Muscular Dystrophy UK is campaigning for faster approval and funding for therapies for children and adults with muscle-wasting treatments, and warns that the wait for a government response to the Accelerated Access Review (AAR) “adds to delays to new drugs”.
The charity is urging the government to “address inefficiencies” surrounding the approval and NHS funding of pioneering new genetic therapies, given the delays patients have faced thus far.
It highlights several cases where patients in the UK had to wait to access new innovations, such as with PTC Therapeutics’ Translarna - the first drug available in the UK to tackle the underlying cause of Duchenne muscular dystrophy - which took three years to be made available to patients after conditional approval by the European Medicines Agency, yet was available “within months” in some other European countries.
Despite government promises to address obstacles identified within AAR, a commitment which the charity notes was reiterated in the Conservatives’ general election manifesto, a formal response to the report - published in October 2016 - is still being awaited.
“A full year after publication, the Accelerated Access Review could now be dubbed the Delayed Access Review, such is the lack of urgency in the Government’s response. Yet the need to act upon this report cannot be overstated,” said Robert Meadowcroft, Muscular Dystrophy UK’s chief executive.
“Our drug approval system is simply not ready for the substantial increase in the number of groundbreaking therapies for genetic conditions that need assessing. The government has been given a blueprint for change and now needs to put it into action.”
The charity has now launched its Fast Track campaign which it hopes will address obstacles to accessing innovative treatment for children and adults with muscle-wasting conditions.
Several calls under the campaign coincide with the AAR, including a new commercial unit at NHS England to handle negotiations with drug companies, a more flexible range of pricing models for treatments, and greater support for the government’s early access to medicines scheme (EAMS).
It also calls for NICE, NHS England, the Scottish Medicines Consortium and other devolved nations to adopt flexible approvals processes to ensure further data can be gathered on drugs impact post approval, and for the Department of Health to increase capacity at the NICE Highly Specialised Technologies Programme (HST).