On 8th December in Westminster Hall, Rachael Maskell MP for York Central spoke in the debate brought by Labour MP for Dudley North Ian Austin, that the House has considered access to medicines for people with cystic fibrosis and other rare diseases. Here is how Hansard reported Racahel's contribution:
It is a pleasure to serve under your chairmanship, Sir Edward.
I thank my hon. Friend Ian Austin for proposing today’s debate on cystic fibrosis and on the future of the drug therapy. I thank the cystic fibrosis team at York hospital. I have met with them and discussed at length their innovative service, which is at the cutting edge of provision for those with cystic fibrosis and takes on board the need for clinical excellence and the sterile conditions that we have heard about—they work the service around the patient, not the patients around the service. I also thank the people at the Cystic Fibrosis Trust for their time.
I emphasise the points made by Mrs Gillan. Her tireless campaigning was triggered by the inspiration of Archie Hill from her constituency and presses for the need to make progress on the right therapeutic responses for those with Duchenne muscular dystrophy. We would all like to see progress with Translarna.
I want to take a wider view of the therapeutic measures for those who experience cystic fibrosis. I am a physiotherapist by training and have worked for 20 years in the NHS with respiratory and neurological conditions, so I have a real understanding of the people who experience cystic fibrosis. There has been massive change in the management of that condition in my time in practice, in particular in physical therapy. Treatment is now more dynamic in support of individuals—physical treatment, rather than a more static treatment, especially when dealing with mucus clearance and building up lung capacity. That is all about the treatment and management of symptoms, however, similar to the drug regime that accompanies the physical therapy.
We have seen progress, therefore, but today we are debating a step change in our approach to cystic fibrosis. We are trying to provide hope to the 10,000 people who happen to have cystic fibrosis. Looking at a new generation of drugs might provide that hope. Orkambi is a drug that targets abnormal proteins, which will deal with the symptoms. When we look at drug therapy for cystic fibrosis, we should be looking not only at the immediate impact, which so many drugs do, but at the long-term effect. Every instance of a chest infection brings about damage to the lungs, as people have to expectorate continually, and that has long-term implications that can be fatal for some.
It is vital that we look at early intervention, which is what Orkambi is all about—about bringing a step change in the treatment process for those with cystic fibrosis. By targeting the proteins we have the opportunity to ensure that the cells in the lungs are healthy, which will produce longevity among patients. It is hoped that the new drug will bring improvement to about 50% of people with cystic fibrosis, which in itself will be a seismic change in the outcomes for them. It will have a profound impact.
I encourage the Government not to be nervous about cost, because costs for someone with cystic fibrosis are already high and cannot be underestimated. I will focus on existing costs, such as the cost of frequent visits to hospital, including the frequent use of intravenous drugs. A large proportion of people are on IV drugs for approximately one month a year, which is costly. People also have to be in sterile conditions, because the risk of further infection is incredibly high. Ongoing therapeutic intervention with drugs or physiotherapy has significant bearing on costs. There are also costs to do with managing a high-calorie but healthy diet.
Another expense is the drugs. Cystic fibrosis is not on the list of diseases for which people get free medication. Will the Minister look at that? When the list was drawn up, people with cystic fibrosis were not living into adulthood, so we should re-examine it. There are the costs of having lung transplants, if people require one, and any drugs that prevent future lung transplants have to be a positive, despite the risks, because people will be brought long-term hope.
There is the cost to an individual of education, which for many will have a disturbed pattern—in and out of school—and the impact on long-term employment opportunities. Even if in work, many people find it difficult to hold down a job, because the nature of the disease often takes them out of the workplace and they have to organise and balance their day with fitting in physio and the demands of drug therapy and diet.
Finally, there is the cost of care. Rarely is only one person involved in care for any of the diseases that we are talking about—a network of care is put around an individual with such a disease. Moving to a precision, early-intervention drug, therefore, is a way to bring in resource management, which can be positive not only for the individual, but for the NHS as a whole.
The result of what is being called for today would be positive economically and for people’s lives. In my short contribution, I want to ask the Minister to address the timeline for progress. There is obviously discussion in Europe, such as on the European regulations for Orkambi, and we want to see the timeline tightened up, so that people can have real hope in the new year that they will get access to the drug, because each time someone has a chest infection it has an impact on their long-term future. Time is not something that so many have, so my plea is for progress on securing access to the drug for those with cystic fibrosis.
To read the whole debate, please visit: Westminster Hall - Cystic Fibrosis Debate