'I was ready to die': B.C.'s uneven coverage for rare disease drugs leaves patients in limbo

When University of Victoria student Lilia Zaharieva's cystic fibrosis escalated in her late twenties, she lost half her lung function, making everyday tasks like walking between classes next to impossible.

Her lung specialist gave her two to five years to live.

"I was ready to transition out of this world, thinking about how to wrap up loose ends in my life,"  Zaharieva, 30, said.

But her grim prognosis changed in Sept. 2016. Zaharieva received coverage for a drug called Orkambi through her UVic student health plan. The drug, which costs $250,000 per year, treats the underlying cause of cystic fibrosis, rather than suppressing its symptoms.

It changed her life. After starting treatment, Zaharieva experienced what to her felt like a minor miracle: "I took a deep breath in and it was nearly effortless."

But in Sept. 2017, Zaharieva learned that Orkambi would no longer be covered under the student health plan. The UVic Student Society, which oversees the students' health plan, voted unanimously to buy her one more month's supply of the drug.

Later, the company that manufactures the drug gave her a 90-day free supply on compassionate grounds.

2 high profile cases

There have now been two high-profile cases involving B.C. university students with rare diseases, whose lives depend on expensive medications approved by Health Canada.

Last week, Shantee Anaquod, a 23-year-old UBC student, became the first person in B.C. to be approved for coverage for Soliris, a $750,000 drug used to treat atypical hemolytic uremic syndrome (aHUS).

Anaquod was diagnosed with the rare disease earlier this fall. As recently as a week ago, her life was on the line.

She's been taking the life-saving medication for six days.

Previously, neither Soliris nor Orkambi were covered in B.C. because both were rejected by a committee of experts that makes recommendations about what drugs should be covered by the province.

Soliris, is now covered on a case-by-case basis in B.C. But Orkambi, which Zaharieva used to treat her cystic fibrosis, is not.

The cases of Zaharieva and Anaquod highlight the complex and uneven way that decisions are made around the coverage of rare disease drugs in B.C. The fact that Anaquod has coverage for the drug she needs, while Zaharieva doesn't further underscores that inequity. 

According to Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders, which advocates for patients living with rare diseases, the system, "simply doesn't make sense." 

Once Health Canada approves a drug for use in Canada, individual provinces decide if they will cover the drug. B.C. relies on the recommendations of two committees: the national Common Drug Review and B.C.'s Drug Benefit Council.

Both those committees chose not to recommend Orkambi and Solaris for provincial coverage.

Drugs that treat rare diseases can be referred to yet another committee for review. In B.C., it's called the Expensive Drugs for Rare Diseases Committee.

'I can't understand the inconsistency'

When Anaquod was first diagnosed with aHUS, she was told that Soliris could not be covered by the province because it had been rejected by the Common Drug Review. 

But days later, Health Minister Adrian Dix announced that he would override that decision, and make Soliris covered on a case-by-case basis.

In Victoria, Zaharieva was happy to hear that another gravely ill woman would get the medication she needed. But her relief was bittersweet because her own case is not resolved.

"I was so happy to hear she'd gotten that coverage because I certainly know the desperation and hopelessness," said Zaharieva.

But she said she couldn't understand the inconsistency. She too was told by Dix that her medication could not be covered because it was not recommended by the Common Drug Review.

"Adrian Dix said to me on the phone, 'Lilia, that's not how we do things here in B.C.'"

The primary difference between the two cases is that while Solaris previously wasn't covered in B.C. even in exceptional cases, it was approved in other provinces on a case-by-case basis. 

Orkambi, on the other hand, isn't covered — even on a case-by-case basis — in any other Canadian province.

Wong-Rieger said the Common Drug Review's framework for making recommendations on whether a province covers a drug was not designed with rare-disease drugs in mind. That's because typically these drugs are extremely expensive and affect few people.

So the benefits are not seen to outweigh the cost — even when, for a patient like Zaharieva, the drug could save her life.

'If you have a rare disease, leave'

Wong-Rieger also has complaints about B.C.'s Expensive Drugs for Rare Diseases Committee. She said little is known about this group, which makes recommendations to the province about when drugs should be made available in exceptional cases. 

"No one knows what the terms of reference for the community are, nobody really knows who is on the committee, and nobody knows how to find it — even the committee members themselves have said that they are not happy with the fact that there's so little known about it," she said.

"We say: 'If you have cancer, B.C. is a great place to be but if you have a rare disease, leave.'"

She said more than 60 per cent of the rare disease drugs that are approved in Canada are not covered in B.C.​

Meanwhile, Zaharieva said she will fight for access to Orkambi "until my last breath."

"I was ready to die before. And now I know how much my life has changed and how many other lives could change."