Recently, Novo Nordisk announced that Rebinyn (Coagulation Factor IX (Recombinant), pegylated), was 1 of 5 drugs that will be available for patients in Canada with hemophilia B.

Making those drugs available in Canada was dependent on patients’ input into the value those treatments brings to the hemophilia B community.

Hemophilia B is a rare bleeding disorder in which patients are missing one of the factors in the coagulation cascade. As a result, their blood does not clot properly and that can lead to excessive bleeding episodes that can be very painful and possibly life threatening.

Recently, I spoke with David Page, national director of health policy at the Canadian Hemophilia Society about the review process for drugs to treat hemophilia B and the value that patient input (and advocacy groups) can provide to the review process.

“We have 2 representatives for the patient organization who sit on the selection advisory committee. That is the committee that reviews all of these submissions in great detail,” stated Page. The committee scores the submissions and makes recommendations Canadian Blood Services and to Hema Quebec.

For the review process, Page provided 3 pieces of advice for those advocacy groups wanting to take an active and effective role in a drug’s review process.

First, Page noted that one thing the patients bring to the review process is a better understanding of the burden of the disease without the treatment (and hopefully with the treatment if they were in the clinical trial). So, the advocacy group should choose people who have a good understanding of the disease and can articulate that during the review process.

Second, patient organizations need to gather their own information on patient reported outcomes. “I can’t speak for other rare diseases, but in hemophilia, if you ask patients what they think are important in how a therapy affects their lives they would generally say they want less pain, more mobility, to partake in activities of normal daily living, get an education, be involved in social activities, work, family, and they want to remove some of the psychosocial aspects of the disease,” noted Page, adding “Those are not things that are typically measured in a clinical trial.”

Finally, Page advised advocacy groups to do their own research on the economics of a treatment. “Don’t always trust the Health Technology Assessment body to get the economics right.” Page said patient groups can provide their own perspective on how a therapy may not only be beneficial in terms of health but may also have some economic advantages. For an upcoming drug review for emicizumab which has a new mechanism of action (as opposed to all current treatments which are replacement therapies), the Canadian Hemophilia Society is doing their own economic assessment to compare this new treatment to current options.  “We don’t want to rely on CADTH or others do that. We will present our own numbers.”

For more information about Hemophilia B, visit