“What Did Maxwell's Equations Really Have to Do With Edison's Invention?”: Addressing the Complexity of Developing Clinical Interventions for Skeletal Muscle Disease

To reach the healthcare market and have a medical intervention reimbursed in any format carries high risk and very low success rates. Even when all regulatory hurdles have been surpassed, there is no guarantee that the product will be purchased; a different body makes that decision using criteria typically unknown to early-stage innovators and intervention developers. In the context of skeletal muscle diseases, the field is at a crossroads; accurate diagnoses are difficult to obtain, patient management and monitoring are equally difficult, cures are evasive, and disease progression is not well enough understood in the human to identify clear targets (irrespective of whether the specific muscle disease is rare or frequent because the progression is slow and the tissue large). Additionally, the generation of fundamental knowledge stemming from pure academic research, which underpins short- and long-term insight and advances, has been stalled or at least slowed. The field also faces challenges common to all healthcare interventions, while there are also some unique barriers to address, in both developmental translation of the therapeutic and obtaining reimbursement approval. This is independent of the number of people globally who suffer directly and indirectly from skeletal muscle degeneration or degradation. This chapter covers key issues facing skeletal muscle intervention translation, problems that seem to be routinely occurring, followed by suggestions on what can and should be done differently.