Access to treatments

Women and Duchenne – Fleur Chandler, Project HERCULES Chair

To celebrate World Duchenne Day on 7th September 2022, with this year’s theme of ‘Women in Duchenne’, Duchenne UK is amplifying the voices of inspiring women working in or impacted by Duchenne muscular dystrophy (DMD). Whether they are scientists, doctors, mothers and caregivers or carriers, women are playing a vital role in the fight against DMD, a rare genetic disease that mainly affects boys.   

Fleur Chandler is a health economist and currently Head of Market Access at Sanofi, a worldwide pharmaceutical and healthcare company. She is also mum to Dominic, a 16-year-old boy living with DMD. 

By bringing this professional and personal experience together, Fleur came up with the concept for Project HERCULES: a global project aiming to increase the chances of DMD patients accessing innovative treatments. As both a mother and industry expert, she is uniquely positioned to support Duchenne UK’s efforts to improve access to life-changing drugs.  

Tell us about Project HERCULES – what does the project aim to do?  

“The project aim (which have now been achieved!) was to create a gold standard disease level evidence base for DMD to be used in health technology assessments and reimbursement decisions across the world.  

In order to decide whether to pay for new treatments (reimbursement), healthcare decision-makers need to see a specific set of data, including how the natural history of the disease, quality of life measures, and health economic modelling (cost effectiveness).  

Through creating this evidence base, the ultimate aim of Project HERCULES is to increase the chances of DMD patients being able to access safe and effective new treatments.” 

Why was the project needed? Why is it difficult to access rare disease drugs? 

“Drug development in rare disease is very challenging, due to low numbers of patients, which means the trials and outcome measures need to be very carefully thought through and evaluated.

Clinical trials are used to measure efficacy and safety, as well as real-world evidence. Regulatory frameworks for approving rare disease medicines on this basis are well established. However, there is a fourth hurdle for pharmaceutical companies: the reimbursement of these drugs. Companies need to establish the value of the medicine and show that it is worth paying for. 

These companies need to recoup drug development costs, so medicines for rare diseases often need high price tags. To demonstrate value for money, this requires very specific evidence, and this is hard to generate in rare diseases with small populations.”  

Why is collaboration so integral to this work?  

“There are different elements to collaboration: one is around bringing the relevant parties together to enable the patient voice to be listened to and heard. Collaboration is essential, across patients, academics, clinicians and industry, so that patient voice can be articulated in a way that a reimbursement authority will understand.

The second is collaborating between companies, to enable evidence to be generated in an efficient and respectful way. In rare diseases, there are multiple pharmaceutical companies needing the same types of disease-level evidence, but only a small pool of patients available to be assessed. This can lead to duplication of, or worse, contradictory, evidence generation which is unhelpful. By working together and asking the questions only once, we can create a coherent and high-quality evidence base. 

Finally, it is essential to be able to access pre-existing data and be able to consolidate it.  Project HERCULES has accessed disease registries from across the world to construct of a natural history model of DMD which can be used to underpin health technology assessment submissions. Excitingly, it has identified an additional disease stage in DMD, called the ‘transfer’ stage.”   

How has your background in the pharmaceutical industry informed this work? 

“I am proud I have worked in the pharmaceutical industry for 30 years, enabling access to many medicines for patients in the UK and across the world.  When I first started looking into how I could use my professional experience, knowledge, capability and contacts to enable access for treatments in DMD, it seemed a little daunting as it was so personal. It has helped that the companies I have worked for have no drugs in development in DMD, which made it easy to be impartial. I spoke to my boss at the time, to ask permission to lead what became Project HERCULES and she was extremely encouraging and said to me that it would be a crime if I didn’t do it!”  

How has your experience as a mother of a boy with a rare disease informed this project, and your career in general?  

“In one word, empathy. Every person’s journey with disease in the family is different, yet we all understand each other.  By having empathy with a patient community and population it gives you the credibility and trust to do a better job.”  

What do you believe can be achieved by initiatives like Project HERCULES? What change would you like to see in future for parents impacted by DMD and other rare diseases?  

“Project HERCULES has been cited globally as an example of best practice.  To my knowledge, it’s the first true collaboration across patient groups, industry, academia, clinicians and authorities, and not only that, but led by a patient organisation! 

It’s a model that works well in rare disease and I would like to see it replicated for other rare diseases too.” 

Find out more about the work Duchenne UK is funding to increase access to effective treatments

World Duchenne Awareness Day is an initiative from the World Duchenne Organization which takes place each year on 7th September.  Find out how to get involved on the WDO website.

Published on 6 September 2022

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