It is for that reason, that the member states must establish a harmonised legal basis for telematics services. Multinational working groups, such as EHTEL, need respective support.

[Legal consequences of suboptimal medical treatment]. / Rechtliche Konsequenzen suboptimaler Arzneimitteltherapie.

The legal framework of the statutory health insurance does not legitimize substandard therapy. There is no civil case law allowing for prescriptions that are not in line with the current "state of the art" procedures for economical reasons.

Teleconsultation practice guidelines: report from G8 Global Health Applications Subproject 4.

This report presents a series of recommendations derived from deliberations of the G8 countries Subproject 4 Group (SP4 Group) of the Global Health Care Applications Project entitled, A Teleconsultation Practice Guideline. The recommendations provide an initial step toward developing a general guideline platform for the practice of telemedicine/teleconsultation.