加入患者倡导团体(Patients Advocacy Groups)对于提醒和教育患者参加试验非常有帮助。患者登记和有关组织也可能很有帮助,其中包括罕见病登记(Rare Disease Registry Program)、Orphanet、国家罕见病组织(National Organization for Rare Disorders)和欧洲罕见病组织(European Organization for Rare Disease)。利用这些资源不仅在...
Upcoming: Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub 美东时间2024年10月16日，FDA的罕见病创新中心将与FDA的Reagan-Udall基金会合作，举办一场题为“通过 FDA 罕见病创新中心推进罕见病疗法的发展”的公开会议。此次会议将共同讨论新成立的罕见病创新中心如何与罕见病社区成员进行最...
罕见儿科疾病优先审评券计划(Rare Pediatric Disease Priority Review Voucher Program)是指,根据《联邦食品、药品和化妆品法案》(《Federal Food, Drug, and Cosmetic Act(FD&C Act)》)第529款规定,获得治疗“罕见儿科疾病”药品或生物制品批准的发起人可以获得一张优先审评券(voucher),该券可以使发起人随后递交的...
FDA和杜克-马戈利斯卫生政策中心（Duke-Margolis Center for Health Policy）将于2023年6月举办一个关于推进新型罕见病终点的研讨会，因为FDA准备启动其罕见病终点推进（Rare Disease Endpoint Advancement Pilot Program ，RDEA）试点计划。据悉，美国《2022年食品和药物综合改革法案》要求FDA建立RDEA试点项目，以增加与罕...
Maynard began stating that of the 37 NME approvals for 2022, 20 were rare diseases. She updated the audience on the CDER-wide effort to connect the new drugs office, office of the Center Director and translational science. That is the Accelerating Rare disease Cures (ARC) Program, which is...
Background: The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children. Prior to requesting a voucher, applicants can request RPD designation, which confirms their product treats or prevents a rare disease in...
Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity. Driven by the calling to “Reimagine Possible”, Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in ...
2024年8月21日，FDA宣布了其罕见神经退行性疾病资助计划 (FDA Rare Neurodegenerative Disease Grant Program)的一个新资助机会，用于资助因解决儿童和成人在罕见神经退行性疾病方面未满足的需求而进行的新适应症开发或标签变更时所进行的疗效和/或安全性评估的临床试验。通过在协作、高效、创新的临床试验方面的资助，FDA...
FDA Rare Neurodegenerative Disease Grants Program 2024年10月1日,FDA宣布其2024财年在罕见神经退行性疾病资助计划(Rare Neurodegenerative Disease Grants Program)下将有超过540万美元的预算用来支持罕见神经退行性疾病(RNDD)的临床研究。2024财年,该计划下FDA收到了15份资助申请,已批准了一项用于皮质下梗死伴白质脑病...
Funding opportunities for rare disease research 2024年8月21日,FDA宣布了其罕见神经退行性疾病资助计划 (FDA Rare Neurodegenerative Disease Grant Program)的一个新资助机会,用于资助因解决儿童和成人在罕见神经退行性疾病方面未满足的需求而进行的新适应症开发或标签变更时所进行的疗效和/或安全性评估的临床试验。通...