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    Home > Active Ingredient News > Study of Nervous System > JNNP: Cost-effectiveness analysis of screening neonatal spinal muscular atrophy with disease-modified therapy

    JNNP: Cost-effectiveness analysis of screening neonatal spinal muscular atrophy with disease-modified therapy

    • Last Update: 2021-08-14
    • Source: Internet
    • Author: User
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    Spinal muscular atrophy (SMA) is a type of disease in which muscle weakness and muscle atrophy are caused by the degeneration of motor neurons in the anterior horn of the spinal cord


    Spinal muscular atrophy (SMA) is a type of disease in which muscle weakness and muscle atrophy are caused by the degeneration of motor neurons in the anterior horn of the spinal cord


    This method aims to economically evaluate the short-term and long-term cost-effectiveness of early identification of SMA through modified disease therapy (ie, nusinersen or gene therapy), which is mainly compared with unscreened nusinersen therapy and previously unscreened and supported by care Case comparison


    A series of Markov cohort simulations were conducted to reflect the health outcomes and costs of each infant in the population: (1) NBS that uses nusinersen for early identification and treatment (2) NBS can identify and initiate gene therapy early (3) Pass Clinical referral for symptom diagnosis, and treatment with nusinersen from the time of diagnosis and (4) symptom diagnosis through clinical referral, and management by supportive care


    SMA NBS Decision Analysis Model

    The parameters of the decision analysis model come from laboratory data from the New South Wales/Australian Capital Territory National Bureau of Statistics pilot project


    Markov queue simulation is a random process that allows individuals to transition from one healthy state to another


    A directed graph representation of the health state modeled in the Markov model

    In the NBS intervention cohort, two disease-modifying treatment options, namely nusinersen or gene therapy, were modeled to obtain the cost and outcome of the SMA patients screened


    To generate QALY


    Compare the incremental cost and QALY with the calculated incremental cost benefit ratio (ICER)


    Cost-effectiveness of NBS and gene therapy

    By using nusinersen or gene therapy to treat a pre-symptomatic SMA infant, compared with the clinically diagnosed late treatment of SMA, an additional 9.


    In short, NBS plus gene therapy can improve the quality of life and longevity of SMA infants.


    In short, NBS plus gene therapy can improve the quality of life and longevity of SMA infants.




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