Trofinetide (Daybue)

What Is the Reimbursement Recommendation for Daybue? Canada’s Drug Agency (CDA-AMC) recommends that Daybue not be reimbursed by public drug plans for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older and weighing at least 9 kg. Why Did CDA-AMC Make This Recommendation? Evidence from 1 clinical trial demonstrated that Daybue resulted in improvements in caregiver-reported behavioural symptoms of Rett syndrome and overall clinical improvement according to clinicians, when compared to placebo. Caregivers of patients with Rett syndrome identified a need for treatments that improve communication skills, motor skills, health-related quality of life (HRQoL), caregiver burden, and other conditions associated with Rett syndrome. However, based on the evidence reviewed in the initial meeting and the reconsideration meeting, the Canadian Drug Expert Committee (CDEC) could not determine if Daybue addresses these needs. The uncertainty was mainly due to a lack of information on whether the results were clinically meaningful, missing data in the study, and a lack of HRQoL data.