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Assessing the economic challenges posed by orphan drugs

Published online by Cambridge University Press:  18 January 2007

Michael F. Drummond ,
David A. Wilson ,
Panos Kanavos ,
Peter Ubel  and
Joan Rovira
Michael F. Drummond
Affiliation:
University of York
David A. Wilson
Affiliation:
Massachusetts General HospitalandGenzyme Corporation
Panos Kanavos
Affiliation:
LSE Health, London School of Economics
Peter Ubel
Affiliation:
University of Michigan, Ann Arbor
Joan Rovira
Affiliation:
University of Barcelona
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Abstract

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

Keywords

Cost-effectiveness analysisEquityHealth policy
Type
GENERAL ESSAYS
Information
International Journal of Technology Assessment in Health Care , Volume 23 , Issue 1 , January 2007 , pp. 36 - 42
Copyright
© 2007 Cambridge University Press

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References

Alcimed. 2005. Study on orphan drugs. Paris: Alcimed;
COMP. Report to the commission in relation to article 10 of regulation 141/2000 on orphan medicinal products. Doc. Ref. EMEA/35218/2005. Available at: http://www.emea.eu.int.
Commonwealth of Australia. Other supply arrangements outside the Pharmaceutical Benefits Scheme (PBS). Department of Health and Ageing, 2006. Available at: http:// www.health.gov.au.
Crump BJ, Panton R, Drummond MF, et al. 1995 Transferring the costs of expensive treatments from secondary to primary care. BMJ. 310: 509– 512.Google Scholar
Department of Health. Drug treatment for multiple sclerosis (beta-interferons and glatiramer acetate)–risk-sharing scheme. HSC 2002/004 briefing note 2002, DH, London. Available at: http://www.dh.gov.uk.
Department of Health. Payment by results breakthrough ends years of uncertainty for MS patients. DH, London, 2002. Available at: http://www.dh.gov.uk.
Devlin N, Parking D. 2004 Does NICE have a cost-effectiveness threshold and what other factors influence its decisions? A binary choice analysis. Health Econ. 13: 437– 452.Google Scholar
Drummond MF, Shannon HS. 1986 Economic aspects of risk assessment in chemical safety. Sci Total Environ. 51: 81– 124.Google Scholar
2005. EurordisCare and EurordisCare2. Survey results. Available at: http://www.eurordis.org, http://www.rare-luxembourg2005.org.
George B, Harris A, Mitchell A. 2001 Cost-effectiveness analysis and the consistency of decision making: Evidence from pharmaceutical reimbursement in Australia (1991 to 1996). Pharmacoeconomics. 19: 1103– 1109.Google Scholar
Haffner ME. 2006 Adopting orphan drugs–two dozen years of treating rare diseases. N Engl J Med. 354: 445– 447.Google Scholar
Hutton J, McGrath C, Frybourg JM, et al. 2006 Framework for describing and classifying decision-making systems using technology assessment to determine the reimbursement of health technologies (fourth hurdle systems). Int J Technol Assess Health Care. 22: 10– 18.Google Scholar
Kanavos P, Saka O. 2005. Providing access to modern treatments and influencing policy in orphan diseases: The international experience and evidence from the UK LSE Health and Social Care Discussion Paper Number 20 LSE Health and Social Care, The London School of Economics and Political Science
McCabe C, Claxton K, Tsuchiya A. 2005 Orphan drugs and the NHS: Should we value rarity? BMJ. 331: 1016– 1019.Google Scholar
2004. National Institute for Health and Clinical Excellence. Citizens council reports: Ultra orphan drugs. London, NICE, Available at: http://www.nice.nhs.uk.
2005. National Institute for Health and Clinical Excellence. Social value judgements. London, NICE, Available at: http://www.nice.org.uk.
Nord E. 1993 The trade-off between severity of illness and treatment effect in cost-value analysis of health care. Health Policy. 24: 227– 238.Google Scholar
Rawlins MD, Culyer AJ. 2004 National Institute for Clinical Excellence and its value judgments. BMJ. 329: 224– 227.Google Scholar
Stolk EA, Busschbach JJ, Caffa M, et al. 2000 Cost utility analysis of sildenafil compared with papaverine-phentolamine injections. BMJ. 320: 1165– 1168.Google Scholar
Ubel PA, Loewenstein G. 1996 Distributing scarce livers: The moral reasoning of the general public. Soc Sci Med. 42: 1049– 1055.Google Scholar
Ubel PA, Richardson J, Prades JL. 1999 Life-saving treatments and disabilities. Are all QALYs created equal? Int J Technol Assess Health Care. 15: 738– 748.Google Scholar