Orkambi

Rachael Maskell Excerpts

Monday 4th February 2019 Commons Chamber

[Bill Wiggin]

I am sure that the hon. Gentleman’s constituents will be grateful to him for raising the issue in the House this evening.

Every week, five babies are born with the disease, according to Great Ormond Street Hospital, and every week two young people die as a result of cystic fibrosis. The disease accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. There are two main ways to treat cystic fibrosis: conventional treatments target the symptoms, and precision medicines such as Orkambi tackle the cause of the condition. For conventional treatment on the NHS, the average waiting time to be admitted to hospital is 45 days.

Orkambi presents a relatively safer, more effective and clinically meaningful alternative. In treating the root causes, it reduces lung damage and cystic fibrosis-related diabetes, and improves pancreatic function. The drug has been approved by the European Medical Association, and the Food and Drug Administration in the United States. It avoids the high risk associated with organ transplants.

Orkambi treats the F508del mutation, which around 50% of people with CF in the UK carry. Essentially, the drug permits more chloride ions to pass into and out of the cells. This helps to keep a balance of salt and water in affected organs. Ivacaftor is one of the active substances in Orkambi. It increases the activity of the defective cystic fibrosis transmembrane conductance regulator protein, thereby making the mucus less thick. Decline in lung function is the most common cause of death for people with cystic fibrosis and, although not a cure, Orkambi has been found to slow the decline in lung function by 42% and reduce hospitalisations by 61%.

[Rachael Maskell (York Central) (Lab/Co-op)]

Before coming to this place, I was a respiratory physio and worked with many people with cystic fibrosis. The cost of hospitalisation and treatment far outstrips the cost of this drug for many people with cystic fibrosis. Should not the National Institute for Health and Care Excellence change its criteria and look at the value of life, instead of only the day-to-day cost of this drug?

[Bill Wiggin]

No, I do not agree. The purpose of this debate is to a show an alternative that allows NICE to spend taxpayers’ money on drugs for other conditions while still allowing cystic fibrosis sufferers to have access to this vital drug—and not just to Orkambi, but to the next generation of the same sort of drugs. Bear with me because we have not got to the good bit yet.

In July 2016, NICE recognised Orkambi as an important treatment, yet was unable to recommend the drug for use within the NHS on grounds of cost-effectiveness. The drug is estimated to cost around £104,000 per patient per year and must be taken for life. Orkambi is not provided by the NHS, except in rare cases on compassionate grounds. It remains patent to its manufacturer, Vertex Pharmaceuticals, under UK patent law. In July 2018, NHS England made what it said was its best and final offer to Vertex of £500 million over five years. This was described by the NHS as the “largest ever financial commitment” in its 70-year history. Tragically, Vertex rejected the offer.

We all know that it is essential that a solution is found as soon as possible to make the drug available, as every day counts in slowing the progress of the disease. In an email to me, Vertex states that it

“is committed to finding a sustainable solution for access to our medicines for Cystic Fibrosis patients, including Orkambi”.

That is not quite the impression I have received so far. I sincerely hope that that is indeed its highest priority.

The drugs that constitute Orkambi—Ivacaftor and Lumacaftor—can be synthetically developed at low cost, yet their price remains inaccessibly high.