Methodological Issues In Modeling Hemophilia Treatment - What We Have Learnt In the Course of a Decade and What We Must Learn In the Decade to Come

The challenges faced by the hemophilia community because of economic constraints will increase in the future. Without disease specific pharmacoeconomic models to inform the decision process important decisions may be taken without taking hemophilia specific issues into account.

To investigate recent advances and current state-of-the-art knowledge in pharmacoeconomic modeling of hemophilia care we conducted a targeted literature review.

Searches were conducted in MEDLINE®, and NHS Economic Evaluation Database using combinations of disease, and economic-related key words, limited to articles published in English between January 1970 and June 2010 (inclusive). A total of 127 articles (from 284 abstracts retrieved) were selected for detailed consideration.

Prior to the year 2000 the focus of the cost side of hemophilia treatment was not given much attention in the scientific literature. However in the last decade pharmacoeconomics have grown in importance and an increasing number of publications specific for this aspect of hemophilia reflect this trend. Still there is limited data on probabilities, costs and outcomes and remarkably few studies comparing QALY estimates of factor VIII prophylaxis in hemophilia. More studies are available specific for the limited inhibitor segment. Recommendations for reporting economic evaluations of hemophilia prophylaxis has been proposed and important ground clearing work has been published on theoretical aspects of hemophilia modeling.

Economic analyses in hemophilia care are limited, but are increasingly in demand to examine the costs and outcomes associated with various treatment regimens. Unresolved questions critical to hemophilia remains such as the appropriate weighting of short term vs. future benefits and equity considerations associated with hemophilia being a rare genetic disease affecting children. Trials designed with pharmacoeconomic objectives in mind will be imperative. Data on generic and disease specific quality of life and resource use/cost data will provide whole new insights to how hemophilia impacts the lives of people living with this rare disease and will provide the data needed to assess the total value of different treatment regimens relevant for hemophilia.

Strandberg-Larsen:Novo Nordisk A/S: Employment.