A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals (HTA) bodies such as the National Institute for Health and Care Excellence (NICE) and the Haute Autorit de Sant (HAS).
Launched today at the 8th World Orphan Drug Congress in Barcelona, this innovative collaboration entitled HERCULES will focus on medicines used to treat Duchenne muscular dystrophy (DMD). It will be the first time a group of pharmaceutical industry partners have ever collaborated to generate, share and align disease level data across an entire condition.
Although this new initiative focuses on DMD, it paves the way for similar approaches in other rare diseases, and has the potential to improve the chances of medicines in rare and orphan diseases receiving positive HTA decisions. This unique collaborative approach has been welcomed by a range of industry health economists, as well as former NICE personnel.
Launching HERCULES at the conference, Emily Crossley, Co-Chair of Duchenne UK, commented:
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. 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.
Rare conditions affect around 3.5 million people in the UK, yet only 5 per cent of rare diseases have a licensed treatment option in the UK. A key issue in developing treatments for rare diseases is the limited evidence base available due to known issues, such as small patient populations for each disease. These obstacles tend to hinder the HTA approval of life-changing treatments and their subsequent availability to NHS patients, even for medicines that have received regulatory approval.
Fleur Chandler, a health economist in the pharmaceutical industry, who leads the steering group for the HERCULES initiative and is the mother of a child (Dominic) with Duchenne muscular dystrophy, added:
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. Its also a great example of a patient organization 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. Were 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.
Josie Godfrey, Director at JG Zebra Consulting Ltd and formerly Associate Director for Highly specialized Technologies and Topic Selection at NICE said:
This project is a great opportunity to create the most robust evidence base for appraising new treatments for DMD. Were 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.