Pharmacoeconomic review report: Nitisinone (Orfadin) (Sobi Canada Inc.)
Although nitisinone can be used in both pediatric and adult populations, the manufacturer's economic evaluation is based on a subset of the labelled indication: the treatment of newborns who are identified and treated within one month of birth, either through a newborn screening program or thro...
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| Format: | eBook |
| Language: | English |
| Published: |
Ottawa (ON)
CADTH
2018, April 2018
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| Series: | CADTH common drug review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | Although nitisinone can be used in both pediatric and adult populations, the manufacturer's economic evaluation is based on a subset of the labelled indication: the treatment of newborns who are identified and treated within one month of birth, either through a newborn screening program or through physical examination, patient history, and specialized tests (including urine succinylacetone levels). The submitted cost-utility analysis compares costs and health benefits associated with nitisinone 1 mg/kg plus best supportive care (BSC; dietary restriction) versus BSC (dietary restriction) alone in the treatment of newborns at least one month old, from a health ministry perspective. The model is based on three health states: diagnosed with HT-1; liver transplant with associated tunnel states; and dead. The time horizon was a patient's lifetime (100 years), and future costs and benefits were discounted at 1.5% annually. Nitisinone (Orfadin) is indicated for the treatment of 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 for oral administration. The submitted price of nitisinone is based on dose: 2 mg ($22.50), 5 mg ($53.30), 10 mg ($100), and 20 mg ($193.33). The recommended initial dosage in pediatric and adult populations is 1 mg/kg body weight daily divided in two doses administered orally. If plasma or urine succinylacetone is still detectable one month after starting treatment, the dosage should be increased to 1.5 mg/kg daily to a maximum dosage of 2 mg/kg daily. If the biochemical response is satisfactory, dosage should be adjusted only according to body weight gain. Comparative treatment effect was based on assumptions regarding premature mortality taken from a study of a Quebec-based cohort (Larochelle et al.) of newborns followed for up to 14 years. Utility values were derived from a cohort of men (average age 54 years) with decompensated cirrhosis resulting from chronic hepatitis B infection and measured with the Health Utility Index Mark 3 (HUI3) instrument. Assumptions regarding resource use and costs were derived from a Quebec-based study of HT-1 patients as well as a British Columbia-based study of liver transplantation costs |
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| Physical Description: | 1 PDF file (29 pages) illustrations |