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ISTH e-Newsletter: Factor 8 and Factor 9 Post-registration Surveillance
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Call for Participants! SSC Factor VIII and IX Subcommittee Project: Standardization of Post-registration Surveillance

A SSC Factor VIII and IX Subcommittee project group, led by Prof. Flora Peyvandi, aims to establish the requirements for an international harmonized post-authorization study using existing national and international database infrastructure and consensus standardized minimum datasets. These data would complement limited pre-authorization data on product safety and hemostatic efficacy, as well as contribute to a greater understanding of immunogenicity and the potential implementation of novel more sensitive antibody detection assays.

In the recent years, many promising strategies are emerging to enhance especially the half-life of therapeutic proteins. These bioengineering strategies have been employed to manufacture novel coagulation factors that prolong the bioavailability with increased potency and resistance to inactivation and potentially reduced immunogenicity. These novel drugs have the potential to dramatically transform the treatment of hemophilia by substantially reducing the frequency of injections.

Promising data from the pre-authorization phase of clinical trials have been presented. Nevertheless, a prolonged period of surveillance will be required to collect more accurate data on the safety and efficacy and additional data to ensure consistency in the long-term of novel drugs. Moreover, the available data on the novel long acting drugs are limited by the relatively few number of patients enrolled in the pre-authorization phase, and for this reason, large-scale post-marketing pharmaco-vigilance studies are necessary to gain sufficient numbers of patients for statistically valid assessments.

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