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A cystic fibrosis sufferer with some of the standard medications he must take to control the disease. (Deborah Baic/The Globe and Mail)
A cystic fibrosis sufferer with some of the standard medications he must take to control the disease. (Deborah Baic/The Globe and Mail)

ANDRÉ PICARD

Drug-funding sob stories make for good reading, but we need hard evidence to set public policy Add to ...

“Cystic fibrosis girl denied life-saving drug.”

“Girl, 12, asks Ontario to cover life-saving cystic fibrosis drug.”

Those two headlines ran from opposite ends of the world: The former was the story of Sinead Brown of Dunedin, New Zealand; the latter about Madi Vanstone of Beeton, Ont.

Yet both recount the pleas of children suffering from cystic fibrosis for government to fund the drug ivacaftor (brand name Kalydeco), costing more than $300,000 a year.

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Madi said the drug had dramatically improved her health and she could no longer imagine life without it; Sinead’s parents said refusal to cover the drug’s cost would be a “death sentence” for their daughter.

It is now a well-worn meme to push for drug funding: Trot out a young girl (they tend to be more eloquent than boys) to tell of its miraculous properties, and parents to decry how politicians are heartless bureaucrats who care only about saving a few bucks. Throw in an opposition politician to say that such a travesty would never happen under their watch, and a consumer group to express shame that we live in a country that puts a price tag on kids’ lives.

It makes for good reading, but is it any way to fashion public policy?

Try instead stepping back and dispassionately examining the Kalydeco story without the sob-story backdrop.

Spurred (and partly funded) by the Cystic Fibrosis Foundation, the manufacturer, Vertex Pharmaceuticals, developed a breakthrough drug to treat some patients with CF. (It’s an inspirational story, brilliantly recounted in Discover magazine). In short, ivacaftor is the first drug to treat the underlying causes of the disease rather than the symptoms; it improves lung function by 10 percentage points – for example, boosting lung function to 30 per cent from 27 per cent. Practically, it means patients with CF spend less time in hospital and reduces their need for intravenous antibiotics. Rhetoric aside, the drug is by no means a cure, or a lifesaver, but it’s progress.

Ivacaftor came to market in 2012; it was fast-tracked in the United States as a breakthrough drug and approved quickly in Canada. Then, as part of the usual process, a review was conducted to determine if it should be funded. The Canadian Drug Expert Committee ruled that, yes, ivacaftor should be covered by public drug plans, but only if a significant reduction in price was negotiated.

Since then, the provinces, led by Alberta, have been trying to negotiate a price. In the past, drug companies worked to get drug approval in one province, then to shame the others. In this case, the provinces, to their credit, have a united front. Still, the company, Vertex, is holding firm at $309,000 – even though it has negotiated lower prices in Britain and Australia (the exact prices are secret). A total of 118 Canadians with cystic fibrosis – out of 3,000 in total – could benefit from this drug. That’s because Kalydeco is only effective for those with a specific mutation, G551D.

The total price tag, $36.5-million per year, would not break the bank, but it’s not insignificant. The bickering over the price is unfortunate but necessary.

Funders – particularly those entrusted with public dollars – need to push back on drug prices. Yes, research and development is expensive and manufacturers deserve a fair return on investment. But one would be hard-pressed to see how the price of Kalydeco is justified, especially considering that development was funded with charitable donations and government grants, not just private dollars.

As heartless as it may sound, there also need to be some lines drawn in the sand on the funding of drugs, particularly so-called orphan drugs like this one; the pricing of drugs for rare disorders is artificial and arbitrary, determined not so much by economics as politics.

Clearly, Vertex does not think the market for Kalydeco is 118 people in Canada and less than 3,000 worldwide. Ideally it would like all 70,000 people around the world who have cystic fibrosis to be customers.

In fact it is already testing the use of Kalydeco, in conjunction with a new drug, VX-809 (brand name Lumacaftor), for the treatment of CF patients with the delta F508 mutation. That’s half of the total patient population.

If you have a combo of two expensive drugs for a fairly large population, suddenly you’re approaching $1-billion or more in Canada alone.

So far, we have a big bill for treatment of a small sub-population. For what? A modest improvement in lung function in the short term, with no solid evidence of long-term benefit or improvements in mortality. Will cost-effectiveness improve as use of Kalydeco expands? That’s not certain.

And the key question in public policy is always: What else could be done with the money that would provide more bang for the buck?

To fund or not fund drugs is not an easy discussion to have. But it is a necessary one. As compelling as the stories of suffering children may be, we have to make decisions based on evidence. We also owe it to ourselves to negotiate firmly with drug-makers.

We cannot continue to fall prey to emotional blackmail, no matter how much the headlines sting.

Follow me on Twitter: @picardonhealth

Editor's note: The drug Kalydeco improves lung function by 10 percentage points in cystic fibrosis patients with a specific genetic mutation, not by 10 per cent as stated in an earlier version of this story.

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