@entity4407 ( @entity4407 ) is a rare, @entity102 of unknown origin characterized by @entity1148 , excessive megakaryocytes, @entity548 , and @entity715 . Because of the high costs of care for @entity1 with @entity395 , policymakers are concerned with both clinical effectiveness and cost-effectiveness of new treatments. Although the clinical efficacy of all new pharmaceutical agents for @entity395 is evaluated extensively in clinical trial settings before approval by the Food and Drug Administration (FDA), comparative phase III trials of a new agent with its major competitor are sometimes not possible to carry out, and estimates of cost-effectiveness are therefore difficult to obtain. We describe methodologic issues associated with the development of economic models of new pharmaceutical agents for @entity395 and illustrate these issues with an analysis of a new therapy for @entity4407 . @entity18829 is a newly approved @entity1423 inhibitor that can be used as primary therapy for @entity4407 . The agent reduces platelet counts by 50% in more than 70% of @entity4407 @entity1 . Economic models suggest that, over the first year of @entity18829 therapy, monthly costs for therapy and complications decreased from  775 to  490, the effectiveness improved to 98%, and the cost-effectiveness improved to  1,505 per major complication ( @entity360 , transient @entity63 attack or @entity64 , or preinfarction @entity951 or @entity583 ) prevented. Sensitivity analyses indicate that, after the first 3 months of treatment, total costs of @entity18829 treatment were in the range of  1,505 to  1,615 per major complication prevented. To make well-informed therapeutic decisions, policymakers and physicians require head-to-head studies of a new pharmaceutical agent with its major competitor. However, economic models can be used to derive estimates of cost-effectiveness of new pharmaceutical agents when such data are lacking. The interpretation of these models raises general issues related to the perspective of the investigator, study design, estimation of costs of care, rates of response, @entity137 , survival, and the ability to generalize the results to other settings, as well as methodologic issues that are unique to @entity395 . If a comparative study found better therapeutic outcomes, then cost-effectiveness models would be of limited usefulness. Almost all physicians would use the drug with the better therapeutic profile.

Cost-effectiveness model of a phase II clinical trial of a new pharmaceutical for essential XXXX : is it helpful to policy makers?

@entity1 :: ('9606', 'Species') :: ['patients', 'persons'] @entity1148 :: ('MESH:D013922', 'Disease') :: ['thrombocytosis'] @entity64 :: ('MESH:D020521', 'Disease') :: ['stroke'] @entity583 :: ('MESH:D009203', 'Disease') :: ['myocardial infarction'] @entity548 :: ('MESH:D006470', 'Disease') :: ['hemorrhages'] @entity1423 :: ('MESH:D001791', 'Disease') :: ['platelet aggregation'] @entity63 :: ('MESH:D007511', 'Disease') :: ['ischemic'] @entity137 :: ('MESH:D064420', 'Disease') :: ['toxicity'] @entity102 :: ('MESH:D002908', 'Disease') :: ['chronic myeloproliferative disorder'] @entity951 :: ('MESH:D000787', 'Disease') :: ['angina'] @entity395 :: ('MESH:D035583', 'Disease') :: ['rare diseases'] @entity4407 :: ('MESH:D013920', 'Disease') :: ['Essential thrombocythemia', 'ET'] @entity18829 :: ('MESH:C021139', 'Chemical') :: ['Anagrelide', 'anagrelide'] @entity360 :: ('MESH:D006471', 'Disease') :: ['gastrointestinal bleed'] @entity715 :: ('MESH:D013927', 'Disease') :: ['thrombotic complications']
@entity1148 :: (MESH:D013922,Disease) :: ['thrombocythemia']
