Cost-effectiveness analysis and prevention effects of ultra-orphan drugs for rare diseases: an in silico model applied to Cryopyrin Associated Periodic Syndromes (CAPS)
نویسندگان
چکیده
Objectives This three-year, international, multicentre, longitudinal, observational, cost-effectiveness study named RaDiCEA (RareDisease &Cost-EffectivenessAnalysis) will assess the economic evaluation (cost of illness COI and costeffectiveness analysis CEA) of innovative therapies (i.e., anti IL-1 agents), quality of life (QoL) and effects of the prevention of otherwise irreversible central nervous system, eye, ear, kidney, and cartilage damages of different treatment strategies for cryopyrin-associated periodic syndromes (CAPS) of adults and children.
منابع مشابه
The RaDiCEA Project: cost of illness (COI) analysis applied to Cryopyrin Associated Periodic Syndromes (CAPS)
Introduction When a disease affects only a few individuals in each country (ultra-orphan disease), it can be very hard to establish the costs of illness (COI) and the cost-effectiveness (CE) of treatments (ultra-orphan drugs). Conventional methods for COI and CE of drugs for common conditions do not apply, and additional factors need to be considered. As expensive medications (biologicals) show...
متن کاملPW02-027 - CAPS and cost-effectiveness analysis project
Introduction Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally very expensive on a per patient basis. European Union (EU) regulations promote the development of ...
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BACKGROUND The Cryopyrin-Associated Periodic Syndromes (CAPS) are a group of rare hereditary autoinflammatory diseases and encompass Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and Neonatal Onset Multisystem Inflammatory Disease (NOMID). Canakinumab is a monoclonal antibody directed against IL-1 beta and approved for CAPS patients but requires post-approval moni...
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Cryopyrin-associated periodic syndrome (CAPS) include a group of rare autoinflammatory disorders, the spectrum of which ranges from the mildest form, ie, familial cold autoinflammatory syndrome to more severe phenotypes, ie, Muckle-Wells syndrome, and chronic infantile neurological cutaneous and articular syndrome, also known as neonatal-onset multisystem inflammatory disease. Three interleukin...
متن کاملRilonacept in the management of cryopyrin-associated periodic syndromes (CAPS)
Cryopyrin-associated periodic syndromes (CAPS) are a subgroup of the hereditary periodic fever syndromes, which are rare autoinflammatory and inherited disorders, characterized by recurrent inflammation and varying degrees of severity. CAPS are thought to be driven by excessive production of interleukin-1β (IL-1β), through over-activation of the inflammasome by gain of function mutations in the...
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عنوان ژورنال:
دوره 13 شماره
صفحات -
تاریخ انتشار 2015