A new initiative between medical charity Duchenne UK and several pharma companies will try to increase the chances of patients with Duchenne muscular dystrophy (DMD) of accessing innovative treatments.
Project Hercules – said to be the first of its kind – will see seven pharma companies and Duchenne UK “generate, align and share” data in a bid to develop a new framework for generating health technology assessment (HTA) data used in reimbursement decisions.
It aims “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)”, according to Duchenne UK, which is investing £200,000 in the initial stages of the project.
The pharma partners – Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, Solid Biosciences, Summit and Wave Life Sciences – all have an interest in DMD as well as other rare and orphan diseases that can have difficulties meeting HTA requirements.
“With a wave of treatments in, or about to enter clinical trials, and with some already gaining approval, the aim of Project Hercules is to address the next challenge after medicines reach regulatory approval; how we get governments and health services to pay for these medicines,” said the charity.
“It is widely recognised that the pace of innovation in treatments and medicines is outstripping the time it takes to approve them for use in the NHS. These delays are worsened for diseases as there is often limited evidence available to meet the requirements for access and pricing decisions.”
The data-sharing model envisaged by the project will focus on the development of common tools and practices, such as mapping clinical trial endpoints to clinical outcomes and developing a common quality-of-life metric for all HTA submissions, that can make it easier for NICE and similar agencies such as the Haute Autorité de Santé (HAS) in France to carry out appraisals.
Pooled data will also provide a clearer picture on the true burden of an illness and help with the development of economic models that can be used by drug developers to make decisions on their products candidates.
The project is being run by Josie Godfrey, an ex-director of NICE who can bring the HTA perspective to the table, with support from the University of Leicester and University of Sheffield.
“HTA agencies can struggle with decisions in the absence of a solid evidence base, be that data on the burden of the disease or the likely disease progression,” she said.
“Through its collaboration with patient organisations, industry, universities and other partners, Hercules will ensure all companies can produce the best possible evidence for HTA agencies – making decisions easier, fairer and hopefully enabling faster access to the best new treatments for DMD.”
The hope is that the model could also be used for other rare diseases and provide greater certainty over the value that new medicines can play in these conditions.