There was a brilliant but controversial opinion piece in The Globe and Mail newspaper this week, by André Picard. The Globe, as it’s known, is Canada’s only national journalism-based news source – aside from the government-funded CBC (Canadian Broadcasting Corporation). As for André Picard, he is this country’s most respected healthcare journalist, having covered health policy and care for almost 40 years.
He has written several books on Canadian healthcare and health policy, six of which were bestsellers – I own several, and have read them all.
André has won the ‘Columns Category’ of Canada’s National Newspaper Awards, twice, as well as the Michener Award for ‘meritorious public service’ in journalism.
The Canadian Public Health Association named him the country’s inaugural ‘Public Health Hero’, and the Canadian Alliance on Mental Illness and Mental Health named him a ‘Champion of Mental Health’. He’s often a panelist or public speaker at healthcare events, and has advised on health policy.
So when he offers an opinion piece, it’s based on his almost four decades of reporting on – and investigating – healthcare, health policy, and health politics.
This week, André Picard wrote about the recent case of a child with a rare disease in British Columbia, and what happened after the provincial government’s initial decision – based on recommendations by an expert medical committee or panel – to discontinue a specific and very expensive drug treatment after six years, when the child’s condition deteriorated; after she got worse.
You might expect that I’d disagree with this kind of decision, because I now live with two different rare diseases (one potentially fatal, the other which has resulted in severe chronic pain and a career-ending Mild Cognitive Impairment)…
But I’m also a medically-retired bioethics professional, who has studied health policy – including how Canada’s mosaic of healthcare funding functions (or doesn’t!) at provincial or territorial and regional levels.
So I do agree with André’s opinion piece:
“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.”
Most other healthcare funding decisions, particularly whether to fund specific medications – for individual patients, groups of patients, or almost entire populations (e.g. H1N1 influenza vaccinations) – are made by government officials based on these kinds of recommendations from expert advisory panels.
These experts consider many issues, like whether a medication shows good efficacy; does it treat or prevent illnesses much better than existing treatments – or no treatment? They’ll look at how cost-effective a medication is; is it cheaper to buy, or less expensive to administer – for example a take-at-home pill to replace an injection that has to be given by a nurse – than current treatment options? They consider Adverse Events (AEs), or side effects, which can sometimes be worse than the disease itself; look up Vioxx, or Thalidomide, for some examples.
You might expect that I’d disagree with this kind of decision, because I now live with two different rare diseases (one potentially fatal, the other which has resulted in severe chronic pain and a career-ending Mild Cognitive Impairment)…
But I’m also a medically-retired bioethics professional, who has studied health policy – including how Canada’s mosaic of healthcare funding functions (or doesn’t!) at provincial or territorial and regional levels.
So I do agree with André’s opinion piece:
“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.”
Most other healthcare funding decisions, particularly whether to fund specific medications – for individual patients, groups of patients, or almost entire populations (e.g. H1N1 influenza vaccinations) – are made by government officials based on these kinds of recommendations from expert advisory panels.
These experts consider many issues, like whether a medication shows good efficacy; does it treat or prevent illnesses much better than existing treatments – or no treatment? They’ll look at how cost-effective a medication is; is it cheaper to buy, or less expensive to administer – for example a take-at-home pill to replace an injection that has to be given by a nurse – than current treatment options? They consider Adverse Events (AEs), or side effects, which can sometimes be worse than the disease itself; look up Vioxx, or Thalidomide, for some examples.
And these experts will evaluate at which point the medication should be discontinued, when a patient should stop taking it. A good example of this is Benzodiazepines for elderly patients; this class of medications can be prescribed for sleep issues in older people, but are also known to cause cognitive impairment, delirium, falls and resulting fractures, and accidents while driving – because how the body processes medication changes as we age. Also in older patients, Benzodiazepines have been shown to be addictive, leading to problems when they’re stopped if a person has been taking them for too long; they were designed for short-term use.
As part of their roles, these expert advisory groups are usually asked to look at whether a medication is safe, effective, and cost-effective – and at what point in disease progression any of these criteria change; when does it no longer make sense to pay for a treatment with taxpayer’s funds?
Why look at cost-effectiveness at all? Governments can’t afford to cover every medication for every condition, which would bankrupt any government, so hard decisions have to be made about which drugs it makes the most sense to cover.
There’s an entire branch of bioethics devoted to ‘resource allocation’, or what to cover with a limited budget – the situation of publicly-funded healthcare everywhere.
“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.”
“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.”
Back the recent media coverage of this one case in B.C… Charleigh Pollock was taking Brineura:
“infused into the brain every two weeks, a treatment that costs $33,000 each session – roughly $850,000 a year…. Brineura was funded for six years, but the experts determined her condition had deteriorated and met the “discontinuation criteria” for the drug.”
“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.”…
“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.”
“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.”
Of course, new research and new information about medications should be considered as it becomes available, and this applies to super-expensive medications for rare diseases as much as to drugs used by large numbers of people – like statins or products to lower blood pressure
References:
“Is denying a drug to a terminally ill 10-year-old cruel – or is it good governance?” André Picard; Health Columnist, The Globe and Mail. 29 Jul 2025; updated 30 Jul 2025. Available online (possibly only with subscription):
https://www.theglobeandmail.com/opinion/article-is-denying-a-drug-to-a-terminally-ill-10-year-old-cruel-or-is-it-good/
The Globe and Mail (website). André Picard. Accessed 02 Aug 2025:
https://www.theglobeandmail.com/authors/andre-picard/
Cindy E. Harnett. Drug coverage to be discontinued for Langford girl with fatal condition. Times Colonist (newspaper). Victoria, BC. 19 Jun 2025. Accessed 02 Aug 2025:
https://www.timescolonist.com/local-news/drug-coverage-to-be-discontinued-for-langford-girl-with-fatal-condition-10830120