37690 - Muscular Dystrophy: Drugs (Answered)

Greg Mulholland
To ask the Secretary of State for Health, following the announcement on Twitter by the Parliamentary Under-Secretary of State for Life Sciences on 15 April 2016 that a Managed Access Agreement had been reached for the Translarna drug, for what reasons NICE granted a further eight weeks on 4 May 2016 for discussions to take place.

George Freeman

On 15 April 2016, the National Institute for Health and Care Excellence (NICE) announced in its final draft guidance that it was recommending Translarna (ataluren) for the treatment of children aged five and over with Duchenne muscular dystrophy caused by a nonsense mutation.

Translarna was recommended only when (a) the company provides it at a discounted price to the National Health Service under the patient access scheme agreed with the Department and (b) that NHS England and the manufacturer, PTC Therapeutics, in conjunction with patient representatives, agree a managed access agreement setting out (i) the criteria for starting and stopping treatment, which individual patients would be required to agree, (ii) data collection to address considerable uncertainty in the evidence base and (iii) additional confidential financial controls between the company and NHS England.

On 4 May 2016, NICE announced that it was allowing extra time for further discussions on access arrangements for Translarna to take place before its final guidance is published. NHS England and PTC Therapeutics have been asked to reach agreement on the cost of the drug to the NHS by 7 July 2016.