Let's talk about Vertex Pharmaceuticals
Updated: Feb 5
A petition calling for global access to a range of life-saving, life-changing drugs called cystic fibrosis modulator therapies is currently live and has gained over 32,000 signatures.
Vertex Pharmaceuticals who manufacture these drugs – namely, Kalydeco, Orkambi, Symdeko (Symkevi)) and Trikafta (Kaftrio) – have made billions of dollars with reimbursement deals in countries such as USA, Germany, UK, France, Ireland and other high income countries. Most of that profit has been generated by sales of their most effective drug, Trikafta (known as Kaftrio in Europe).
However, there are tens of thousands of cystic fibrosis (CF) patients living in low and middle income countries, whose governments cannot afford the exorbitant price tags that Vertex Pharmaceuticals attach to their drugs. View VIDEO.
The petition mentioned above is part of a campaign coordinated by patient advocacy groups Vertex Save Us and Just Treatment, but driven by the passion and commitment of CF families and groups around the world - in countries both with and without access. This campaign, calling for a global access plan to Vertex’s modulator therapies, has managed to unite CF patients and groups in countries that previously felt isolated in their fight for equitable treatment options.
CF modulators work at the root cause of CF, attempting to correct problems caused by the basic genetic fault. How Trikafta works.
Treatment with Trikafta is linked with fewer infection related hospitalisations and a reduced need for antibiotics, among people with cystic fibrosis, a study based on real-world US data indicated. (Source: Article)
So the earlier a person with cystic fibrosis can be started on Trikafta, the better chance they have of maintaining their health and avoiding frequent hospitalisations, which of course, impacts on every aspect of both the patients life and the lives of their families.
The main problem is that Vertex Pharmaceuticals have a complete monopoly in this field. A situation that is likely to continue for some considerable time. But time is one luxury that people living with cystic fibrosis don’t have - sudden, frightening declines with CF are not an uncommon occurrence.
A recent presentation at the North American Cystic Fibrosis Conference, revealed that Trikafta could be profitably produced for LESS than $6,000.
“The estimated minimum cost of production of Vertex Pharmaceutical’s most effective modulator therapy Trikafta is $5,700 - 98% lower than current US list price of $311,000”
So when does profit become greed?
Vertex currently have around $9 billion in the bank. Their former CEO Jeffrey Leiden has been quoted as saying that the company "has a good problem of accumulating money quickly.”
One way that Vertex Pharmaceuticals have found to spend their money, is in sponsoring any CF related event or providing grants to any cystic fibrosis association. You could throw a stone blindfold in the CF community and you would hit someone whose event/organistion has received financial support in one way or another from the company.
On the surface, this seems to be incredibly generous. But you don’t have to ponder for too long to see the motivation here.
Vertex cultivate a ‘Don’t bite the hand that feeds you’ mentality. They act as a friend and benefactor to the cystic fibrosis community on the one hand, while the other hand is gleefully counting the billions they are making from selling those drugs to the same people at such elevated prices. Not so much robbing from Peter to pay Paul - more like ‘Mugging Paul and giving him a couple of dollars back to compensate.’
The downside for the CF community is that they feel unable to criticise their 'benefactor'. This is even more ominous in the USA where Vertex offer a copay assistance program for CF patients. This is also currently the subject of much intense criticism as Vertex have recently taken the decision to decrease copay assistance - see: CF Foundation Urges Vertex Pharmaceuticals to Address Its Decision to Decrease Copay Assistance for People with Cystic Fibrosis
But the fact that many CF patients rely on this copay assistance, makes it incredibly hard for them to question, stand-up to or criticise Vertex Pharmaceuticals. During the course of campaigning for global access a real climate of fear and trepidation - particularly in the United States - has come through in anecdotal evidence from the CF community.
While negotiating their deals around the world, the company have offered ‘compassionate access’ to patients too ill to wait for protracted negotiations. However, the same compassionate access is not offered in low or middle income countries, where Vertex have no interest in pursuing a deal – because they know that they will not achieve the high prices they aspire too.
So ‘compassion’, in Vertex Pharmaceuticals rulebook, seems only to exist where they see huge profits at the end of their compassionate rainbow.
So why are we calling for a ‘global access plan’ for CF modulator therapies?
A campaign coordinated by patient advocacy groups Vertex Save Us and Just Treatment, is calling for all countries to have access, at a reasonable price.
According to a study published in the ‘Journal of Cystic Fibrosis’ in February 2022 “Of the 162,428 people estimated to have CF worldwide, currently 64.9% are diagnosed and 12% are receiving triple combination therapy”. See study here: https://www.cysticfibrosisjournal.com/article/S1569-1993(22)00031-5/fulltext
Inevitably Vertex Pharmaceuticals have sought reimbursement deals with high income countries, who also have high rates of diagnosed CF patients – the USA has around 30,000 people diagnosed with cystic fibrosis, the UK – around 10,500. But as the above study shows, that leaves many thousands around the world without access to the medicine that could transform and even save their lives.
But Vertex have no altruistic intentions towards the global cystic fibrosis community. In fact, by taking out patents in third world countries which often lack a proper medical infrastructure, where they have no intention of pursuing a reimbursement deal, Vertex are effectively blocking other companies from producing a more affordable generic version of Trikafta.
Or, to put it more bluntly, blocking any possible route to the most effective treatment for thousands upon thousands of CF patients.
In the UK, the cystic fibrosis community fought for several years for access to Orkambi.
Their campaigning made the issue of access to CF modulator therapies so high profile, that when Trikafta/Kaftrio was approved for use in this country, the reimbursement deal followed fairly swiftly.
But patients and families of those with CF, will remember only too clearly the pain of being left behind while patients in the USA and Ireland gained access.
As campaigning mum Christina Walker said of her son at the time: “To know there are drugs available that could extend and improve his life, yet Luis can’t access them, is torture in the extreme and I can’t allow that to be the case. It’s life and death for Luis and thousands of others.”
The following video from This Morning will remind us of that desperate time – and perhaps anyone with a child under the age of three with cystic fibrosis, who might not be aware of some of the more unsavoury details of our battle with Vertex Pharmaceuticals, might find this interesting viewing too: This Morning interview with Christina and Luis
Perhaps this video from Dr. Paul Quinton - who discovered the basic defect in CF in 1983 - best sums up many people's feelings about Vertex and their excessive prices.
"On the one hand we would like to think of Vertex Pharmaceuticals as our hero. In Trikafta/Kaftrio they have produced a drug that is life-saving and life-altering.
On the other hand the extortionate prices they charge, and some of the methods they use during negotiations, do not show much 'compassion' or care for the patients they are seeking to help."
Join Dr. Paul Quinton in challenging Vertex Pharmaceuticals and their CEO Dr. Kewalramani to devise a system by which any patient with cystic fibrosis can obtain the drug - in every country worldwide.
Please sign and share the PETITION
Remember - “The enjoyment of the highest attainable standard of health is one of the fundamental rights of every human being without distinction of race, religion, political belief, economic or social condition”.
Almost 70 years after these words were adopted in the Constitution of the World Health Organization, they are more powerful and relevant than ever.