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Is the high price of drugs for rare diseases justified?

Afflicted with a rare blood disorder, Barry Katsof relies on an intravenous medication every two weeks that affords him the active lifestyle of his pre-symptom days almost a decade ago. The only problem is the drug therapy comes at a hefty price tag to taxpayers - $500,000 every year for the rest of his life.

Is that cost justified? Mr. Katsof believes so.

The Montreal man has paroxysmal nocturnal hemoglobinuria (PNH), a potentially life-threatening disease characterized by anemia caused by the destruction of red blood cells. He is being treated with Soliris, the only drug on the market that can control the body from attacking its red blood cells.

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Health experts wonder whether enough is being done to rein in pharmaceutical companies from gouging the public-health system.

Soliris, produced by U.S. pharmaceutical firm Alexion and approved by Health Canada, made headlines this week after Lucas Maciesza, a 26-year-old man in Guelph, Ont., was fighting for his life in hospital because his family could not afford the drug and Ontario refused to cover the costs. The hospital eventually stepped in to pay for the treatment.

The blame was squarely focused on the provincial government, which has repeatedly come under fire for inequitable access to life-saving drugs. What was missing, however, were questions around the company's drug pricing.

"If I was in government and had absolute control, I would tell the drug companies that they had to show me the figures to justify the cost," said Joel Lexchin, a professor of health policy at York University. "They're entitled to a reasonable return, but I certainly wouldn't start paying that kind of money without seeing some reason why it's that expensive."

Alexion argued that its price is reasonable, but did not itemize its costs. The company said it spent $1-billion (U.S.) for research, development and manufacturing, an amount "invested with substantial risk, as most drug development ends in failure."

"Governments and private insurers around the world recognize that, because of the large costs and extensive risks of developing drugs for patients with ultra-rare diseases and the very small numbers of patients suffering from those diseases, reimbursement levels need to be commensurately higher on a per-patient basis," the general manager of Alexion Pharma Canada, John Haslam, wrote in an e-mail.

PNH affects about 90 Canadians. Patients with PNH lack a protective protein around their red blood cells, which leads to a host of complications, including blood clots and kidney failure. Those on Soliris did not need as many transfusions (Mr. Katsof hasn't had one since he started treatment two years ago). But the drug has one severe side effect: an increased risk of meningococcal infections.

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Mr. Katsof, whose treatment is paid for by the Quebec government, hasn't had any adverse effects. "This drug saved my life. It gave me back my life. It gave me back to my family," he said from Montreal.

As president of the Canadian Association of PNH Patients, a non-profit group, he has been fighting for provinces to fund the drug. The group hired a public-relations firm, Cohn and Wolfe, to solicit media outlets last week to publicize the plight of Mr. Maciesza and Ontario's refusal to fund the drug. The association recently received a $15,000 grant from Alexion, Mr. Katsof said.

"I look it at this way: In our society, the way it's structured, the government is mandated to provide health care, the pharma industry is mandated to do the research and development and develop these life-saving medications," Mr. Katsof said. "The drug company has done their job. Now, it's up to the government to step up to the plate to do their job."

Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders, said one way to justify the cost is to have drug plans determine a limit on how much they are willing to pay for medications. The key, she said, is not to hurt the patient.

"The sticker shock can be huge, there's no doubt about it. We want patients to have access. We want to continue to do development. But are there some ways in which we can do a better job of really deciding what is appropriate value for the price?" Ms. Wong-Rieger asked. "There are lots of innovative ways we can do this that won't penalize the patient."

Health experts said governments and pharmaceutical companies can be more innovative in treating patients. For example, patients could initially get access to a particular drug, funded by the government, to determine if it works for them over a fixed period of time. If it proves beneficial, then the government would agree to pay for it long-term.

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