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Putting our best foot forward: Clinical, treatment‐based and ethical considerations of nusinersen therapy in Canada for spinal muscular atrophy
Author(s) -
Vukovic Sonya,
McAdam Laura,
ZlotnikShaul Randi,
Amin Reshma
Publication year - 2019
Publication title -
journal of paediatrics and child health
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.631
H-Index - 76
eISSN - 1440-1754
pISSN - 1034-4810
DOI - 10.1111/jpc.14226
Subject(s) - spinal muscular atrophy , medicine , sma* , life expectancy , pediatrics , orphan drug , clinical trial , disease , physical therapy , intensive care medicine , physical medicine and rehabilitation , bioinformatics , population , mathematics , environmental health , combinatorics , biology
Spinal muscular atrophy (SMA) is the most common genetic cause of infant mortality. SMA is a spectral disorder and is categorised based on symptom onset and severity. The median life expectancy for infants with SMA presenting before 6 months of age is less than 2 years without respiratory support. To date, there is no cure for SMA. In June 2017, nusinersen was approved in Canada as the first disease‐modifying drug for SMA because of its demonstrated benefits on motor function and survival in clinical trials. However, with a price tag of almost 1 million dollars for the first year of therapy, careful clinical, treatment‐based and ethical consideration of the principles of (i) best interests; (ii) universality; (iii) portability; (iv) public administration; (v) accessibility; and (vi) comprehensiveness are important guideposts to ensure transparent and equitable allocation of health‐care resources for nusinersen and all other future orphan drugs.