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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. First, the systematic literature review by Ryder et al. only considered records published up until June 2015. As a consequence, several recent key publications on the economic burden and patient quality of life were not identified. Examples include our own work [2, 3], as well as the work of others [4–9]. In addition, several studies of costs and quality of life in DMD published between 2005 and June 2015 are not included in the review (e.g. [10–16]). For these reasons, the outcomes of the review provide an incomplete description of the current evidence base. Second, Ryder et al. claim they only included studies of patients with DMD, not mixed populations of e.g. patients with muscular dystrophy. This is a reasonable criterion for robust evidence synthesis and meaningful comparison, as e.g. Becker muscular dystrophy and

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