39151 - Muscular Dystrophy: Drugs (Answered)

Greg Mulholland
To ask the Secretary of State for Health, what recent discussions he has had with NHS England about making Translarna available for use in England.

George Freeman

National Health Service funding decisions for treatments for rare and ultra-rare conditions, such as Translarna (ataluren) for Duchenne muscular dystrophy (DMD), are made by NHS England, as the responsible commissioner for specialised services.

I have been working with the National Institute for Health and Care Excellence (NICE) and NHS England to understand and expedite what can be done to help people access Translarna and have met with MPs, patient groups, drug companies, and parents of children with rare diseases, including DMD, to discuss their concerns about this specific issue.

Following stakeholders’ concerns regarding NHS England’s evaluation processes of treatments for rare conditions, NHS England decided in December 2014 to hold a 90-day consultation on prioritisation for specialised services (held from 27 January to 27 April 2015). This was to ensure that the principles and process for making such decisions were well informed, evidence-led and in line with the expectations of patients and the public.

The Department and the NICE took steps to ensure that Translarna was referred to the NICE as quickly as possible (March 2015) so that final guidance would be available without undue delay. NHS England announced in July that it would wait until the NICE’s guidance was available before developing a funding policy on Translarna.

On 15 April 2016, the NICE announced in its final draft guidance that it was recommending Translarna (ataluren) for the treatment of children aged five and over with DMD caused by a nonsense mutation.

Translarna was recommended only when a) the company provides it at a discounted price to the NHS under the patient access scheme agreed with the Department and b) 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, the 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.

If the NICE’s final guidance recommends Translarna, NHS England will be legally required to fund it.