ASSESSING THE COST-EFFECTIVENESS OF PRIMARY PROPHYLAXIS FOR THE TREATMENT OF SEVERE HAEMOPHILIA A.

Author(s)

Axelsen F1, Miners A21Baxter Healthcare SA, Zurich, Switzerland, 2London School of Hygiene and Tropical Medicine, London, United Kingdom

OBJECTIVES: The objective of this analysis was to understand the economic consequences of different products in primary prophylaxis in severe Haemophilia A patients. Miners’ (2002, 2009) haemophilia economic Markov model was revised to compare two different factor VIII products - a full-length recombinant FVIII (Advate) and a B domain-deleted recombinant FVIII (BDDrFVIII) (Refacto AF). Based on documented differences in half life in a pharmacokinetic cross-over license study [1] with results submitted to EMA[2], this research evaluated the amount of clotting factor required to maintain the patient factor VIII trough levels above 1%. If a product has a shorter half-life, more clotting factor is required to prevent factor VIII levels falling below the minimum trough level. METHODS: The same basic model structure of Miners’ haemophilia economic Markov model was used to analyse the effects of differences in half-life in an Italian economic country setting, full-length recombinant FVIII (Advate) half-life 13.3 hours at a list price of €0.75 per IU versus BDDrFVIII (Refacto AF) half-life 11.2 hours at a list price of €0.69 per IU. The model considers the amount of factor required to prevent trough clotting factor levels from falling below 1%. RESULTS: The analysis estimated that the mean expected, discounted lifetime (70 years) cost of primary prophylaxis in a severe Haemophilia A patient was €0.88 million less using full length recombinant FVIII (Advate) when compared to a BDDrFVIII (Refacto AF), assuming a 2.1 hour shorter half-life; i.e. equal to over €12,500 in cost savings per patient per year. CONCLUSIONS: This analysis shows that prophylaxis therapy using full-length recombinant FVIII (Advate) may be cost saving compared to BDDrFVIII (Refacto AF) based on the difference in half-life reported, even if BDDrFVIII has a lower price per IU. [1] Recht et al. Haemophilia 2009:1-12; [2] EMA Assessment report Refacto AF. London March 2009. http://www.ema.europa.eu/humandocs/PDFs/EPAR/Refacto/refactoaf-H-232-II-59-68-AR.pdf  

Conference/Value in Health Info

2010-11, ISPOR Europe 2010, Prague, Czech Republic

Value in Health, Vol. 13, No. 7 (November 2010)

Code

PSY36

Topic

Economic Evaluation

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis

Disease

Systemic Disorders/Conditions

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