Newfoundland and Labrador provides coverage for SMA treatments through their Prescription Drug Program (NLPDP) via Special Authorization. Please see below for coverage details of each of the available SMA treatments. Please discuss your eligibility with your health-care provider.

SMA Treatments:

Evrysdi (generic name: risdiplam)

• Coverage criteria can be found here by downloading the PDF and searching for ‘Evrysdi’ in the document. For convenience, full coverage criteria is posted below.

 

Full coverage criteria

 

For the treatment of 5q spinal muscular atrophy (SMA), if the following criteria are met:

• Genetic documentation of 5q SMA homozygous gene deletion or

compound heterozygote.

• Patient does not currently require permanent invasive ventilation.
• Patient who is symptomatic with two or three copies of the SMN2 gene and is:

– 2 months to 7 months of age, or

– 8 months to 25 years of age and non-ambulatory.

 

Discontinuation Criteria:

Treatment should be discontinued upon meeting any of the following circumstances:

• There is no demonstrated achievement in, or maintenance of, motor milestone

function as assessed using an age-appropriate measurement after treatment

initiation in patients aged between 2 months and 2 years at the time of treatment

initiation;

• There is no demonstrated maintenance of motor function (as assessed using an

age-appropriate measurement) after treatment initiation in patients aged between

2 years and 25 years at the time of treatment initiation; or

• permanent invasive ventilation is required.

 

It should be noted that the decision to discontinue reimbursement should be based

on 2 assessments separated by no longer than a 12-week interval, with the first

evaluation taken close to (i.e. within 3 months) of the date of renewal of funding. The

second assessment is only required for patients who demonstrated a decline in

motor milestones/motor function at the time of the first evaluation.

 

Clinical Notes:

• An age-appropriate scale is defined as 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).

• A baseline assessment using an age-appropriate scale must be completed prior to

initiation of treatment.

• Yearly assessments must be completed using an age-appropriate scale no more

than 12 weeks prior to the renewal date.

• 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

 

Claim Notes:

• The patient must be under the care of a specialist experienced in the treatment of SMA.
• Combination therapy with nusinersen will not be reimbursed.
• Requests for risdiplam will not be considered for patients who have received adenoassociated virus (AAV) vector-based gene therapy.
• Patients currently receiving SMA drug therapy may be eligible to switch to an

alternate SMA drug therapy; however, patients will not be permitted to switch back to

a previously trialed SMA drug.

• Approvals will be for a maximum of 0.2 mg/kg/day for patients 2 months to less than

2 years of age, 0.25 mg/kg/day for patients greater than or equal to 2 years of age

weighing less than 20 kg, or 5 mg/day for patients greater than or equal to 2 years of

age and weighing greater than or equal to 20 kg.

• Approval period: 1 year

 

Please visit the link below if you require our standard special authorization form:

http://www.health.gov.nl.ca/health/prescription/standard_specauth_form.pdf.

 

Spinraza (generic name: nusinersen)

Coverage criteria can be found here by downloading the PDF and searching for ‘Spinraza’ in the document. For convenience, full coverage criteria is posted below.

 

Full coverage criteria (please click to expand) [hyperlink to expand]

 

For the treatment of Sq spinal muscular atrophy (SMA), if the following criteria are met:

• Genetic documentation of Sq SMA homozygous gene deletion, homozygous

mutation , or compound heterozygous mutation; AND

• Patient who:

Are pre-symptomatic with genetic documentation of two or three copies of the survival motor neuron 2 (SMN2) gene, OR has had disease duration less than 6 months, two copies of the SMN2 gene, and symptom onset after the first week of birth and on or before 7

months of age, OR is 18 years of age or younger with symptom onset after 6 months of age.

AND

• Patient is not 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 nusinersen treatment.

* 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.

 

Discontinuation Criteria: For continued coverage, the patient must meet the following

criteria:

• Prior to the fifth dose or every subsequent dose
• There is demonstrated achievement or maintenance of motor milestone

function as assessed using age-appropriate scales since treatment initiation

in patients who were pre-symptomatic at the time of treatment initiation; OR

• There is demonstrated maintenance in motor milestone function as assessed

using age-appropriate scales: HINE Section 2, CHOP INTEND, or HFMSE)

since treatment initiation in patients who were symptomatic at the time of

treatment initiation; or

• The patient does not require permanent invasive ventilation*.

Treatment should be discontinued if, prior to the fifth dose or every subsequent dose of

nusinersen, the above renewal criteria are not met.

 

Claim Notes:

• The patient must be under the care of a specialist experienced in the treatment of

SMA.

• Approval Period: 1 year.
• Requests for patients who do not meet the criteria may be considered on a case by-case basis as outlined in the Exceptional Review Process.

 

Please visit the link below if you require our standard special authorization form:

http://www.health.gov.nl.ca/health/prescription/standard_specauth_form.pdf.