WFH releases statement on orphan medicine status for hemophilia treatment products
July 2012 – The WFH has communicated to the US Food and Drug Administration and the European Medicines Agency its position on orphan medicine status for hemophilia treatment products. The WFH recommends that orphan drug designation should not be used to hinder, for the same condition or indication, the development, licensing and marketing of other products that are demonstrably different. WFH Statement
The World Federation of Hemophilia Product Selection Statement
February 2012 – The WFH BPSSA and Executive Committee revised and updated this statement in February 2012. It is intended for Ministries of Health and regulatory agencies involved in the selection of treatment products for bleeding disorders. WFH Statement
WFH Statement on Expiry Dates
March 2003 – The World Federation of Hemophilia is an international not-for-profit organization. The WFH is not a regulatory agency and cannot make recommendations relating to specific clotting factor products. Each country’s regulatory authority must make these judgments based on domestic legislation, national health policies and clinical best practices.
The following information may assist regulators in making determinations with regard to clotting factor concentrates and expiry dates. An expiry date on a clotting factor concentrate is a guarantee of potency if stored according to manufacturer’s instructions. After the expiry date, although the manufacturer can no longer guarantee potency, the WFH is not aware of any safety problems with clotting factor concentrates. Clinical experience reported to the WFH suggests that, under appropriate storage conditions, the loss in potency immediately after the expiry date is negligible. Any loss in potency is gradual over months and factor concentrates may still be used safely and effectively in many clinical situations after their labeled expiry date. In critical situations, like life threatening bleeds or major surgery, use of these products should be accompanied by close clinical monitoring and appropriate laboratory assays.
Approved by WFH Executive Committee March 15, 2003