The last few days have seen some dramatic breaking news for the cystic fibrosis community, with results from the gene therapy Phase 2b clinical trial being published, NHS England announcing funding for ivacaftor for eight rare mutations, and the FDA in the US licensing the combination treatment Orkambi. In this blog, Trust Chief Executive Ed Owen looks back on a momentous couple of days, and how the power of the CF community shines through.
The last 48 hours has proved a significant step forward in our fight for a life unlimited by cystic fibrosis – and one that reinforces the collective power we have as a community, both national and global.
On Friday morning the results of the gene therapy Phase 2b clinical trial were published in the Lancet Respiratory journal. While not a surprise to those who took part in our sneak preview a few weeks ago, the outcome is certainly encouraging.
More clinical trials are needed before we can say gene therapy can be a viable treatment for people with cystic fibrosis, but it successfully establishes proof of concept that demonstrates genuine promise.
Future trials are now needed and that will almost certainly require the sort of resources and expertise that only a pharmaceutical company can provide. We also have to be realistic and accept that this will take some time.
But it is good news and one that has not come this far by accident.
First and foremost, it is a tremendous tribute to Professor Eric Alton and the wider Gene Therapy Consortium team for their ground-breaking work. Yet, as they would be the first to say, this could not have happened without the participation of people with cystic fibrosis, young and old, who have volunteered to take part in this and earlier clinical trials.
And, vitally, it has only come about because of the extraordinary support of the wider cystic fibrosis community raising many millions of pounds over the last 15 years.
This is continuing and the Trust has pledged a further £500,000 to support the GTC’s Wave 2 gene therapy product which – while about five years behind the product that is the focus of this week’s news – has potentially much greater impact.
The Trust will continue to work tirelessly to ensure everyone with cystic fibrosis gets access to life-changing therapies however long it takes. As with gene therapy and other promising scientific development, that means investing funds raised in research. But it also means lobbying and campaigning to ensure the NHS funds therapies approved by regulators.
So we were delighted with the news on Thursday that NHS England has agreed to extend access to Kalydeco (ivacaftor) to about 40 people who have non-G551D gating mutations of cystic fibrosis.
It has taken far too long, and demonstrates the inadequate system in place to ensure such transformational drugs are appraised. What’s more, a handful of people in Wales are still waiting – and we will continue to fight for them until the right decision is made.
But the voice of people with cystic fibrosis supported by clinical teams and backed by clear evidence of Kalydeco’s transformational impact rightly won the day.
There are many more issues like this to come over the next years and a further big potential campaigning issue on the horizon is Orkambi – the so-called combination therapy eligible for those with two copies of the F508delta gene, the most common mutation of cystic fibrosis.
Orkambi has yet to be approved by EU regulators but it was good to hear this week that the US regulators, the FDA, have given the green light for its use in America.
Its effect is less dramatic than that seen with Kalydeco on those with gating mutations. But the evidence from Phase 3 trials shows that it significantly reduces hospitalisations for those who take the drug and stabilises lung function.
We are expecting a decision from European regulators by the end of this year and, if approved, the key issue will be whether it is reimbursed by the NHS. It is by no means certain that it will, and we will be pushing Vertex and the NHS in the four parts of the UK to do the right thing to enable Orkambi to get to those who need it as soon as possible at an affordable price.
This week has shown that when we come together as one we can change the world for many with cystic fibrosis. The Power of Us will continue to be our key weapon to secure further changes vital to our mission to transform the lives of all.