Meeting of the Parliament 20 June 2018
It was only a week ago that we had a members’ business debate on access to Orkambi. The Minister for Public Health and Sport’s response then was disappointing and pre-scripted, and it offered little comfort to those for whom that drug could be life saving. I believe that the cabinet secretary has moved on from that today, which is welcome, although I would, of course, encourage her to go further.
The issue is not just access to Orkambi, important though that is, but access to the next generation of drugs to treat cystic fibrosis—the drugs that are being trialled as we speak, which will transform lives, and the drugs that will follow them in a few months, which will treat the underlying causes of cystic fibrosis rather than simply the symptoms. That really will be life changing. According to clinicians, instead of someone with cystic fibrosis dying before they reach 31 years of age, they could live into their 70s or 80s. They would have a normal life expectancy, which is simply extraordinary. We have an opportunity and a duty to do something about that.
Orkambi was licensed for use in 2015. The SMC recognised that it was an important therapy but rejected it on cost grounds in 2016. Two years on from that, Orkambi is available from the drugs company only on compassionate grounds. Meanwhile, people with cystic fibrosis are dying.
Time is something that cystic fibrosis sufferers do not have. A resubmission to the SMC would take six months, and I am not convinced that the appraisal process recognises the contribution to the economy that someone living and working for an additional 30 to 40 years would make. The way in which the SMC measures cost and benefit does not even begin to capture that fully.
I welcome the roll-out of the ultra-orphan medicine pathway. It is great, but the truth is that it applies only to conditions affecting fewer than 100 people. There is a gap in the system in how we treat orphan conditions. Orkambi is appropriate for about 300 cystic fibrosis sufferers, so it does not qualify as an ultra-orphan medicine. It is clear that there is a gap between the ultra-orphan medicine pathway and the SMC process.
I hear that the cabinet secretary is asking for an exemption to be made in the case of Orkambi. That is a start, but it does not address the underlying problem, which is where I would like her to go further. We need a portfolio agreement, not just an agreement about Orkambi. We need an agreement about the next generation of medicines that are coming down the line. I know that that is a new concept that does not fit the processes that the Scottish Government has, but let us not be hidebound by systems if they are not flexible.
Allow me to correct the cabinet secretary as gently as I can. All new treatments in a portfolio agreement have to be licensed first. I would have the same safety concerns as she has, and I would not be recommending that course if I did not think that it was appropriate. At the end of the day, it is about patients.
Here are the countries that have agreed a portfolio deal: Austria, Denmark, Germany, Luxembourg, the Netherlands, Italy, Greece, the United States, the Republic of Ireland and—just this week—Sweden. I ask the cabinet secretary: are all those countries wrong? Do we know better? Are cystic fibrosis sufferers in Scotland different from sufferers in any of those countries?