Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy

Erik LandfeldtEmail author View ORCID ID profile,
Hanns Lochmüller and
Peter Lindgren

Orphanet Journal of Rare Diseases201914:75

https://doi.org/10.1186/s13023-018-0975-3

Received: 3 October 2017
Accepted: 10 December 2018
Published: 2 April 2019

Recently, Ryder et al. [1] published results from a systematic literature review of the burden, epidemiology, costs, and treatment of Duchenne muscular dystrophy (DMD), a rare, terminal, neuromuscular disease for which several molecules currently are being tested in trials. The review provides a somewhat peculiar perspective of the current body of evidence of these aspects of DMD, in particular the economic burden and patient quality of life, which in our opinion may result in misconceptions of prevailing data gaps concerning evidence employed in health technology assessments. In addition, there are several components of the synthesis and reporting of the identified evidence which warrant clarification to avoid confusion. The purpose of this commentary is to highlight some of the shortcomings in the search strategy and synthesis of cost and quality of life data reported by Ryder et al. that ultimately result in a description that we find is at odds with our understanding of the health economic context of DMD.