Evrysdi – Risdiplam
Available:
EVRYSDIⓇ (risdiplam) has been added to the PEI High Cost Drug Program formulary effective March 27th, 2023 For patients diagnosed with 5q Spinal Muscular Atrophy (SMA) under the care of a specialist with experience in the diagnosis and management of SMA, if the following clinical criteria are met: • Genetic documentation of 5q SMA homozygous gene deletion or compound heterozygote, AND • Patients who: o are symptomatic and have genetic documentation of two or three copies of the SMN2 gene, AND o aged between 2 months and 7 months (inclusive), OR o aged 8 months up to 25 years and are non-ambulatory • Patient is not currently requiring permanent invasive ventilation*, AND • A baseline assessment using an age-appropriate scale (the Hammersmith Infant Neurological Examination [HINE] Section 2, Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND], or Hammersmith Functional Motor Scale-Expanded [HFMSE]) must be completed prior to initiation of risdiplam treatment. • For continued coverage, the patient must meet the following criteria: There is demonstrated achievement or maintenance of motor milestone function (as assessed using age-appropriate scales: the [HINE] Section 2, CHOP INTEND, or HFMSE) after treatment initiation in patients aged between 2 months and 2 years at the time of treatment initiation; OR o There is demonstrated maintenance of motor milestone function (as assessed using age-appropriate scales: the HINE Section 2, CHOP INTEND, or HFMSE) after treatment initiation in patients aged between 2 years and 25 years at the time of treatment initiation; AND o Patient does not require permanent invasive ventilation*. The decision to discontinue reimbursement should be based on 2 assessments separated by no longer than a 12-week interval. Claim Notes: • Approval: 12 months
The specific criteria for reimbursement are in line with recommendations made by CADTH in 2021.
*Permanent invasive ventilation is defined as the use of tracheostomy and a ventilator due to progression of SMA that is not due to an identifiable and reversible cause.