Two Dudley campaigners for better cystic fibrosis treatments have prompted a House of Commons debate on access to new treatments for rare diseases.
Dudley MP Ian Austin has organised the debate to push for changes to the way that the NHS assesses new drugs that promise improved quality and length of life for people suffering from rare diseases.
Cystic fibrosis is a life-shortening inherited disease affecting over 10,000 people in Britain. It causes the lungs and digestive system to become clogged with mucus, making it hard to breathe and digest food. The damage cystic fibrosis causes to the lungs means many people with condition come to rely on a lung transplant to stay alive. There is no cure for the condition and although it can be managed with physiotherapy, exercise, medication and nutrition the average survival age is around 41.
The debate was inspired by two of Austin’s constituents, Samantha Carrier and Carly Jeavons.
Samantha Carrier is campaigning to raise awareness and raise funding after her young daughter Daisy was diagnosed with cystic fibrosis. Samantha spends hours every day giving her daughter extra care and support making work impossible, and she is campaigning for access to new drugs and treatments so daughter can live as full a life as possible.
Carly Jeavons suffers from cystic fibrosis but has benefited from the clinical trial of a new treatment called Orkambi. Three years ago, Carly was faced with a choice between leaving work and facing financial difficulties or staying in work to the detriment of her health. Despite taking around 90 tablets and undertaking two hours of physiotherapy a day she had a lung function of around 44 per cent and spent two weeks in hospital every three months.
In September 2014, Carly was offered an opportunity to participate in a clinical trial for a new type of treatment that targets the genetic mutations that cause cystic fibrosis called Orkambi. This treatment has enabled Carly to halve the number of hospital visits she makes and go on holiday with her family for the first time.
In the debate Austin has joined other MPs to call on the Government to listen to the recommendations of the NHS Accelerated Access Review that would change the way that new drugs like Orkambi are assessed. Under the current system new treatments are assessed based on clinical trials lasting just 24 weeks, but organisations like the Cystic Fibrosis Trust are arguing that these short trials do not take account of the long-term benefits of new treatments.
Ian Austin said:
“This is exactly how Parliament should work. It’s my job to listen to my constituents and stand up in Parliament to push for changes to help them. People with cystic fibrosis like Carly Jeavons and Samantha’s daughter Daisy struggle to do things that the rest of us take for granted because they have to undergo hours of treatment every day.
“New treatments offer them the opportunity to live their lives as they wish, but thousands of people are set to miss out on these new drugs under the current rules.
“I want the Government to listen to the recommendations of its own review and change the NHS rules on commissioning new treatments so they take account of the longer and more productive lives that these drugs offer people suffering from rare diseases.”