Lobby for Translarna
Yesterday Empower: Access to Medicine were proud to have joined the Duchenne Muscular Dystrophy community for a powerful lobby of Parliament and 10 Downing Street.
Dozens of children with Muscular Dystrophy, and their families, joined MPs and Peers in Portcullis House for a summit on a promising treatment - Translarna - as well as delivering a 24,000 strong petition to Number 10.
Translarna is a drug already shown to have a significant effect on 13% of Muscular Dystrophy sufferers (there are many different mutations of the condition). One parent described how her son (the condition mostly affects boys) was able to walk 30 metres further than he had been able to before taking the drug.
Muscular Dystrophy is progressive, causing the muscles to gradually weaken over time which affects mobility and usually leads to some sort of disability over time. The severity of conditions and how they affect individuals varies greatly from person to person, but unfortunately leads to greatly shortened life expectancy due to respiratory failure or other complications. For Duchenne patients, deterioration is a question of when not if.
Translarna is the first treatment in some time to demonstrate real efficacy in a patient cohort, shadow secretary of health; Andy Burnham MP, described it as a "glimmer of hope." It passed all the appropriate clinical hurdles to get to this point, only then to be blocked by NHS England due to funding issues.
No-one is denying that the NHS faces difficult financial challenges, no-one at the event was naïve enough to suggest that the NHS should fund every treatment for every condition. But to deny the first lifeline the Duchenne community have had in some time was hard to take – particularly when it was on financial grounds for the upfront costs of the drug.
The economic argument caused considerable ire amongst parents for a number of reasons. But first and foremost because NHS England’s numbers don’t stack up in the long term. Yes the treatment was expensive at the point of use, but the mitigating effect it appeared to be having on the symptoms of Duchenne Muscular Dystrophy were considerable.
Duchenne is incredibly expensive to manage for both the NHS and parents. From steroids and wheelchairs to ventilators and end-of-life care, the condition has significant ongoing financial consequences for the health service. Therefore the logic dictates that the more patients you can save earlier in the condition, the less demand for their needs later in life.
Nobody likes to talk numbers when considering children’s lives - but yesterday the Duchenne community fought fire with fire.