Seven pharmaceutical companies are banding together to sponsor Project Hercules — an initiative that addresses evidence generation for Duchenne muscular dystrophy (DMD) treatments.
The project, led by the non-profit organization Duchenne UK, aims to simplify the way evidence is generated for health technology assessments by authorities such as Great Britain’s National Institute for Health and Care Excellence (NICE).
Throughout the European Union, local health authorities — assessing both benefits and cost of new medications — have the right to rule against a treatment, even if it has been approved by the 28-member trade bloc.
The companies sponsoring Project Hercules are Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, Solid Biosciences, Summit Therapeutics and Wave Life Sciences. In addition, researchers from England’s Universities of Leicester and Sheffield will provide a tool for measuring and analyzing quality of life data.
Duchenne UK and its partners hope the work will create more certainty over the value of Duchenne therapies, which would help them being endorsed by local health authorities.
“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,” Emily Crossley, co-CEO of Duchenne UK, said in a press release. “By pooling evidence through Hercules, all partners involved can avoid ‘reinventing the wheel’ by producing evidence for similar submissions.”
As for other rare diseases, the generation of robust data for DMD treatments is hampered by the scarcity of affected patients and limited resources.
“We’re delighted that our industry partners have joined us to lead the way in this collaborative approach,” Crossley said. “This could not only help industry and assessment bodies such as a NICE, but ultimately ensure that new treatments for DMD get to the boys who need them the most.”
Added Fleur Chandler, future pipeline head of value evidence and outcomes at GlaxoSmithKline: “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 modeling 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.”