The main aim of this project is to develop a disease based model capable of quantifying the socio-economic burden and Health-Related Quality of Life (HRQOL) for patients in Europe with rare diseases (RD) and their caregivers. The model that will be generated will not only be suitable to apply a wide range and hopefully all RD known to date, but it will also be sufficiently flexible to identify and adapt to the challenges faced by the different Health and Social Care Systems of EU Member States.
The importance of RD has long been recognised in the EU. BURQOL-RD will provide new tools to study the socio-economic burden and Health-Related Quality of Life (HRQOL) for RD patients and their caregivers. In this way BURQOL-RD will help to harmonise the measurement of the impact of new policies (programmes, new organizations, interventions and treatments) on socioeconomic and HRQOL issues for RD patients and their caregivers at different EU countries with different health and social care systems. These contributions will help to improve the knowledge and recognition of RD, and to assess the potential effects of new health plans focused on RD. For that reason BURQOL-RD has been developed in close interaction with EUROPLAN. By testing the model in different countries, the approach used by BURQOL-RD will evolve until it can convincingly measure: social and health care costs and the HRQOL of patients and caregivers in different countries.
The model will incorporate information on: Medical resources related to RD (e.g. hospitalisation, consultations, drugs), Non-medical resources (e.g. walking sticks, wheelchairs, modifications to house and car), Services (e.g. home care, transportation), Informal care by relatives, Productivity losses, Intangible costs, Overall HRQOL (utilities), measured both in RD patients and their caregivers. This information will be collected using a standardized pre-tested questionnaire from samples of patients registered in RD associations. Accordingly, the mean annual costs will be estimated from a society perspective.
This project will generate new key indicators regarding RD in Europe for an array of EU countries that differ in their cultural, social, economic and organisational characteristics. These indicators will permit the updating of existing estimates and a better extrapolation to the rest of the EU, as well as anticipating future information needs. The results and deliverables that emerge from this project will stimulate the future comparability and monitoring of RD in Europe. In addition, they will be crucial for further studies on the cost-effectiveness of new treatments to prevent diagnosis or provide better health care to patients with RD.