Canadians living with pulmonary arterial hypertension (PAH) — a rare, progressive lung disease — gathered at Queen’s Park in Ontario recently to urge their province’s health minister, Dr. Eric Hoskins, to publicly fund Actelion’s Opsumit (macitentan) as a treatment option in Ontario. The drug is currently the only oral therapy proven in a randomized controlled trial to decrease PAH’s long-term morbidity and reduce symptom worsening.

“On behalf of the Pulmonary Hypertension Association of Canada and the Scleroderma Society of Ontario, we are calling on the Ontario government to urgently fund Opsumit as an option for those living with PAH, a rare, serious and complex lung disease,” said Anna McCusker, executive director of the Scleroderma Society of Canada and Ontario, in a press release. “Our community should be feeling hopeful today to have an important new therapy for this highly-individualized condition, made more complex when caused by scleroderma. Instead, we are extremely concerned that the lack of access to Opsumit will lead to undue and avoidable patient suffering.”

Despite official support — Health Canada approved Opsumit in 2013 and the Common Drug Review recommended in 2015 that Opsumit be publicly funded as a means of reducing PAH severity — Ontarians still do not have access to Opsumit through the Ontario Drug Program (OPDP). As such, only patients with private health insurance or of sufficient means can be prescribed the medication, even though evidence shows that those who depend on public funding could also benefit.

“It is unacceptable that for the past year, Ontario PAH patients who rely on the public drug plan have been prevented from accessing Opsumit because we as PAH specialists are unable to exercise the individual clinical judgment that is essential to the proper treatment of this disease,” Dr. Sanjay Mehta, MD, FRCPC, FCCP, director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Ontario, and chair of Pulmonary Hypertension Association Canada, said at the Dec. 3, 2015, gathering. “Our community is very willing to work with government to resolve this situation, but we are also here today to remind elected officials and decision-makers of the devastating consequences of taking treatment decisions out of the experienced hands of PAH medical experts.”

This is not the first time the PAH community in Ontario as experienced delays in accessing new and important therapies.  An OPDP ruling restricted PAH patients’ options for treatment in 2008-09 to a stepped method, meaning that specialists were required to prescribe medications in a specific order rather than choosing those they thought of best benefit to patients.

The OPDP changed its position, but only after one woman’s death was attributed to her inability to access appropriate treatment.

“The PAH community was grateful to see the province abolish the procedure that enforced a one-size-fits-all approach to treating this complex disease, but six years later, we are worried that … we find ourselves in a similarly precarious position,” said Ruth Dolan, director at PHA Canada, co-leader of the Toronto PH Support Group, and the mother of a PAH patient. “We urge Health Minister Eric Hoskins to immediately make this treatment accessible to PAH patients who rely on public funding. Treatment decisions for this complex disease must be left in the hands of specialists who are best able to assess their patients’ individual needs, benefiting their long-term health at diagnosis and beyond.”