Muscular Dystrophy UK’s FastTrack campaign is focused on faster access to treatments.
For people with muscle-wasting conditions, every day is vital and we are committed to ensuring urgent action is taken so that people are able to access the support and treatments that they want and need. The FastTrack campaign would focus on FastTrack to treatments for people with muscle-wasting conditions.
This is an encouraging time with many clinical trials in development, and several treatments either available or emerging on the horizon. Translarna, a drug to treat Duchenne muscular dystrophy, is now approved across the UK. Exondys 51, another drug to treat Duchenne, and Spinraza, a treatment for spinal muscular atrophy, still waiting to be assessed in the UK.
Once treatments are available it is crucial that people are able to access them quickly. The NHS needs more powers and resources to negotiate good deals with pharmaceutical companies so that access to treatments becomes more streamlined. This can help to control the costs of new medicines and could also speed up patient access to treatments, for example via the Early Access to Medicines Scheme.
FastTrack calls to action
Backed by families, clinicians, researchers and politicians across the country, Muscular Dystrophy UK’s FastTrack campaign is calling for:
- The Department of Health to update on the implementation of the new Commercial Unit at NHS England without delay to ensure earlier negotiations on price can take place.
- 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.
- The Department of Health and NHS England to work with pharmaceutical companies to meet the additional NHS costs of going through early access and expanded access schemes.
- The Department of Health to increase capacity at the NICE Highly Specialised Technologies Programme (HST).
- Pharmaceutical companies to engage with regulators when they identify data to support proof of concept for exon-skipping drugs.
- The UK Government to ensure that UK drug licensing continues to linked to the European Medicines Agency (EMA) and that decisions are taken on a parallel timeframe.
- An urgent review of the conditions, processes and criteria for Individual Funding Requests (IFR) and Critically Clinically Urgent Funding Requests (CCUFR).
How you can get involved
- Support the petition by eight-year-old Charlie, who has muscular dystrophy. He’s asking for the Prime Minister’s help in removing barriers to all life-changing treatments that he and other people would benefit from.
- If you wanted to meet your MP, we can help and support with this. You could meet them in Westminster or in their constituency office.
- In Spring 2018, we will be holding some campaigning workshops across the UK.
Contact your parliamentarian
- If you live in England, you can support the campaign by writing to your MP using our template letter.
- If you live in Scotland, you can support the campaign by writing to your MSP using our template letter.
- If you live in Wales, you can support the campaign by writing to your AM using our template letter.
- If you live in Northern Ireland, you can support the campaign by writing to your MLA using our template letter.
It would be helpful if you could share a copy of your email to your parliamentarian with us by emailing Jonathan Kingsley at firstname.lastname@example.org so that we can also help to enlist their support.
Support for the campaign
Professor Dame Kay Davies, Dr Lee’s Professor of Anatomy at the University of Oxford:
Muscular Dystrophy UK continue to make significant contributions to muscle disease research. Through their Fast Track strategy they now plan to make a difference in the delivery of the fruits of this research to the clinic. This is a vital step in getting novel treatments for patients. I am delighted to hear that Muscular Dystrophy UK continues to be at forefront in this challenging area.
Professor George Dickson, Chair of Molecular Cell Biology at Royal Holloway:
We are in a promising time with new therapies finally coming to the market. It is vital that people with muscle-wasting conditions are able to access life-changing new treatments as quickly as possible, this is why I fully support Muscular Dystrophy UK’s new Fast Track campaign.
Dr Adnan Manzur, Consultant Paediatric Neurologist at Great Ormond Street Hospital:
“Muscular Dystrophy UK have been at the forefront of bringing novel treatments from clinical trials to the patient. Their work with NICE was instrumental in developing and implementing the Managed Access Agreement which allowed ataluren (Translarna) treatment to be made available to boys with nonsense mutation Duchenne muscular dystrophy. Further advocacy of early access to novel medicines and initiated is like the fast track campaign, are most important.”
Professor Francesco Muntoni, Professor in Paediatric Neurology and Director of the Dubowitz Neuromuscular Centre:
The new Fast Track campaign is very timely and welcome. There are currently very few treatment options for many muscle-wasting conditions. When new treatments become available it is crucial that they get to those who need them as quickly as possible. Without these drugs, people’s conditions can decline at least for one condition, SMA1, there are major implications for the survival of affected individuals if drugs are not started in a timely fashion. The focus needs to be on removing barriers and accelerating access to these new treatments, and identify appropriate mechanisms to deal with rare and very rapidly progressive conditions, an area which there does not appear to be a dedicated path in this country.
Mary Glindon MP, Chair of the All Party Parliamentary Group for Muscular Dystrophy:
“As the Chair of the All-Party Parliamentary Group for Muscular Dystrophy I strongly welcome the new Fast Track campaign from Muscular Dystrophy UK. Without the right assessment processes and funding in place we risk individuals and families enduring agonising delays before people with muscle-wasting conditions can benefit from cutting-edge therapies, which could drastically improve their quality of life.”
For more information on FastTrack, and ways in which you can support the campaign, please contact Clare Lucas at email@example.com or on 020 7803 4838.