Die Arzneimittelversorgung für gesetzlich Krankenversicherte und im Speziellen die Problematik der Bildung der Erstattungspreise bleibt auch nach den jüngsten gesetzlichen Änderungen in Deutschland ein zentrales Thema der gesundheitspolitischen Diskussion. Aktuell werden Erstattungsbeträge für Arzneimittel mit neuen Wirkstoffen zwischen GKV-Spitzenverband und pharmazeutischen Unternehmen im Rahmen der AMNOG-Verhandlungen vereinbart. Grundlage dieser Verhandlungen sind die Beschlüsse des Gemeinsamen Bundesausschusses über den Zusatznutzen eines neuen patentgeschützten Arzneimittels. Für Arzneimittel ohne festgestellten Zusatznutzen, die einer Festbetragsgruppe zugeordnet werden können, finden diese Verhandlungen nicht statt. Das übergreifende Ziel der Studie ist es, die Arzneimittelversorgung auf Grundlage eines systematischen Ländervergleichs anhand ausgewählter Kriterien darzustellen und ein umfassendes Bild der regulatorischen Mechanismen aufzuzeigen, welches die Arzneimittelversorgung in europäischen Ländern im Vergleich zu Deutschland prägt. Im Fokus der vergleichenden Analyse stehen hierbei Regulierungsmechanismen, welche auf die Zulassung, Postlizenzierungsevaluation, Preisbildung und -aktualisierungen, finanzielle Beteiligungen der Patienten, Maßnahmen der Wirtschaftlichkeits- oder Qualitätssicherung sowie Regulierungsmechanismen für Generika fokussieren. Des Weiteren wird untersucht, wie sich die Preise bedingt durch Regulierungsmechanismen in den Vergleichsländern unterscheiden. Die Studie basiert auf einer umfassenden Stichprobe von 16 europäischen Gesundheitssystemen (Belgien, Dänemark, Deutschland, England, Finnland, Frankreich, Griechenland, Irland, Italien, Niederlande, Österreich, Portugal, Schottland, Schweden, Spanien und Polen). Um eine grundlegende Informationsbasis für die Vergleichsländer zu beschaffen, wurden quantitative Daten von der OECD, landesspezifische regulatorische Dokumente sowie publizierte und graue Literatur herangezogen. Aufbauend auf der Systematisierung und tabellarischen Darstellung der länderspezifischen Informationen, wurden Experten im jeweiligen Land zum Review dieser Informationen herangezogen. Die Arzneimittelversorgung in der deutschen GKV weist im Vergleich zu den betrachteten europäischen Ländern auch nach den Änderungen durch das AMNOG eine sehr geringe „Verzögerung“ zwischen Marktzulassung eines neuen Arzneimittels und der tatsächlichen, öffentlich finanzierten Nutzung dieses Präparates auf. Eine Erstattungsfähigkeit besteht für praktisch alle Präparate und je Produkt für alle zugelassenen Indikationen, während Einschränkungen auf bestimmte Indikationen oder Patientengruppen in anderen Ländern häufig auftreten. Diese vergleichsweise „großzügige“ Erstattungsfähigkeit von rezeptpflichtigen Arzneimitteln – sowie die verhältnisweise mäßigen Zuzahlungen – spiegeln sich in sehr hohen öffentlich finanzierten Arzneimittelausgaben pro Kopf wider. Auch unter Einbezug der privaten Gesundheitsausgaben weist Deutschland überdurchschnittlich hohe Arzneimittelausgaben auf. Da der Anteil an Generika in Deutschland auf verhältnisweise hohem Niveau liegt, dürfte dies zum einen auf das überdurchschnittliche Preisniveau und zum anderen auf die Nutzung von neuen (und teureren) Arzneimitteln zurück zu führen sein. Insgesamt sieht sich Deutschland primär weder vor der Herausforderung einen besseren Zugang zu neuen Arzneimitteln zu gewährleisten, noch niedrigere finanzielle Hürden für Patienten einzuführen. Kosten und Qualitätsaspekte verdienen weiterhin die meiste Aufmerksamkeit. Um den „Value for Money“ zu erhöhen, könnte eine gezieltere Nutzungsteuerung bei neuen Arzneimitteln etwa durch eine Differenzierung der Erstattungsfähigkeit erwogen werden. Da die Nutzenbewertung bereits auf der Ebene von Subgruppen stattfindet, liegen die hierfür notwendigen Informationen vor und können bei der Entscheidung über die Erstattungsfähigkeit (und nicht nur über den Erstattungspreis) genutzt werden. Pharmaceutical care in the statutory health insurance system remains among the main topics in the health policy debate in Germany, not least regarding the benefit-oriented pricing and reimbursement amount negotiations introduced by the most recent changes in legislation. Prices for newly authorized pharmaceuticals are to depend on patient benefit. The Federal Joint Committee determines whether new patented pharmaceuticals have an additional benefit compared to existing alternatives and what it amounts to. Reimbursement amounts are agreed on by the manufacturers and the Federal Association of Sickness Funds (AMNOG negotiations), unless no additional benefit has been demonstrated and the pharmaceutical can be incorporated into a reference price group. The objective of this systematic country comparison was to contextualize pharmaceutical care in the statutory health insurance system in Germany by illustrating its main characteristics and underlying regulatory mechanisms in relation to other European health systems. The investigation spans measures related to marketing authorization; pricing and price updates; post-marketing evaluations guiding coverage decisions (health technology assessment); patient cost-sharing; specific cost and quality control measures targeting individual stakeholder groups (industry, wholesalers/pharmacists, prescribers); generic substitution; and resulting price levels. Including Germany, a sample of 16 European health systems was selected (Austria, Belgium, Denmark, England, Finland, France, Germany, Greece, Ireland, Italy, the Netherlands, Poland, Portugal, Scotland, Spain, and Sweden). Quantitative data from the OECD, country-specific regulatory documents as well as published and grey literature were combined to form an initial evidence base in the form of health system profiles, which were then sent to relevant experts for review and validation. Compared to the other health systems in the sample, pharmaceutical care in the statutory health insurance system in Germany is characterized by a very short delay between marketing authorization of new pharmaceuticals and their statutory reimbursement (i.e. actual patient access), as well as high availability of such products. While in other health systems reimbursement restrictions based on indication or patient group are not uncommon, virtually all authorized products and all authorized indications per product are eligible for reimbursement in the German statutory system. This comparatively generous reimbursement, in conjunction with moderate copayments, is reflected in the public pharmaceutical expenditure per capita, which is among the highest in the sample. Total pharmaceutical expenditure is also relatively high, both per capita and as a share of GDP. Given that the share of generics in Germany is quite high, it is likely that above-average price levels and the utilization of new (and more expensive) pharmaceuticals contribute to this phenomenon. Thus, enabling better access to innovative pharmaceuticals and lowering financial hurdles for patients do not seem to be the main challenges for the German pharmaceutical market. However, cost and quality aspects merit continued attention. To increase value for money, a more targeted utilization control could be considered, for example by differentiating reimbursement eligibility for newly authorized pharmaceuticals. Since benefit assessments by the Federal Joint Committee already consider evidence at subgroup level, the necessary information is already available and could be used to inform decisions on reimbursement eligibility in addition to pricing.

Eine unabdingbare Voraussetzung für die Beantwortung der Forschungsfragen der Versorgungsforschung sind das Vorhandensein und die Zugänglichkeit von umfassenden Daten hoher Qualität. Es ist davon auszugehen, dass die Versorgungsforschung in den kommenden Jahren auf der Verlinkung (Datenlinkage, „data linkage“), also der Verknüpfung mehrerer Datenquellen auf Basis von geeigneten gemeinsamen Schlüsselvariablen beruhen wird. In Deutschland werden umfassende Daten hoher Qualität routinemäßig gesammelt, allerdings sind diese für Forschungszwecke nur bedingt geeignet und eingeschränkt verfügbar. Daher liegt der Ursprung dieses Gutachtens in den Grundfragen, welche Fragestellungen der Versorgungsforschung im Ausland auf Basis besserer Daten besser beantwortet werden können, und auf welchen Wegen eine solche Verbesserung der Datengrundlage in Deutschland erzielt werden könnte. Fallbeispiele interessanter Verlinkungsansätze aus zwölf Ländern wurden identifiziert und anhand eines vordefinierten Konzeptes analysiert. Informationen wurden für alle Vergleichsländer bzw. Fallstudien zunächst auf Basis von öffentlich zugänglichen Quellen identifiziert. Anschließend wurde pro Land ein Profil erstellt und geeigneten Länderexperten zur Validierung und Ergänzung weitergeleitet. Das Gutachten stellt eine Übersicht der eingeschlossenen Fallstudien vor (Kapitel 3), sowie im Anhang wichtige Eckdaten pro Land und Fallstudie. Es folgen eine nähere Betrachtung der Möglichkeiten der Nutzung von Routinedaten (Kapitel 4), der Verlinkungsmöglichkeiten mit und ohne eindeutigem personenbezogenen Merkmal (Kapitel 5), der unterschiedlichen Zugriffswege für Forscher (Kapitel 6), der Ausgestaltung des Einsatzes von Daten aus elektronischen Patienten- bzw. Gesundheitsakten für die Forschung (Kapitel 7) und ein Zwischenfazit (Kapitel 8). Das Gutachten endet mit einer Wunschliste für mögliche regulatorische Änderungen in Anbetracht der aktuellen politischen Diskussion (Kapitel 9). Wichtig ist hier zu beleuchten, dass die thematisierten Fallstudien aus dem internationalen Vergleich wichtige Aspekte hervorheben, die für die Problematik in Deutschland von Bedeutung sein könnten. Neben dem Mehrwert von vergleichsmäßig einfach zugänglichen, umfangreiche(re)n Abrechnungsdaten, die schneller als in Deutschland vorliegen, sektorenübergreifende Analysen erlauben und personenbezogen mit Gesundheits- sowie auch mit Regionaldaten verknüpfbar sind, wird auch das Potential von Datenbanken beleuchtet, die auf Basis der routinemäßigen Dokumentation (i.d.R. elektronischen Gesundheitsakten) von Leistungserbringern entstehen. Eine Investition in diese Richtung kann nicht nur für das eigene Gesundheitssystem Einsichten ermöglichen, sondern auch die internationale Kooperation fördern und zur internationalen Sichtbarkeit wissenschaftlicher Exzellenz beitragen. Eine Nebeneinanderstellung der in Deutschland vorhandenen Datenbestände und der in anderen Ländern eingesetzten Daten deutet darauf hin, dass sich die Diskussion hierzulande an erster Stelle mit der Ausgestaltung der zur Verfügung stehenden Daten befassen sollte und ergänzend die Erfassung zusätzlicher Informationen bedenken kann. The availability and accessibility of comprehensive, high-quality data are indispensable prerequisites for effective health services research. It can be assumed in the coming years that health services research will increasingly be based on data linkage, i.e. the linking of several data sources based on suitable common key variables. In Germany, comprehensive high-quality data are routinely collected, but their suitability and availability for research purposes is limited. Therefore, the motivation for this report lies in the answering of two basic questions: which questions of health services research can be better answered abroad on the basis of better data, and in which ways such conditions could be achieved in Germany. Case studies of interesting linkage approaches from twelve countries were identified and analyzed based on a predefined conceptual framework. Information on all comparison countries or case studies was first identified from publicly available sources. Profiles were then created for each country and case study and forwarded to appropriate country experts for validation and completion. The report presents an overview of the included case studies (chapter 3), as well as key information per country and case study in the appendix. This is followed by a closer look at the possibilities of using routine data (chapter 4), linkage approaches with and without unique personal identifiers (chapter 5), the different access routes for researchers (chapter 6), options for using data from electronic patient or health records for research (chapter 7), and an interim conclusion (chapter 8). The report ends with a wish list for possible regulatory changes in light of the current policy debate in Germany (Chapter 9). It is important to underline here that case studies from the international comparison were selected to highlight important aspects that could be relevant to the challenges in Germany. In addition to the added value of comparatively easily accessible, extensive claims data, which are available more quickly than in Germany, allow cross-sector analyses and can be linked to health data and regional data on a person-by-person basis, the potential of databases created on the basis of the routine documentation of service providers (usually electronic health records) is also discussed. Investing in this direction can not only provide insights for a country’s own healthcare system, but also promote international cooperation and contribute to the international visibility of scientific excellence. A juxtaposition of the data sets available in Germany and those used in other countries suggests that the discussion in Germany should focus first and foremost on optimizing the availability and accessibility of existing data and consider new approaches to the collection of additional information as a supplement.

1. Although the primary responsibility for health systems within the European Union (EU) lies with its Member States, the EU also has many tools that can support the strengthening of health systems.2. Many of the EU's tools can provide support even though strengthening health systems is not their primary objective, such as the European Semester, the Cohesion Policy Funds, Horizon Europe and the Recovery and Resilience Facility.3. The available tools address different parts of change processes. Making best use of these instruments typically requires combining various EU tools with different objectives across multiple stages of the change process.4. The need to combine diverse tools creates the challenge for Member States of being aware of many different tools and their potential to support health systems, and in aligning objectives and processes between health objectives and the requirements of those tools. There are some examples of technical assistance from the EU to help with doing this, although none are specifically focused on health systems strengthening.5. There is potential to combine support from the EU with support from other sources, such as national and regional instruments or other international bodies like the World Health Organization (WHO), although this also presents countries with the challenge of combining instruments with diverse objectives and processes.6. The EU's support to health systems respects the primary responsibility of EU countries for their own health systems. Nevertheless, being able to draw on EU support has been increasingly important, in particular for Member States that have joined since 2004, and will become even more vital in the coming years.7. As health systems across Europe deal with the consequences of the COVID-19 pandemic, there is scope for greater collaboration between individual countries and at EU level to make best use of EU tools to strengthen health systems.

1. Public payers and pharmaceutical manufacturers have a shared interest in enabling timely access to necessary and innovative medicines. At the same time, there is a tension between the interests of purchasers (to contain costs) and of manufacturers (to provide return on investment and maximize profits).2. The most common pricing mechanisms in Europe - such as external reference pricing (ERP) and managed entry agreements (MEAs) - tend to create incentives for pharmaceutical companies to give confidential discounts on the official list prices. This obscures the actual prices paid for medicines.3. Net price transparency (NPT) for pharmaceuticals - the public disclosure of prices paid to manufacturers - is seen by many as key to overcoming the opacity of pharmaceutical systems and to addressing the imbalance between countries with different levels of negotiating power (as larger or smaller purchasers, or richer or poorer countries). It is also regarded as a means of ensuring that public payers purchasing pharmaceuticals for their populations can be held accountable.4. However, policy action on NPT is not straightforward:1.1. Empirical evidence on the effect of NPT is extremely limited. Economic simulations do not provide a clear answer and evidence from natural experiments only comes from very specific settings, not least because NPT policies have never been fully implemented.1.2. Some policy-makers are concerned that moves towards increased price transparency would have a negative impact on accessibility, because pharmaceutical companies may then withdraw from markets or set prices at unaffordable levels, in particular in cases of less attractive (e.g. smaller) markets.1.3. Payers in different health systems may consider measures to increase price transparency for pharmaceuticals as more or less necessary to increase affordable access to medicines.5. Any movement towards NPT requires a re-examination of the established trade-offs in pharmaceutical policy in Europe and worldwide. It is important to consider:1.1. the differing needs and negotiating capacities across countries1.2. the complexities of the interactions between stakeholders1.3. the particularities of specific market segments1.4. the way the process of implementation may shape the policy's impact1.5. the consequences for different countries and the 'knock on' implications of these effects for availability and affordability in other (national) health systems1.6. the likely implications for innovation.6. Increasing transparency in the pharmaceutical system will require greater European and international collaboration - strengthening and going beyond existing initiatives. It also demands a clear focus on maintaining access, innovation and sustainability. Recent experience with joint purchasing, such as in the case of the COVID-19 vaccines, may bolster similar initiatives in future.

1. Policy-makers and purchasers increasingly face very high price tags for new medicines, and some worry that these prices challenge the financial sustainability of solidarity-based, publicly funded health care systems.2. EU Member States use a mix of policy instruments to regulate the prices and determine the reimbursement of medicines. Different policies are applied for different sectors, market segments and medicines. Some pricing, procurement and reimbursement policies are more frequently used for new, potentially high-priced medicines.3. Popular policies and tools include external price referencing, managed entry agreements, health technology assessment (HTA) and tendering, but each one of these comes with limitations.4. Adaptations of these policies, such as value-based pricing, strategic procurement and differential pricing traits, are also being explored and could be a first step in this direction.5. Further, more far-reaching measures and initiatives may also be needed to fundamentally tackle the issue of high medicine prices and to overcome information asymmetry and a lack of transparency about the real prices paid and the real development costs of medicines.6. Collaborative efforts, at both intra-country and cross-country levels, in terms of joint price negotiations and procurement and enhanced collaboration for horizon scanning and HTA appear to be promising but require strong commitment from national policy-makers.7. Pricing, reimbursement and procurement policies are important elements in the 'tool-box' to improve affordable access to new high-priced medicines but further interventions in other pharmaceutical policy areas may be required to supplement them.

1. The development of innovative medicines is essential for making progress in preventing and treating diseases. However, the high price tags put on new medicines do not always reflect the value added for patients. Also, persisting unmet clinical need in the population suggests a misalignment of pharmaceutical research and development efforts.2. There is a growing consensus that existing policies need to be rethought and new approaches need to be found to strike the delicate balance between stimulating true innovation, particularly towards addressing unmet needs, and ensuring both financial sustainability for health systems and accessibility for patients.3. While small and medium-sized enterprises (SMEs), academia and public institutions play a major role in driving innovation and enriching the industry's pipeline, the commercialization of new products is almost exclusively in the hands of large companies, often as a result of mergers and acquisitions. This is a concern for the sustainability of pharmaceutical research and development (R&D) infrastructure.4. The impact of public funding on pharmaceutical innovation cannot be underestimated. A stronger implementation of public interest provisions along the life cycle of pharmaceuticals, including "fair return of investment", is required.5. For public finance of R&D to facilitate better alignment of innovation with unmet clinical needs, more discrimination could be introduced in the reward system. Through limiting the risks, reducing R&D costs and/or increasing the innovation potential, incentives can be created for industry to re-embrace revolutionary - or even disruptive - innovation.6. More can also be done to improve coordination and priority-setting across R&D efforts, ideally globally, but with further refinement from an EU level to reflect regional priorities. In this respect, the creation of an entity to monitor clinical need, in conjunction with inequalities in access to essential medicines, merits consideration.7. Improving the efficiency of evidence generation in clinical research is not only good for driving down the costs of clinical trials, it can also help to remediate some of the related technical and ethical challenges, such as the fragmentation and duplication that unnecessarily expose patients to risk; the lack of comparative effectiveness data; the evidence gaps regarding specific patient groups and therapeutic areas; or the perceived conflicts of interest and related publication bias.8. Raising the bar for market entry by requiring that a new product demonstrate its superiority or equivalence to existing alternatives could encourage manufacturers to focus more on areas with limited treatment options and facilitate increased alignment with specifications applied in post-marketing evaluations for pricing and/or reimbursement (for example, Health Technology Assessment). Increased collaboration and alignment on evidentiary requirements between and within EU Member States are likely to simplify evidence generation for manufacturers as well as increase efficiency on the evaluators' side.9. Only a comprehensive approach that combines initiatives to guarantee funding, optimize evidence generation and align regulatory requirements can effectively tackle innovation deficits. An overall vision with greater policy coherence and backed by strong political commitment and transparency is needed.