Up to 5,000 children and young people in England with the life-shortening disease cystic fibrosis will soon get access to the new drug Orkambi, after the US manufacturer ended years of bitter wrangling and agreed a price with the NHS.
Campaigners celebrated the news, which NHS England’s chief executive said meant cystic fibrosis patients would get the drug within the month. “Given the time it has taken for the company to reach this agreement, there must be no further delay,” wrote Sir Simon Stevens to the chair of the House of Commons health select committee, Sarah Wollaston. The committee held hearings on the impasse earlier this year.
The families of children and young adults with the disease, which gradually clogs the lungs, have fought a heart-rending campaign for the first drugs that target the cause of the disease rather than just improving symptoms. The drugs were approved four years ago but Vertex set a price tag of more than £100,000 per patient per year, which NHS England refused, saying it was unaffordable.
Both sides said details of the deal and how much NHS England would pay Vertex were commercially confidential. But Stevens’ letter made it clear that NHS England was happy with the terms. “We have only been able to finalise this negotiation because the company has now agreed confidential commercial terms that constitute good value for British taxpayers,” he said in his letter.
The last offer NHS England made to the company was £500m over five years for access to Orkambi and other drugs in the pipeline.
Crucially, Vertex has agreed to submit its forthcoming drugs, believed to be more promising than Orkambi, to the National Institute for Health and Care Excellence (Nice), which assesses the cost-effectiveness of drugs and can recommend or bar them from NHS use. Vertex had withdrawn its future drugs from the process during the long standoff.
Nice will now assess all the drugs, using data not only from the trials but also from the patients who will now get them. That will enable experts to decide how well the drugs work and what they are worth.
Stevens said in his letter that the deal would apply to Wales and Northern Ireland as well. However, it will not apply to Scotland, which recently made its own agreement with Vertex. Stevens implied NHS England had done better.
“Unfortunately, we have not been able to extend this option for Scottish patients. That is because six weeks ago a decision was taken there to step away from coordinated UK-wide approach by ignoring the independent expert medical advice of the Scottish Medicines Consortium – their equivalent of Nice. We are, however, legally able to share with the Scottish government confidential details of the improved deal we have now negotiated for England,” he wrote.
The health secretary, Matt Hancock, said it was wonderful news. “We’ve agreed a deal to provide Orkambi and other lifesaving cystic fibrosis drugs on the NHS. The deal is great value for money for the NHS and, crucially, will improve thousands of lives. This deal – on the back of several others this summer – shows why we get some of the best-value drugs in the world, and is another reason to be so proud of our NHS,” he said.
Ludovic Fenaux, a senior vice-president at Vertex International, said it was “a significant day for the cystic fibrosis community in England. This important agreement, reached in collaboration and partnership with NHS England and Nice, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease.”
