评估囊性纤维化患者胰酶替代疗法疗效和安全性的研究设计考量
Study design considerations for evaluating the efficacy and safety of pancreatic enzyme replacement therapy in patients with cystic fibrosis.
作者信息
Konstan Michael W, Borowitz Drucy, Mayer-Hamblett Nicole, Milla Carlos, Hendeles Leslie, Murray Susan, Kronmal Richard A, Casey Susan, Rose Lynn M, Morgan Wayne J, Ramsey Bonnie W
机构信息
Case Western Reserve University School of Medicine, Cleveland, OH, USA.
State University of New York at Buffalo, Buffalo, NY, USA ; Cystic Fibrosis Foundation Data Safety Monitoring Board, Tucson, AZ, USA.
出版信息
Clin Investig (Lond). 2013 Aug;3(8):731-741. doi: 10.4155/cli.13.63.
In 2006, the US FDA issued a 'Guidance for Industry' regarding submission of New Drug Applications for pancreatic enzyme replacement therapy (PERT) products. Five oral delayed-release PERT products have been approved by the FDA, and several others are under development and/ or evaluation for New Drug Application submission. We present in this paper recommendations of the Cystic Fibrosis Foundation's Cystic Fibrosis (CF) Therapeutics Development Network and Data Safety Monitoring Board regarding study design considerations for evaluating PERT products in patients with CF. Careful attention to study design and accuracy of the outcome measures has confirmed our understanding of the efficacy and safety of PERT for the treatment of exocrine pancreatic insufficiency of CF.
2006年，美国食品药品监督管理局（US FDA）发布了一份关于提交胰酶替代疗法（PERT）产品新药申请的“行业指南”。美国食品药品监督管理局已批准了五种口服缓释PERT产品，还有其他几种产品正在研发和/或评估，准备提交新药申请。我们在本文中介绍了囊性纤维化基金会的囊性纤维化（CF）治疗研发网络和数据安全监测委员会关于评估CF患者PERT产品的研究设计考量的建议。对研究设计和结果测量准确性的仔细关注，证实了我们对PERT治疗CF外分泌性胰腺功能不全的疗效和安全性的理解。
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