The modern management of haemophilia has greatly influenced not only survival of patients, their clinical symptoms and orthopaedic outcome but also their perceived quality of life (QoL). QoL measures recently became an essential part of clinical trials being one of the most important patient-rated outcomes (PROs). Moreover, QoL assessment is essential in pharmacoeconomics. In clinical trials, not only clinical outcomes but also the so-called PROs and health-economic outcomes are included. Different types of economic evaluations may be conducted in order to describe the economic burden of a disease condition, to determine the cost of care and to assess and evaluate alternative treatments in terms of both costs and effects as well as benefits and outcomes with the aim of optimizing the use of resources. For these evaluations, the following analyses are performed: descriptive cost of illness study, incremental cost-effectiveness analysis, incremental cost-utility analysis and incremental cost-benefit analysis. By contrast, PROs are derived from direct patient reports and they allow to evaluate the impact of a disease and its treatment on patients' well-being and functioning. PROs include health-related quality of life, patient preferences/utilities, treatment satisfaction and other PROs such as functional assessment, etc. Choosing a QoL measure, study- and instrument-related aspects have to be taken into account. Finally, QoL assessment will become a part of the regular clinical assessment of persons with haemophilia, in order to provide trustworthy data of perceived well-being to be compared over time and in order to assess treatment efficacy and quality of care.
The modern management of haemophilia has greatly influenced not only survival of patients, their clinical symptoms and orthopaedic outcome but also their perceived quality of life (QoL). QoL measures recently became an essential part of clinical trials being one of the most important patient-rated outcomes (PROs). Moreover, QoL assessment is essential in pharmacoeconomics. In clinical trials, not only clinical outcomes but also the so-called PROs and health-economic outcomes are included. Different types of economic evaluations may be conducted in order to describe the economic burden of a disease condition, to determine the cost of care and to assess and evaluate alternative treatments in terms of both costs and effects as well as benefits and outcomes with the aim of optimizing the use of resources. For these evaluations, the following analyses are performed: descriptive cost of illness study, incremental cost-effectiveness analysis, incremental cost-utility analysis and incremental cost-benefit analysis. By contrast, PROs are derived from direct patient reports and they allow to evaluate the impact of a disease and its treatment on patients' well-being and functioning. PROs include health-related quality of life, patient preferences/utilities, treatment satisfaction and other PROs such as functional assessment, etc. Choosing a QoL measure, study- and instrument-related aspects have to be taken into account. Finally, QoL assessment will become a part of the regular clinical assessment of persons with haemophilia, in order to provide trustworthy data of perceived well-being to be compared over time and in order to assess treatment efficacy and quality of care.