Lucas Cortial, Pierre-Olivier Boyer, Sylvain Forget, Ronan Le Joubioux, O. Blin, F. Mouthon
{"title":"罕见病:患者可持续获得治疗的具体挑战","authors":"Lucas Cortial, Pierre-Olivier Boyer, Sylvain Forget, Ronan Le Joubioux, O. Blin, F. Mouthon","doi":"10.20517/rdodj.2022.08","DOIUrl":null,"url":null,"abstract":"In late 2021, the health technology assessment of the French National Authority for Health was seized by the French Ministry of Solidarity and Health to address a specific challenge, the identification of solutions contributing to the development of the methodological expertise in new types of clinical studies for rare diseases. Experts from the rare diseases environment were gathered by OrphanDev, the French network of expertise dedicated to rare diseases. They allowed to identify some of the current issues in France concerning rare diseases, and then present different solutions, in particular related to the evaluation process of orphan drugs and the collection of data on rare diseases.","PeriodicalId":74638,"journal":{"name":"Rare disease and orphan drugs journal","volume":"1 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Rare diseases: specific challenges for sustainable accessibility of treatments for patients\",\"authors\":\"Lucas Cortial, Pierre-Olivier Boyer, Sylvain Forget, Ronan Le Joubioux, O. Blin, F. Mouthon\",\"doi\":\"10.20517/rdodj.2022.08\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"In late 2021, the health technology assessment of the French National Authority for Health was seized by the French Ministry of Solidarity and Health to address a specific challenge, the identification of solutions contributing to the development of the methodological expertise in new types of clinical studies for rare diseases. Experts from the rare diseases environment were gathered by OrphanDev, the French network of expertise dedicated to rare diseases. They allowed to identify some of the current issues in France concerning rare diseases, and then present different solutions, in particular related to the evaluation process of orphan drugs and the collection of data on rare diseases.\",\"PeriodicalId\":74638,\"journal\":{\"name\":\"Rare disease and orphan drugs journal\",\"volume\":\"1 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2022-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Rare disease and orphan drugs journal\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.20517/rdodj.2022.08\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Rare disease and orphan drugs journal","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.20517/rdodj.2022.08","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Rare diseases: specific challenges for sustainable accessibility of treatments for patients
In late 2021, the health technology assessment of the French National Authority for Health was seized by the French Ministry of Solidarity and Health to address a specific challenge, the identification of solutions contributing to the development of the methodological expertise in new types of clinical studies for rare diseases. Experts from the rare diseases environment were gathered by OrphanDev, the French network of expertise dedicated to rare diseases. They allowed to identify some of the current issues in France concerning rare diseases, and then present different solutions, in particular related to the evaluation process of orphan drugs and the collection of data on rare diseases.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
