Financial Coverage for Rare Disease Treatments Inconsistent in British Columbia

The Canadian Agency for Drugs and Technologies in Health (CADTH) organization’s Common Drug Review (CDR) protocol often leaves rare disease patients in British Columbia without governmental payment coverage for their expensive treatments.

The CDR process analyzes clinical, economic and patient evidence on a drug to determine if the treatment should be eligible for public reimbursement. Once the CDR approves a drug, then the province’s Drug Benefit Council offers a recommendation to the Ministry of Health. Rare disease treatments go through an additional committee in called the Expensive Drugs for Rare Diseases Committee.

Many rare disease therapies are rejected by the CDR because they are more expensive than common disease drugs and affect a very small population, making it economically inefficient to cover the costs.

An example of this came to the attention of the public recently when Natalie Essex, spinal muscular atrophy (SMA) Type 2 patient, began to gain strength in her limbs after participanting in a clinical trial of the drug Spinraza. When the trial ends in 5 years, however, she will not be eligible for financial coverage since she is not diagnosed with the most severe form of the disease, a decision recommended by CADTH.

"[The CDR] also really disregards the fact that this is the only therapy available for a patient population that has a very debilitating condition, and without any other kinds of treatment available," said Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders.

Lilia Zaharieva, a University of Victoria student with cystic fibrosis, was told that her treatment, Orkambi, which coses $250,000 per year, would not be covered, while a University of British Columbia student named Shantee Anaquod was given coverage for Soliris, a $750,000 treatment for atypical hemolytic uremic syndrome (aHUS), a drug that is now covered on a case-by-case basis in the province.

The inconsistency among rare disease drug approvals is apparent, and Wong-Rieger advocates for all patients to receive financial support in treating their illnesses. She also notes that the British Columbia Expensive Drugs for Rare Diseases Committee has little information available to patients regarding what their process in making drug approval decisions.

"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," Wong-Rieger said.

Zaharieva and Essex’s families continue to fight for treatment coverage for all rare diseases with the hope of changing the drug approval process into one that is more rare disease inclusive.

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