Silvia Manea, Laura Visonà Dalla Pozza, Cinzia Minichiello, Linda Altieri, Monica Mazzucato, Mauro Bonin, Paola De Ambrosis, Elio Borgonovi, Paola Facchin
{"title":"治疗罕见病的高成本药物:基于地区研究的支出和价值。","authors":"Silvia Manea, Laura Visonà Dalla Pozza, Cinzia Minichiello, Linda Altieri, Monica Mazzucato, Mauro Bonin, Paola De Ambrosis, Elio Borgonovi, Paola Facchin","doi":"10.1177/09514848231151814","DOIUrl":null,"url":null,"abstract":"<p><p><b>Background</b>: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). <b>Objectives</b>: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. <b>Methods</b>: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs' list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. <b>Results</b>: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million (\"blockbuster drugs\"). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. <b>Conclusions</b>: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing \"blockbuster drugs\". Some therapeutical areas seem to be lacking in the drug research field.</p>","PeriodicalId":45801,"journal":{"name":"Health Services Management Research","volume":null,"pages":null},"PeriodicalIF":1.6000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study.\",\"authors\":\"Silvia Manea, Laura Visonà Dalla Pozza, Cinzia Minichiello, Linda Altieri, Monica Mazzucato, Mauro Bonin, Paola De Ambrosis, Elio Borgonovi, Paola Facchin\",\"doi\":\"10.1177/09514848231151814\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p><b>Background</b>: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). <b>Objectives</b>: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. <b>Methods</b>: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs' list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. <b>Results</b>: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million (\\\"blockbuster drugs\\\"). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. <b>Conclusions</b>: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing \\\"blockbuster drugs\\\". Some therapeutical areas seem to be lacking in the drug research field.</p>\",\"PeriodicalId\":45801,\"journal\":{\"name\":\"Health Services Management Research\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-02-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Health Services Management Research\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/09514848231151814\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/1/10 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH POLICY & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Health Services Management Research","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/09514848231151814","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/1/10 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH POLICY & SERVICES","Score":null,"Total":0}
High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study.
Background: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). Objectives: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. Methods: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs' list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. Results: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million ("blockbuster drugs"). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. Conclusions: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing "blockbuster drugs". Some therapeutical areas seem to be lacking in the drug research field.
期刊介绍:
Health Services Management Research (HSMR) is an authoritative international peer-reviewed journal which publishes theoretically and empirically rigorous research on questions of enduring interest to health-care organizations and systems throughout the world. Examining the real issues confronting health services management, it provides an independent view and cutting edge evidence-based research to guide policy-making and management decision-making. HSMR aims to be a forum serving an international community of academics and researchers on the one hand and healthcare managers, executives, policymakers and clinicians and all health professionals on the other. HSMR wants to make a substantial contribution to both research and managerial practice, with particular emphasis placed on publishing studies which offer actionable findings and on promoting knowledge mobilisation toward theoretical advances.