Real World Data can impact economic analyses of Huntington’s Disease
November 5, 2019

Studies on direct and indirect costs are lacking in Canada

Huntington’s disease (HD) is a fatal, autosomal dominant disorder that causes progressive neuronal dysfunction and death in areas of the brain underlying voluntary movement. HD has an estimated disease prevalence of 10.4-13.7 individuals per 100,000 in Canada. Current evidence suggests that the prevalence of HD may be increasing in Canada, as well as globally. Estimates extrapolated from a previous study suggest that there are up to 4,700 individuals affected with HD and 14,000 at 50% risk for HD in Canada. The typical onset of HD occurs in the prime of adult life. Subtle motor, cognitive and behavioural changes may manifest up to 15 years before a formal clinical diagnosis of motor dysfunction. However, once overt motor dysfunction is present the average survival is 18 years. In the absence of an effective disease-modifying therapy, the management of HD focuses on treating symptoms. Management requires an extensive, multidisciplinary approach through medical and non-medical means to support optimal function and quality of life. Consequently, the burden of HD on patients, families, and healthcare utilization is significant, and increases as patients lose functionality and independence. Direct healthcare costs for HD have been estimated in the US to be between $3,257-4,947 USD in early stages, rising steeply to $22,582-37,495 USD in late stages (annualized cost per patient). However, these costs do not include informal care or other societal costs. A study from the UK that included indirect costs found that later stages reached an average cost of £89,760 per person per year, with informal care as the cost driver. While there are significant differences between the US and UK health care systems, collectively, these results highlight a significant burden on healthcare utilization, as well as patients and caregivers. Similar studies on direct and indirect costs of HD are lacking in Canada. In addition, the incidence and prevalence of Huntington’s Disease in Canada is poorly understood. A Real World Evidence study examining the incidence, prevalence, direct and indirect costs, and impact of disease on patients, families and caregivers is needed. Real World Data on the impact of disease progression, treatment patterns, healthcare utilization, and societal burden will be vital to future economic analyses. References

  1.     Bates GP, Dorsey R, Gusella JF, et al. Huntington disease. Nat Rev Dis Primers 2015; 1: 15005.
  2.     Fisher ER, Hayden MR. Multisource ascertainment of Huntington disease in Canada: prevalence and population at risk. Mov Disord 2014; 29(1): 105-14.
  3.     Rawlins MD, Wexler NS, Wexler AR, et al. The Prevalence of Huntington’s Disease. Neuroepidemiology 2016; 46(2): 144-53.
  4.     Langbehn DR, Brinkman RR, Falush D, Paulsen JS, Hayden MR, International Huntington’s Disease Collaborative G. A new model for prediction of the age of onset and penetrance for Huntington’s disease based on CAG length. Clin Genet 2004; 65(4): 267-77.
  5.     Nopoulos P, Epping EA, Wassink T, Schlaggar BL, Perlmutter J. Correlation of CAG repeat length between the maternal and paternal allele of the Huntingtin gene: evidence for assortative mating. Behav Brain Funct 2011; 7: 45.
  6.     Ross CA, Aylward EH, Wild EJ, et al. Huntington disease: natural history, biomarkers and prospects for therapeutics. Nat Rev Neurol 2014; 10(4): 204-16.
  7.     Divino V, Dekoven M, Warner JH, et al. The direct medical costs of Huntington’s disease by stage. A retrospective commercial and Medicaid claims data analysis. J Med Econ 2013; 16(8): 1043-50.
  8.     Jones C, Busse M, Quinn L, et al. The societal cost of Huntington’s disease: are we underestimating the burden? Eur J Neurol 2016; 23(10): 1588-90.

Image Source