WHAT IS THE REIMBURSEMENT RECOMMENDATION FOR WAINUA?: Canada’s Drug Agency (CDA-AMC) recommends that Wainua be reimbursed by public drug plans for the treatment of polyneuropathy (PN) associated with stage I or stage II hereditary transthyretin amyloidosis (hATTR) in adults, if certain conditions are met. WHICH PATIENTS ARE ELIGIBLE FOR COVERAGE? Wainua should only be covered to treat adults with stage I or stage II genetically confirmed hATTR with PN (hATTR-PN) who are symptomatic with early-stage neuropathy, do not have severe heart failure symptoms, and have not had a liver transplant. A patient’s response to treatment with Wainua should be assessed at least every 6 months to determine whether they would benefit from continued treatment. Treatment with Wainua should not be continued in patients who are permanently bedridden and dependent on assistance for basic activities of daily living or who are receiving end-of-life care. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Wainua should only be reimbursed if the patient is under the care of a specialist with experience in the diagnosis and management of hATTR-PN, and should not be reimbursed if it is used in combination with interfering ribonucleic acid (RNA) drugs or transthyretin stabilizers. The cost of Wainua should be reduced so that it does not cost more than other drugs for hATTR. WHY DID WE MAKE THIS RECOMMENDATION? • Evidence from a clinical trial demonstrated that, in patients with hATTR-PN, treatment with Wainua reduces TTR protein production and improves neuropathy-related neurologic function and health-related quality of life (HRQoL), as measured by modified Neuropathy Impairment Score plus 7 (mNIS+7) score and Norfolk Quality of Life questionnaire – Diabetic Neuropathy (QoL-DN) score, when compared to treatment with placebo. • Wainua may meet some needs that are important to patients because it provides another subcutaneous (SC) drug option that can be administered in a patient’s home, which addresses a need identified by patients. • Based on our assessment of the health economic evidence, Wainua does not represent good value to the health care system at the public list price. The committee determined that there is not enough evidence to justify a greater cost for Wainua compared with currently available treatments for hATTR-PN. • Based on public list prices, Wainua is estimated to cost the public drug plans approximately $800,000 over the next 3 years; however, the actual budget impact is uncertain.
WHAT IS HATTR-PN? hATTR is an inherited condition caused by alterations in a gene that makes a protein called TTR, and results in the misfolding of the TTR protein. In people with hATTR, this misfolded protein forms into abnormal fibrous tissue called amyloids, which can build up in the body’s organs and peripheral nerves causing organs to not function properly as well as nerve damage. In patients with hATTR-PN, amyloids primarily build up in the peripheral nerves. hATTR is considered a rare disease, affecting about 10,000 people worldwide. UNMET NEEDS IN HATTR-PN: Patients with hATTR-PN need effective treatments that slow disease progression, have a low risk of adverse events (AEs), improve HRQoL, improve convenience and independence, and have less frequent dosing. HOW MUCH DOES WAINUA COST? Treatment with Wainua is expected to cost approximately $572,164 per patient per year.
