| Summary: | Patisiran (Onpattro) is indicated for the treatment of polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis. Patisiran is administered through IV infusion at a dosage of 0.3 mg per kg of body weight once every three weeks up to a recommended maximum dose of 30 mg. Patisiran is supplied in single-use 2 mg/mL 10 mL vials at a cost of $13,022.02 per vial. The annual cost is between $451,430 and $677,145 per patient, depending on whether two vials (for those between 34 kg and 66 kg) or three vials (for those greater than 66 kg) are required, respectively. The manufacturer submitted a cost-utility analysis of patisiran compared with inotersen in its base case and compared with best supportive care (BSC) in a scenario analysis. The goal of BSC is symptomatic management and may include treatments for polyneuropathy, gastrointestinal disorders, and bladder dysfunction such as pregabalin, metronidazole, and anticholinergics
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