WHAT IS THE REIMBURSEMENT RECOMMENDATION FOR AYVAKYT?: Canada’s Drug Agency (CDA-AMC) recommends that Ayvakyt be reimbursed by public drug plans for the treatment of adult patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL), only if certain conditions are met. WHICH PATIENTS ARE ELIGIBLE FOR COVERAGE? Ayvakyt should only be covered to treat adult patients with AdvSM who have a diagnosis of ASM, SM-AHN, or MCL based on WHO criteria and have an Eastern Cooperative Oncology Group (ECOG) performance status score (which estimates patients’ ability to perform activities of daily living) of 0 (no symptoms, fully active) to 3 (symptomatic, confined to bed or chair for 50% or more of waking hours). Patients with AdvSM who have low platelet counts are not eligible for Ayvakyt. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Ayvakyt should only be reimbursed if prescribed by medical teams with access to expertise in the diagnosis, treatment, and response evaluation of patients with AdvSM. It should only be reimbursed if the cost of Ayvakyt is reduced and the economic feasibility of Ayvakyt is addressed. WHY DID CDA-AMC MAKE THIS RECOMMENDATION? • Evidence from 2 clinical trials suggested that treatment with Ayvakyt causes tumours to shrink or disappear, and response to treatment is durable in patients with AdvSM. • Ayvakyt meets some patient needs as it is an additional treatment option that may offer improved disease control. • Based on the CDA-AMC assessment of the health economic evidence, Ayvakyt does not represent good value to the health care system at the public list price. A price reduction is therefore required. • Based on public list prices, Ayvakyt is estimated to cost the public drug plans approximately $149 million over the next 3 years.
WHAT IS ADVSM? AdvSM is a rare disease with which mast cells (a type of white blood cell) are overproduced and accumulate in bone marrow and other parts of the body. The overproduction of mast cells can cause variable symptoms, such as skin hives or rash, bone and/or muscle pain, fatigue, diarrhea, nausea, vomiting, allergic reactions, anxiety, and depression. The estimated AdvSM incidence rate in Canada is 0.06 cases per 100,000 adults. UNMET NEEDS IN ADVSM: There is a need for effective treatments that improve disease control with fewer symptoms, have fewer adverse effects, and improve quality of life. HOW MUCH DOES AYVAKYT COST? Treatment with Ayvakyt is expected to cost $37,614 per 28-day cycle ($490,362.40 annually).
