{"title":"孤儿药法案》40 周年：立法的胜利与成功的挑战","authors":"PETER SALTONSTALL, HEIDI ROSS, PAUL T. KIM","doi":"10.1111/1468-0009.12680","DOIUrl":null,"url":null,"abstract":"<div>\n<div>\n<h3>Policy Points</h3>\n<p>\n</p><ul>\n<li>The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration–approved treatment.</li>\n<li>The ODA's success led to sustained criticism of high drug prices, often for products that have orphan drug indications. Critics misconstrue the ODA's intent and propose reducing its incentives instead of pursuing policies focused on addressing broader prescription drug price challenges that exist in both the orphan and nonorphan drug market.</li>\n<li>Patients and their families will continue to defend the purpose and integrity of the ODA and to drive investments into rare disease research and clinical development.</li>\n</ul><p></p></div>\n</div>","PeriodicalId":501846,"journal":{"name":"The Milbank Quarterly","volume":"34 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2023-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success\",\"authors\":\"PETER SALTONSTALL, HEIDI ROSS, PAUL T. KIM\",\"doi\":\"10.1111/1468-0009.12680\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n<div>\\n<h3>Policy Points</h3>\\n<p>\\n</p><ul>\\n<li>The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration–approved treatment.</li>\\n<li>The ODA's success led to sustained criticism of high drug prices, often for products that have orphan drug indications. Critics misconstrue the ODA's intent and propose reducing its incentives instead of pursuing policies focused on addressing broader prescription drug price challenges that exist in both the orphan and nonorphan drug market.</li>\\n<li>Patients and their families will continue to defend the purpose and integrity of the ODA and to drive investments into rare disease research and clinical development.</li>\\n</ul><p></p></div>\\n</div>\",\"PeriodicalId\":501846,\"journal\":{\"name\":\"The Milbank Quarterly\",\"volume\":\"34 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2023-12-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"The Milbank Quarterly\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1111/1468-0009.12680\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"The Milbank Quarterly","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1111/1468-0009.12680","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
政策要点《孤儿药法案》（ODA）是患者倡导的结果，从许多方面来看都取得了巨大成功。然而，在已知的 7,000 多种罕见病中，仍有约 95% 的罕见病没有得到美国食品药品管理局批准的治疗方法。ODA 的成功导致了对高药价的持续批评，而高药价往往是针对孤儿药适应症产品的。批评者曲解了 ODA 的初衷，并建议减少其激励措施，而不是推行侧重于解决孤儿药和非孤儿药市场中存在的更广泛的处方药价格挑战的政策。患者及其家属将继续捍卫 ODA 的宗旨和完整性，并推动对罕见病研究和临床开发的投资。
The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success
Policy Points
The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration–approved treatment.
The ODA's success led to sustained criticism of high drug prices, often for products that have orphan drug indications. Critics misconstrue the ODA's intent and propose reducing its incentives instead of pursuing policies focused on addressing broader prescription drug price challenges that exist in both the orphan and nonorphan drug market.
Patients and their families will continue to defend the purpose and integrity of the ODA and to drive investments into rare disease research and clinical development.
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