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Schoser B, Hahn A, James E, et al. A Systematic Review of the Health Economics of Pompe Disease. Pharmacoecon Open. 2019;3(4):479-493doi: 10.1007/s41669-019-0142-3.
Schoser, B., Hahn, A., James, E., Gupta, D., Gitlin, M., & Prasad, S. (2019). A Systematic Review of the Health Economics of Pompe Disease. PharmacoEconomics - open, 3(4), 479-493. https://doi.org/10.1007/s41669-019-0142-3
Schoser, Benedikt, et al. "A Systematic Review of the Health Economics of Pompe Disease." PharmacoEconomics - open vol. 3,4 (2019): 479-493. doi: https://doi.org/10.1007/s41669-019-0142-3
Schoser B, Hahn A, James E, Gupta D, Gitlin M, Prasad S. A Systematic Review of the Health Economics of Pompe Disease. Pharmacoecon Open. 2019 Dec;3(4):479-493. doi: 10.1007/s41669-019-0142-3. PMID: 31049836; PMCID: PMC6861413.
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Pharmacoecon Open. 2019 Dec;3(4):479-493. doi: 10.1007/s41669-019-0142-3.

A Systematic Review of the Health Economics of Pompe Disease.

PharmacoEconomics - open

Benedikt Schoser, Andreas Hahn, Emma James, Digant Gupta, Matthew Gitlin, Suyash Prasad

Affiliations

  1. Friedrich-Baur-Institut, Neurologische Klinik, Klinikum der Universität München, Ludwig-Maximilians-University Munich, Munich, Germany. [email protected].
  2. Department of Child Neurology, University of Giessen, Feulgenstrasse 12, 35385, Giessen, Germany.
  3. Audentes Therapeutics, 600 California Street, Floor 17, San Francisco, CA, 94108, USA.
  4. Bridge Medical Consulting Ltd, Gainsborough House, 2 Sheen Road, Richmond, London, UK.
  5. BluePath Solutions, 10951 West Pico Blvd, Suite 120, Los Angeles, CA, 90064, USA.

PMID: 31049836 PMCID: PMC6861413 DOI: 10.1007/s41669-019-0142-3

Abstract

BACKGROUND: Pompe disease is a rare, severe neuromuscular disease with high mortality and substantial clinical and humanistic burden. However, the economic burden of Pompe disease and the health economic value of its treatments are not well understood. The objectives of this systematic review were to characterize the health economic evidence on Pompe disease, including healthcare resource use and costs (direct and indirect), health utilities, and the cost-effectiveness of current treatments used to manage patients with Pompe disease.

METHODS: A systematic search of MEDLINE

RESULTS: Eight publications evaluated patients with infantile-onset Pompe disease (IOPD) (two studies), late-onset Pompe disease (LOPD) (four studies), or both (two studies). In IOPD, total cost of supportive therapy (excluding treatment) was €32,871 (equivalent to US$41,667 when adjusted for currency and inflation to 2017 US dollars) over a life expectancy of 0.4 years. In adult LOPD, the average annual cost per patient of supportive therapy was €22,475 (adjusted $28,489). Resource use in LOPD was high, with nursing home admissions accounting for 19% of annual direct medical costs. Health economic evaluations estimating incremental costs per quality-adjusted life year (QALY) gained with enzyme-replacement therapy (ERT) versus supportive therapy ranged from £109,991 (adjusted, $186,851) per QALY gained in Columbia to €1,043,868 (adjusted, $1,323,207) in the Netherlands.

DISCUSSION: Despite a full systematic literature search, only eight relevant publications were identified, most of which were of relatively poor quality. However, a significant economic burden of Pompe disease on patients, families, healthcare systems, and society was found, with the majority of costs driven by the only currently approved treatment, ERT. Health economic evaluations of ERT versus supportive therapy vary significantly, with the majority well above willingness-to-pay thresholds. New therapies and approaches to care are needed to address the persistent and lifelong economic burden of Pompe disease and the large incremental cost-effectiveness ratios observed.

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