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- Title
Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies.
- Authors
Pierce, G. F.; Ragni, M. V.; Berg, H. M.; Weill, A.; O'Mahony, B.; Skinner, M. W.; Pipe, S. W.
- Abstract
The authors emphasize the need for a drug evaluation standard that can directly measure the effect of the applied gene therapy for hemophilia. Topics covered include blood coagulation factor VIII and factor IX activity levels, limitations of annualized bleeding rate (ABR) in prelicensure studies of new factor VIII and IX products, and proposal for gene therapy trials to utilize clotting factor activity as primary endpoint to assess efficacy than ABR.
- Subjects
HEMOPHILIA treatment; GENE therapy; BLOOD coagulation factor VIII; BLOOD coagulation factor IX; PHARMACEUTICAL research
- Publication
Haemophilia, 2017, Vol 23, Issue 5, p643
- ISSN
1351-8216
- Publication type
Editorial
- DOI
10.1111/hae.13313