The Yale Cancer Center has recently presented the findings of their study of the cost-effectiveness of caplacizumab at the 62nc ASH Annual Meeting and Exposition. Their conclusion, which is that caplacizumab is ultimately not the most cost-effective treatment option for thrombotic thrombocytopenia purpura (TTP), can help doctors do the best thing for their patients and the general health system.
About Thrombotic Thrombocytopenia Purpura (TTP)
TTP is a rare and severe blood disease that is characterized by small blood clots that form in the smaller arteries of the body. These clots cause symptoms like headaches, mental changes, speech abnormalities, coma, confusion, seizures, slight or partial paralysis, proteinuria, fever, hematuria, rash-like patches on the skin (purpura), weakness, fatigue, hemorrhaging, pallor, nausea, vomiting, and abdominal pain. Medical professionals are unsure as to what causes this condition, but they believe that there is a familial form and non-inherited form. Regardless of cause, treatment consists of plasmapheresis, steroids, Cablivi, and the blood product SD plasma.
About the Study
The purpose of this study was to evaluate whether the addition of caplacizumab to the current standard of care for TTP was cost-effective. It is important to analyze this, as cost-effective treatment is for the best for the community, patients, and the entire healthcare system.
Researchers at the Yale Cancer Center developed multiple cost models with the use of data from the TITAN and HERCULES studies, two major trials of TTP treatments. These models were able to incorporate the price of multiple TTP therapies, including caplacizumab, therapeutic plasma exhange, and more.
By the end of the study, researchers discovered that the addition of caplacizumab to the current standard of care made the incremental cost effectiveness ratio $1.5 million dollars at 5 years with a 95% confidence interval of $1.3-1.7 million. This is much above the current standard in the United States, which is $195,330.
Hopefully more research will be conducted so that medical professionals can best understand both the effects and cost effectiveness of caplacizumab. Read more about this study here.