Patisiran and inotersen are considered medically necessary when ALL of the following are met:
Individual has a confirmatory diagnosis of hATTR by a genetic test AND tissue biopsy showing amyloid deposition
Presence of clinical signs and symptoms of polyneuropathy characterized by ONE of the following:
Baseline polyneuropathy disability (PND) IIIb or lower (see Table 1 in background section)
Baseline FAP Stage one or two (see Table 1 in background section)
Individual does not have ANY of the following
New York Heart Association (NYHA) class III or IV heart failure
Sensorimotor or autonomic neuropathy not related to hATTR amyloidosis (monoclonal gammopathy, autoimmune disease, etc.).
Prior liver transplantation.
The prescriber is a specialist in the area of the patient’s diagnosis (e.g. neurologist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
The patient does NOT have any FDA labeled contraindications to the requested agent and is intended to be used consistently with the FDA approved label (see policy guidelines).
Patisiran and inotersen are considered investigational in all other situations.
