It took five years for Ronald Laberge to be diagnosed with a life-threatening autoimmune disease that has left him riddled with infection, and it came none too soon.
In December 2018, Laberge was emaciated, in pain and close to death.
One of the few Canadian doctors familiar with his rare condition — immunodeficiency with anti-interferon-gamma autoantibodies — recommended rituximab, a cancer and arthritis drug sold under the brand name Rituxan.
Doctors at the Hotel Dieu Hospital in Kingston, Ont., administered one course of the drug over four weeks, and in April Laberge was discharged.
He was able to walk on his own two feet for the first time in almost six months.
But he needs more courses of treatment, which the hospital pharmacy estimates would cost about $15,000 every six months.
Only one problem: as an outpatient, the hospital no longer covers his medicine, and because the drug is not approved to treat Laberge's rare illness specifically, the province won't pay for it, either.
"You wait five years for the diagnosis, then they tell you, 'We're not going to fund you,'" Laberge said in an interview at his home near Brockville, Ont.
Denied exceptional coverage
Rituximab is approved for cancer and arthritis treatment in Ontario but not for Laberge's condition.
In a statement to CBC News, the Ministry of Health explained it was reviewed by Health Canada and other experts for medical, economic and "societal/patient values" and funding it wasn't recommended.
Getting approval to use rituximab in Laberge's situation requires applying through Ontario's Exceptional Access Program.
Dr. Anne Ellis applied to have Laberge covered but he was denied. She appealed, and he was denied again.
Ellis said the review process for exceptional cases is hit and miss — often the requesting doctor knows more about the diagnosis and treatment than the people reviewing the applications.
"The ministry sends out the request to five different reviewers for their own expert opinion, but if none of us have seen the condition because it is so rare, it could be hard to do an objective evaluation," Ellis said.
Manufacturer funds treatment for 1 year
Ellis went to the manufacturer of rituximab to ask for funding on humanitarian grounds, and it agreed to pay for one year of treatment.
"I don't know whether a one-year course will be sufficient to produce long-term benefits," she said.
"I'm hopeful and optimistic, but we'll have to see how things go."
Advocates want that uncertainty to end.
Last week, former Ontario health minister Dr. Eric Hoskins unveiled an advisory council's recommendation to establish a universal, single-payer public pharmacare system.
The council's report called for a national strategy for expensive drugs for rare diseases, noting the difficulty in conducting clinical trials for diseases that affect so few people.
The report recommended creating a national expert panel to review cases and monitor treatments.
The national strategy would establish "a distinct pathway" for "innovative, life-changing drugs for rare diseases," and "support more consistent access" to them.
If accepted, it would only come into effect in 2022 — too late to help the Laberge family after their year of free treatment runs out.
'Seamless system' needed
"I think it would be really helpful to have some sort of centralized process to help support patients who have such rare conditions," Ellis said.
Durhane Wong-Rieger, CEO of the Canadian Organization for Rare Disorders (CORD), called the idea a good first step.
"We need a seamless system," she said.
Her group was consulted for the advisory council's pharmacare report, and she said the acknowledgement that drugs available in hospital should not be discontinued when patients are discharged is "very important."
She also called Laberge's case "very typical."
When patients are diagnosed with rare illnesses, Wong-Rieger said it's not uncommon to have a limited treatment option involving a drug approved for more common illnesses, but not the rare ones.
Call for 'more consistent access'
Laberge's wife Arlene Laberge, who has been called to hospital bedsides to say goodbye to her husband multiple times over the last five years, said the whole ordeal has been unnerving.
"We've come this far, and now they're going to say to us, 'No, you can't have it,' after everything we've persevered through," she said.
"We've learned you can't plan too far ahead, and you have to really be grateful for every day you get."
Ron Laberge's second course of treatment, courtesy of the rituximab manufacturer, will go ahead in July.
For now, they're trying not to worry about who will pay for next year's treatment.