Access to medicines for people with cystic fibrosis

Medical science continues to show astounding progress and hold the potential to change the lives of people in the UK. The human genome project, a major global achievement to which the UK contributed a huge role, is now starting to show the first signs of promise in treatments. The challenge for government and the NHS is how we can make these innovations available in a fair and equitable way.

A lot of the focus of public debate has been thrown on cancer medicines in recent years, with even a special fund set aside to make new medicines available. But today in parliament I am hosting a Westminster Hall debate looking at an often overlooked condition that is in the midst of a potentially game changing medical revolution thanks to genetic medicine.

Cystic fibrosis (CF) care has long been limited to managing symptoms and decline. Now, After 25 years of research, we have a pipeline of precision medicines that target particular mutations of CF and seek to correct the basic underlying genetic defect.

Patients using these medicines have shown increased lung function, slower progression of lung disease and reduced number of hospital admissions – making a huge impact on their lives. The promise is that other medicines in development could make these benefits available to almost all people with CF.

One of my constituents, Carly Jeavons, was among the first to benefit having been enrolled on clinical trials. Three years ago this 28-year-old part-time worker and full-time mum from Dudley, was at a crossroads. She didn’t know whether to leave work and face financial turmoil or continue but risk her physical and mental health. Struggling to breathe with a lung function of around 44%, Carly was taking around 90 tablets and undertaking two hours of physiotherapy a day, spending two weeks in hospital every three months.

Carly’s health was dramatically declining. She started to worry about looking after her four-year-old son, Corey, and the future. Since being on the treatment plan, she has noticed significant improvement and even had three holidays with her family – something that would have been unthinkable before.

One of the cruellest aspects of CF is that those with the condition can never meet in person as the risks of passing respiratory infections is too high. For parliamentarians it means a delegation of people with CF is simply impossible and too many parliamentarians are not aware of the condition. Today’s debate is among the first of its kind, involving an online “live tweet” discussion beforehand to share experiences and opinions between the CF community.

The Cystic Fibrosis Trust, who are helping support and promote this debate to their members, are hopeful that the detailed registry of clinical data they hold about the condition means the government will see the CF treatments as the opportunity to test out new ways of assessing medicines being discussed as part of Science Minister George Freeman’s Accelerated Access Review. For the sake of people like Carly, I hope this debate adds weight to those considerations.