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• W2000873524 abstract "Appraising rare disease treatments involves multiple issues and represents a significant challenge for HTA agencies. Multicriteria-approaches are uniquely suited to assess their real life value. were to develop a framework adapted to rare diseases while remaining compatible with other therapeutic areas for broad application. Adaptation of the framework to rare diseases was based on methodological and ethical principles underlying the EVIDEM framework, informed by issues and policies specific to rare diseases, which were identified through literature review and survey, and guided by pragmatic considerations of real life application in participatory processesCriteria selection followed MCDA principles: completeness; non-redundancy; operationalizability; and independence. MCDA model mechanics and sensitivity analyses were designed based on a review of MCDA modeling. Quantitative criteria of the framework are organized into a hierarchical MCDA model consisting of six domains of value (top-level criteria): Need, Type of benefit, Outcomes, Economic consequences, Knowledge, and Established priorities. Each domain includes criteria and subcriteria, each contributing to the final output of the model, i.e., the Value Estimate. The model explicitly takes into account aspects of rare diseases, including: disease complexity; treatment outcomes complexity; multiple economic and social consequences; data limitations and innovative approaches to tackle these; and health care system priorities. Weighting and scoring methodologies capture individual perspectives and judgments on the meaning of data while allowing for full exploration of uncertainty through six types of sensitivity analyses. Qualitative criteria support consideration of the impact of contextual issues. This framework promotes a comprehensive, transparent and systematic appraisal of rare disease treatments while remaining applicable to any therapy. Although numerical outputs are produced, the framework is intended to support deliberative processes that allow sharing of perspectives and rationales for decisions. Intended to measure value in its broad sense, the framework supports sustained application of MCDA in health care decisionmaking." @default.
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• W2000873524 date "2014-05-01" @default.
• W2000873524 modified "2023-10-18" @default.
• W2000873524 title "Assessing The Value Of Treatments For Rare Diseases Using An Mcda-Based Approach: Methodological And Ethical Foundations Of Criteria Selection And Framework Development" @default.
• W2000873524 doi "https://doi.org/10.1016/j.jval.2014.03.046" @default.
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