Why the study?
Most therapies developed in preclinical stages fail to reach clinical trials or achieve marketing approval, highlighting challenges in translational research for neuromuscular disorders despite recent advances.
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Design
Review article
Low translation rates persist despite neuromuscular advances; leaves open need for better preclinical-to-trial pipelines.
Although around half of US Food and Drug Administration (FDA)-approved drugs originate from discoveries made in academic research laboratories, the US National Institutes of Health (NIH) estimates that nearly 90% of therapies developed in preclinical stages never reach clinical trials. From those in clinical trials, only 10% obtain marketing approval. Despite the recent advances in our understanding and diagnosis of neuromuscular disease, and the development of rational therapies in clinical trials, these numbers have not changed dramatically over the past two decades. This article discusses the advantages and challenges for translational research initiated from academia, and the trend towards bridging the gap between discovery and translation to the clinic. A focus is made on recent advances in therapeutic development for neuromuscular disorders.
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Cowling et al. (2019) studied this question.
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