NHS England successfully negotiates access to new drug treatment for children with duchenne muscular dystrophy

Children in England suffering from a rare form of muscular dystrophy should soon be able to access the only drug for their condition after a ground-breaking agreement was reached between NHS England and the manufacturer.

NHS England and the manufacturer, PTC Therapeutics, have successfully negotiated a managed access agreement (MAA) for ataluren (brand name Translarna) for the treatment of children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.

The five year commercial agreement will see a managed access scheme put in place for patients, which includes an outcomes-based incentive for the manufacturer as well as a mechanism to monitor how well the medicine has worked in practice before future funding decisions are taken.

The independent medicines assessor, NICE, recommended ataluren in principle in April 2016, if NHS England was able successfully to negotiate an economically acceptable MAA with PTC Therapeutics.

This agreement will now trigger the publication of final NICE guidance, and plans are being put in place to allow treatment to commence in designated specialist centres soon afterwards.

Confirming the deal, NHS England’s Director of Specialised Commissioning and Deputy Medical Director, Dr Jonathan Fielden, said: “This hard won agreement is good news for patients and their families living with this debilitating and distressing condition, for whom no treatment option currently exists.

“Our innovative agreement with PTC makes the treatment available for patients now, provides us with further evidence on effectiveness to inform future decisions, and importantly allows us to effectively manage our resources across this and other new treatments for other patients.”

“This also signals the approach NHS England will be taking for a wider range of decisions regarding new high cost medicines, where we will look for better ‘win-win’ arrangements with companies that link payments to outcomes, while driving value for taxpayers.”


  1. naif says:

    I need my 4 years Baby Just like others to life normal and enjoying his life.

    No time to waste.

    I can’t see my baby Die every day.

  2. Viv Wicklen says:

    I should think So too!! My 4year old great nephew has just been diagnosed with DMD.My own Father died at 39 with Muscular Atrophy, but he did have children to love and be loved by. These children deserve the same chance.

  3. Mary liquorish says:

    Now get this to these boys ASAP!!! No time to waste!! They need it NOW!!! X