Rome, 21st November 2016
Over the last years, there has been a remarkable evolution of the substitutive therapy with factors of the coagulation for treatment of haemophilia patients; the increasing diffusion of more effective therapeutic approaches and/or new therapies is also fated to produce a potential impact on the pharmaceutical expense.
Anything described above puts the necessity and provides impulse to lead studies of economic assessment and to use the results by various individuals having decision-making and/or management roles in haemophilia patient care.
The outcomes research studies and the economic assessments set as their objectives the integration of data of clinical effectiveness with those of economics and the definition of synthetic indexes considering concurrently both aspects, making them available in the decision-making process of the therapeutic choices.
At aggregate level, the ESCHQoL study has shown how the frequency of bleedings and the orthopaedic status are linked to levels of consumption per person of factor VIII (Schramm et al.). Otherwise, still during last decade Scalone and colleagues showed how the orthopaedic status of patients was related to the quality of life in general (Scalone et al.).