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A novel method for predicting the budget impact of innovative medicines: validation study for oncolytics

Author

Listed:
  • Joost W. Geenen

    (Utrecht University)

  • Svetlana V. Belitser

    (Utrecht University)

  • Rick A. Vreman

    (Utrecht University
    National Health Care Institute)

  • Martijn Bloois

    (Brabers)

  • Olaf H. Klungel

    (Utrecht University)

  • Cornelis Boersma

    (Health-Ecore
    University of Groningen)

  • Anke M. Hövels

    (Utrecht University)

Abstract

Background High budget impact (BI) estimates of new drugs have led to decision-making challenges potentially resulting in restrictions in patient access. However, current BI predictions are rather inaccurate and short term. We therefore developed a new approach for BI prediction. Here, we describe the validation of our BI prediction approach using oncology drugs as a case study. Methods We used Dutch population-level data to estimate BI where BI is defined as list price multiplied by volume. We included drugs in the antineoplastic agents ATC category which the European Medicines Agency (EMA) considered a New Active Substance and received EMA marketing authorization (MA) between 2000 and 2017. A mixed-effects model was used for prediction and included tumor site, orphan, first in class or conditional approval designation as covariates. Data from 2000 to 2012 were the training set. BI was predicted monthly from 0 to 45 months after MA. Cross-validation was performed using a rolling forecasting origin with e^|Ln(observed BI/predicted BI)| as outcome. Results The training set and validation set included 25 and 44 products, respectively. Mean error, composed of all validation outcomes, was 2.94 (median 1.57). Errors are higher with less available data and at more future predictions. Highest errors occur without any prior data. From 10 months onward, error remains constant. Conclusions The validation shows that the method can relatively accurately predict BI. For payers or policymakers, this approach can yield a valuable addition to current BI predictions due to its ease of use, independence of indications and ability to update predictions to the most recent data.

Suggested Citation

  • Joost W. Geenen & Svetlana V. Belitser & Rick A. Vreman & Martijn Bloois & Olaf H. Klungel & Cornelis Boersma & Anke M. Hövels, 2020. "A novel method for predicting the budget impact of innovative medicines: validation study for oncolytics," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(6), pages 845-853, August.
  • Handle: RePEc:spr:eujhec:v:21:y:2020:i:6:d:10.1007_s10198-020-01176-x
    DOI: 10.1007/s10198-020-01176-x
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    References listed on IDEAS

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    1. Stuart Keeping & Paul N. Deslandes & Kathryn E. Haines & Philip A. Routledge, 2019. "Estimated Versus Observed Expenditure Associated with Medicines Recommended by the All Wales Medicines Strategy Group," PharmacoEconomics - Open, Springer, vol. 3(3), pages 343-350, September.
    2. Johanna Wiss & Lars-Ake Levin & David Andersson & Gustav Tinghög, 2017. "Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making," Medical Decision Making, , vol. 37(5), pages 567-576, July.
    3. Katelijne Vooren & Silvy Duranti & Alessandro Curto & Livio Garattini, 2014. "A Critical Systematic Review of Budget Impact Analyses on Drugs in the EU Countries," Applied Health Economics and Health Policy, Springer, vol. 12(1), pages 33-40, February.
    4. Maarse, Hans & Jeurissen, Patrick & Ruwaard, Dirk, 2016. "Results of the market-oriented reform in the Netherlands: a review," Health Economics, Policy and Law, Cambridge University Press, vol. 11(2), pages 161-178, April.
    5. Doug Coyle & Matthew C. Cheung & Gerald A. Evans, 2014. "Opportunity Cost of Funding Drugs for Rare Diseases," Medical Decision Making, , vol. 34(8), pages 1016-1029, November.
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    Blog mentions

    As found by EconAcademics.org, the blog aggregator for Economics research:
    1. Chris Sampson’s journal round-up for 3rd August 2020
      by Chris Sampson in The Academic Health Economists' Blog on 2020-08-03 11:00:00

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    More about this item

    Keywords

    Budget impact; Oncology; Medicines; Budget impact estimation; Prediction modeling; Validation study;
    All these keywords.

    JEL classification:

    • I18 - Health, Education, and Welfare - - Health - - - Government Policy; Regulation; Public Health
    • I13 - Health, Education, and Welfare - - Health - - - Health Insurance, Public and Private
    • I10 - Health, Education, and Welfare - - Health - - - General

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