Pfizer and Roche are among the pharma companies throwing their weight behind a new project aiming to pioneer novel approach to health technology assessments (HTA).
Known as HERCULES, the collaboration will focus on medicines for Duchenne muscular dystrophy – a genetic disorder characterised by progressive muscle degeneration.
The initiative, launched by the Duchenne muscular dystrophy research charity Duchenne UK, aims to radically simplify the way 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).
While this new initiative focuses on Duchenne muscular dystrophy, it paves the way for similar approaches in other rare diseases, and has the potential to better demonstrate the value of medicines for rare and orphan diseases.
It is hoped that greater certainty over the value of these medicines could help to improve their chances of receiving positive HTA decisions.
Rare conditions affect around 3.5 million people in the UK and 30 million people across Europe. Yet only 5% of rare diseases have a licensed treatment option in the UK.
Small patient populations and limited resources to build a robust evidence base are key hurdles in developing treatments for rare diseases.
These obstacles tend to hinder the HTA approval of potentially life-changing treatments for rare diseases and their subsequent availability to NHS patients, even for medicines that have received regulatory approval.
Emily Crossley, Co-CEO of Duchenne UK, said: “By pooling evidence through HERCULES, all partners involved can avoid ‘reinventing the wheel’ by producing evidence for similar submissions.”
She added: “HERCULES has the potential to transform health technology assessment submissions in Duchenne muscular dystrophy and other rare diseases.
“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.”