PDF(Pubmed)
Abstract：
Treatments for emerging and rare invasive fungal diseases (IFDs) represent a critical unmet medical need. For IFDs that occur less frequently than invasive aspergillosis, such as mucormycosis, hyalohyphomycosis, and phaeohyphomycosis, randomized controlled clinical trials are impractical and unlikely to meet urgent public health needs. Understanding regulatory approaches for approval of drugs for rare cancers and rare metabolic diseases could help meet the challenges of studying drugs for rare IFDs. A single-arm, controlled clinical trial with a high-quality external control(s), with confirmatory evidence from nonclinical studies, including pharmacokinetic/pharmacodynamic data in predictive animal models of the disease may support findings of effectiveness of new drugs and biologics. Control populations may include historical controls from published literature, patient registries, and/or contemporaneous external control groups. Continuous engagement among clinicians, industrial sponsors, and regulatory agencies to develop consensus on trial design and innovative development pathways for emergent and rare invasive fungal diseases is important.
摘要：
新出现的和罕见的侵袭性真菌病(IFD)的治疗代表了关键的未满足的医疗需求。对于发生频率低于侵袭性曲霉病的IFD，如毛霉菌病，透明真菌病,和phaeophyphysporcosis，随机对照临床试验是不切实际的,不可能满足紧迫的公共卫生需求.了解批准用于罕见癌症和罕见代谢疾病的药物的监管方法可能有助于应对研究用于罕见IFD的药物的挑战。单臂,具有高质量外部对照的对照临床试验，非临床研究的确证证据,包括疾病预测动物模型中的药代动力学/药效学数据可能支持新药和生物制剂有效性的发现。控制人群可能包括已发表文献中的历史控制，病人登记处,和/或同期外部对照组。临床医生之间的持续参与，工业赞助商,和监管机构就紧急和罕见侵袭性真菌病的试验设计和创新开发途径达成共识非常重要。
