IDEAS home Printed from https://ideas.repec.org/p/liu/liucec/265.html
   My bibliography  Save this paper

The economics of orphan drugs: the case of osteosarcoma treatment

Author

Abstract

There is still an unmet need for intensified research into health economic evaluation models of orphan drugs. In the first part of the paper some main issues related to the limits of cost-effectiveness in rare diseases are developed. In order to investigate the theoretical aspect in practice, a cost-effectiveness analysis has been performed to a new drug in osteosarcomas.Osteosarcomas are rare tumors: approximately 750 to 900 new cases are diagnosed each year in the United States, of which 400 arise in children and adolescents younger than 20 years of age. In the second part of the paper the cost-effectiveness of mifamurtide as add-on treatment to standard chemotherapy for the high grade resectable non metastatic osteosarcoma, compared to standard chemotherapy alone has been evaluated by using a Markov model. The horizon was lifetime and the perspective of the Italian National Health Service has been applied. The undiscounted results show that the ICER for mifamurtide added on maintenance chemotherapy over maintenance chemotherapy alone is € 32,111per Quality Adjusted Life Year (QALY). When discounting is included, the ICER changes to € 73,151 per QALY. The primary reason that discounting (of the outcomes) has a significant effect on the ICER is that the majority of the treatment costs are incurred within the first two years of the model but the clinical outcomes are obtained throughout the whole time horizon and thus discounting the outcomes (benefits) reduces the QALY difference between the treatments which adversely affects the cost-effectiveness of mifamurtide. The Probabilistic Sensitivity show that at a WTP threshold of €£50,000 around 76.4% of the iterations were below this limit (undiscounted), but only 19.4% when discounted. 19.4% of the iterations were below this limit. However, the value of € 73.151 per QALY gained seems to be relatively low when compared to the ICER of orphan drugs already marketed and reimbursed in Europe.

Suggested Citation

  • Stefano Capri, 2013. "The economics of orphan drugs: the case of osteosarcoma treatment," LIUC Papers in Economics 265, Cattaneo University (LIUC).
    • Handle: RePEc:liu:liucec:265
    as

    Download full text from publisher

    File URL: https://biblio.liuc.it/liucpap/pdf/265.pdf
    Download Restriction: no
    ---><---

    References listed on IDEAS

    as
    1. Paul Kind & Geoffrey Hardman & Susan Macran, 1999. "UK population norms for EQ-5D," Working Papers 172chedp, Centre for Health Economics, University of York.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Bishal Mohindru & David Turner & Tracey Sach & Diana Bilton & Siobhan Carr & Olga Archangelidi & Arjun Bhadhuri & Jennifer A. Whitty, 2020. "Health State Utility Data in Cystic Fibrosis: A Systematic Review," PharmacoEconomics - Open, Springer, vol. 4(1), pages 13-25, March.
    2. Dina Jankovic & Pedro Saramago Goncalves & Lina Gega & David Marshall & Kath Wright & Meena Hafidh & Rachel Churchill & Laura Bojke, 2022. "Cost Effectiveness of Digital Interventions for Generalised Anxiety Disorder: A Model-Based Analysis," PharmacoEconomics - Open, Springer, vol. 6(3), pages 377-388, May.
    3. Billingsley Kaambwa & Julie Ratcliffe, 2018. "Predicting EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L) Utilities from Older People’s Quality of Life Brief Questionnaire (OPQoL-Brief) Scores," The Patient: Patient-Centered Outcomes Research, Springer;International Academy of Health Preference Research, vol. 11(1), pages 39-54, February.
    4. Carsten Hirt & Sergio Iannazzo & Silvia Chiroli & Lisa J. McGarry & Philipp Coutre & Leif Stenke & Torsten Dahlén & Jeffrey H. Lipton, 2019. "Cost Effectiveness of the Third-Generation Tyrosine Kinase Inhibitor (TKI) Ponatinib, vs. Second-Generation TKIs or Stem Cell Transplant, as Third-Line Treatment for Chronic-Phase Chronic Myeloid Leuk," Applied Health Economics and Health Policy, Springer, vol. 17(4), pages 555-567, August.
    5. Billingsley Kaambwa & Gang Chen & Julie Ratcliffe & Angelo Iezzi & Aimee Maxwell & Jeff Richardson, 2017. "Mapping Between the Sydney Asthma Quality of Life Questionnaire (AQLQ-S) and Five Multi-Attribute Utility Instruments (MAUIs)," PharmacoEconomics, Springer, vol. 35(1), pages 111-124, January.
    6. Miqdad Asaria & Susan Griffin & Richard Cookson, 2016. "Distributional Cost-Effectiveness Analysis," Medical Decision Making, , vol. 36(1), pages 8-19, January.
    7. Donna Rowen & John Brazier & Clara Mukuria & Anju Keetharuth & Arne Risa Hole & Aki Tsuchiya & Sophie Whyte & Phil Shackley, 2016. "Eliciting Societal Preferences for Weighting QALYs for Burden of Illness and End of Life," Medical Decision Making, , vol. 36(2), pages 210-222, February.
    8. Abdullah Pandor & Matt Stevenson & John Stevens & Marrissa Martyn-St James & Jean Hamilton & Jenny Byrne & Claudius Rudin & Andrew Rawdin & Ruth Wong, 2018. "Ponatinib for Treating Chronic Myeloid Leukaemia: An Evidence Review Group Perspective of a NICE Single Technology Appraisal," PharmacoEconomics, Springer, vol. 36(8), pages 903-915, August.
    9. Nádia Simões & Nuno Crespo & Sandrina B. Moreira & Celeste A. Varum, 2016. "Measurement and determinants of health poverty and richness: evidence from Portugal," Empirical Economics, Springer, vol. 50(4), pages 1331-1358, June.
    10. Miqdad Asaria & Susan Griffin & Richard Cookson, 2013. "Distributional cost-effectiveness analysis: a tutorial," Working Papers 092cherp, Centre for Health Economics, University of York.
    11. Andrea Manca & Nigel Rice & Mark J. Sculpher & Andrew H. Briggs, 2005. "Assessing generalisability by location in trial‐based cost‐effectiveness analysis: the use of multilevel models," Health Economics, John Wiley & Sons, Ltd., vol. 14(5), pages 471-485, May.
    12. Laura Bojke & Andrea Manca & Miqdad Asaria & Ronan Mahon & Shijie Ren & Stephen Palmer, 2017. "How to Appropriately Extrapolate Costs and Utilities in Cost-Effectiveness Analysis," PharmacoEconomics, Springer, vol. 35(8), pages 767-776, August.
    13. Amy Dymond & Will Green & Mary Edwards & Maria Angeles Lopez Pont & Girish Gupta, 2023. "Economic Evaluation of Tirbanibulin for the Treatment of Actinic Keratosis in Scotland," PharmacoEconomics - Open, Springer, vol. 7(3), pages 443-454, May.
    14. Brouwer, Werner B.F. & van Exel, N. Job A., 2005. "Expectations regarding length and health related quality of life: Some empirical findings," Social Science & Medicine, Elsevier, vol. 61(5), pages 1083-1094, September.
    15. Jamie Elvidge & Ash Bullement & Anthony J. Hatswell, 2016. "Cost Effectiveness of Characterised Chondrocyte Implantation for Treatment of Cartilage Defects of the Knee in the UK," PharmacoEconomics, Springer, vol. 34(11), pages 1145-1159, November.
    16. Paul Wallace & Elizabeth Murray & Jim McCambridge & Zarnie Khadjesari & Ian R White & Simon G Thompson & Eleftheria Kalaitzaki & Christine Godfrey & Stuart Linke, 2011. "On-line Randomized Controlled Trial of an Internet Based Psychologically Enhanced Intervention for People with Hazardous Alcohol Consumption," PLOS ONE, Public Library of Science, vol. 6(3), pages 1-8, March.
    17. Merili Tamson & Rainer Reile & Diana Sokurova & Kaire Innos & Eha Nurk & Kaia Laidra & Sigrid Vorobjov, 2022. "Health-Related Quality of Life and Its Socio-Demographic and Behavioural Correlates during the COVID-19 Pandemic in Estonia," IJERPH, MDPI, vol. 19(15), pages 1-12, July.
    18. Mengjun Wu & John Brazier & Benjamin Kearns & Clare Relton & Christine Smith & Cindy Cooper, 2015. "Examining the impact of 11 long-standing health conditions on health-related quality of life using the EQ-5D in a general population sample," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 16(2), pages 141-151, March.
    19. Bromley, Hannah L. & Petrie, Dennis & Mann, G.Bruce & Nickson, Carolyn & Rea, Daniel & Roberts, Tracy E., 2019. "Valuing the health states associated with breast cancer screening programmes: A systematic review of economic measures," Social Science & Medicine, Elsevier, vol. 228(C), pages 142-154.
    20. Tomasz Fundament & Paul R Eldridge & Alexander L Green & Alan L Whone & Rod S Taylor & Adrian C Williams & W M Michael Schuepbach, 2016. "Deep Brain Stimulation for Parkinson’s Disease with Early Motor Complications: A UK Cost-Effectiveness Analysis," PLOS ONE, Public Library of Science, vol. 11(7), pages 1-18, July.

    More about this item

    NEP fields

    This paper has been announced in the following NEP Reports:

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:liu:liucec:265. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Laura Ballestra (email available below). General contact details of provider: https://edirc.repec.org/data/liuccit.html .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.