On the few occasions when Nicole Dempsey has used her accessible parking pass, perfect strangers have openly accused her of abusing the system.
After all, Dempsey looks like a healthy woman.
What those strangers didn't realize is that Dempsey, a 41-year-old mother of two who lives in Cambridge, Ont., sometimes can't make it up the stairs without gasping for air. Or that she had to give up a career she loved as an elementary school teacher because of a life-threatening illness. Her children, ages 6 and 7, don't realize there's anything unusual about the fact their mom often can't take them to the park or play with them because she is too tired and out of breath.
"They're not mad. They don't know any different," Dempsey said. "I find that hard."
Dempsey has pulmonary arterial hypertension, a progressive disease caused by narrowed, constricted arteries in the lungs, which forces the heart to work harder to pump oxygenated blood through the body. It can lead to heart failure and death, and according to the Pulmonary Hypertension Association of Canada, anywhere from 5,000 to 10,000 Canadians have the condition, which affects twice as many men as women.
Dempsey is telling her story as part of an advocacy campaign to secure funding for an expensive new drug called Opsumit, which research has shown works well for many patients with her condition. In December, she attended a news conference at Queen's Park in Toronto urging Ontario Health Minister Eric Hoskins to resume negotiations with Actelion, the drug's manufacturer, to publicly fund the drug.
Although Dempsey doesn't take Opsumit, she said she believes patients like her should have access to anything that might help. "If they can't have access to a drug, this particular drug," she said, "there will be a lot of fatalities. That's the bottom line."
Her stance is shared by medical experts who say this drug may work better than its predecessors. And yet, the push to get funding for Opsumit is also an example of a much larger issue playing out across Canada. As research yields a growing number of drugs to treat rare diseases, drug companies are putting pressure on cash-strapped provinces to fund them, often with public campaigns that utilize non-profit patient advocacy groups and patients.
The Queen's Park news conference, for example, was led by the Pulmonary Hypertension Association of Canada, a charitable organization that has enlisted public relations firm Cohn & Wolfe to get attention to its cause. Much of the organization's funding comes from pharmaceutical companies, including the manufacturer of Opsumit.
When patient advocates and the medical community align with drug makers behind that common purpose, it raises some challenging questions about whose best interests are being represented.
"There is an important role for patient groups, no question," said Steve Morgan, an expert in drug policy at the University of British Columbia. "There is also, unfortunately, a conflict of interest that a lot of patient groups find themselves in as they accept more and more money from pharmaceutical manufacturers."
Opsumit, manufactured by Actelion Pharmaceuticals, a Swiss company, was approved by Health Canada in November, 2013, after a large study in the New England Journal of Medicine showed it worked well for many patients.
Doctors say they are excited about the promise of this new drug. Patients with this disease also respond differently to various medications, so having as many options on the table as possible is important, said Dr. Sanjay Mehta, chair of the board of directors of the Pulmonary Hypertension Association of Canada and director of the Southwestern Ontario Pulmonary Hypertension Program.
"If you don't treat them well, they're going to get worse and they're going to die," he said. "All we want is the government to sit down and negotiate." (Hoskins was not available for an interview. Actelion did not respond to an interview request.)
But cost is an issue. One tablet of Opsumit costs $128, according to a July, 2015, report from the Canadian Agency for Drugs and Technologies in Health's Common Drug Review.
But Actelion also sells another drug, Tracleer, that for years has been a mainstay drug for many patients with pulmonary hypertension. The patent for Tracleer expired in June, 2012, about a year before Opsumit came on the market. Now, five generic companies are producing lower cost versions of Tracleer at about $45 a tablet, according to the Common Drug Review report.
The difference between the price for generic Tracleer, known as bosentan, and brand name Opsumit is nearly $30,500 a year, the report found.
The timing of the public campaign to secure government funding for Opsumit, which has patents that extend to the late 2020s, so soon after the expiry of Tracleer's patent is "not a coincidence," said Dr. Stephen Archer, head of the department of medicine at Queen's University in Kingston, and pulmonary hypertension expert.
He doesn't dispute the fact that Opsumit may be a good option for some patients, particularly those who don't do well on some of the other existing medications on the market.
But Archer is concerned the benefits of Opsumit are greatly exaggerated. If more doctors start prescribing it, patients won't necessarily fare much better, but the price they – and, potentially, the public-health system – pay will grow exponentially.
"Someone has to actually be willing to speak about this," Archer said.
One of the biggest issues that few seem willing to acknowledge, he said, is the way evidence was portrayed in the New England Journal of Medicine study that helped get the drug approved in Canada and around the world. The study concludes that Opsumit improves morbidity and mortality of patients, which sounds like a major achievement.
But the study data don't correspond to that rosy conclusion, Archer said. Study participants either took a placebo, a low dose of Opsumit or a high dose of the drug. When the trial ended, the death rate from any cause was highest among those who took the low dose Opsumit, and it was virtually identical between those on the high dose and those who took a placebo.
Despite this, the study authors conclude that Opsumit "significantly reduced morbidity and mortality among patients with pulmonary arterial hypertension."
Archer said he is concerned that many doctors will mistakenly believe that Opsumit does offer patients a significantly lower risk of death.
"What it does not do is prevent you from dying and yet it's being sold as a drug that reduces morbidity and mortality," Archer said.
A more reliable indicator, according to Archer, is the six-minute walk test, which measures how far patients can walk during a six-minute time period.
Doctors often rely on that test to assess the degree to which drugs can improve the quality of life for pulmonary hypertension patients. According to the Opsumit website, study patients who took the drug walked, on average, 22 metres farther than those who weren't on the drug.
Archer questions whether this relatively short distance is enough to justify putting many patients on this expensive medication when generic versions of Bosentan, which is comparable to Opsumit, are available at a fraction of the cost.
He is concerned that many doctors will unquestioningly gravitate toward the new expensive Opsumit because of the misleading statement that it reduces death rates.
"If it were me, I'd probably prescribe another drug," Archer said.
To UBC's Morgan this situation is just one example of a much larger issue facing the country: the ever-growing availability of expensive drugs to treat rare diseases, unsustainable costs to the health-care system and drug companies that, in some cases, are helping to exert public pressure on governments to fund more and more drugs.
It's becoming increasingly common to see headlines chronicling the campaigns to get more funding for expensive drugs, such as Soliris, which is used to treat the rare disease atypical hemolytic uremic syndrome and costs $700,000 a year, or Kalydeco, a cystic fibrosis drug, which costs about $300,000 a year.
At an estimated cost of $45,000 a year, Opsumit seems like a drop in the bucket. But it's one of dozens – possibly hundreds – of new drugs that will have doctors, patients and drug companies clamouring for provincial coverage in the coming years.
One fact the Pulmonary Hypertension Association of Canada didn't mention during its Queen's Park news conference in December is that 70 per cent of its funding comes from pharmaceutical companies – including Actelion.
Mehta said that such funding is necessary to conduct campaigns and help patients across the country. The potential conflict of interest is mitigated by the fact the organization is upfront about its relationships with drug companies, he said.
"Our simple be-all end-all motivation is a better life for patients affected by [the disease]," he said.
But Morgan said the relationship with drug makers does put patient groups in a tricky position. When they urge governments to extend funding to new, expensive drugs, shouldn't they also publicly urge drug companies to lower the prices of their medications?
That message is not part of the Pulmonary Hypertension Association of Canada's campaign for Opsumit funding.
In fact, Mehta argued in an interview that while Opsumit may seem expensive, it is "no different" than pricey chemotherapy drugs that have public funding and that, relatively speaking, there aren't very many pulmonary hypertension patients in Canada to even take the drug.
"This is not just a marketing thing," he said.
Back in Cambridge, Dempsey said she understands the difficult position policy-makers face when it comes to the growing pressure to fund more expensive drugs for a growing roster of niche diseases. But that won't derail her campaign for Opsumit.
"I'm sure it's challenging," she said. "We're not taking away from all these other diseases, but right now, we're advocating for our disease. And this is what we need right now."