Instead, other issues, mainly those related to the practical aspects of treatment (for example, mode of distribution, number of infusions to stop bleeding) were considered differently by the different categories of subject, allowing a highlight on how the different opinions and preferences depend on interacting factors, which not only are attributable to clinical issues, but are also related to practical aspects and with the different points of view considered. To allow for optimal decisions for the management of haemophilia, every relevant aspect must be considered.
Conclusion particular, the CONAN study14 aimed to
assess and compare preferences between adult patients with moderate or severe haemophilia, haematologists and pharmacists with experience in the delivery and storage of coagulation factor concentrates. Instead, the COHIBA study13
Haemophilia is a chronic, lifelong condition that dramatically contributes to increasingly high costs for healthcare systems and society as a whole. Actually, the management of haemophilia and related consequences should be considered as a long-term investment,
involved adult and parents/tutors of paediatric patients with inhibitors, and again haematologists and pharmacists. In both studies, to design the main survey, a limited number of treatment
characteristics was preliminarily selected with a small survey, showing already some different needs and related preferences in the two categories of patient.
In particular, in the CONAN study, the following characteristics were selected and valued in the main study: perceived viral safety, risk of inhibitor development, factor infusion frequency on prophylaxis, pharmaceutical drug form, distribution modes and price of coagulation factor concentrates (which, in Italy, is paid for by the National Health Service). Instead, the characteristics selected and then valued during the main COHIBA study were: perceived risk of viral infection, risk of anamnestic response, number of infusions to stop bleeding, time to stop bleeding, time to pain recovery, frequency of infusions if used in prophylaxis, possibility of undergoing major surgery and increase of general healthcare costs contributing to the costs of treatment of haemophilia. In both studies, generally the characteristics related to effectiveness and safety were recognised as important in every subgroup of participant. However, in the COHIBA study, unlike with the patients, the physicians did not find relevant the difference of safety between modern recombinant and highly purified plasma-derived products.
gain all the information necessary for making decisions that lead to efficient investments. l
References 1. Gringeri A et al. Quality of life assessment in clinical practice in haemophilia treatment. Haemophilia 2006;12:22–29.
2. Scalone L et al. Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia 2006;12:154–162.
3. Scalone L et al. Cost and quality of life of hemophilia: comparison between patients with and patients without inhibitor. Value Health November 2005;8(6):A5.
4. Scalone L. Investing in hemophilia care: benefits and costs for patients and society. Semin Hematol.2008;45:S31-S3.
5. Mannucci PM et al. How we choose factor VIII to treat hemophilia. Blood 2012;119:4108-14.
6. Gringeri A et al. Costs and Outcomes of Patients With Haemophilia A (HA) and Factor VIII Inhibitors Treatment: The Immune Tolerance and Economics
“Two multicentre observational studies have been carried out to assess and compare preferences for the characteristics of treatment of patients with haemophilia A or B”
requiring the conduct of complex decisions for the optimisation of benefits, while using the available resources that, by definition, are scarce, hence applicable to alternative uses. To make better decisions, both present and future benefits and related costs should be taken into account when evaluating and comparing treatment options. For instance, prophylaxis is recognised for its potential benefits in terms of bleeding reduction, prevention of future disabilities and consequent improvement of patients’ health and wellbeing. Subsequently, further benefits could also be expected from both the patients’ and society’s point of view, as a reduction in the cost of managing complications and productivity could be expected. However, prophylaxis can be a particularly challenging regimen and appears to be very expensive, at least in the short term. Information on cost and on both present and future benefits is thus necessary to understand the relative value of the compared options and then to make rational decisions on which one to apply.4
The performance of appropriate
economic evaluations, like those reported in the present section, together with clinical research, is strongly encouraged to
Retrospective Registry (ITER) Results. Value in Health 2012;15(4):A103.
7. Gringeri A et al. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003;102:2358-63.
8. Smith PS et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996; 129:424-31.
9. Globe DR et al. Utilization of care in haemophilia: a resource-based method for cost analysis from the Haemophilia Utilization Group Study (HUGS). Haemophilia.2004;10:63-70.
10. Schramm W et al. Haemophilia Care in Europe: the ESCHQoL study. Haemophilia 2012;18:729-37.
11. Gringeri A et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011;9:700–10.
12. Astermark J et al. Current use of by-passing agents in Europe in the management of acute bleeds in patients with haemophilia and inhibitors. Haemophilia 2007;13:38–45.
13. Scalone L et al. Patients, physicians, and pharmacists preferences towards coagulation factor concentrates to treat haemophilia with inhibitors: results from the COHIBA Study. Haemophilia 2008;15:473-86.
14. Mantovani LG et al. Differences between patients’, physicians’ and pharmacists’ preferences for treatment products in haemophilia: a discrete choice experiment. Haemophilia 2005;11:589-97.
| Page 2
| Page 3
| Page 4
| Page 5
| Page 6
| Page 7
| Page 8
| Page 9
| Page 10
| Page 11
| Page 12
| Page 13
| Page 14
| Page 15
| Page 16
| Page 17
| Page 18
| Page 19
| Page 20
| Page 21
| Page 22
| Page 23
| Page 24
| Page 25
| Page 26
| Page 27
| Page 28
| Page 29
| Page 30
| Page 31
| Page 32
| Page 33
| Page 34
| Page 35
| Page 36