A B.C. woman with a rare, serious skin disease can't understand why the province refuses to cover a one-time treatment that would likely put it into remission — but will pay for much more expensive treatment that only helps relieve her symptoms. 

"I am totally confused — particularly because it would so quickly save them so much money," said Elizabeth Williamson from Lasqueti Island.

"We all know money is tight — especially in health care."

Williamson was diagnosed in 2007 with pemphigus — a rare, painful, disfiguring and potentially life-threatening autoimmune disorder. At her worst point, much of Williamson's body was covered with painful sores.

"When I was covered with a lot of lesions, they were on my face, they were on my scalp. It was very extremely embarrassing because it looks bad. It looks really ugly," she said.

"The pemphigus itself doesn't kill you. What kills you is the lesions — the sores — get infected."

Current drug costly

Williamson's dermatologist initially prescribed a medication that caused severe side effects, but later switched her to CellCept — a $1,000-per-month drug paid for by B.C.'s PharmaCare Plan.

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This photo of an unnamed Pemphigus sufferer shows the lesions when left untreated. (CBC)

"I still am experiencing these lesions," she said. "It's not a cure. It's just dealing with the symptoms."

Dermatologist Gabriele Weichert wrote to PharmaCare, recommending a one-time treatment with Rituximab instead. The drug is approved for treatment of rheumatoid arthritis and other conditions, and Weichert said the drug has also shown much better results in treating pemphigus.

"We have a better therapy that is available for her," said Weichert. "It may take her off all treatment altogether — for life."

The Rituximab would cost the province a total of $2,400 — and Williamson's doctor said it could put  her disease in remission.

"When [these drugs] have been approved for other disease states — their safety issues have been rubber stamped already," said Weichert.

"I'd really like to get into remission and not have to take any medication and get on with my life and live a normal life," Williamson said.

Williamson, 54, estimates B.C. taxpayers have paid $30,000 for her CellCept prescriptions to date. Without any other treatment options, Williamson expects the cost of the drug will continue to mount for the rest of her life.

Recommended drug much cheaper

"I feel bad that I am taking so much of our taxpayer money," said Williamson.

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B.C. Health Minister Mike de Jong said that as a result of CBC News inquires, the province will reconsider Williamson's case. (CBC)

Pacific Blue Cross has agreed to pay 80 per cent of the new cost of the Rituximab, with coverage Williamson has through her employer. However, Pharmacare has refused to pay the remaining 20 per cent.

"All I'm asking for is another $2,400," said Williamson. "Which would then mean that I wouldn't need any more money from them again."

A spokesperson for PharmaCare told CBC News approval was denied because Health Canada has yet to approve Rituximab for treatment of pemphigus. Using it to treat that condition is considered "off-label".

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In order to get federal government approval, the drug marketing company, Hoffman-LaRoche, would have to apply. However, Weichert said that is unlikely because pemphigus is very rare and therefore not worth that investment.

"There are many, many, many rare conditions for which a drug will never get an indication for it — because the numbers of patients with these rare conditions are so small," said Weichert.

"We need to use a lower degree of evidence to support these treatments, because we just don't have that gold standard — randomized control trials — for most [skin] conditions."

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Dermatologist Gabriele Weichert said many patients with rare skin diseases can't get coverage for drugs that could help them. (CBC)

She understands the concern over side effects, but said most drugs come with that risk, including those that are fully approved, like CellCept for pemphigus.

After Go Public brought Williamson's case to the attention of B.C. health minister, Mike de Jong, who said Pharmacare would reconsider.

"We'll have a look. Because it's about ensuring that the balance is there and - from everyone's perspective, including the patient and their family," said de Jong. "It's a really tough situation for the patient, of course."

Many patients fight for coverage

Weichert said Williamson is not alone is her struggle.

"It's frustrating," she said. "I've had patients want to mortgage their house to access some of these therapies."

"It’s a very common occurrence," said Sharon Manson Singer, a former B.C. government deputy minister and a health care policy expert with the Evidence Network.

"I am sure that the letters that have been written to deny [Williamson] have cost more than the drug."

Singer’s group agrees with medical professionals calling for a national pharmacare system. She said it would give patients with rare conditions power in numbers — and track their treatments on a national scale.

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Health policy expert Sharon Manson Singer said a national pharmacare program would be more effective for patients with rare disorders. (CBC)

"We should be a nationalized system — that can compare rare diseases internationally," said Singer. "It’s much less expensive for us to deal with this as a national issue than to deal with it as one-offs."

Williamson said if the government won't pay for her treatment she will have to start saving up to pay for it herself.

"It’s like knocking my head against a wall," said Williamson. "I can’t understand it. I truly cannot understand it."