Budget impact of prophylactic treatment of rVIII-SingleChain in moderate and severe hemophilia A in Italy.

INTRODUCTION: rVIII-SingleChain, a recombinant factor VIII (rFVIII), has demonstrated safety and efficacy in patients with hemophilia A in clinical trials and real-world evidence. This analysis aimed to estimate the potential budget impact of increasing the usage of rVIII-SingleChain for the prophylactic treatment of hemophilia A over 3 years in Italy. METHODS: Patients with moderate and severe hemophilia A receiving prophylaxis were included in the analysis. Epidemiological data were obtained from published literature. Mean product consumption and mean annual bleeding rate for rVIII-SingleChain, rFVIIIFc, octocog alfa and BAY 81-8973 were based on pooled real-world data from Italy, Germany and US. A budget impact model has been developed in order to compare two scenarios: a base-case scenario where current rVIII-SingleChain shares are kept constant over 3 years and an alternative scenario where rVIII-SingleChain shares increase by taking from other rFVIII products. Analysis 1 was based on the current Italian list prices and Analysis 2 considered current regional acquisition prices for both scenarios. RESULTS: Annually, adult patients treated with rVIII-SingleChain prophylaxis are expected to consume 324,589 units per patient, resulting in annual costs of €240,196 per patient. In Analysis 1, comparing the base case (constant market share of 9% rVIII-SingleChain over time) with the alternative scenario (higher rVIII-SingleChain market share and increasing from 15% in the first year to 25% in the third year), the total expenditure for prophylaxis using rFVIII products is expected to decrease by €1.4 million in Year 1, by €3.1 million in Year 2 and by €5.4 million in Year 3. In Analysis 2 based on regional prices, the results remained consistent. DISCUSSION/CONCLUSION: This analysis suggests that increasing utilization of rVIII-SingleChain in hemophilia A patients may lead to cost savings as a result of reduced consumption with uncompromised efficacy in bleed protection.

Why was the study done? Hemophilia A is a rare inherited bleeding disorder. People with severe hemophilia are more likely to bleed compared to people without hemophilia and bleeds can occur spontaneously or in response to trauma. Patients are treated with medication to reduce the chance of bleeding. However, the cost of treating patients with hemophilia can be high and place demands on the healthcare system.What did we do and find?This study looked at the cost of treating people with hemophilia in Italy and used a type of economic analysis (called budget impact modelling) to estimate the effect of increasing the use of a particular medication (rVIII-SingleChain), compared to other medications that are available. Different variations of the model were tested to compare a range of scenarios.The results of this analysis suggested that increasing the use of rVIII-SingleChain may lead to cost-savings for the Italian healthcare system, compared to using the other currently available treatments. This analysis suggests that the use of rVIII-SingleChain enables people with hemophilia A to remain protected from bleeds, whilst using less product compared to other available medications.What is the influence of this study on the wider field?This type of analysis can be useful to healthcare systems, to guide the decision-making process regarding which medications to use or when making decisions related to healthcare policy.

Clinical and cost implications of target joints in Canadian boys with severe hemophilia A.

OBJECTIVE: To compare target joint-associated costs incurred by boys with severe hemophilia A 1 year before and 1 year after development of a target joint (pre-TJ, post-TJ). STUDY DESIGN: Resource utilization data were extracted retrospectively from medical and hemophilia clinic charts and patient diaries for 16 boys attending the Hospital for Sick Children (HSC)'s comprehensive care hemophilia program. Resources examined included drugs, medical care, hospitalization, laboratory tests, therapies, and transfusions received. All costs were figured using standard price lists and were discounted using an annual rate of 3%. RESULTS: Fifteen of the 16 boys developed at least one target joint, defined as three bleeds into any single joint within a consecutive 3-month period, at an average age of 54 months (range, 15-94 months), with ankles being most often affected, followed by elbows and knees (46% vs 28% and 23%, respectively). The total cost of treating a boy with on-demand Factor VIII (FVIII) increased by 119% after development of a target joint, from $20,091 (in 2002 Canadian dollars [$CDN]) in the year before to $43,890 in the year after target joint development. Factor VIII use accounted for 87% of the total cost in the year before target joint development and 93% in the year after. CONCLUSIONS: This study identified substantial increased costs of care associated with target joint development. This finding provides further support for more aggressive treatment aimed at reducing target joints-either more aggressive treatment of joint bleeds or institution of primary prophylactic therapy at an early age.

Financial aspects of haemophilia care in Puerto Rico and other Latin American countries.

The present cost of optimal care in haemophilia is very high. The paradigm of comprehensive care approach, including the essential elements of continuous integrated multidisciplinary health services and the provision of home care with early use of antihaemophilic products requires abundant economic resources that usually are not readily available in nonaffluent countries. A cost-effective comprehensive paediatric haemophilia programme has been operating in Puerto Rico during the last 15 years that provides quality care to over 90% of paediatric haemophiliacs, and is financed mainly by the local government health system. Efforts are also being made to provide and/or coordinate health care to all adult haemophilic patients through the Puerto Rico Pediatric Hemophilia Treatment Center. Information on the haemophilia care available in Latin American countries is scanty. The data available indicate that with a few exceptions, haemophilia care in this vast region is suboptimal. Apparently, early diagnosis is not common, there is lack of accessibility of services in most countries, comprehensive care is not the rule, safe high-purity AHF concentrates are used infrequently, and home care is used rarely. The main antihaemophilic products used are fresh frozen plasma and cryoprecipitate due to the high cost of modern antihaemophilic factor concentrates. The average per capita income of the main Latin American countries is about one-quarter of that of the USA and Canada. The existing economic situation of most Latin American countries would make it very difficult for them to purchase modern antihaemophilic products regularly and provide quality comprehensive care to their haemophilia patients, unless they make special efforts and get some type of external help. As a result of this study recommendations are made to improve the quality and accessibility of services to haemophilia patients in Latin America and other nonaffluent countries.