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Project Topic
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Patients with rare diseases face common problems such as diagnosis wandering, complex health care needs, uncertain prognosis, side effects of treatments, with an overall impact on their work or personal life, especially when they are still young and professionally active. However, data on their Quality of life (QoL) are still scarce and focus on disease-related variables, failing to incorporate the social and the subjective dimensions of QoL. People living with Vascular liver diseases (VLD), who are mainly young and active patients, share these difficulties but no study has been published on their QoL. Our main hypothesis is that the QoL of patients with VLD is poor, with an impact on their work situation. Our main objective is to develop a questionnaire highlighting the social impact of rare diseases, which will be tested in the setting of VLD, and which will be transferable to other diseases. Our secondary hypothesis is that the subjective dimension of QoL is not completely captured by questionnaires alone, but requires qualitative and participatory methods to appropriately integrate the patients’ perspective. Our secondary objective is to comprehensively assess QoL of VLD patients and their unmet needs by conducting a peer-based research study: in-depth interviews with patients will be conducted and analyzed by members of PAOs (patients’ organizations) previously trained by qualitative research experts. The project gathers partners from different Social sciences and Humanities (SSH) disciplines, clinicians, and patients’ representatives in a balanced Work Plan involving 5 European countries. Our expected result is to design a questionnaire that can be transferable to other disease contexts and that will be accessible for the scientific community so that any research team can use it easily and freely. At the end of the project, the questionnaire will be available in English, French, German, Italian, Spanish and Dutch. Expected impacts are to: 1) encourage research on the QoL of patients living with a rare disease, 2) harmonize research on the social component of QoL, 3) design appropriate interventions for patients with VLD, 4) provide data for advocacy and national disease care plans, 5) improve knowledge on the effects of patient and public involvement.
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