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

NHS England. Published online: 7 July 2016

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Image shows histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells.

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.

Read the full release here

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