Access to treatments

Emily Crossley's ISPOR Blog

5th November 2019

Being a graduate of Modern History, the thought of getting familiar with spreadsheets is something that has always terrified me. And I never – in my wildest dreams – believed I would actually get excited by one. But that’s exactly what has happened in the last few months and it’s the reason why I’m in Copenhagen this week for the ISPOR conference. 

ISPOR is the go-to conference for those who work in Health Technology Assessment – by which I mean the people who work out whether or not newly approved medicines offer value for money. This is a very tricky question to answer and it’s one that has plagued the rare disease and DMD communities. 

In 2014, a drug called Translarna (then Ataluren) was given Conditional Marketing Authorisation (CMA) by the European Medicines Agency. Families living in the UK celebrated – this was the first approved drug to treat a subsection of the boys with the devastating muscle wasting disease, Duchenne Muscular Dystrophy. The label on the drug meant you could take it from the age of five, until you lost ambulation. However it took the National Institute for Clinical Excellence (NICE), the body that reviews new drugs for cost effectiveness in England, more than two and half years to make the decision on whether or not to the fund the drug. In that time, boys who were walking and eligible for the drug, stopped walking and became ineligible. A doorway to hope slammed shut in their faces. And I knew in that moment there was something worse than having no drug approved for your disease. And that was having a drug that had been approved for use as safe and effective but that was locked away in a cupboard because no-one could work out how to pay for it. 

So Fleur Chandler came up with a fantastic idea. To get competing companies to work together and build TOGETHER the evidence base they would need to take to bodies like NICE, and give them the strongest possible chance of proving the cost effectiveness of their drugs.

And so Project HERCULES was born.

We now have NINE industry partners.

We have completed most of our workstreams.

And this is where the spreadsheet comes in – in the form of a fully comprehensive economic model that can be tailored to any specific product. It has taken two years to build and is based on two years worth of hard work, collaboration and gathering data.

We’ve also defined a new disease progression model that really describes the patient experience of the disease.

We’re finalising a new Quality of Life Instrument to better capture the impact of a progressive paediatric disease.

And we’ve embarked on a new Burden Of Illness study to properly measure the devastating impact of this disease. 

So now you can see why I’m suddenly interested in spreadsheets; But please don’t’ just take my word for it. Last month, we presented our work to a panel of independent experts. Here’s what Professor Alan Brennan Professor of Health Economics and Decision Modelling had to say: “The work you are doing is inspiring … the commitment and the whole style of the approach is unusual, impressive and beautiful”.


What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) 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.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

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 visit www.duchenneuk.org


Published on 11 May 2019

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