| Summary: | Recommendation: The CADTH Canadian Drug Expert Committee (CDEC) recommends that patisiran be reimbursed for the treatment of polyneuropathy in adult patients with hATTR amyloidosis only if the following conditions are met. Conditions for Reimbursement Initiation Criteria 1. Confirmed genetic diagnosis of hATTR. 2. Patients who have all of the following characteristics: 2.1 are symptomatic with early-stage neuropathy, defined as 2.1.1 polyneuropathy disability [PND] stage I to [less than or equal to sign] IIIB, or 2.1.2 familial amyloidotic polyneuropathy [FAP] stage I or II 2.2 do not exhibit severe heart failure symptoms (defined as New York Heart Association [NYHA] class III or IV) 2.3 Have not previously undergone a liver transplant. 3. Patisiran should not be used in combination with other interfering ribonucleic acid drugs or transthyretin stabilizers used to treat hATTR. Discontinuation Criteria 1. An initial assessment of treatment response should occur nine months after treatment initiation. Thereafter, patients should be assessed at least every six months to determine whether they would benefit from continued treatment with patisiran. 2. Treatment with patisiran should be discontinued for patients who are: 2.1 permanently bedridden and dependent on assistance for basic activities of daily living, or 2.2 receiving end-of-life care. Prescribing Conditions 1. The patient must be under the care of a specialist with experience in the diagnosis and management of hATTR. Pricing Conditions 1. Reduction in price
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