Pharma industry leaders join forces in support of DMD project, HERCULES
Leaders from the pharmaceutical industry are set to join forces for the generation, alignment and sharing of disease-level data across an entire condition for health technology assessments (HTA).
The project, led by Duchenne UK, will be known as HERCULES and will focus on medicines for Duchenne muscular dystrophy (DMD) — a rare and fatal genetic disease that almost exclusively affects boys. Sponsors of the project from industry include Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, Solid Biosciences, Summit (Oxford) and Wave Life Sciences USA.
In addition to sponsors from industry, the project has gained the support from the University of Leicester and University of Sheffield, which will be involved in the development of a quality of life metric and data analysis. HERCULES will be aimed at simplifying the way in which evidence is generated for submissions to HTA bodies, such as the National Institute for Health and Care Excellence (NICE) and the Haute Autorité de Santé (HAS).
“As a parent of a child with Duchenne, it can be heartbreaking to know that access to life-changing treatments might be delayed because the assessment bodies don’t have the right evidence to make a decision. By pooling evidence through HERCULES, all partners involved can avoid ‘reinventing the wheel’ by producing evidence for similar submissions,” said Emily Crossley, co-CEO of Duchenne UK. “We’re delighted that our industry sponsors have joined us to lead the way in this collaborative approach. HERCULES has the potential to transform health technology assessment submissions in DMD and other rare diseases. This could not only help industry and assessment bodies such as a NICE, but ultimately ensure that new DMD treatments get to the boys who need them the most.”
“With HERCULES, we have a patient organisation taking the lead to solve a problem that the industry has talked about for a long time, but where limited progress has been made to date,” added Fleur Chandler, future pipeline head, VEO at GSK R&D and a member of Duchenne UK’s Patient Advisory Board. “HERCULES gives us the opportunity to develop a much more robust evidence base for Duchenne muscular dystrophy. Some companies can dedicate considerable time and resource to developing the economic modelling and quality of life measures for their submissions. However, this is not the case for all, which runs the risk of uncertainty in decision making and, ultimately, patients losing out.”
Janis Clayton, General Manager at PTC Therapeutics, commented: “HERCULES, led by Duchenne UK, provides an excellent opportunity for innovation and collaboration, bringing together as it does patient organisations, industry and academia. PTC are very pleased to be supporting, and actively involved in, this appropriately ambitious project.”