LONDON: Soon after Emma Corr, a teacher in northeast England, gave birth to her second daughter in 2014, she was devastated to learn that her baby had cystic fibrosis, a life-shortening genetic disease that causes severe lung damage.
Then she heard about a promising new drug nearing the market that might transform her child’s life.
“It was a glimmer of hope at a very dark time,” says Corr, whose daughter, Harriet, is one of 10,000 people in the UK afflicted with the illness.
Yet more than three years after the medicine, Orkambi, was cleared for sale, it remains unavailable in England.
It’s an agonising wait on top of an already heart-breaking diagnosis.
Cystic fibrosis leads to a build-up of thick and sticky mucus, breathing difficulties and lung infections.
Children born today with the disease can only expect to live into their mid-40s.
The long delay is all because of a dispute over the drug’s price between Vertex Pharmaceuticals and the country’s health authorities.
That’s left the Corrs with little choice: Like other families, they are preparing to leave England and move to Scotland or Ireland in a desperate bid to obtain the treatment.
“How long can we wait?” says Corr, 38.
Unless the drug is made available soon, she and her husband plan to permanently relocate to Edinburgh 100 miles from their Newcastle home.
“It’s so frustrating,” she says.
“I just want to secure a future for my little girl.”
Wildly expensive drugs are commonplace in the US, where a year’s treatment of Orkambi costs US$272,000.
There, the problem has gotten so severe that several top pharmaceutical executives are being called this week to testify before the Senate Finance Committee for the first time ever.
But in countries with universal health coverage, high prices manifest differently.
In financially-constrained England, when confronted with a pharmaceutical company such as Boston-based Vertex seeking to recoup billions of dollars to develop a new drug, the answer is sometimes brutally simple: almost no one gets the medicine.
It’s a global conundrum that’s intensifying, says Karl Claxton, a health economist at the University of York.
“There’s a massive mismatch between what is coming out of the pipeline and what health-care systems can afford,” he says.
“Ultimately the sector is going to have to change to realign with those realities.’’The National Institute for Health and Care Excellence, an independent body that advises the government on drug coverage, says Vertex isn’t doing enough to negotiate.
The group has also said Orkambi’s benefits appeared only modest.
Vertex argues that England’s drug-review system isn’t set up to evaluate modern rare-disease treatments.
What’s more, the company asserts the drug helps extend lives and cut hospital visits while tackling the root cause of cystic fibrosis.
“Precision therapies like ours are delivering promising results, but they also present new economic challenges,” says Rebecca Hunt, a vice president of Vertex’s international business.
“We recognise fully the constraints that health systems around the world are working under, which create unenviable choices.”
The drugmaker has succeeded in getting Orkambi coverage in 10 nations and said it’s determined to find a solution in England.
Across the border in Scotland, the medicine is available at a discount on a case by case basis and may be more broadly available within the year, according to the company.
While Vertex’s profit more than doubled last year to US$1.06 billion, the company said its drugs are the result of almost two decades of work and billions of dollars of spending.
Even so, Vertex is an example of pharma’s use of reimbursement deals as a “cudgel’’ to influence holdouts like England to pay for expensive treatments, says Peter Bach, director of Memorial Sloan Kettering Cancer Center’s health policy group in New York.
“If we could have access at a reasonable price, that would be better than the alternative,” he says.
“But giving in to hostage-takers has led to this endless increase in prices of drugs vastly beyond what’s appropriate.”
While an older Vertex drug, Kalydeco, has been available in the country for years, it treats only a small number of patients with specific genetic mutations.
Orkambi and another new drug, Symkevi, introduced in 2018, help a much broader pool of people.
The three drugs are forecast to generate about US$3.5 billion in global sales this year.
The two sides could still reach an accord but remain far apart for now.
England’s National Health Service called Vertex an “extreme outlier in both their pricing and behaviour.”
The country offered about 100 million pounds (US$130 million) per year for all of Vertex’s current and future medicines.
That’s roughly double the amount it spent on Kalydeco.
Settling the dispute is particularly important in England.
The island nation is home to 12% of the patients globally with the disease, which is more common in Celtic and Anglo-Saxon populations.
Orkambi also isn’t available in the UK region of Northern Ireland.
That’s driving Jen Banks, whose two-year-old son Lorcan suffers from cystic fibrosis, to move across the border into Ireland.
Her husband faces a long daily commute, but the couple has decided that’s a small price to pay for their son’s health.
“It’s going to have an effect on probably every aspect of our lives, but it’s worth it,” says Banks, 34, a citizen of both countries.
“There’s hopelessness around it all, knowing he’ll only get sicker. Without these drugs, it really casts a shadow over our time with him.”
Corr, the teacher who is getting ready to move from Newcastle, worries about finding work in Scotland and displacing her daughters.
But she says she has no choice, “As a parent, you are going to do everything you can to get your hands on those drugs.”