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Comment: Politics, price, process: Why Orkambi is worth it

Re: “ Sky-high prices don’t mean a drug is effective ,” comment, Nov. 23.

Re: “Sky-high prices don’t mean a drug is effective,” comment, Nov. 23.

Of all the scientists I have ever met who work in the pharmaceutical industry, most are there for exactly the right reasons — they want to make a positive difference in the world.

Unfortunately, the corporate behaviour of some pharmaceutical companies has created an air, sometimes merited, of an industry out of control.

Last week, Alan Cassels painted a cynical portrait of one such situation that misrepresented the facts. Only someone not intimately familiar with cystic fibrosis could describe it as he did — as making breathing difficult.

Cystic fibrosis kills people. Half of those who died from CF in 2015 were under the age of 30. Respiratory failure kills more than 90 per cent of patients. Lung transplants are the last hope of end-stage disease. That’s more than difficulty breathing.

He goes on to question the drug’s effectiveness, while ignoring the fact 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. Cassels acknowledges that every individual responds differently to a drug, then promptly forgets that and cites only high-level statistics to buttress his contention that the drug isn’t effective.

However, the Canadian Agency for Drugs and Technologies in Health’s own data states clearly that 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. Cassels’ and the government’s contention appears to be that if everyone can’t benefit from this drug, then no one should get it.

What is also 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 exacerbations, 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. Compare that with your mortgage interest. Would a 42 per cent drop in the interest rate make a difference? And that’s just where you live — for people with CF, it’s their life.

But most of all, I compare Orkambi with the drug that preceded it — Kalydeco. Both Kalydeco and Orkambi treat the basic defect in cystic fibrosis, but Kalydeco is better at it. But because Kalydeco only treats certain mutations (there are more than 2,000 mutations in CF), it is only indicated for about five per cent of the CF population, or about 210 individuals in Canada, and about 160 will benefit.

Orkambi, on the other hand, treats the most common mutation and is indicated for 50 per cent of the CF population, or 2,091 individuals, and one-quarter of those, or 520 individuals, will benefit. Which drug has the bigger positive impact on the CF community and a bigger clinical impact on the health-care system? The drug that provides the same clinical benefit in the same disease to 160 people, or to 520 people?

And yet Kalydeco is paid for by the same provinces that are refusing to cover Orkambi.

The problem isn’t that Orkambi doesn’t work or that its list price is outrageously expensive. 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. And patients continue to suffer.

Three weeks ago, the company put forward an unsolicited offer to the provinces with what it says is a significant price reduction. It’s time to put patients first and to negotiate so that Orkambi can get to the people who do not have the one thing that this bickering is taking: time.

Dr. John Wallenburg is chief scientific officer of Cystic Fibrosis Canada.