Journal article
Joint WFH‐ISTH session: issues in clinical trial design
Abstract
Haemophilia therapy is experiencing an unprecedented expansion in the number and novelty of clotting factor concentrates. Every product must be licensed by regulatory authorities, primarily on the basis of its safety and efficacy profiles. The low prevalence of haemophilia, and other inherited bleeding disorders, presents a significant challenge to patient recruitment for preauthorization clinical trials, especially given the low frequency of …
Authors
Peyvandi F; Farrugia A; Iorio A; Key NS; Srivastava A
Journal
Haemophilia, Vol. 20, No. s4, pp. 137–144
Publisher
Wiley
Publication Date
May 2014
DOI
10.1111/hae.12415
ISSN
1351-8216