Duchenne muscular dystrophy (DMD) patients and their families have welcomed a decision by NICE making PTC Therapeutics’ Translarna available on the NHS.
The UK’s cost-effectiveness watchdog has backed the use of Translarna (ataluren) for DMD patients aged five and over whose disease is caused by ‘nonsense’ mutations and who are able to walk, provided the drug is prescribed within the terms of a managed access deal based on ‘financial and clinical details’.
Translarna remains the only treatment to be approved for marketing – after a series of setbacks for other late-stage clinical candidates – that modifies the underlying cause of DMD and it is estimated that around 50-60 children could receive the drug over five years under NICE’s access plan.
The drug has a list price of £220,000 ($290,000) a year, and it is understood the access deal includes an agreed discount as well as a commitment by PTC to generate real-world evidence on the drug’s benefits over the five-year period. Thereafter a new finding decision will be made.
“When we published our draft recommendations in April we acknowledged that ataluren represents a significant cost to the NHS at a time of increased pressure on funding, especially given the uncertainties of the drug’s potential long-term benefits,” commented NICE chief executive Andrew Dillon on the decision.
“We are therefore extremely pleased NHS England and the company have agreed the terms of a five-year managed access agreement for ataluren.”
DMD is a muscle-wasting disease that usually affects boys in early childhood and is often fatal by age 30.
The Muscular Dystrophy UK charity described the decision as ‘history in the making’, and welcomed the news that as part of the agreement, NHS England has waived the usual three-month funding period, meaning the drug could be available within weeks.
Northern Ireland has already confirmed it will also make the drug available and Wales is expected to follow suit shortly. Elsewhere in the UK, the Scottish Medicines Consortium (SMC) turned down Translarna for NHS funding in April and the Isle of Man has also decided to deny treatment with the drug.
“While NICE’s announcement is welcome, it is simply unacceptable that Scotland is forced to continue fighting, with a cruel postcode lottery for families to endure,” said the charity’s chief executive Robert Meadowcroft.
“We will not rest until all eligible boys can access this drug,” he added.