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Towards sustainability and affordability of expensive cell and gene therapies? Applying a cost-based pricing model to estimate prices for Libmeldy and Zolgensma

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اقرأ أكثر حفظ في قائمتي
  • المؤلفون: Thielen, Frederick W.; Heine, Renaud J.S.D.; Berg, Sibren van den; Ham, Renske M.T.ten; Groot, Carin A.Uyl de
  • المصدر:
    Thielen , F W , Heine , R J S D , Berg , S V D , Ham , R M T T & Groot , C A U D 2022 , ' Towards sustainability and affordability of expensive cell and gene therapies? Applying a cost-based pricing model to estimate prices for Libmeldy and Zolgensma ' , Cytotherapy , vol. 24 , no. 12 , pp. 1245-1258 .
  • نوع التسجيلة:
    Electronic Resource
  • الدخول الالكتروني :
    https://pure.eur.nl/en/publications/5cfd65f3-22d5-4b9e-822d-f36036c2edfa
  • معلومة اضافية
    • Publisher Information:
      2022-12
    • نبذة مختصرة :
      Background aims: Drug prices are regarded as one of the most influential factors in determining accessibility and affordability to novel therapies. Cell and gene therapies such as OTL-200 (brand name: Libmeldy) and AVXS-101 (brand name: Zolgensma) with (expected) list prices of 3.0 million EUR and 1.9 million EUR per treatment, respectively, spark a global debate on the affordability of such therapies. The aim of this study was to use a recently published cost-based pricing model to calculate prices for cell and gene therapies, with OTL-200 and AVXS-101 as case study examples. Methods: Using the pricing model proposed by Uyl-de Groot and Löwenberg, we estimated a price for both therapies. We searched the literature and online public sources to estimate (i) research and development (R&D) expenses adjusted for risk of failure and cost of capital, (ii) the eligible patient population and (iii) costs of drug manufacturing to calculate a base-case price for OTL-200 and AVXS-101. All model input parameters were varied in a stepwise, deterministic sensitivity analysis and scenario analyses to assess their impact on the calculated prices. Results: Prices for OTL-200 and AVXS-101 were estimated at 1 048 138 EUR and 380 444 EUR per treatment, respectively. In deterministic sensitivity analyses, varying R&D estimates had the greatest impact on the price for OTL-200, whereas for AVXS-101, changes in the profit margin changed the calculated price substantially. Highest prices in scenario analyses were achieved when assuming the lowest number of patients for OTL-200 and highest R&D expenses for AVXS-101. The lowest R&D expenses scenario resulted in lowest prices for either therapy. Conclusions: Our results show that, using the proposed model, prices for both OTL-200 and AVXS-101 lie substantially below the currently (proposed) list prices for both therapies. Nevertheless, the uncertainty of the used model input parameters is considerable, which translates in a
    • الموضوع:
      dk/atira/pure/sustainabledevelopmentgoals/good_health_and_well_being; name=SDG 3 - Good Health and Well-being; article
    • Availability:
      Open access content. Open access content
      info:eu-repo/semantics/openAccess
    • Note:
      application/pdf
      English
    • Other Numbers:
      QGQ oai:pure.eur.nl:publications/5cfd65f3-22d5-4b9e-822d-f36036c2edfa
      https://pure.eur.nl/en/publications/5cfd65f3-22d5-4b9e-822d-f36036c2edfa
      https://doi.org/10.1016/j.jcyt.2022.09.002
      https://pure.eur.nl/ws/files/69115638/PIIS1465324922007861.pdf
      1349453082
    • Contributing Source:
      ERASMUS UNIVERSITEIT ROTTERDAM
      From OAIster®, provided by the OCLC Cooperative.
    • الرقم المعرف:
      edsoai.on1349453082
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