Herculean task for DMD
How Duchenne UK’s HERCULES consortium hopes to improve access to DMD therapies
Driven by the delayed reimbursement decision for Europe’s only approved DMD therapy, the patient advocacy group Duchenne UK has brought together seven companies in the space to create a set of shared tools that should expedite HTA evaluations for all of their therapies.
Project HERCULES was launched in January with Pfizer Inc., PTC Therapeutics Inc., Roche, Sarepta Therapeutics Inc., Solid Biosciences Inc., Summit Therapeutics plc and Wave Life Sciences Ltd. Steering Committee members include CureDuchenne and Parent Project Muscular Dystrophy (PPMD).
Duchenne UK co-founder and co-CEO Emily Crossley told BioCentury the impetus for the consortium arose out of the mounting frustration of parents of children with Duchenne muscular dystrophy (DMD) during NICE’s health technology assessment (HTA) of Translarna ataluren from PTC.
The small molecule