Mónica Inês1, Teresa Coelho2,3, Isabel Conceição1,4, Filipa Landeiro5, Mamede de Carvalho1,4, João Costa1,6. 1. Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal. 2. Andrade's Center for Familial Amyloidosis, Hospital de Santo António, Centro Hospitalar Universitário do Porto, Porto, Portugal. 3. Department of Neurosciences, Hospital de Santo António, Centro Hospitalar Universitário do Porto, Porto, Portugal. 4. Department of Neurosciences and Mental Health, Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Lisboa, Portugal. 5. Nuffield Department of Population Health, Health Economics Research Centre, University of Oxford, Oxford, UK. 6. Laboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.
Abstract
Background: Hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN) is a rare life-threatening disease that imposes considerable mortality and morbidity associated with increased costs, high social support and productivity losses. This study aims to estimate the societal costs and burden of ATTRv-PN. Methods: A cost-of-illness (COI) and burden of disease model were specified from a societal perspective, using a prevalence-based approach. Direct and indirect costs were included. Healthcare resource use was retrieved from public databases, previous Portuguese studies and the literature. The burden of disease was expressed in terms of disability-adjusted life years (DALYs), as defined by the World Health Organisation. Results: In 2016, the total annual COI of ATTRv-PN in Portugal was 52,502,796€ and the mean cost per patient was 28,152€ (79% direct; 21% indirect costs). Treatments accounted for 52% of total costs, while 0.18% were devoted to disease prevention. A total of 2056 DALYs were lost, 26% due to disability and 74% due to death.Conclusions: Annual costs and burden of ATTRv-PN were considerable but within the range of other rare diseases. Policies and public interventions to prevent and reduce the burden of disease should be prioritised, since patients experience excess morbidity, mortality and total costs will likely increase in the future.
Background: Hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN) is a rare life-threatening disease that imposes considerable mortality and morbidity associated with increased costs, high social support and productivity losses. This study aims to estimate the societal costs and burden of ATTRv-PN. Methods: A cost-of-illness (COI) and burden of disease model were specified from a societal perspective, using a prevalence-based approach. Direct and indirect costs were included. Healthcare resource use was retrieved from public databases, previous Portuguese studies and the literature. The burden of disease was expressed in terms of disability-adjusted life years (DALYs), as defined by the World Health Organisation. Results: In 2016, the total annual COI of ATTRv-PN in Portugal was 52,502,796€ and the mean cost per patient was 28,152€ (79% direct; 21% indirect costs). Treatments accounted for 52% of total costs, while 0.18% were devoted to disease prevention. A total of 2056 DALYs were lost, 26% due to disability and 74% due to death.Conclusions: Annual costs and burden of ATTRv-PN were considerable but within the range of other rare diseases. Policies and public interventions to prevent and reduce the burden of disease should be prioritised, since patients experience excess morbidity, mortality and total costs will likely increase in the future.
Authors: Sheila R Reddy; Eunice Chang; Marian H Tarbox; Michael S Broder; Ryan S Tieu; Spencer Guthrie; Montserrat Vera-Llonch; Michael R Pollock Journal: Neurol Ther Date: 2020-05-25