Nitisinone (MDK-Nitisinone) is an inhibitor of the tyrosine catabolic pathway and is indicated for patients with hereditary tyrosinemia type 1 (HT-1), in combination with dietary restriction of tyrosine and phenylalanine. Nitisinone is available as 2 mg, 5 mg, 10 mg, and 20 mg capsules. The submitted price of nitisinone is based on dose: 2 mg ($14.78), 5 mg ($34.18), 10 mg ($64.70) and 20 mg ($128.10). The recommended initial daily dose of nitisinone is 1 mg/kg body weight divided into two doses administered orally. The dosage of nitisinone should be increased to 1.5 mg/kg/day in patients whose plasma and urine succinylacetone are still detectable one month after starting treatment; a maximum of 2 mg/kg/day may be needed based on evaluation of all biochemical parameters. If the biochemical response is satisfactory, dosage should be adjusted only according to body weight gain.
The manufacturer submitted a cost-utility analysis (CUA) that compared the costs and quality of life associated with nitisinone plus dietary restriction with dietary restriction alone in infants less than 30 days of age over a six-year time horizon, from a public health care system perspective (Quebec used as proxy). The manufacturer did not apply a discount rate to costs and benefits in the CUA. The manufacturer also presented a supplementary cost-consequence analysis, based on a 2010 master’s thesis (and subsequent publication) evaluating the costs associated with nitisinone for the treatment of HT-1. Nutritional therapeutic products containing amino acid supplements free of tyrosine and phenylalanine are reimbursed in some jurisdictions but not included as comparators.
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