Cystic Fibrosis Drugs: Orkambi

Catherine McKinnell Excerpts

Monday 10th June 2019 Westminster Hall

[Paul Scully]

My hon. Friend is absolutely right. Before I led a similar debate for the Petitions Committee roughly this time last year, I went to the Cystic Fibrosis Trust, as I did this morning, where I met and spoke to a lot of parents whose children are suffering, as he has mentioned. This morning, I met a number of adult sufferers, who I will speak further about in a minute. One of them, who is 43, described the disease—this is harsh, especially for any sufferers watching—as his lungs filling up and effectively drowning. That is pertinent to me, because my father died of mesothelioma 30 years ago, after a year of suffering. This man is 43; I cannot imagine that suffering. Early treatment for children, however, stops that build-up in the first place and allows them to keep their lung capacity higher for longer, so they can have a proper standard of living.

[Catherine McKinnell (Newcastle upon Tyne North) (Lab)]

I suspect that my fellow Petitions Committee member shares my concern that families are being put in the terrible position of having not only to deal with a devastating illness and diagnosis, but fight for a treatment that they know is available and that other people across Europe and across the world are receiving. Whether it is cystic fibrosis, Batten disease or phenylketonuria, or PKU, it is wrong that families are wasting their precious energy, which they would like to channel into looking after their children, into fighting for a treatment that could solve many of those medical issues.

[Paul Scully]

I totally agree with the hon. Lady. It is frustrating to look at access around the world; there is even an interim solution in Scotland. It would be interesting to hear from the Minister about how that may pan out. Scotland has given interim access, including for compassionate use, and further access to some sufferers who can use Orkambi with a view to reporting back in August.

It is disappointing to find that people living with CF, in their ingenuity, have had to resort to looking at a buyers’ club. The Vertex drug is patent pending in Argentina, so another company is making a copy that can be sourced for £18,000 a patient—still a lot of money—rather than £104,000. That £18,000 comes out of their pockets, however, which defeats the idea of the NHS being free at the point of need.