Project Hercules to lead way in Duchenne muscular dystrophy

For the first time, pharmaceutical companies are sponsoring a project, led by Duchenne UK, to generate, align and share disease-level data across an entire condition for health technology assessments (HTA). The collaboration, known as HERCULES, will focus on medicines for Duchenne muscular dystrophy.

The unique initiative 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). The University of Leicester and University of Sheffield have also partnered on HERCULES in the development of a quality of life metric and data analysis. 

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 UK1 and 30 million people across Europe2. Yet only 5 percent of rare diseases have a licensed treatment option in the UK.3 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.

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