This morning, the Government announced a new, fast-track route into the NHS for “breakthrough” medicines and technologies. Muscular dystrophy UK welcomes this news, which is an early campaign win in our fight to FastTrack treatments for muscle wasting conditions.
What the Government has said
The new scheme is called the Accelerated Access Pathway and will come into effect from April 2018. It is intended to speed up the time it takes for patients to benefit from ground-breaking products for conditions. Each year, the scheme would see technologies and treatments deemed to have the greatest potential to change lives designated as “breakthrough.” This would unlock a comprehensive package of support that will allow firms to accelerate clinical development and benefit from a fast-track route through the NHS’s approval processes.
Health Minister, Lord O’Shaughnessy, said:
I want the UK to be the best place in the world to develop new drugs and medical technology – but despite the innovation happening here, our uptake in the NHS can be too slow.
Today’s new measures will not only benefit patients by improving how quickly and easily we can get innovative products from the lab to the bedside, but will guarantee future collaboration between the life sciences sector and the NHS post-Brexit – benefiting the British economy and creating jobs.
In addition to the Accelerated Access Pathway, the Government is also providing an £86 million funding package to help innovators of all sizes to access the NHS market, and help ensure that these products get to the patients that need them. This includes £6 million to support clinicians to use new treatments and technologies in everyday practice.
FastTrack campaign win
The news of the Accelerated Access Pathway is a positive campaign win for our FastTrack to treatments campaign which launched last month. We know that for people with muscle-wasting conditions, every day is vital. Anything that helps speed us access to emerging treatments is welcome. We would like to thank everyone who has supported the campaign so far and we hope to see more progress in the future.
Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, said:
This news could be of tremendous significance to people with progressive muscle-wasting conditions. In a year waiting for a treatment, the ability to walk could be lost, or respiratory muscles irreversibly damaged. We hope the new scheme will go some way to ending bureaucratic delays and speeding up access to new drugs.
What we do need is urgent clarification on what a ‘breakthrough’ treatment is and how these will be evaluated by NICE. There are currently no cures for muscle-wasting conditions, but emerging therapies are buying time and slowing their devastating and often life-shortening effects. The scheme’s criteria and capacity needs to reflect this. The opportunity to accelerate progress in treating rare diseases must not be lost.
You can read more about Muscular Dystrophy UK’s position in this briefing document. We know there is still more to be done to improve access to treatments but the new Pathway is a welcome and positive step.
What does this announcement mean for families?
This is an encouraging time with many clinical trials in development and several treatments either available or emerging on the horizon. The Accelerated Access Review could mean that new treatments become available up to four years earlier by reducing the time taken to negotiate the evaluation and financial approvals necessary before the NHS can purchase them.
Claire Liggett, whose son, Charlie, has a muscle wasting condition, started a petition last month in support of our FastTrack to treatments campaign. The petition has had over 16,000 signatures. The family are awaiting a review of an emerging therapy for the condition, Exondys 51, which could help Charlie. They fear delays to and NHS funding, which have affected other drugs for Duchenne. Claire said:
There are no cures for Duchenne. Charlie will need to use a wheelchair within a few years, and his life expectancy is in his early thirties. His muscles are weakening and every day that passes before he is treated takes him closer to losing an ability. We are against the clock. Exondys 51 is not a cure. We know that. For some people, the opportunity to slow things down, even just a little, might not make a drug sound ‘breakthrough’. To us and to Charlie though, that would be enormous. It could buy time for other things too. We need to know that treatments that hold potential like Exondys 51 will get a fair hearing.
Make your voice heard
- You can still support Claire and Charlie’s petition on Change.org- every signature counts.
- For more information on FastTrack, and ways in which you can support the campaign, please contact Clare Lucas, Campaigns and Engagement Manager, on 020 7803 4838 or email firstname.lastname@example.org