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Bonnie Cameron, photographed with daughter Alica Mehlenbacher, 12, last week at Toronto General Hospital, has incurable PAH. (Charla Jones/The Globe and Mail)
Bonnie Cameron, photographed with daughter Alica Mehlenbacher, 12, last week at Toronto General Hospital, has incurable PAH. (Charla Jones/The Globe and Mail)

MDs press Ontario to fund rare-disease therapy Add to ...

The specialist physicians who treat patients suffering from a deadly heart and lung disease are pleading with provincial officials to allow them to continue "pushing the envelope" with under-studied treatments that can drastically help their sickest patients.

Some patients diagnosed with fatal pulmonary arterial hypertension (PAH) see their life expectancies more than double - from three years to seven or 10 in some cases - when doctors treat them with drug combinations.

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While the often expensive drugs are all approved for use in Canada, the effects of combining them have not been well studied. Because of that, Ontario recently decided to cut off support for PAH patients who require more than one drug - even in cases where patients were not asking for coverage of more than one medication.

The decision remains illogical to most patients and their doctors, who were not given a detailed explanation. The province responded to queries about the criteria used to make the funding decision by repeatedly citing "a lack of evidence with respect to combination therapy."

But the public has a right to know the real details, said Arthur Schafer, the director of the Centre for Professional and Applied Ethics at the University of Manitoba and an expert in biomedical ethics.

"The standards or criteria should be publicly known. Transparency is important," he said. "People have to understand the basis on which their requests are refused - the scientific basis, the moral basis - they should be entitled to look at the evidence and they should be able to appeal."

Doctors, who can appeal funding decisions by writing letters to Helen Stevenson, the provincial deputy minister in charge of Ontario's drug funding programs, have been lobbying hard to persuade her office to soften its position in special and severe cases. Those include patients who lost secondary medication due to the crackdown and have suffered severe declines in health.

A Health Ministry subcommittee has been struck to study the issue, but the process has been slow and for some patients, the clock is literally running out.

"We need to do something now for these people who are dying waiting for combination drug therapy," said Sanjay Mehta, a respirologist who treats patients with PAH at the London Health Sciences Centre in London, Ont. "We're asking them to consider, very simply, on a case-by-case basis, if a patient is failing the first drug therapy to add and pay for a second drug for three to six months."

Doctors, patients and their advocates are concerned the committee review will be stalled by the same dearth of scientific studies on combination therapy that health officials have cited in past decisions not to fund treatment, which can hover around $100,000 annually per patient.

"In rare diseases it is difficult to find studies that meet the criteria that you would utilize [to determine funding]in more widespread diseases," said Greg Engel, a former pharmaceutical executive specializing in PAH therapy who is now a freelance consultant. Mr. Engel said funding deliberations are too often hung up on the lack of large studies, which is "not just a situation unique to PAH. Indirectly, it's also important for all rare diseases."

Earlier this year, a review of the existing combination therapy studies was published by Canadian Agency for Drugs and Technologies in Health, a national body mandated to advise Canada's health-care decision makers on the efficacy and effectiveness of drugs. While it acknowledged the lack of volumes of research on the subject, the paper noted that international guidelines for PAH treatment support combination therapy in cases where a single drug is deemed ineffective.

"Decisions to fund combination therapy may be best considered on a case-by-case basis until further evidence is available," it states.

That is exactly what doctors in Ontario are hoping officials will come around to.

"The data for combination therapy is not overwhelming," said Lisa Mielniczuk, a PAH specialist at the University of Ottawa Heart Institute, adding: "The reality is that we are not going to abuse these drugs. We really do save these therapies for the people who we think need the benefit."

Two years ago, Dr. Mielniczuk said, it was possible to get approval in Ontario for patients who required combination therapy. Now it's "impossible" to get patients "the same therapy that may be offered say, in Quebec, or south of the border."

John Granton, a Toronto-based doctor who treats PAH and is widely viewed as one of the continent's top experts, is the lead doctor liaising with the province on the issue. He said he remains optimistic.

"My anticipated outcome is that patients in the province of Ontario will have the same access to medication that is provided to patients in other provinces," he said.

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