“So guess what’s been approved by NHS England today? What have we been fighting for?” said Beth to her anxious looking daughter, Isabelle. “In 30 days we’ll be able to get a prescription” Beth continued, as Isabelle broke down in tears of joy and palpable relief. Captured on video and posted on social media, this was 13-year-old Isabelle’s moving reaction to the announcement last week that the medication Orkambi (lumacaftor-ivacaftor) will be available on the NHS before Christmas to patients like her suffering from cystic fibrosis (CF).
CF, a genetic condition affecting over 10,000 people in the UK, causes breathing difficulty and recurrent chest infections. Half of those affected won’t make it past the age of 40. Orkambi slows progression and improves the quality of life of CF sufferers. You’d be forgiven for thinking its availability to children like Isabelle would be a certainty.
However, it’s taken four long, hard years of campaigning for NHS England to finally reach an agreement with Vertex - the US pharmaceutical corporation that holds the intellectual property rights to exclusively produce it. Vertex has a patent on Orkambi, and for the past four years they’ve charged over £100,000 per year for every patient treated (though a report from Just Treatment calculated that a generic could be produced for just £5000 a year). This is an excessive and unaffordable amount for the NHS and, in this time, hundreds of people with CF that have been denied treatment have suffered unnecessarily, and died.
This is despite the fact that the development of Orkambi (like many other drugs) was heavily subsidised by both public (US) and charitable (Cystic Fibrosis Foundation) sectors. A report by Aidan Hollis at the University of Calgary suggested that Vertex recouped their R&D costs years ago (net cost, adjusting for risk and cost of capital, was in the range of $2.5bn). We know that the price tag that pharmaceutical corporations attach to medicines internationally bears little relation to R&D costs.
The public pay twice, and pharmaceutical corporation CEOs, like Vertex’s Jeff Leiden, are left with “a nice problem of accumulating cash very rapidly.” Despite this, the Secretary of State for Health and Social Care, Matt Hancock MP, stated that the agreement reached was “proof” that the system works and that we should “reject” calls for reform. I am not sure in what way four years of campaigning to get access to a life saving drug is proof of a system that works!
Just Treatment, who worked on the Orkambi campaign, together with economics Professor Mariana Mazzucato and others, are calling for progressive reform to prevent this happening again. One of their proposals is the use of compulsory licences to allow the NHS to buy a generic (identical but much lower priced) version of a medicine, even if it’s protected by a patent-backed monopoly. This would have offered an important legal safeguard to protect public health and secure affordable access to Orkambi, and Labour’s recently announced conference policy would go some way towards addressing this point. This Government, however, rejected the proposal, leaving my young patients struggling to breathe.
Whilst we rightly celebrate the success of the Orkambi campaign and acknowledge the hard work of campaigners, let’s also ensure that we question why this ordeal was even necessary. Should we have to fight so hard in order to get government to fulfil it’s basic responsibility of protecting us from harm, and allowing the healthcare service to do what it’s meant to: heal us?
Vertex is not the real villain here. They are simply operating within a system that prioritises profit over public health. This government’s commitment to protecting private interests is implicit both in Hancock’s opposition to systemic reform of the pharmaceutical industry, and in the defeat last week in Parliament of a motion put forward by Labour that would have protected the NHS from privatisation in future trade deals (Conservatives voted against the motion, and Liberal Democrats abstained). And this week’s report by Channel 4’s Dispatches, exposing secret meetings between UK trade negotiators and US corporations discussing drugs pricing, further highlighted the potential risk to the NHS posed by a post-Brexit US-UK drugs trade deal.
Where public money has been spent on research, we should expect to pay a reasonable price for the drugs that we’ve helped to develop. And when pharmaceutical corporations insist on extortionate pricing, we should expect our government to stand up for its citizens and exercise its legal powers to obtain the drug generically, rather than cower in servitude to pharma.
Kuvan (generic name sapropterin hydrochloride) is another new drug produced by the US corporation, BioMarin. It’s effective in the treatment of a rare genetic condition called phenylketonuria (PKU), but it remains unavailable on the NHS due to its £70,000 price tag. “I helped test a wonder drug - then I was denied it”, says Louise Moorhouse, a PKU patient.
So whilst Isabelle’s battle with Vertex may have ended, Louise’s battle with BioMarin continues. The system remains broken and in need of urgent reform.