A Pipeline of Therapies for Cystic Fibrosis

被引：18

作者：

Ashlock, Melissa A. ^{[1
]}

Beall, Robert J. ^{[2
]}

Hamblett, Nicole M. ^{[3
,4
]}

Konstan, Michael W. ^{[5
]}

Penland, Christopher M. ^{[2
]}

Ramsey, Bonnie W. ^{[3
,4
]}

Van Dalfsen, Jill M. ^{[4
]}

Wetmore, Diana R. ^{[1
]}

Campbell, Preston W., III ^{[2
]}

机构：

[1] Cyst Fibrosis Fdn Therapeut Inc, Bethesda, MD 20814 USA

[2] Cyst Fibrosis Fdn, Bethesda, MD USA

[3] Univ Washington, Dept Pediat, Seattle, WA 98195 USA

[4] Cyst Fibrosis Fdn Therapeut Inc, Seattle Childrens Res Inst, Therapeut Dev Network Coordinating Ctr, Seattle, WA USA

[5] Case Western Reserve Sch Med, Cleveland, OH USA

来源：

SEMINARS IN RESPIRATORY AND CRITICAL CARE MEDICINE | 2009年 / 30卷 / 05期

关键词：

Cystic fibrosis; drug development; clinical trials; SURROGATE END-POINTS; DRUG DEVELOPMENT; PULMONARY GUIDELINES; OUTCOME MEASURES; CLINICAL-TRIALS;

D O I：

10.1055/s-0029-1238919

中图分类号：

R4 [临床医学];

学科分类号：

1002 ; 100602 ;

摘要：

Therapeutics development for cystic fibrosis (CF) involves a coordinated effort among many groups, including individuals with CF and their caregivers, clinical research teams, and those in academia and industry who have discovered and developed the therapeutic strategies. In the United States, the Cystic Fibrosis Foundation (CFF) has devoted over $875 million to facilitate and coordinate this process since 1986, resulting in the clinical development and/or assessment of similar to 50 drug candidates during that time. The more than 30 compounds currently in the pipeline of Foundation-funded therapeutics are used as a platform to discuss why and how therapeutic strategies are brought into clinical development. Consideration is also given to the funding, management, and infrastructure necessary and practical to support the progression of drug candidates and the availability of therapeutics for use by individuals with CF. The importance of the clinical trial process and relevant outcome measures to assess the efficacy of drug candidates is also discussed. Finally, the potential impact of the pipeline for individuals with CF is summarized.

引用

页码：611 / 626

页数：16

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