Accelerated Access Review

Kerry McCarthy Excerpts

Tuesday 13th December 2016 Westminster Hall

[Kerry McCarthy (Bristol East) (Lab)]

It is a pleasure, as ever, to see you in the Chair, Sir Alan. I thank the Backbench Business Committee for granting us this debate. I also thank the Cystic Fibrosis Trust, which has not just been instrumental in getting MPs here today, but does great work, as my hon. Friend the Member for Dudley North (Ian Austin) said, in supporting people with cystic fibrosis and their families. I think that Ed Owen is now moving on to other things and I particularly pay tribute to him for his work.

As I have mentioned before in this place, I have a 12-year-old niece, Maisie, who was diagnosed at Christmas in 2004 as one of the 10,500 people in the UK with cystic fibrosis. Thankfully, she is doing very well. In recent weeks, the number of constituents who have contacted me whose children, grandchildren or friends’ children have cystic fibrosis has been revealing to me. Sadly, I also have constituents whose children have died from the condition. The youngest cystic fibrosis patient I have been contacted about was born in April this year. I have also been contacted by a couple of people who have 19-year-olds who are both at university, and the issue of prescription charges mentioned by my hon. Friend the Member for Dudley North has particularly hit home. One constituent said that, although she was very proud that her son had now gone to university hundreds of miles away, he was now responsible for managing his condition, which created some anxieties for her. She said that he was very angry that, despite being classed as a disabled student, he had to pay for his prescription. I hope that the Minister takes that on board because we have been promised a review of exemptions for many years.

As we have heard, Kalydeco, the drug that treats one particular mutation of cystic fibrosis, helps about 4% of patients in the UK. It was approved back in 2013 and the results have been dramatic, increasing lung function and reducing the time people have to spend in hospital on intravenous antibiotics. It has made a huge difference to patients’ quality of life and all the signs indicate that it could significantly improve life expectancy. Now we have Orkambi, which is a combination of the drug that is marketed as Kalydeco and another drug. Orkambi targets a different mutation that affects more than 4,000 people, so it could help almost half the CF population in the country.

The clinical studies that became available last year indicated a significant reduction of 30% to 39% in lung infections and inflammations that lead to irreversible lung scarring and the need for a lung transplant. One in three patients who need a double lung transplant because of cystic fibrosis die while they are still on the waiting list. I hope that organ donation is also on the Minister’s radar. Orkambi could be absolutely life-transforming. Despite that, NICE rejected it back in June because of doubts about its cost-effectiveness. It recognised that the drug is potentially a “valuable new therapy” with “significant clinical benefits”, but it concluded that the cost per “quality-adjusted life year” is too high.

I acknowledge that NICE has a difficult job in assessing all the potential treatments for a range of conditions, and that it operates on a tight budget, but, as the Cystic Fibrosis Trust has argued and as we have heard this morning, the processes that NICE uses can be flawed because they rely on short-term data for a lifelong condition. We heard from my right hon. Friend the Member for Leigh (Andy Burnham) that new data show that after 96 weeks of treatment—NICE only looked at the 24-week clinical trials—the decline in lung function, which is the main cause of death for people with cystic fibrosis, slows by 42%. If NICE had had that data, it would have rated Orkambi’s cost-effectiveness much higher.

I am partly speaking from personal experience, but I also know from my constituents’ very sad stories that people are desperate for something to address these problems. The accelerated access review was commissioned because the Government recognised the weaknesses in the NICE process. For patients whose life depends on the outcome of the report and its implementation, it has been a long wait. I urge the Minister not to delay any longer and to consider the Cystic Fibrosis Trust’s request to apply the AAR recommendations to the deadlocked Orkambi negotiations. Confidential commercial agreements would free Vertex to make the NHS its best offer, and commercial agreements under the Cancer Drugs Fund are confidential. Flexible pricing arrangements would allow the consideration of alternative models to manage costs, such as a price cap that is not exceeded even if patient numbers rise, and the collection of real-world data would allow a more accurate assessment of the drug’s effectiveness. The hon. Member for Bury St Edmunds (Jo Churchill), who has now left the Chamber, mentioned the fact that 99% of cystic fibrosis patients are on the database and that there is a lot of available information about them, which makes them ideal to pilot the scheme. I hope that the Minister considers that.