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The elephant in the room: ethical issues associated with rare and expensive medical conditions*

Published online by Cambridge University Press:  16 December 2015

Constantine D. Mavroudis ,
Constantine Mavroudis  and
Jeffrey P. Jacobs
Constantine D. Mavroudis*
Affiliation:
Department of Surgery, Division of Cardiovascular Surgery, University of Pennsylvania, School of Medicine, Philadelphia, Pennsylvania, United States of America
Constantine Mavroudis
Affiliation:
Johns Hopkins Children’s Heart Surgery, Florida Hospital for Children, Orlando, Florida, United States of America
Jeffrey P. Jacobs
Affiliation:
Johns Hopkins Children’s Heart Surgery, All Children’s Hospital, St Petersburg, Florida, United States of America
*
Correspondence to: Dr C. D. Mavroudis, MD, MSc, Hospital of the University of Pennsylvania, 4 Maloney Building, 3400 Spruce St, Philadelphia, PA 19104, United States of America. Tel: 215 776 9285; Fax: 215 614 1861; E-mail: Constantine.mavroudis@uphs.upenn.edu
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Abstract

The treatment of rare and expensive medical conditions is one of the defining qualities of paediatric cardiology and congenital heart surgery. Increasing concerns over healthcare resource allocation are challenging the merits of treating more expensive forms of congenital heart disease, and this trend will almost certainly continue. In this manuscript, the problems of resource allocation for rare and expensive medical conditions are described from philosophical and economic perspectives. The argument is made that current economic models are limited in the ability to assess the value of treating expensive and rare forms of congenital heart disease. Further, multi-disciplinary approaches are necessary to best determine the merits of treating a patient population such as those with significant congenital heart disease that sometimes requires enormous healthcare resources.

Keywords

Congenital heart diseaserare diseasesresource allocation
Type
Original Articles
Information
Cardiology in the Young , Volume 25 , Issue 8: HeartWeek 2015 , December 2015 , pp. 1621 - 1625
Copyright
© Cambridge University Press 2015 

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Footnotes

*

Presented at the Johns Hopkins All Children’s Heart Institute 15th Annual International Symposium on Congenital Heart Disease, Saint Petersburg, Florida, United States of America, Friday 6 February, 2015 to Monday 9 February, 2015.

References

1.US Department of Health & Human Services National Institutes of Health. Rare Diseases Act of 2002; Public Law 107-280, November 6, 2002. Retrieved June 8, 2015, from http://www.gpo.gov/fdsys/pkg/PLAW-107publ280/pdf/PLAW-107publ280/pdf.Google Scholar
2.Griggs, RC, Batshaw, M, Dunkle, M, et al. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab 2009; 96: 20–26.CrossRefGoogle ScholarPubMed
3.US Department of Health & Human Services National Institutes of Health. NIH establishes rare diseases. Clinical research network. November 3, 2003. Retrieved June 8, 2015, from http://www.nih.gov/news/pr/nov2003/ncrr-03.htm.Google Scholar
4.Centers for Disease Control and Prevention. Congenital Heart Defects. Retrieved June 8, 2015, from http://www.cdc.gov/ncbddd/heartdefects/data.html.Google Scholar
5.National Organization for Rare Disorders. Tetralogy of Fallot. Retrieved June 8, 2015, from https://rarediseases.org/rare-diseases/tetralogy-of-fallot/.Google Scholar
6.US Department of Health & Human Services National Institutes of Health. Research Portfolio Online Reporting Tools (RePORT). Estimates of Funding for Various Research, Condition, and Disease Categories (RCDC). Retrieved June 8, 2015, from http://report.nih.gov/categorical_spending.aspx.Google Scholar
7.Centers for Disease Control and Prevention. Leading causes of death. Retrieved June 8, 2015, from http://www.cdc.gov/nchs/fastats/leading-causes-of-death.htm.Google Scholar
8.National Conference of State Legislatures. Heart disease and stroke-an overview of our nation’s leading killers. Retrieved June 8, 2015, from http://www.ncsl.org/research/health/heart-disease-and-stroke-an-overview.aspx. .Google Scholar
9.Pasquali, SK, Jacobs, ML, He, X, et al. Variation in congenital heart surgery costs across hospitals. Pediatrics 2014; 133: e553–e560.CrossRefGoogle ScholarPubMed
10.Keren, R, Luan, X, Localio, R, et al. Prioritization of comparative effectiveness research topics in hospital pediatrics. Arch Pediatr Adolesc Med 2012; 166: 1155–1164.CrossRefGoogle ScholarPubMed
11.Robbins, JM, Bird, TM, Tilford, JM, et al. Centers for Disease Control and Prevention (CDC). Hospital stays, hospital charges, and in-hospital deaths among infants with selected birth defects – United States, 2003. MMWR Morb Mortal Wkly Rep 2007; 56: 25–29.Google Scholar
12.Hyry, HI, Stern, AD, Cox, TM, Roos, JCP. Limits on use of health economic assessments for rare disease. Q J Med 2014; 107: 241–245.CrossRefGoogle Scholar
13.Mill, JS. Utilitarianism, 1st edn. Parker, Son & Bourn, West Strand, London, 1863.Google Scholar
14.Kernick, D. An introduction to the basic principles of health economics for those involved in the development and delivery of headache care. Cephalalgia 2005; 25: 709–714.CrossRefGoogle Scholar
15.Beauchamp, TL, Childress, JF. Principles of Biomedical Ethics, 6th edn. Oxford University Press, New York, New York, 2009: 222–223.Google Scholar
16.Williams, A. The importance of quality of life in policy decisions. In: Walker SR, Rosser RM (eds), Quality of Life: Assessment and Application. MTP Press, Boston, Massachusetts, 1988: 285.Google Scholar
17.Neumann, PJ, Cohen, JT, Weinstein, MC. Updating cost-effectiveness – the curious resilience of the $50,000-per-QALY threshold. New Engl J Med 2014; 371: 796–797.CrossRefGoogle ScholarPubMed
18.Hadorn, DC. Setting health care priorities in Oregon: cost effectiveness meets the rule of rescue. JAMA 1991; 265: 2218.CrossRefGoogle ScholarPubMed
19.Menzel, P, Gold, MR, Nord, E, Pinto-Prades, JL, Richardson, J, Ubel, P. Toward a broader view of values in cost-effectiveness analysis of health. Hastings Cent Rep 1999; 29: 7–15.CrossRefGoogle Scholar