{"title":"欧盟罕见病患者药品的获取和定价：行业视角。2025年更新。","authors":"Giovanni Tafuri, Andrea Bracco, Jens Grueger","doi":"10.1080/14737167.2025.2479131","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>In a previous publication, we provided an overview of the current situation of patient access to orphan drugs in the European Union (EU), of the evolving rare disease policy landscape and recommendations for areas of improvement throughout the lifecycle of orphan drugs. This article aims to provide an update on evolving new policies impacting access to orphan drugs in the European Union from a health technology developer perspective.</p><p><strong>Areas covered: </strong>We provide an update on the emerging competitiveness gap in the pharmaceutical sector between the EU and the United States. We discuss the latest developments of the European Commission's revision proposal of the EU's pharmaceutical legislation and the threats and opportunities of the new HTA Regulation with a focus on orphan drugs. Additionally, we present the latest policy updates from Germany and the United Kingdom impacting orphan drug developers.</p><p><strong>Expert opinion: </strong>While our initial set of recommendations remains unchanged, the policy landscape for orphan drugs seems to be deteriorating both at the EU and at the Member State level.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-7"},"PeriodicalIF":1.8000,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective. A 2025 update.\",\"authors\":\"Giovanni Tafuri, Andrea Bracco, Jens Grueger\",\"doi\":\"10.1080/14737167.2025.2479131\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>In a previous publication, we provided an overview of the current situation of patient access to orphan drugs in the European Union (EU), of the evolving rare disease policy landscape and recommendations for areas of improvement throughout the lifecycle of orphan drugs. This article aims to provide an update on evolving new policies impacting access to orphan drugs in the European Union from a health technology developer perspective.</p><p><strong>Areas covered: </strong>We provide an update on the emerging competitiveness gap in the pharmaceutical sector between the EU and the United States. We discuss the latest developments of the European Commission's revision proposal of the EU's pharmaceutical legislation and the threats and opportunities of the new HTA Regulation with a focus on orphan drugs. Additionally, we present the latest policy updates from Germany and the United Kingdom impacting orphan drug developers.</p><p><strong>Expert opinion: </strong>While our initial set of recommendations remains unchanged, the policy landscape for orphan drugs seems to be deteriorating both at the EU and at the Member State level.</p>\",\"PeriodicalId\":12244,\"journal\":{\"name\":\"Expert Review of Pharmacoeconomics & Outcomes Research\",\"volume\":\" \",\"pages\":\"1-7\"},\"PeriodicalIF\":1.8000,\"publicationDate\":\"2025-03-19\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Expert Review of Pharmacoeconomics & Outcomes Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/14737167.2025.2479131\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Review of Pharmacoeconomics & Outcomes Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/14737167.2025.2479131","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective. A 2025 update.
Introduction: In a previous publication, we provided an overview of the current situation of patient access to orphan drugs in the European Union (EU), of the evolving rare disease policy landscape and recommendations for areas of improvement throughout the lifecycle of orphan drugs. This article aims to provide an update on evolving new policies impacting access to orphan drugs in the European Union from a health technology developer perspective.
Areas covered: We provide an update on the emerging competitiveness gap in the pharmaceutical sector between the EU and the United States. We discuss the latest developments of the European Commission's revision proposal of the EU's pharmaceutical legislation and the threats and opportunities of the new HTA Regulation with a focus on orphan drugs. Additionally, we present the latest policy updates from Germany and the United Kingdom impacting orphan drug developers.
Expert opinion: While our initial set of recommendations remains unchanged, the policy landscape for orphan drugs seems to be deteriorating both at the EU and at the Member State level.
期刊介绍：
Expert Review of Pharmacoeconomics & Outcomes Research (ISSN 1473-7167) provides expert reviews on cost-benefit and pharmacoeconomic issues relating to the clinical use of drugs and therapeutic approaches. Coverage includes pharmacoeconomics and quality-of-life research, therapeutic outcomes, evidence-based medicine and cost-benefit research. All articles are subject to rigorous peer-review.
The journal adopts the unique Expert Review article format, offering a complete overview of current thinking in a key technology area, research or clinical practice, augmented by the following sections:
Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results
Article Highlights – an executive summary of the author’s most critical points.
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