IDEAS home Printed from
MyIDEAS: Login to save this article or follow this journal

Pharmacoeconomic Considerations in Treating Iron Overload in Patients with β-Thalassaemia, Sickle Cell Disease and Myelodysplastic Syndromes in the US: A Literature Review

  • Bin Zhang

    (Abt Bio-Pharma Solutions, Inc., Lexington, Massachusetts, USA)

  • Prina Z. Donga

    (Abt Bio-Pharma Solutions, Inc., Lexington, Massachusetts, USA)

  • Mitra Corral

    (Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA)

  • Medha Sasane

    (Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA)

  • Jeffrey D. Miller

    (Abt Bio-Pharma Solutions, Inc., Lexington, Massachusetts, USA)

  • Chris L. Pashos

    (Abt Bio-Pharma Solutions, Inc., Lexington, Massachusetts, USA)

Registered author(s):

    Patients with β-thalassaemia, sickle cell disease (SCD) and myelodysplastic syndromes (MDS) require chronic blood transfusions, which can lead to iron overload and substantial morbidity and mortality. To reduce the excess iron and its deleterious effects, available iron chelation therapy (ICT) in the US includes oral deferasirox or infusional deferoxamine (DFO). The aim of this study was to review and synthesize the available pharmacoeconomic evidence on ICT in patients with β-thalassaemia, SCD and MDS in the US. We systematically identified and reviewed pharmacoeconomic studies of ICT in patients with β-thalassaemia, SCD and MDS that either were published in MEDLINE-indexed, English-language journals from 1999 to 2009, or appeared in medical society websites and scientific meeting abstracts. We assessed available cost-of-illness, cost-of-treatment, cost-consequence, cost-effectiveness, utility and patient-satisfaction studies. The majority of the 20 identified studies assessed cost of treatment, mainly focusing on acquisition and administration costs of ICTs. Gaps in the published literature include current data on direct medical costs for patients with MDS, direct medical costs associated with complications of iron overload, direct non-medical costs, indirect costs and patient utilities. Different underlying model assumptions, methodologies and comparators were found in the cost-effectiveness studies, which yielded a broad range of incremental cost-effectiveness ratios for different ICTs. Comprehensive cost-of-illness studies are needed to address data gaps in the published literature regarding the economic burden of iron overload. Comparative-effectiveness studies that evaluate clinical, economic and patient-reported outcomes would help the medical community to better understand the value of different ICTs.

    If you experience problems downloading a file, check if you have the proper application to view it first. In case of further problems read the IDEAS help page. Note that these files are not on the IDEAS site. Please be patient as the files may be large.

    File URL:
    Download Restriction: Pay per view

    File URL:
    Download Restriction: Pay per view

    As the access to this document is restricted, you may want to look for a different version under "Related research" (further below) or search for a different version of it.

    Article provided by Springer Healthcare | Adis in its journal PharmacoEconomics.

    Volume (Year): 29 (2011)
    Issue (Month): 6 ()
    Pages: 461-474

    in new window

    Handle: RePEc:wkh:phecon:v:29:y:2011:i:6:p:461-474
    Contact details of provider: Web page:

    No references listed on IDEAS
    You can help add them by filling out this form.

    This item is not listed on Wikipedia, on a reading list or among the top items on IDEAS.

    When requesting a correction, please mention this item's handle: RePEc:wkh:phecon:v:29:y:2011:i:6:p:461-474. See general information about how to correct material in RePEc.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: (Dave Dustin)

    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 references are entirely missing, you can add them using this form.

    If the full references list an item that is present in RePEc, but the system did not link 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 profile, as there may be some citations waiting for confirmation.

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

    This information is provided to you by IDEAS at the Research Division of the Federal Reserve Bank of St. Louis using RePEc data.