Pharmacare ‘cost-savings’ should not trump appropriate drug use

The final report of the Advisory Council on the Implementation of National Pharmacare, due in spring 2019, is meant to provide the federal government with recommendations on how best to implement a national drug insurance program. Despite the Trudeau government’s mantra of “affordability, accessibility and appropriate use of prescription drugs,” a crucial question remains. If a national drug insurance program is implemented, will cost-savings outweigh appropriate drug use, pushing patients towards less-expensive drug options over more costly—but appropriate—medicines?

Let’s consider a relevant example to illustrate how influence can be applied.

Age-related macular degeneration (AMD) is a progressive eye disease and leading cause of vision loss in people over 50, affecting about 1.4 million Canadians. Standard-of-care therapies for the most severe type of AMD are called anti-VEGF drugs (VEGF stands for vascular endothelial growth factor). VEGF is a substance normally produced in humans and is responsible for telling new blood vessels to form. However, uncontrolled blood vessel growth in the retina of the eye causes vision loss.

Anti-VEGF drugs remove excess VEGF from the eye, preventing or reducing vision loss. Two of these drugs, known as Eylea and Lucentis, have been developed. Health Canada has reviewed and approved the efficacy and safety of these drugs, and they are available to Canadian patients.

And yet, the Canadian Agency for Drugs and Technologies in Health (CADTH) recommends Avastin, a Health Canada-approved cancer drug—instead of Eylea or Lucentis—as the preferred initial therapy for retinal conditions such as AMD. (CADTH provides recommendations to the federal and provincial governments about what drugs should be covered in their public drug plans.)

The reason for CADTH’s recommendation?

Avastin costs much less than Eylea and Lucentis, and is reasonably effective as an anti-VEGF therapy. Saving public money sounds good but, unlike Eylea and Lucentis, Avastin’s manufacturer has not applied to Health Canada for marketing approval for the drug’s use in eye diseases.

Most significantly, Avastin’s product monograph (the official description of how a drug should be appropriately used in Canada) for its use as a cancer therapy has a serious safety warning explicitly saying that Avastin is “not formulated and has not been authorized” to treat eye diseases. The monograph also says that Avastin’s unauthorized use as retinal therapy has been linked with increased risks of eye inflammation, stroke and death. And a clinical trial found an increased risk of serious adverse events such as heart attack and stroke in patients using Avastin for vision loss.

Avastin’s use for AMD would be “off-label,” which means a medicine is being used to treat a health condition for which the drug does not have marketing approval. Off-label drug use is common when patients have life-threatening illnesses where no alternative treatment exists. In these circumstances, such use may be the last hope for a patient. But for less-serious conditions, off-label use may lead to greater harm than benefit.

Only one drug out of 154 (less than one per cent) in the Government of Ontario’s two programs that provide funding for costly medicines is covered for off-label use contrary to a serious safety warning, and prescribers are required to explain to the drug plan why such use is necessary. Accepting CADTH’s recommendation to cover off-label Avastin as initial treatment for retinal diseases, as some other provinces have, would be contrary to Ontario’s normal practice. Not only that, such use would expose individuals with diabetes, who frequently have vision loss from diabetes complications and are at risk for heart and circulatory problems, to even greater cardiovascular dangers.

CADTH is not an independent agency— it’s funded and owned by the federal and provincial governments to which it makes recommendations. The agency can be used as part of government efforts to influence drug costs. If Canada’s governments are set on a national pharmacare program to provide affordable access to prescription drugs to more patients, it’s crucial that decisions are not made where cost-savings trump appropriate drug use.