SMA: A very useful compilation of medico-economic studies

SMA has recently experienced a spectacular acceleration at the therapeutic level. Genetically determined, this neuromuscular disease causes paralysis related to the degeneration of peripheral motor neurons and, for some affected children, earlybouleverser death. The arrival on the market of three drugs, Spinraza®, Zolgensma®, and Evrysdi® disrupts the natural history of the disease and raises many questions to families, clinicians and payers, the common denominator of these three products being a very high price. 

In an article published in January 202,1 researchers from the Benelux reviewed all the recent medico-economic-scientific literature related to SMA. They were initially interested in the costs linked to the disease and noted a great disparity in average costs, due to a lack of standardization of studies and due to the variability of the severity, and therefore of the burden of care according to the SMA subtypes. The cost could thus vary, for a population however more homogeneous like the types I, from 75,000 to 196,000 dollars per year. Studies examining the impact of innovative therapies on these costs are only just beginning to be published but are difficult to compare with each other. The only certainty is that the therapies mentioned, at the price level practiced, are not likely to reduce costs significantly when they are administered at a post-symptomatic stage. Another major challenge will be to assess the impact of current newborn screening programs. 


Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments, T Dangouloff, C Botty, C Beaudart et al. Orphanet J Rare Dis. 2021 (Janv).16(1):47.