It is the day after the latest Westminster debate (watch here) on access to Orkambi and other life-saving drugs, and I thought it would be useful to share some reflections on what has at times felt like a rollercoaster ride over the last two weeks.
A good place to start is on Thursday 30 May, when Vertex Pharmaceuticals released the latest results from their clinical trials of ‘triple therapies’, one of which they are submitting for FDA approval. These drugs have the potential to improve the lives of up to 90% of people with cystic fibrosis across the world and the results provided compelling evidence of the impact they could have.
At what should be a moment of excitement, however, we still face the reality that there is not a permanent deal in place in any part of the UK that ensures universal and rapid access to the therapies that already exist.
There is an interim agreement in place in Scotland, which allows some access through a system called PACS Tier 2, and we expect a decision from the Scottish Medicines Consortium in August on whether Orkambi and Symkevi will be available for medical teams to prescribe beyond this.
There are signs of progress in Wales. Today, the Petitions Committee discussed access to cystic fibrosis medicines and confirmed Vertex intend to submit evidence to the All Wales Medicine Strategy Group (AWMSG) in the coming weeks. The Committee have pledged to write to Vertex to ensure this happens as soon as possible. You can read our latest correspondence with the Petitions Committee here.
What we do know is that over the last few weeks and months NHS England and Vertex have been meeting on a regular basis. As recently as last Friday, they both described these talks as “ongoing and intensive” but the sliver of hope that this offers is clearly not enough after this desperate, enforced and prolonged wait.
It is three and a half years since Orkambi was approved for use, and the lack of a deal has led to an appalling situation where, worn down by the delay, some people within the CF community have decided that waiting is no longer an option and are putting in place arrangements to personally pay thousands of pounds out of their own pocket to get generic drugs from overseas.
Everyone knows the quickest, most reliable and most equitable way of getting life-saving drugs to the thousands that need them remains comprehensive deals between the NHS in all four nations of the UK and the company that manufacturers them.
We don’t want any more empty and repetitive commentary from the negotiating parties, we just need action and access. That means a deal.
Which brings me to last night and the debate in Westminster Hall. Firstly, I should say that the very fact that the event took place was a tribute to the drive and commitment of the CF community.
On a day when there were plenty of political distractions, it was important to see a strong turnout and a wide range of contributions that crossed party political lines and the borders of the UK’s nations. A central theme of the debate was captured in the comment that this is not about names on spreadsheet, but real people simply wanting to live their lives.
The Government Minister present, Seema Kennedy, Parliamentary Under-Secretary of the State for Health and Social Care, in her response emphasized that Vertex must re-engage with the NICE process. She was also clear that a deal was the best option, but if it was not achieved, she had a "moral obligation" to look into other options that could deliver access. The Minister also agreed to meet with a cross-party group of MPs and Trust representatives as soon as possible.
The other element of the rollercoaster ride I mentioned above was the European Cystic Fibrosis Society’s Annual Conference that took place in Liverpool last week. It was an amazing event, with nearly 3,000 delegates from around the world coming to share and discuss the latest clinical and scientific breakthroughs in cystic fibrosis.
It is 30 years since the CFTR gene was discovered and the conference showed how far things have come and held out the promise of further significant scientific advances, clinical approaches and new treatments in the years ahead.
That progress should be celebrated, but we know it is not enough. From funding research, promoting the best care, providing information and support and campaigning for everyone with cystic fibrosis, we will continue the fight until we have truly delivered a life unlimited for all.
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