Policy alternatives for treatments for rare diseases

Abbas H. Panju; Chaim M. Bell

doi:10.1503/cmaj.081429

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PNH Prevalence
Nadeem Chaudhry

Posted on: 18 August 2010

Posted on: (18 August 2010)

PNH Prevalence
Nadeem Chaudhry, Ann Arbor, USA
Agreeably, we need a Canadian policy for treating rare diseases. Most developed nations have adopted such policies by defining criteria for rare diseases.
Canadian prevalence of 2000 PNH patients is significantly high as stated.
Currently, no epidemiological studies exist that clearly establish the incidence and prevalence of PNH in Canada. The most recent publication on PNH prevalence and incidence is...
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Agreeably, we need a Canadian policy for treating rare diseases. Most developed nations have adopted such policies by defining criteria for rare diseases.
Canadian prevalence of 2000 PNH patients is significantly high as stated.
Currently, no epidemiological studies exist that clearly establish the incidence and prevalence of PNH in Canada. The most recent publication on PNH prevalence and incidence is a UK study published in 2006. The study identified all PNH patients by flow cytometry in a population of 3.7 million.1 Based on incidence and survival estimates, the study found the prevalence of patients with a PNH clone in this population to be 15.9/million. Patients with PNH who exhibit evidence of hemolysis are at the highest risk to suffer from the debilitating and life-threatening complications of PNH. While patients with lower clone sizes are generally less likely to exhibit evidence of hemolysis,2 there will be specific patients in this group who are important exceptions since they will be at higher risk for the severe complications of PNH and demonstrate hemolysis. Enriching the population for patients with clone sizes of 10% or greater (43% of all PNH patients and who are hemolytic (82.5% of all PNH patients) provides an estimate that 35% of all PNH patients, or approximately 5.6 patients/million population, are at highest risk for the severe and life-threatening complications of PNH.
References 1. Hill A, Platts PJ, Smith A, et al. The incidence and prevalence of PNH and survival of patients in Yorkshire. Haematologica 2007;92 (suppl 2): abstr 0067. 2. Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005;106:3699-709.
Conflict of Interest:
None declared
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Competing Interests: None declared.