There is a moment early in a newborn baby’s life when their mother willingly lets a stranger hurt them. She does it because she loves her child and wants to keep them safe.
The baby’s pain is fleeting, but it’s real – a needle pricks the skin of the child’s heel, the baby cries, the blood wells up and is collected by a healthcare visitor. And then the mother soothes her baby with strokes and kisses and feeds.
For the vast majority of mothers that’s the end of it, because the nine diseases being looked for in the UK’s national newborn screening programme are all very rare.
We were one of the families for whom the heel-prick test opened a new and brutal chapter. Our baby, a boy named Griffin, was diagnosed with a genetic condition affecting just 1-in-2,500 born in the UK.
We took Griffin into the local specialist centre for a confirmation of diagnosis. The doctors were kind but they also told us the truth. As we sat there, in our mid-thirties, we likely had more years left on this planet than our wriggling five-week-old baby boy. Before he’d even learned to walk or talk, our child’s journey towards death had been given its form: lung infections, long hospitalisations and then respiratory failure. This death had a name: cystic fibrosis (CF).
The grief that followed a diagnosis like this was of a bitter and peculiar kind. We hadn’t lost our child… but we knew that in due course we would experience losing him. Consequently there was a dark lining around the silver of his fresh existence.
We work hard, daily, to keep him well. Physiotherapy to keep his lungs clear, pills to help him digest food, antibiotics and bacterial surveillance at the first signs of a common cold. We do anything we can to stave off the day when an infection in his chest can’t be shifted and then becomes something that eats away at him, until his lungs fail. We carry that dread and that responsibility.
But two years after Griffin was born, something happened that promised to change things, something that gave us hope for a joyfully different kind of ending. In America, the FDA approved a new drug called “Orkambi”, made by the pharma company Vertex.
This drug wasn’t perfect by any means, but it offered stability – and even mild improvements – to patients with Griffin’s kind of CF. Even more excitingly, this was only a first-generation therapy in a pipeline of drugs that have spectacular promise. Vertex’s drugs would soon be moving beyond offering ‘stability’ to making major, life-changing improvements to patients, achieved through fixing the genetic problem at its root – forcing the body’s faulty cells to function as they do in healthy people. A very modern miracle.
These drugs wouldn’t just reduce the daily treatment burden or result in fewer hospital stays. They offer whole decades of extra, healthy life, provided therapy begins when children are still young and relatively healthy.
If that first prick with a needle at five-days-old had initiated a fairy-tale kind of curse, then this was the moment where the curse might lift. We could see an end to the quiet grief that we carry daily and the beginning of a brighter, unburdened future for our child.
Many people celebrated Orkambi’s approval. Patients with CF and those who love them, of course. The amazing scientists at Vertex who have dedicated their working lives to fixing this horrible disease. And also shareholders in Vertex’s rocketing stock (shares which hovered around $70 in 2013 but today are worth $180).
In our naivety, we didn’t know that this stock-price surge might be an ominous omen for British CF patients.
You see, American pharma companies operate on a high-risk high-reward basis. The vast majority of new drugs fail at trial stage and all those failures are extraordinarily costly. The drugs that do become ‘hits’ then have to pay for those failures.
Drugs for rare conditions like CF end up being the highest-priced of all, precisely because the market is so small. In America this system is just about workable, as insurers can spread the cost of high-price patients across patient pools. They pay a lot for healthcare, but access to drugs is pretty much guaranteed.
In the UK it’s a different deal. Our socialised healthcare system, the NHS, refuses to guarantee access to the best new drugs. Here, drugs have to pass an additional “value for money” test, via assessment by an independent review board, NICE. It’s procedures are opaque, the prices put on lives arbitrary, and its recommendations are non-binding… but nevertheless politicians have found it a very useful fig-leaf, because no health minister wants dead bodies on his or her doorstep. “Respecting NICE’s independence” has allowed politicians to duck full responsibility for making life-or-death decisions about drug funding.
In the summer of 2016 NICE ruled that, despite being both effective and the only treatment targeting the root cause of CF, ‘Orkambi’ was too expensive for the UK.
We were devastated.
We had grown up believing that, for all its travails, our NHS was a world-class healthcare system. We had paid our taxes. There were no private health insurance packages for chronic conditions that we might have bought. In short, we had done everything we were supposed to as citizens and parents.
And yet we now faced a situation where our child would suffer earlier, and die sooner, than his peers in the USA and most of Europe.
Since NICE’s recommendation, two hundred people with CF have died in the UK, many of whom might have lived longer, healthier lives, had they had these drugs.
With every day that passes, children with CF pick up infections that they won’t ever be able to shift, and thus their prospects for a long and healthy life dim radically.
It’s not over yet, though.
This Thursday representatives from Vertex and NHS England will meet once again to discuss reaching a deal, with health minister Matt Hancock ultimately responsible for the outcome of these talks.
Both sides need to listen. Vertex need to help the NHS adapt to this new era of high-priced rare-disease medicines. If they can help cut a deal, they’ll both honour the work done by their scientists and deliver value to their shareholders, through accessing the world’s second-largest CF patient population.
NHS England and Matt Hancock need to be realistic as well. We know that American biotech pricing is a poor fit with NHS budgets, but patients cannot wait for those long-term problems to be fixed. Politicians need to offer a fair price for Vertex’s drugs, accepting that if the UK can’t or won’t take on curing CF using public funds, then a reasonable price ought to be paid to those who take on that task.
Many countries have struggled with the cost of treating CF patients in the new era. Ireland, Germany, Sweden, Italy and many others besides have weighed these arguments and decided, on balance, that saving the lives of their citizens matters most. As a result, children born with CF in those countries are now offered more than a slender dream of things getting better – they have access to drugs that will likely see them living well into retirement.
Children born with CF in the UK currently have no such prospects.
Thursday’s meeting is a chance to change that, to reach a deal that gives us hope again. To unpick the black-lining from around our children’s days. To strip the name ‘cystic fibrosis’ from their destinies and free them, to live their lives like any other.
Hannah Begbie is a novelist. Her first book, “Mother” (Harper Collins), tells the story of a woman’s dark descent into life-risking behaviours, following the diagnosis of her baby with CF. Her husband Tom Edge is an award-winning screenwriter whose work includes the TV shows THE CROWN, STRIKE and LOVESICK.