Doctors, not politicians, should be making decisions on patient care.

And the B.C. government’s handling of a controversial, costly treatment for 10-year-old Charleigh Pollock just showed why.

Charleigh has a form of Batten disease. It’s genetic, extremely rare, incurable and kills at an early age, with cruel and worsening symptoms.

Since 2019, the Langford girl has been receiving Brineura, a medication that has shown some success in slowing her disease progression, at a cost of $800,000 a year. It’s made by BioMarin Pharmaceutical Inc., an $11-billion California-based company with $2.9 billion in sales in 2024, up 18 per cent from a year earlier.

Charleigh’s parents — understandably — want to continue the treatment. She can no longer walk or speak, but they say it has reduced her seizures. And there is always the hope of buying time until more effective treatments might be discovered.

Drug companies like BioMarin justify high prices for rare disease treatments by pointing to high research and development costs and limited markets. (Raising questions about whether more of the research should be publicly funded and not dependent on profit potential.)

Given the extraordinary costs, limited health budgets and intense drug company marketing efforts, governments need to be sure treatments are effective.

Since 2007, the B.C. government has relied on assessments from the Expensive Drugs for Rare Diseases committee, with more than 50 members. The researchers, doctors and experts are unpaid and tasked with assessing whether drugs are effective and making recommendations to the health ministry. It’s challenging, in part because there is limited data on drugs used by only a handful of patients.

The committee panel concluded Brineura was no longer effective as Charleigh’s illness progressed. The ministry asked for another review and the findings were unchanged.

So funding for Charleigh’s treatment ended, a decision Health Minister Josie Osborne publicly defended despite political and media pressure.

Until last month, when the government reversed course and agreed to pay for the drug. Osborne said she made the decision after getting a letter from 12 doctors and one neuroscientist from around the world saying Charleigh’s treatment should continue.

That showed “a significant disagreement between health experts on Brineura, and it is not acceptable that Charleigh and her family suffer as a result,” she said.

Premier David Eby offered the same justification, claiming the government had to deal with arguments between “two groups of experts fighting it out” in deciding on funding.

But reporting from the Vancouver Sun’s Gordon Hoekstra showed “two groups of experts fighting it out” doesn’t really cover the situation.

The Expensive Drugs for Rare Diseases committee offered evidence-based advice based on the science. They were physicians, researchers and other experts, volunteers willing to provide independent guidance in the public interest.

The letter that prompted Osborne to change her mind came from 13 people.

And as Hoekstra reported “eight have declared conflicts in the past five years in papers… because they have consulted for, been paid by, or received grants from California-based BioMarin Pharmaceutical Inc.”

Another doctor reported being a consultant for BioMarin for four years and was paid by BioMarin to be on a medical podcast.

“Three other doctors who signed the letter have relationships with foundations that advocate and raise money for Batten disease research,” Hoekstra reported.

Why would Eby and Osborne deem those two equivalent voices?

Some members of the Expensive Drugs for Rare Diseases committee asked the same question after their work was rejected in favour of the advocates aligned with the pharma company’s interests. At least 10 resigned. A program that has served British Columbians well for decades is in shambles because the government threw the participants under the bus once the political heat got too intense.

Eby called it a “secret committee within the Ministry of Health speaking only through politicians” and trashed the committee process.

And he chose to overrule the expert committee and turn what had been a scientific, evidence-based decision into a political one, based on lobbying efforts from scientists linked to the drug company and other pressure.

There are two big questions here.

One is scientific and medical. Drug companies are developing treatments for rare diseases that can cost millions per patient. B.C. spent $153 million dollars on rare disease drugs in the 2024 fiscal year ending in March 2024, up 52 per cent from the previous year. At the current growth rate, Colin Dormuth and Ken Bassett of the Therapeutics Initiative wrote in the Vancouver Sun, B.C. will pass $1.6 billion per year in rare disease drug costs in five years. “It will pass $11.3 billion annually in 10 years, solely devoted to drugs for less than one per cent of the population, equivalent to almost a third of today’s total health-care budget.”

Emerging treatments are a cause for celebration. But they also mean governments need to ensure effectiveness.

The B.C. government didn’t do that.

The other is integrity. The government could have decided to fund Charleigh’s continued treatment and acknowledged it was responding to pressure and choosing compassion over evidence.

Instead, it suggested the committee had botched its work and failed the public. Some media joined in, accusing members of lacking compassion. That claim stung and was false, said Dr. Sandra Sirrs, one of the members who resigned.

“Like holy God, you have no idea the amount of consideration and thoughtful discussion,” she said. “What we want is we want Charleigh not to have that disease, and if we couldn’t have that, what we want is a drug that reverses the changes she suffered from Batten disease.”

And the review found Brineura would no longer do that.