8 July 2016 - Children in England suffering from a rare form of muscular dystrophy will soon be able to access the only drug licensed for their condition after a ground-breaking agreement was reached between NHS England and PTC Therapeutics.

Under the five-year commercial agreement, 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 made, Translarna (ataluren) can be used to treat children aged five and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.

The move follows a recommendation by the National Institute for Health and Care Excellence earlier this year that the drug, which costs £222,000 per year (before discount) should be available on the NHS if NHS England and PTC could successfully negotiate an economically acceptable managed access agreement.

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, NHS England noted.

VIew Pharma Times article