Flawed Logic and Treatment Denial in Cystic Fibrosis: More Evidence for National Public Pharmacare

Governments across Canada have recently been under pressure to cover new and VERY expensive treatments for cystic fibrosis.

Government decision-makers are faced with the plights of Canadians like 24-year old Katrina Secord in Alberta or 30-year old Lilia Zaharievais in British Columbia, just two of the 4000 cystic fibrosis patients literally begging for their lives.

And CF patients have much more to fight for. Fifty years ago life expectancy for those with CF was 17. Treatment and life expectancy have improved significantly, with median age for survival in Canada now 51. And that may increase even more with novel treatments like Kalydeco™ and its analogues, Orkambi™ and Symdeko™.

But there IS a catch.

These novel treatments cost about $250,000 per year. Every year. Per Patient. For the rest of their potentially longer, better lives.


Kalydeco was approved by Health Canada in 2012 and became available through many private plans almost immediately, but not through public plans until a price was negotiated in 2014.

Orkambi which is actually just Kalydeco with an additive that promotes absorption was approved in 2016. It was also made available through private drug plans that covered it quite quickly, but rather than having a price negotiated for public plans, access became a nasty political and moral blame-fest. A quick note, Symdeko (which is also just Kalydeco with a non-curative additive) was approved by Health Canada in 2018, but has not yet been considered for public coverage.

Act I: The Private Insurers

As quick as private plans were to add these drugs, the mess for Katrina and Lilia started with private drug insurance. Katrina’s and Lilia’s plans both provided access to Orkambi. Both saw their lives change dramatically. Their health improved and—for the first time in their lives—they started to make long-term plans.

To no avail.

Katrina began having trouble when her husband had to switch employers as his new job didn't provide drug insurance for almost a year.

For Lilia her plan at the University of Victoria was, like many, a self-insured group plan. That meant that each year’s premiums were based on how much was paid for drugs in total the year prior, with a percentage of about 4% tacked on as an “administration fee.”

Due to an “unprecedented increase,” including Lilia’s medication, the student managers running the plan decided that their only option was to adopt the BC provincial drug formulary which, conveniently, excluded Orkambi.

The private insurers who provided the plan to the student union likely adopted a typical “who us?” posturing, all the while scuttling furtively behind their student client, claiming innocence and pointing out that they were only providing the service the client asked for.

Although, I’m sure they lament the loss of the quick and easy $10,000 in “administration fees” from that coverage ending.

But this highlights just one of many important and often overlooked reasons why private insurance is a significant drain our economy and offers Canadians no value-added. They have no incentive to save money by keeping people as healthy as possible and out of the hospital. In fact, with many plans operating on a cost-plus basis, sicker insured people likely mean more money in their pockets at the end of the day.

Act II: The Drug Company

Vertex, for its part, well, is doing what pharmaceutical companies do. They trot out the same old tired rationale, which I’d paraphrase as ‘we sell hope, the cost of development is astronomical, we have to cover the loses from all our failures, there’s such a small market…’

A recent paper entitled “How to Beat Science and Influence People: Policy Makers and Propaganda in Epistemic Networks” shows that propaganda like this can easily lead to “… scenarios [where] we find that while the community of scientists converges on true beliefs about the world … policy makers reach near certainty in false belief.”

The more the drug industry repeats their “narrative” uniformly, the more it picks up traction as repetition breeds a certain level of believability simply out of familiarity. Industry is not about to change its game now.

Vertex DOES have a Special Access Program for Orkambi. If a patient’s lung function falls below 30 percent, they will provide the drug at no cost for short periods on a renewable basis. Laudable, but with a caveat. They will give access only IF—and this is the curious part—the drug had NOT been covered by a payer previously.

I keep circling back to this, wondering why Vertex would have this stipulation. Getting a straight answer from them would be difficult—if not impossible. Drug pricing worldwide, as well as the finances of the drug industry in general are notoriously opaque.

So given the lack of information I can only speculate.

Could it be as simple as their part in this morbid game of policy chicken? Or maybe it has something to do with the Byzantine rules that govern corporate taxation in Canada? Maybe they can write-off the entire price as a “donation,” rather than just writing-off the cost-of-goods (although that too is an unknown), so long as they can show evidence that the list price of the drug has actually been paid elsewhere (thanks a lot private insurance). Of course, if that were the case in Canada, this largess from industry would actually be a form of government funding due to the equivalent loss of tax income. Hmmmm.

Act III: The Canadian, Provincial and Territorial Governments

After negotiating a “reduced” price for Kalydeco, the provinces took a perversely united front on Orkambi refusing to even enter into negotiations with Vertex Pharmaceutical. A drug that, may I remind you, was previously approved, only this time it has an additive to improve absorption. Kind of like next year’s car with better cup holders.

Really provinces? THIS is where the line is drawn?

The provinces kept insisting that Vertex provide better data on which to base an updated decision. But, maybe the decision-makers should have gone back, read the recommendation again and checked some statistics first.

A single statistically weak clinical trial was used in recommending Kalydeco. It showed a mean improvement in lung function of 5 percent in an adult sub-population of 50 participants. The weakness of the study means that that actual percentage could be anywhere between 1.1 and 8.8.

Orkambi used pooled data from two larger trials. These studies concluded that in the total population aged 12 and up, there was a mean improvement in lung function of 2.8 percent. The lower volatility meant the actual percentage was between 1.8 and 3.8 percent.

Dusting off my 25-year-old probability classes, statistically speaking there’s really no difference between any of these numbers.

When assessing Kalydeco, CADTH clearly said that the “minimally clinically important difference” for lung function was UNKNOWN. Yet, the expert for their review suggested that the “magnitude of improvement reported in the adult subgroup [5 percent]…can be considered clinically meaningful.”

So out of thin air, an arbitrary standard is adopted based on the opinion of a single expert using the results of the very same study they are meant to be assessing.

... head spins.

This in turn is based on the best result in a single weak study, which statistically speaking may well be LESS than 5 percent and indistinguishable from the values in the studies of Orkambi.

And that very same arbitrary standard plucked out of the ether was ultimately used as the “objective” criteria that ruled AGAINST Orkambi.

...still spinning.

For the love of Pete, please read your “evidence” and stop looking for someone to point the finger at.

Act IV: The Cystic Fibrosis Patients

Almost EVERYONE in this mess holds some blame.

Everyone—that is—except the Katrinas, Lilias and all the other CF patients in the same boat. They sit in the middle of callous private insurers, intransigent and mis-informed governments and a drug company that actually believes its own hype.

They have been denied the help they clearly need because the MEAN absolute change in lung function in the studies did not meet some arbitrary minimum.

Am I the only person that remembers what a ‘mean’ is? An average…? Anyone…? Anyone…? There will be patients who do worse, patients who do average and patients who do better!

Given that there are almost 2000 identified mutations associated with CF of which 242 are known to actually cause CF, is it any wonder that individual response varies? So why would you be flummoxed by the positive impact that Orkambi had on Katrina’s or Lilia’s life, an impact you can see with your very own eyes?

And these Canadians who have OBJECTIVE proof that Orkambi improves their very health but no longer have access. Well—as my great Aunt Anna from Cape Breton used to say—tough beans sweetie. OK. She didn’t say beans.

Act V: Prologue and a Humble Call to Action

To be clear, the scandalous list price is NOT the fault of Katrina, Lilia or others with CF.

So whose fault is it?

The real underlying cause of this failure is the collective 52 year procrastination over national public drug coverage by the federal, provincial and territorial governments.

Think about this. Had we added drug coverage in 1966, we would not be in this position:

  • We would have strong single-buyer leverage to get the best prices
  • We would have had 52 years to learn the art of drug price negotiation
  • A single-buyer would mean no issues with pricing transparency as it would be the same for everyone in Canada
  • We would not have a private drug insurance sector that is, in reality, a significant drain on the economy as it offers no value-added and siphons away hard-earned dollars into bonuses, net income and grossly inefficient administration
  • We would have had 52 years to work out the kinks in our strategy

And for those who argue that we can’t possibly afford national drug coverage, let me just say this: There is already MORE than enough money in our drug system to cover everyone in Canada for all effective medications. It’s just allocated mind-bogglingly badly.

Case in point, the amount paid out to private insurance last year—about one billion dollars—is enough to cover every single person with cystic fibrosis for Orkambi (although clearly not all of them would benefit from it).

But, rather than getting our act together now (because while the best time for pharmacare was 1966, the second best IS now) on a true national public drug plan, our decision makers use cystic fibrosis patients as pawns in a losing battle against an adversary who—despite everything you may think or are told—holds all the cards.

The most compassionate Canadian thing that our leaders can do is to take a trip to meet a Katrina or Lilia—in person. See what REAL evidence looks like rather than the rationalization that you have convinced yourselves matters more.

Then cover their damn drugs right away and get on with setting up national public drug coverage.