Making sense of the Orkambi debate in British Columbia

Making sense of the Orkambi debate in British Columbia[1]

By PATRICK S. WOLFE

We are served by innumerable organizations that overlap and interlock, but which don’t always co-operate—sometimes far from it. The ongoing and bleakly fascinating Orkambi debate, which has occurred in a variety of jurisdictions and is now playing out in British Columbia, is a case of such friction trying to sort itself out.

A new, super-expensive drug that became available in 2016, Orkambi “treats the most common [cystic fibrosis] mutation and is indicated for 50 per cent of the CF population, or 2091 individuals,” according to Dr. John Wallenburg, chief scientific officer, Cystic Fibrosis Canada. Manufactured by Vertex Pharmaceuticals, it’s available for $250,000.00 per patient per year.

Based on scientific reviews of the drug’s “efficacy, therapeutic value and cost,” the BC government decided in March 2017 not to list Orkambi as a PharmaCare benefit. In response, private insurance plans stopped covering Orkambi, leaving two BC women, Melissa Verleg, 34, a wife and mother of two from Vernon, and Lilia Zaharieva, 30, a UVic student, in what CF Canada calls “a dire situation.” CF Canada, which regards Orkambi as a “life-changing” medication, launched a campaign to support Verleg and Zaharieva and to pressure government to change its stance.

The Victoria Times Colonist’s Katie DeRosa has characterized the situation in BC as “a high-stakes poker game, with a giant pharmaceutical company on one side and big government on the other.”

Dr. Wallenburg says “The problem is that the company accuses the government of denying patients access to a life-saving drug, and the government accuses the company of trying to skirt due process. The company over-priced the drug at the outset and the government has been using a flawed review process as an excuse to reject an effective drug.”

Orkambi’s astronomical price has been a focal point. Alan Cassels, the well-known pharmaceutical researcher, quotes a Boston Globe report from a few years ago that says Orkambi is “slated to help 12 Vertex senior executives secure over $53 million in one-time bonuses if Vertex is profitable over the next four quarters.” Cassels says the money for those bonuses helps explain Orkambi’s “extortionate price.”

On the other hand, Vertex recently offered to reduce Orkambi’s price by a “significant” but unspecified amount. It also provided Zaharieva with a free 12-week supply, which will run out by Christmas [2017]. Readers can be forgiven if they’re tempted to see these steps as strategic moves in the aforementioned poker game. In this cynical scenario people like Zaharieva are little more than pawns, although they are also the reason why drugs like Orkambi are developed in the first place.

Things are complex on the government side, too. While it argues that “all provinces and territories across Canada have decided not to cover Orkambi in [their] drug plans at this time,” Dr. Wallenburg points out “that Health Canada, and pretty much every other regulatory body in the world, has approved the drug for sale because the clinical benefits outweigh any risks.” If BC breaks rank with the other provinces and territories, it would weaken the position of all of them. BC Health Minister Adrian Dix has stated: “Using public funds to pay for drugs that have been rejected for coverage by the scientific process and pay whatever price the manufacturer wishes to set… would undermine any public prescription drug plan.”

Important as price is, it appears the primary sticking point is the review process. Dix has stressed that pharmaceutical companies “must demonstrate that their [drugs result] in clinically meaningful improvements in outcomes. There is no alternative for Vertex or any other drug company but to go through the Common Drug Review process.”

Summarizing the results of the independent reviews of Orkambi, Times Colonist columnist Lawrie McFarlane writes: “Orkambi increased lung function, on average, by only two to three per cent.  Anything less than a five per cent improvement is considered clinically insignificant….On the other hand, the clinical trials of Orkambi did show that a small minority of patients gained a somewhat greater measure of benefit, at least for a time.” Cassels contends Orkambi “has a minimal impact on quality of life and shows no impact on survival.”

Dr. Wallenburg explains the review results differently. He says, “one in four patients sees a clinically significant five per cent improvement in FEV1, the most commonly used clinical measure of a CF patient’s health, and one in eight patients are ‘super-responders,’ seeing a dramatic 10 per cent or more increase.” He goes on to say:

“What is overlooked by critics of this drug is the manner in which Orkambi works. It corrects enough of the faulty protein to change the course of the disease. Individuals don’t simply see improvements in one symptom, they feel better because any number of symptoms are improved all at once.

“Depending on the individual, Orkambi leads to significant improvements in lung function, improved body mass index, a reduction in pulmonary exacerbation, increased energy, better nutrition and overall health. It can rescue individuals from the lung-transplant list, and in contrast to Cassels’ statements, can extend life.

“A 2017 study demonstrated that Orkambi can reduce that rate of progression by 42 per cent.”

Zaharieva appears to be one of those to have benefitted significantly from Orkambi. It enabled her “to clear a lung infection that was very serious and resistant to treatment” and to gain new stability in her life. She has said: “for the first time in my life I can get through the day with energy, a smile, and fuller breath.”

Minister Dix hasn’t agreed that the review process is flawed, but his ministry did urge Vertex “to pursue resubmission.” In October, the ministry also “raised and offered support to implementing changes to the resubmission process. It is anticipated that these changes… will be implemented shortly.”

Although it is far from clear, does this suggest that a previously closed door might now be slightly ajar? Might there be a reasonable way to acknowledge that Orkambi has therapeutic value in some cases and to reinstate coverage for it on that basis? If this is possible, Vertex and the pan-Canadian Pharmaceutical Alliance would still have to negotiate an acceptable price.

So, given the pace of developments within the topic organizations that exist to serve our collective interests, nothing is going to happen quickly. Meanwhile, people like Zaharieva twist in the wind. However, as Vertex professes “a deep-rooted passion for making the lives of patients better,” hopefully it will provide Zaharieva and others like her with additional free supplies of Orkambi in recognition of the current stalemate.

An author and historian, Patrick Wolfe lives in Victoria, British Columbia, Canada.

  1. This article was originally published on my LinkedIn page in December 2017. It updated my views on this subject, which were first expressed in a commentary, “Super-costly drugs make for tough moral choices,” that appeared in the Victoria Times Colonist on October 5, 2017. That commentary put the onus for resolution and cost on the British Columbia Ministry of Health, a conclusion that I soon after came to regard as wrong. My initial view was a case of passion outpacing judgment. Per “UVic student, spurned by B.C., given drug free by U.S. maker” (Times Colonist, Saturday, March 10, 2018, p. A1), Vertex Pharmaceuticals has decided, on compassionate grounds, to continue to provide Orkambi to Lilia Zaharieva. Melissa Verleg, however, has “lost her coverage for Orkambi.” The latest development is Zaharieva and Verleg’s decision to file “a class-action lawsuit against the B.C. government, the federal government and two drug-approval bodies for failing to provide coverage for Orkambi under public drug plans.” See “UVic student takes lead in $60-million class-action suit over costly drug,” Times Colonist, July 25, 2018, p. A3. This article also notes that Verleg, who has been without Orkambi since February, has “said her lung capacity has declined dramatically.”
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