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Cost Effectiveness of Monoclonal Antibody Therapy for Rare Diseases: A Systematic Review

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Abstract

Background

Monoclonal antibody (mAb)-based orphan drugs have led to advances in the treatment of diseases by selectively targeting molecule functions. However, their high treatment costs impose a substantial cost burden on patients and society.

Objectives

The study aimed to systematically review cost-effectiveness evidence of mAb orphan drugs.

Methods

Ovid MEDLINE®, EMBASE®, and PsycINFO® were searched in June 2014 and articles were selected if they conducted economic evaluations of the mAb orphan drugs that had received marketing approval in the USA. The quality of the selected studies was assessed using the Quality of Health Economic Studies (QHES) instrument.

Results

We reviewed 16 articles that included 24 economic evaluations of nine mAb orphan drugs. Six of these nine drugs were included in cost-utility analysis studies, whereas three drugs were included in cost-effectiveness analysis studies. Previous cost-utility analysis studies revealed that four mAb orphan drugs (cetuximab, ipilimumab, rituximab, and trastuzumab) were found to be cost effective; one drug (bevacizumab) was not cost effective; and one drug (infliximab) was not consistent across the studies. Prior cost-effectiveness analysis studies which included three mAb orphan drugs (adalimumab, alemtuzumab, and basiliximab) showed that the incremental cost per effectiveness gained for these drugs ranged from $US4669 to $Can52,536 Canadian dollars. The quality of the included studies was good or fair with the exception of one study.

Conclusions

Some mAb orphan drugs were reported as cost effective under the current decision-making processes. Use of these expensive drugs, however, can raise an equity issue which concerns fairness in access to treatment. The issue of equal access to drugs needs to be considered alongside other societal values in making the final health policy decisions.

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Author contributions

Dong-Churl Suh is responsible for the integrity of the data in the study and the accuracy of the data analyses. Study conception and design: Taehwan Park and Dong-Churl Suh. Collection and assembly of data: Taehwan Park and Scott K. Griggs. Data analysis and interpretation: Taehwan Park, Scott K. Griggs, and Dong-Churl Suh. Manuscript writing and critical revision: Taehwan Park, Scott K. Griggs, and Dong-Churl Suh. Final approval of manuscript: Taehwan Park, Scott K. Griggs, and Dong-Churl Suh.

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Authors and Affiliations

  1. Pharmacy Administration, St. Louis College of Pharmacy, St. Louis, MO, USA

    Taehwan Park & Scott K. Griggs

  2. College of Pharmacy, Chung-Ang University, 84 Heukseok-ro, Dongjak-gu, Seoul, South Korea

    Dong-Churl Suh

Authors
  1. Taehwan Park
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Corresponding author

Correspondence to Dong-Churl Suh.

Ethics declarations

All authors (Taehwan Park, Scott K. Griggs, and Dong-Churl Suh) have nothing to declare. There is no conflict of interest. No funding has been received to conduct this study and prepare this manuscript.

Appendix

Appendix

Table 6 lists monoclonal antibody drugs and their designated indications.

Table 6 Monoclonal antibody-based orphan drugs and their designated indications
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Park, T., Griggs, S.K. & Suh, DC. Cost Effectiveness of Monoclonal Antibody Therapy for Rare Diseases: A Systematic Review. BioDrugs 29, 259–274 (2015). https://doi.org/10.1007/s40259-015-0135-4

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