Abstract
In this chapter, the focus is on the application of decision analytic tools to assist in reimbursement decisions related to drugs for rare diseases. Focus is on the evaluative framework developed by the Ontario Ministry of Health's Drugs for Rare Diseases Working Group. The chapter describes the framework and illustrates the role of decision analytic methods through the application of the framework to idursulfase treatment of Hunter disease, an enzyme deficiency syndrome. The chapter highlights the development of a Markov model designed to mirror the natural disease history and to simulate the possible benefits of treatment. This process led to the Ministry of Health developing funding recommendations for the treatment of Hunter disease.
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Sonnenberg FA, Beck JR (1993) Markov models in medical decision making: a practical guide. Med Decis Making 13(4):322–338
Briggs A, Sculpher M (1998) An introduction to Markov modelling for economic evaluation. Pharmacoeconomics 13(4):397–409
Laupacis A (2005) Incorporating economic evaluations into decision-making: the Ontario experience. Med Care 43(7 Suppl):15–19
Clarke JT (2006) Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy. CMAJ 174(2):189–190
Cooper N, Coyle D, Abrams K, Mugford M, Sutton A (2005) Use of evidence in decision models: an appraisal of health technology assessments in the UK since 1997. J Health Serv Res Policy 10(4):245–250
McCabe C, Claxton K, Tsuchiya A (2005) Orphan drugs and the NHS: should we value rarity? Br Med J 331:1016–1019
McCabe C, Tsuchiya A, Claxton K, Raftery J (2006) Orphan drugs revisited. QJM 99(5):341–345; discussion 350–351
Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J (2007) Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care 23(1):36–42
Hughes DA, Tunnage B, Yeo ST (2005) Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 98(11):829–836
Canadian Organization for Rare Disorders (2005). Canada’s Orphan Drug Policy: Learning from the Best. http://www.raredisorders.ca/documents/CanadaOrphanDPFinal.pdf. Accessed 19 Dec 2011
Panju AH, Bell CM (2010) Policy alternatives for treatments for rare diseases. CMAJ 182(17):E787–E792.
CADTH (2011) Procedures for common drug review. http://www.cadth.ca/media/cdr/process/CDR_Procedure_e.pdf. Accessed 19 Dec 2011
Ontario Ministry of Health (2010) The committee to evaluate drugs: terms of reference and administrative guidelines. http://www.health.gov.on.ca/english/providers/program/drugs/how_drugs_approv/documents/ced_terms.pdf. Accessed 17 Mar 2011
Hunter C (1917) A rare disease in two brothers. Proc R Soc Med 10:104–116
Bach G, Eisenberg F Jr, Cantz M, Neufeld EF (1973) The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase. Proc Natl Acad Sci U S A 70(7):2134–2138
Martin R, Beck M, Eng C, Giugliani R, Harmatz P, Muñoz V, Muenzer J (2008) Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics 121(2):e377–e386
Morini SR, Steiner CE, Gerson LB (2010) Mucopolysaccharidosis type II: skeletal-muscle system involvement. J Pediatr Orthop B 19(4):313–317
Young ID, Harper PS (1982) Mild form of Hunter’s syndrome: clinical delineation based on 31 cases. Arch Dis Child 57(11):828–836
Young ID, Harper PS (1983) The natural history of the severe form of Hunter’s syndrome: a study based on 52 cases. Dev Med Child Neurol 25(4):481–489
Muenzer J, Wraith JE, Beck M et al. (2006) A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med 8(8):465–473
CADTH (2007) CEDAC recommendation and reasons for recommendation: idursulfase. http://www.cadth.ca/media/cdr/complete/cdr_complete_Elaprase_Dec-19-2007.pdf. Accessed 19 Dec 2011
Ontario Ministry of Health (2009) Committee to evaluate drugs (CED) recommendations and reasons: idursulfase—September, 2009. http://www.health.gov.on.ca/english/providers/program/drugs/ced/pdf/idursulfase.pdf. Accessed 19 Dec 2011
Daniels N, Sabin J (2002) Setting limits fairly: can we learn to share medical resources. Oxford University Press, Oxford, UK,
Ontario Citizen’s Council (2010) Considerations for funding drugs for rare diseases: a report of the Ontario citizens’ council. http://www.health.gov.on.ca/en/public/programs/drugs/councils/report/report_201003.pdf. Accessed 19 Dec 2011
Ontario public drug programs exceptional access program elaprase (idursulfase)—reimbursement guidelines version 2—October, 2009. http://www.health.gov.on.ca/english/providers/program/drugs/pdf/elaprase_reimburse.pdf. Accessed 19 Dec 2011
Acknowledgments
Dr. Bell is supported by a Canadian Institutes of Health Research and Canadian Patient Safety Institute Chair in Patient Safety and Continuity of Care.
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2013 Springer Science+Business Media New York
About this chapter
Cite this chapter
Coyle, D. et al. (2013). Application of Operations Research to Funding Decisions for Treatments with Rare Disease. In: Zaric, G. (eds) Operations Research and Health Care Policy. International Series in Operations Research & Management Science, vol 190. Springer, New York, NY. https://doi.org/10.1007/978-1-4614-6507-2_13
Download citation
DOI: https://doi.org/10.1007/978-1-4614-6507-2_13
Published:
Publisher Name: Springer, New York, NY
Print ISBN: 978-1-4614-6506-5
Online ISBN: 978-1-4614-6507-2
eBook Packages: Business and EconomicsBusiness and Management (R0)