For the first time in history, Health Canada has approved a gene therapy treatment for people with Hemophilia B. This is a tremendous moment for medicine and for the bleeding-disorders community that can now imagine a life without pain and difficulty.
People with hemophilia are at risk for severe and potentially life-threatening bleeding episodes. Many also bleed into their joints, resulting in chronic, debilitating joint pain. This is because their ability to clot is impaired due to an inherited genetic defect. As a result, patients rely on chronic medication or injections of clotting factor for their entire life.
For more than a decade, gene therapy for Hemophilia B has been rigorously studied in clinical trials and has consistently been shown to help patients clot. For patients who respond to gene therapy, they no longer require additional treatment and can live like a regular person.
However, gene therapy is not cheap. Pfizer is seeking more than $4 million per dose and the government is reviewing whether it will cover this cost.
Current treatment options for hemophilia are expensive and cost the government millions of dollars over the lifetime of a hemophiliac. Procurement details for these treatment options are confidential in Canada. However, one study estimated that the cost to treat patients with Hemophilia B in Canada for medications alone is anywhere from $350,000 to $500,000 per year.
There are also additional costs to the medical system related to hemophilia such as joint replacements, emergency department visits, doctors’ appointments, long-term disability payments and other costs for social services. These costs are not paid by the same source of funds that covers the cost of treatment, which can make it difficult for governments to accurately assess the actual cost of caring for hemophiliacs. As a result, despite its high cost, gene therapy may end up supporting cost-savings for the health-care system over time.
Another cost is that hemophiliacs are more likely to miss school and work and more likely to be unable to be productive members of society.
The human toll of suffering from a bleeding disorder is also worthy of consideration. Failing to provide access to the best treatment possible leaves hemophiliacs with preventable pain and severely decreases their quality of life.
The cost to treat patients with Hemophilia B in Canada for medications alone is anywhere from $350,000 to $500,000 per year.
If governments consider these factors seriously, the decision to fund gene therapy for hemophiliacs becomes clear. In Canada, our perspective on health care is based on a collective recognition that we are all better when we support those who are most in need. When considering the true cost of caring for hemophiliacs, along with the decreased pain and suffering when their condition is treated properly, government funding for gene therapy is well worth the cost.
This will not be the first time we will question whether to fund gene therapy. As we continue to innovate, gene therapy may emerge as the answer for other diseases as well. For example, Johnson & Johnson is currently working on a gene therapy treatment for inherited retinal disease. Astellas is working on a gene therapy for X-linked myotubular myopathy, which is a serious neuromuscular condition. When the time comes for these innovations to enter the market, we hope that the precedent established here will demonstrate how Canada prioritizes getting patients the treatments they need and ensures that those who suffer from rare disease are not left behind.
In this respect, Canada needs to champion the market viability of these innovative and technologically advanced solutions. Not everyone is so lucky, and Canada needs to set an example for the rest of the world, especially in areas where rare diseases like hemophilia are underprioritized.
“Living with hemophilia is all about where one is born,” says Glenn Pierce, vice-president of the World Federation of Hemophilia in Montreal, told Scientific American. We need to make sure that Canada becomes a place where patients with hemophilia thrive.
Hemophilia is a genetic condition and those that suffer from it face the difficulties of living with their condition every day. For these patients, the right decision is to fund gene therapy – because patients deserve not just treatment for their disease, but the best possible treatment to help them lead a regular life.
