#MarksManInsights

Hemophilias are a group of inherited disorders of blood clotting wherein the coagulation cascade following injury becomes abnormal due to low amounts of clotting factors such as factor VIII (hemophilia A), factor IX (hemophilia B), and so on. While current management largely involves replacing the missing clotting factors, non-factor therapies (such as monoclonal antibodies and RNA interfaces) and gene therapy are being studied as possible therapeutic options for these disorders. Our client was interested to compile evidence surrounding the efficacy, safety, and patient-reported outcomes of factor replacement therapies and various non-factor therapies (excluding plasma derivatives and supportive therapies) for both hemophilia A and hemophilia B, in terms of bleeding rates and other surrogate markers. For this project, we screened over 2550 articles concerning both the disorders over 2 rounds of screening, based on eligibility criteria shared by the client, and identified the most relevant articles for data extraction, quality assessment, and report preparation.
Therapy area: Hematology