Canadian Organization for Rare Disorders Responds to CADTH Recommendation for SMA Gene Therapy

Gaping Hole for SMA Babies: Why Canada needs National Rare Drug Strategy NOW

CADTH Restricts Access to Zolgensma gene therapy. Canada needs National Rare Disease Drug Strategy Now!

Toronto, Ontario, March 31, 2021 – The positive recommendation by the Canadian Agency for Drugs and Technologies in Health (CADTH) for reimbursement of Zolgensma gene therapy by the public drug plans is eclipsed by the negative impact of the surprising and devastating age restriction. CADTH does recognize the Health Canada approved indications, namely, all SMA-diagnosed pediatric patients with double mutations of SMN1 gene or 3 copies of SMN2 gene. But unlike the funding recommendations in other countries, CADTH has restricted their recommendation to babies under 6 months old. In so doing, they have denied treatment to a significant number of SMA children who are included in Health Canada’s approval for young children up to 21 kg, European Medicines’ approval for 21kg, and the US FDA for children up to age two. 

The good news is that there is a potential lifeline extended by some provinces to those families whose children are over six months old. Families have been petitioning for Zolgensma even prior to its submission to Health Canada in June 2020. Over the past year, some eight to 10 Canadian children have received the gene therapy thanks to Novartis’ compassionate access program or “angel donors.” Now, the patient community is counting on provincial “case-by-case” approvals, previously announced by Alberta, Quebec, and Ontario. 

Among these, only Alberta has actually made treatment available. CORD salutes Alberta for putting the interests of children first and calls upon Quebec and Ontario to follow through and make Zolgensma available immediately to those children over (or near) 6 months who would qualify under the Health Canada approval. We call on all other jurisdictions to make similar provisions for individual access while final criteria are being negotiated. 

CORD also reinforces CADTH’s recommendation for all provinces to immediately implement newborn screening (NBS) for SMA, which would identify most infants at birth and, hopefully, allow them to be treated prior to six months of age. Currently, only Ontario has approved NBS for SMA. Nevertheless, it can be expected that some infants will not be detected or families may move into Canada without a diagnosis, so a pathway for children over six months will continue to be needed.

Coincidentally, this CADTH recommendation comes on the same day that submissions were due to Health Canada on the implementation of a $1 billion National Rare Disease Drug Strategy. CORD has called for an access pathway that would provide access to some treatments immediately upon Health Canada approval, or before based on urgent individual need. Access to Zolgensma for SMA babies and children would have definitely met that criteria … as would other life-saving and life-altering rare disease therapies that need to get to patients as soon as possible.

CONTACT 
Durhane Wong-Rieger
Canadian Organization for Rare Disorders
(647) 801-5176
durhane@raredisorders.ca