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Forecasting spending on orphan diseases to maintain the long-run financial sustainability of healthcare system

Author

Listed:
  • Rasstrigin, M.

    (The Center for Strategic Research, Moscow, Russia)

  • Kitaev, A.

    (The Center for Strategic Research, Moscow, Russia)

  • Pleshackova, E.

    (The Center for Strategic Research, Moscow, Russia)

Abstract

The paper deals with the rare diseases' burden forecasting in the context of information gaps on these diseases. There are many countries pumping up public funding for rare diseases' treatment despite lack of accurate data on patients numbers. This study is aimed at the formulation the one-size-fi ts-all approach for forecasting treatment costs of rare NCD. The approach is based on cohort component method and requires minimum exogenous data set. The model utilizing the proposed approach was developed for forecasting The Circle of Good Foundation's treatment costs on cystic fibrosis and Pompe's disease in 2022-2050. The authors found that forecasted number of patients in 2022 does not deviate dramatically from actual data for 2019-2021 and predicted cost pattern does not conflict with experts up to 2050. This supports study's hypothesis that the quality of treatment costs forecast does not depend signifi cantly on prevalence and degree of knowledge of rare disease. The results will be of interest to government authorities, charity foundations and pharmaceutical companies for upgrading decision-making on funding rare diseases' treatment costs and improvement market planning for new drugs in pharmaceutical companies.

Suggested Citation

  • Rasstrigin, M. & Kitaev, A. & Pleshackova, E., 2023. "Forecasting spending on orphan diseases to maintain the long-run financial sustainability of healthcare system," Journal of the New Economic Association, New Economic Association, vol. 59(2), pages 120-141.
    • Handle: RePEc:nea:journl:y:2023:i:59:p:120-141
    DOI: 10.31737/22212264_2023_2_120-141
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    References listed on IDEAS

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    1. Weinstein, M.C. & Coxson, P.G. & Williams, L.W. & Pass, T.M. & Stason, W.B. & Goldman, L., 1987. "Forecasting coronary heart disease incidence, mortality, and cost: The coronary heart disease policy model," American Journal of Public Health, American Public Health Association, vol. 77(11), pages 1417-1426.
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    More about this item

    Keywords

    rare diseases; orphan diseases; burden forecast; treatment costs forecast; NCDs; Cohort component method; The Circle of Good Foundation; orphan drugs;
    All these keywords.

    JEL classification:

    • C53 - Mathematical and Quantitative Methods - - Econometric Modeling - - - Forecasting and Prediction Models; Simulation Methods
    • H51 - Public Economics - - National Government Expenditures and Related Policies - - - Government Expenditures and Health
    • I18 - Health, Education, and Welfare - - Health - - - Government Policy; Regulation; Public Health

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