Cystic fibrosis and your treatment – what matters to you?

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New precision medicine tezacaftor-ivacaftor has completed clinical trials, but before any decisions on its use in the NHS are made, it must be granted a license for sale by the European Medicines Agency, which is due to be rubberstamped this year. We are launching a new survey to help us represent you as the case for using the drug in the NHS is evaluated by health watchdogs. Nick Medhurst, Head of Policy at the Cystic Fibrosis Trust, explains how the information you share will help us to fight for you.

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With a whirlwind of activity around new CF drugs, it can be a struggle to keep up with what is happening when and why it’s important.

As discussions progress between the NHS and Vertex, the pharmaceutical company’s most recent product to complete clinical trials, the tezacaftor-ivacaftor combination therapy (known as Symdeko in North America), is due to be analysed for use in the UK.

The world of drug evidence reviews is very confusing. There are different processes in all four nations of the UK, different advisory bodies, and different politicians and decision-makers. Our job is to make sure all these groups and individuals understand how CF affects your life, what matters to you, and what can be done about it.

To do that, we need to listen to you and we hope you will use our new short survey to make your voice heard.

What is the review process, and how does it work?

Organisations such as the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and All Wales Medicines Strategy Group (AWMSG), all perform an important role in advising our NHS through evidence reviews for drugs. This process is often called a Health Technology Appraisal (HTA).

A HTA will usually analyse clinical trial data presented by a drug company, combined with evidence submissions from patient groups and clinical experts. The Cystic Fibrosis Trust is one of these groups.

How have your views helped in the past?

Back in October 2015, we asked you to complete a short survey to tell us what mattered to you when taking your CF treatments. Your response to our survey was phenomenal; nearly 1,500 people shared their experiences, covering everything from how you feel about doing your daily nebs and physio to how you know you’ve got a chest flare up coming. You also told us what you want your treatments to do for you and why you do them.

Information like this helps these organisations, who have been given the job of defining the clinical value of new drugs, to see beyond the data to understand the impact a new drug might have on a person’s life. The results of the survey in 2015 have helped us to argue how important people with CF feel it is to stay out of hospital and gain some stability in their lives. They have also helped us to describe what life is like with the complications of CF and what a drug that could delay or avoid these complications would mean.

It is the job of these organisations to weigh a drug’s clinical value against the company’s asking price and make a recommendation as to whether it represents value for money for the NHS, whether it doesn’t, or whether more information is needed. By sharing your experiences with us again this year, you can help us to continue to fight for these organisations to truly recognise what you value most in the treatments you take.

These reviews could and should be the foundation of positive negotiations that deliver value for money and access to new drugs.

Help us to represent you

Please do take the time to make sure that the evidence we provide represents you by completing our survey.

It’s been an incredible year, but not a successful one – yet. We know that only one success really counts: people with cystic fibrosis with their hands on the most advanced treatments available to them.

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