Charity-industry initiative launched focusing on DMD treatment

A new charity-industry initiative has been launched by Duchenne UK, a muscular dystrophy charity, aimed at pioneering a new approach to health technology assessment.

The ‘HERCULES’ project — a collaboration of pharma industry partners — will focus on medicines used to treat Duchenne muscular dystrophy (DMD). This will be the first collaboration of its kind where companies will generate, share and align disease level data across an entire condition.

“Larger pharma companies can often commit considerable internal resources to the health technology appraisal process, creating bespoke economic models and quality of life measures for example. This is not always the case for smaller companies, which risks their submissions being more uncertain and patients losing out,” explained Emily Crossley, Co-Chair of Duchenne UK. “HERCULES aims to pool resources in advance for the benefit of every company involved, drastically reducing the time spent ‘reinventing the wheel’ on evidence for similar appraisals down the line. This has the potential to completely transform the way we look at HTA submissions to bodies like NICE, initially in DMD. But it could be equally transformative if adopted in other rare and orphan diseases.”

“With HERCULES, we have a unique opportunity to develop the evidence for DMD collaboratively, reducing duplication and ultimately helping to get new treatments for DMD to the boys who need them most,” added Fleur Chandler, a health economist in the pharmaceutical industry, who leads the steering group for the HERCULES initiative and mother of a child (Dominic) with DMD. “It’s also a great example of a patient organisation taking the lead in solving a problem that the industry has talked about for a long time, but where limited progress has been made to date. We’re absolutely delighted to be working with Duchenne UK to make this happen, as it has the potential to make an enormous difference to the way treatments for rare diseases are assessed.”

“This project is a great opportunity to create the most robust evidence base for appraising new treatments for DMD. We’re doing this as part of our mission to help bring hope to families affected by DMD, and look forward to the future where our HERCULES data package can provide efficient and streamlined access to innovations in this devastating disease,” summarised Josie Godfrey, director at JG Zebra Consulting and formerly associate director for Highly specialised Technologies and Topic Selection at NICE.

Although this new initiative is focused on DMD, it is hoped that it will pave the way for similar approaches to be started in other rare diseases.