JULY 2019

This week, Fleur Chandler, member of our Patient Advisory Board and chair of Project HERCULES, attended a meeting in Boston, hosted by the Institute for Clinical and Economic Review (ICER). 

ICER is an American organization that reviews new medicines for their cost effectiveness. This review process is known as Health Technology Assessments (HTA). Much like bodies such as the National Institute of Clinical Excellence (NICE) in the UK, they use health economics to assess the cost effectiveness and value of new drugs.

ICER is an independent body, but US insurance companies and states that pay for medicines, use its reviews and outputs to make decisions on whether they will fund the drugs.

ICER is currently reviewing deflazacort, eteplirsen and golodirsen in DMD for cost effectiveness and held their public meeting in Boston yesterday (25th July).

Fleur Chandler attended the meeting to represent the views of Project HERCULES on the economic modelling and evidence used at a disease level.

Project HERCULES is a collaborative global project set up by Duchenne UK to increase the chances of patients with Duchenne Muscular Dystrophy (DMD) of accessing innovative treatments. It brings together pharmaceutical companies to develop a better evidence base to help support their case for the pricing and reimbursement of new treatments for DMD. 

Project HERCULES works internationally: we partner with patient groups from across the world, including PPMD in the US. The majority of the data accessed is from the US via the Critical Path Institute. One of our academic advisers is Professor Jalpa Doshi, a leading US Health economist.

We have shared our findings to date with ICER and offered support during their assessment. However, they are relying on published data that is both sparse and weak evidence, and is a cause for concern. We do not believe the resulting economic modelling is a sound basis for decision making. Through Project HERCULES, we are working to create a better evidence base.

Project HERCULES economic team (Prof Ron Akehurst, Prof Keith Abrams, Juliet Mumby Croft and SOURCE HEOR, along with Dr Philip Powell) have commented extensively on the economic model produced by ICER. Fleur attended the public meeting yesterday to ensure that the teams concerns were heard. Fleur was able to support the US patient organisations by representing patients using her health economist perspective.

It is important that Project HERCULES is represented as Health Technology Assessments start to happen in in DMD across the world.

Our focus is on ensuring the right methodology and evidence are used to fully articulate the impact of DMD on patients, and highlight the need for a robust evidence base to enable high quality HTA.

For more information please visit projecthercules.org

NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

• Direct Debit – Duchenne Direct
• Individual Donation – Donate
• If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
• Take part in one of our fundraising events – Events
• Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information and interview requests:

Visit www.duchenneuk.org

Molly Hunt – Head of Communications, Duchenne UK E: [email protected]