Cystic Fibrosis Drugs: Orkambi

Emma Hardy Excerpts

Monday 10th June 2019 Westminster Hall

[Paul Scully]

One of the reasons why NICE was set up in the first place was to take politics out of drug development. We need to ensure that we have the balance right between the Government intervening and the clinicians—the people who can make their assessments without political interference—making their decision. None the less, we clearly must have a view.

One of the things that some people have suggested—I think it was intimated in an earlier intervention—is Crown use of patents, to allow the use of generic drugs and effectively remove patents from pharmaceutical companies. Obviously, that is in extremis. There is an inherent problem with the potential lack of investment in future research, should we start taking away patents from private companies.

[Emma Hardy (Kingston upon Hull West and Hessle) (Lab)]

The hon. Gentleman is making a very powerful speech, and I stand here today representing my constituents. Does he agree that when factoring in costs, NICE should also factor in the ongoing costs that not treating cystic fibrosis will bring? It needs to factor in the cost of the additional mental health treatment and other health treatments that will be needed, and possibly even the cost of lung transplants. This is not a simple comparison with the cost of treatment; other things need to be taken into account.

[Paul Scully]

The hon. Lady has hit on a really crucial point. The underlying thing that I took away from my meeting with the people living with CF this morning was mental health, which ran through all their situations.

I heard from Oli Rayner, who talked about the fact that he is 43 and has dedicated his whole life to staying alive; he had effectively been told that he would not make 10 years. He was then told that he would not make 20, then 30. This is a guy who has now got cystic fibrosis-related diabetes and a number of other conditions. He has had a lung transplant, and he had Orkambi to get him to that stage. The fact is that his lungs are now doing what he wants them to do, without his having to think about it. We can imagine the mental issues that he had before.

Jessica Jones told me that people with CF are very good at living. Yvonne Hughes said she felt broken. One lady, Carly Beale, told me that she had been on the original Orkambi trial. The NHS had not prepared her for when Orkambi stopped at the end of the trial. She had suddenly improved and started to get her life back—perhaps a life that she had not had in the first place—and she was not ready to have it taken away from her. She said that it is almost worse that this drug exists but she cannot get access to it. She said, “I’d rather it didn’t exist than have it dangled in front of me in expectation.”