Eight industry partners to support Phase 2 of Project HERCULES in 2019
Please note: Project HERCULES is an ongoing project. Please visit the Project HERCULES website for the latest information.
19th December 2018
Duchenne UK are delighted to announce that eight industry partners will support Phase 2 of Project HERCULES in 2019
Catabasis Pharmaceuticals, Pfizer, PTC Therapeutics International Ltd, Roche, Sarepta Therapeutics, Solid Biosciences LLC, Santhera Pharmaceuticals Holding AG and Wave Life Sciences USA, Inc will join Duchenne UK and other partners in this ground breaking global project.
Project HERCULES will develop a disease level economic model and other tools to better demonstrate the real value of new treatments for DMD for Health Technology Assessments and reimbursement decisions.
Project HERCULES has already achieved real advances in our understanding of DMD. We are bringing together possibly the largest collection of clinical data on DMD to develop a better understanding of which aspects of the disease are most important to patients and how they might best inform an economic model. We interviewed people living with DMD to understand better what has the most impact on their quality of life and will be publishing this in 2019. We have identified key evidence gaps and commissioned a burden of illness study to address these in 2019.
This project is a multi-stakeholder collaboration with international applicability. By engaging with global patient organisations, we hope it will pave the way forward for more evidence generation to aid reimbursement decisions worldwide for DMD and other rare diseases.
We were delighted that, in November, Project Hercules was awarded the EURORDIS Black Pearl Award for Patient Engagement in recognition of its innovative approach to bringing patients, clinicians, industry, academics, HTA organisations and others together to address the challenges of assessing new treatments in DMD.
Project HERCULES will deliver:
- A bespoke, validated, Quality of Life metric being developed by the University of Sheffield
- A natural history model developed for bringing together the largest collection of clinical data in DMD for multiple registries and trials. This work is led by the University of Leicester
- A burden of illness study that will better capture the true impact of DMD on patients and their families being developed by HCD Economics
- A disease level economic model that will be produced by SourceHEOR
Together these tools will support companies taking new treatments through HTA and reimbursement processes and should lead to faster, better and more consistent decision making and improved access to medicines for patients.
The concept of Project HERCULES could also benefit other rare conditions struggling to develop strong evidence of the value of new treatments.
Emily Crossley, Co-founder of Duchenne UK said:
Project HERCULES has gone from an idea to a real working collaboration in just over a 12 months which is a huge achievement. Duchenne UK is thrilled to be leading such a ground breaking initiative and are grateful to our industry partners for seeing the value of Hercules and for working with us to address one of the major challenges of drug development. We would like to thank all the boys, men and their families who have taken part in some of the data generation for HERCULES, and all of our charity and academic partners who have helped drive this forward.
Josie Godfrey, Project Director for Project HERCULES said:
I am delighted to be working with such engaged patients, companies, academics, clinicians and HTA agencies on a project that will help ensure new treatments for DMD have the best possible tools and evidence to support access and reimbursement decisions.
Dr Elin Haf Davies, CEO of Aparito said:
Project HERCULES is an extremely exciting initiative that has all the ingredients to transform patient access to drugs through collaborative, cross-industry working. We’re delighted to offer regulatory support in ensuring the EMA/ HTA Qualification of this approach and the various elements it consists of.
Janis Clayton, VP & GM UK Region, PTC Therapeutics Limited said:
This highly ambitious and visionary project is committed to providing the materials needed to address the challenges of patient access to new therapies for Duchenne. It successfully brings together the knowledge and perspectives of patient organisations, industry and academia. PTC have supported the project from its initiation and it is wonderful to see the progress made to date, as well as the enthusiastic engagement from all parties for Phase 2. Many congratulations to Duchenne UK on this great achievement.
For more information visit: hercules.duchenneuk.org/
Phase 1 of the project involved seven leading pharmaceutical companies developing treatments for Duchenne Muscular Dystrophy, are collaborating with the charity on Project HERCULES, to develop a robust and relevant evidence base for payers to accelerate the time it takes for payers to make decisions about which medicines to fund.
Q & A on Hercules:
What is Project HERCULES?
Project HERCULES is the name given to a collaboration between Duchenne UK, and pharmaceutical companies who are developing medicines to treat Duchenne Muscular Dystrophy, DMD, to increase the chances of patients with DMD of accessing innovative treatments.
With a wave of treatments in, or about to enter clinical trials, and with some already gaining approval, the aim of HERCULES is to address the next challenge after medicines are approved: how we get governments to pay for these medicines. This is called reimbursement.
In order to persuade government bodies to pay for new medicines, companies often have to provide complex data analysis and burden of care studies which are costly and time-consuming. The aim of HERCULES is for companies to collaborate together on building such models, to save time, money and ensure that the collaboration speeds up access for patients.
What does HERCULES stand for?
HERCULES stands for HEalth Research Collaboration United in Leading Evidence Synthesis.
Why do we need HERCULES?
We all share the hope that new medicines coming through will improve the lives of those living with DMD and their families. However, we have concerns about how difficult it has been to get countries to make decisions about whether or not to pay for these new medicines.
And we’re concerned that changes to these procedures in the UK could further prevent or delay those with DMD with access to effective new treatments.
Organisations like NICE, the National Institute of Clinical Excellence (NICE) in England, run what’s called Health Technology Assessments
(HTA) to determine whether new medicines offer value for money and should be paid for, known as reimbursement, on the NHS.
In order to get NICE to pay for their medicines, companies are required to develop a vast amount of highly specialized and detailed work which includes data collection, economic modelling and quality of life measurements, all of which can be difficult, time consuming and expensive. The processes are well established and need to be followed. However, we hope that through the collaboration in Hercules, we can help industry better engage with this process and actually speed up the time it takes for industry to engage with bodies like NICE to make decisions
What we propose with HERCULES, is to bring companies together to develop a SHARED model that will focus on developing common tools and practices, including:
*Data analysis, including mapping clinical trial endpoints to clinical outcomes
* An HTA relevant Quality of Life metric
* Cross-sectional study looking at the true Burden of Illness
* Economic model for individual companies to adapt to their products * Training, expert input and mock appraisal committees
How does HERCULES fit in with other campaigns?
We recognise that many patient organisations are active in this area and will be having conversations with HTA agencies and other agencies over the coming months. HERCULES is intended to complement and build on the work already underway within the DMD community. Project HERCULES aim is to provide both practical and tactical support to companies, and to also to all patient groups, so that interactions with HTA bodies are cohesive, persuasive and supported with a strong and relevant evidence base.
Who is running project HERCULES?
Duchenne UK is delighted that, Josie Godfrey, a former Associate Director at NICE, who set up the Highly Specialised Technologies Appraisals System, is running HERCULES. The Steering Group will be chaired by Fleur Chandler who works in Health Value Outcomes at GSK and who also sits on the Patient Advisory Board of Duchenne UK. Together they bring much valuable experience of HTA methods and processes.
How to contact Project HERCULES?
We’re also pleased to share with you an article in Pharmaco- economics, by Anthony Hatswell and Fleur Chandler:
Duchenne UK is extremely grateful to Anthony Hatswell, Professor Ron Akehurst and the Bresmed Foundation for their early support for the project.
We would also like to thank the legal team at CMS for their advice and support.
For more information please read our HERCULES FACTSHEET.
Published on 19 December 2018Share this articleCategories Accelerating drug development