Economic burden and health-related quality of life in tenosynovial giant-cell tumour patients in Europe: an observational disease registry

Background Tenosynovial Giant-Cell Tumour (TGCT) is a benign clonal neoplastic proliferation arising from the synovium, causing a variety of symptoms and often requiring repetitive surgery. This study aims to define the economic burden-from a societal perspective-associated with TGCT patients and their health-related quality of life (HRQOL) in six European countries. Methods This article analyses data from a multinational, multicentre, prospective observational registry, the TGCT Observational Platform Project (TOPP), involving hospitals and tertiary sarcoma centres from six European countries (Austria, France, Germany, Italy, the Netherlands, and Spain). It includes information on TGCT patients' health-related quality of life and healthcare and non-healthcare resources used at baseline (the 12-month period prior to the patients entering the registry) and after 12 months of follow-up. Results 146 TGCT patients enrolled for the study, of which 137 fulfilled the inclusion criteria. Their mean age was 44.5 years, and 62% were female. The annual average total costs associated with TGCT were euro4866 at baseline and euro5160 at the 12-month follow-up visit. The annual average healthcare costs associated with TGCT were euro4620 at baseline, of which 67% and 18% corresponded to surgery and medical visits, respectively. At the 12-month follow-up, the mean healthcare costs amounted to euro5094, with surgery representing 70% of total costs. Loss of productivity represented, on average, 5% of the total cost at baseline and 1.3% at follow-up. The most-affected HRQOL dimensions, measured with the EQ-5D-5L instrument, were pain or discomfort, mobility, and the performance of usual activities, both at baseline and at the follow-up visit. Regarding HRQOL, patients declared a mean index score of 0.75 at baseline and 0.76 at the 12-month follow-up. Conclusion The results suggest that TGCT places a heavy burden on its sufferers, which increases after one year of follow-up, mainly due to the healthcare resources required-in particular, surgical procedures. As a result, this condition has a high economic impact on healthcare budgets, while the HRQOL of TGCT patients substantially deteriorates over time.Orthopaedics, Trauma Surgery and Rehabilitatio

Using a stated preference discrete choice experiment to assess societal value from the perspective of decision-makers in Europe. Does it work for rare diseases?

Objectives: To pilot the feasibility of using a discrete choice experiment (DCE) design to investigate individual preferences from the decision-maker perspective regarding the use of public funding for orphan drugs and generate prior information for future experimental designs. Methods: A DCE was used on a convenience sample of participants from five European countries (England, France, Germany, Italy and Spain), exploring their preferences in distinct healthcare scenarios involving orphan drugs. A preliminary review of the empirical literature on distributive preferences informed the selection of attributes and their levels in the design. An online questionnaire was used to conduct the DCE survey. Results: A total of 199 questionnaires were completed. The five country model showed relative preference for some attributes over others: cost of treatment, improvement in health, value for money and availability of treatment alternatives received the greatest attention. However, disease severity, beginning of life, waiting times and side effects were also shown to be important social values that should not be ignored. Conclusions: The findings presented in this study provide insight about the preferences that can influence decisions on orphan drugs in different countries. This study also provides valuable prior information that could inform future DCE designs in this area

Considering the Societal Perspective in Economic Evaluations: A Systematic Review in the Case of Depression-Related Interventions

Lopez-Bastida J, Düvel J, Hasemann L, et al. Considering the Societal Perspective in Economic Evaluations: A Systematic Review in the Case of Depression-Related Interventions. Value in health. 2020;23(Suppl. 2):S586