A compelling drama played out in B.C. over the past few months features several people suffering from cystic fibrosis calling on the new NDP government to cover a new and very expensive drug.
Some CF sufferers, including a woman in Victoria and one in Vernon, consider Orkambi to be an immense benefit to them. And we may expect that if a drug has a price tag of $250,000 per patient per year, it must work really well, right?
Well, not so fast. The media narrative that has unfolded characterizes the drug as a “lifesaver” and slams the unfairness of a government that refuses to pay for it.
Unfortunately, both those views are flawed, and while we all want fairness in our public health-care system, using compelling patient stories to determine which health services get funded is not a reliable approach to providing the best care.
Let’s look at some of the facts of the case. Undeniably, cystic fibrosis is a terrible disease in which, due to a defective gene, mucus builds up in the lungs, making breathing difficult. As there is no cure, dealing with the disease typically focuses on managing symptoms and in the most extreme cases, patients face a lung transplant.
While everyone responds differently to a drug, the evidence suggests this new drug is marginal at best. The two main studies in more than 2,000 patients show it might improve lung function by about three per cent, a benefit considered by experts to be of “uncertain clinical significance.”
The trials of Orkambi have been extensively reviewed by independent scientific committees in Canada and around the world. Those reviews have found it has a minimal impact on quality of life and shows no impact on survival.
Further, compared to patients given placebo, those who took the drug had higher rates of breathing problems, higher rates of upper-respiratory-tract infections and had more serious adverse events related to abnormal liver function.
B.C. is not alone in not providing coverage for this drug. None of the other provincial drug plans in Canada and most insurers in the U.S. don’t cover it, either. Probably, drug plans are weighing the negligible effects shown in trials against the significant impact on their budgets that would come with paying for it. It seems most are standing up to the manufacturer, Vertex Pharmaceuticals, and saying they will refuse to be held hostage to a company charging outrageous prices for a drug.
You will hear people say that Vertex has to charge such immense prices because it needs to recoup its research investments in Orkambi, but that’s bogus, too. A report from the Boston Globe a few years ago found that Vertex’s market value is about $30 billion, and 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.” That explains part of the extortionate price.
We have seen this scenario played out scores of times. In one case, breast-cancer patients, in front-page stories, argued for coverage of a new $40,000-per-year treatment. They won their case, only for evidence to show a few years later the patients who took the drug did not live longer and had higher rates of heart disease.
Right now in B.C., patients are paying out of their pockets for a whole range of newer diabetes drugs (not covered by public money) that we know do virtually nothing to alter the course of that disease.
And let’s not forget the dozens of drug disasters such as Vioxx or Prepulsid, where regulators ignored safety signals only to later learn the drugs increased your chances of dying.
I could go on, but the key message here is that the future portends a steady stream of new and expensive drugs coming to market for many diseases. Independent drug research that proves the benefits and harms of the drug will be crucial to guide decision-making on those therapies.
The good news is that with better knowledge and research, Canadians with cystic fibrosis live nearly 20 years longer than they did two decades ago. We should do what we can to help improve the lives of people with this dreaded disease, but we should not allow ourselves to be held hostage by pharmaceutical companies.
Alan Cassels researches and writes about pharmaceuticals. His latest book is The Cochrane Collaboration: Medicine’s Best Kept Secret.
