Is denying a drug to a terminally ill 10-year-old cruel – or is it good governance?

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Much ink has been spilled and many pixels alighted in recent weeks about the case of Charleigh Pollock, a 10-year-old B.C. girl who suffers from a rare disorder called Batten disease.

Long story short: B.C. stopped funding Charleigh’s drug treatment, deeming it was no longer effective; her parents objected and launched a GoFundMe campaign to continue treatment; the media and opposition politicians expressed outrage about heartless politicians and bureaucrats condemning a severely ill child to death to save a few bucks; the government caved to public pressure and overruled an expert committee, announcing that funding for the drug would continue.

A happy ending? Not really.

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There are few things more heart-wrenching than the plight of children with rare neurological disorders like Batten disease, a fatal condition that progressively robs them of the ability to see, speak, walk and, eventually, swallow.

Doubly tragic is that there are few effective treatments, and the drugs that do exist are often exorbitantly costly.

Charleigh, only one of 13 children in Canada with Batten disease, is being treated with Brineura, manufactured by BioMarin Pharmaceuticals. The drug helps slow the seizures she suffers from. It improves her quality of life. It could potentially extend her life expectancy, which is eight to 12 years.

Brineura is infused into the brain every two weeks, a treatment that costs $33,000 each session – roughly $850,000 a year.

Because drugs for rare disorders – also known as orphan drugs – are used by a very small number of patients, and tend to be very costly, they are subject to particular scrutiny.

Every province has a review process – a group of expert advisers who determine if a patient is eligible for a drug, and if it should be funded.

In the case of Charleigh Pollock, Brineura was funded for six years, but the experts determined her condition had deteriorated and met the “discontinuation criteria” for the drug.

Many patients are denied public reimbursement for orphan drugs because the treatments are deemed to not be efficacious and/or cost-effective.

These are tough decisions, but they need to be made. If we’re going to list drugs, we have to be able to delist them.

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Understandably, Charleigh’s family was upset. The treatment gave them hope. They wanted it to continue.

So they went public, and the girl’s story captured the public imagination.

On the surface, it looks heartless for a government to say “no” to a severely disabled child. But from a public policy perspective, we have to make evidence-based funding decisions. We have to be able to say “no” sometimes.

What is the alternative? To simply fund every drug that comes along, regardless of how effective and costly?

Initially, B.C. Health Minister Josie Osborne backed the expert committee. So did Premier David Eby, saying politicians shouldn’t be interfering in complex medical decisions. And he’s quite right – just look at Alberta’s awful Bill 26, which limits how doctors can provide gender-affirming care for young people.

But the B.C. government took a beating in the media. One columnist rebuked the province for “picking a fight with an ailing 10-year-old.” Opposition Leader John Rustad said they were creating “GoFundMe health care.”

The government caved. Suddenly, it became okay for politicians to make medical decisions. Ms. Osborne justified the flip-flop by saying U.S. experts questioned the criteria used for judging the effectiveness of the drug Charleigh was taking.

There is a legitimate debate worth having about how a drug is determined to be working, if it’s more beneficial than harmful and if it provides value for money.

But these are esoteric debates that have to be hashed out by experts. Not by public opinion.

There needs to be a transparent process for such decisions. But there also needs to be a broader recognition that there is no perfect way of making them. When it comes to when and how to fund drugs for rare disorders, no country in the world has managed to get it quite right.

What is clear is that B.C. got it quite wrong. By opting for political expediency, the province has sent a message that the way to get a treatment funded is to make a stink.

When announcing the policy reversal, Ms. Osborne said the treatment will be available to Charleigh “for as long as the treating physician and family deem it appropriate.”

Really? Is that how decisions will be made now? Can someone who is told a drug won’t work demand that it continue, anyhow? Can a patient deemed unfit for a transplant demand it?

That approach is neither equitable nor sustainable.

Governing responsibly means making tough decisions, not just popular ones. And nowhere is that more important than in health care.