Archive for December, 2015

Equitable drug access urged for patients with rare, potentially fatal lung disease

Physicians, patients and caregivers remind Health Minister of devastating consequences when treatment decisions for PAH are taken out of medical experts’ hands

TORONTO, Dec. 3, 2015 /CNW/ – Ontarians affected by pulmonary arterial hypertension (PAH), a rare but serious, progressive and potentially fatal lung disease, gathered at Queen’s Park today to urge Health Minister Dr. Eric Hoskins to make Opsumit (macitentan) publicly accessible as a treatment choice for patients in Ontario. It is the only oral treatment proven in a randomized controlled trial to reduce long-term morbidity in patients, including reductions in PAH worsening and hospitalizations.1

“On behalf of the Pulmonary Hypertension Association of Canada and the Scleroderma Society of Ontario, we are calling on the Ontariogovernment to urgently fund Opsumit as an option for those living with PAH, a rare, serious and complex lung disease,” says Anna McCusker, Executive Director of the Scleroderma Society of Canada and Ontario. “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.”

Drug not accessible in Ontario, despite positive funding recommendation 

Two years ago, Health Canada’s approval of Opsumit offered new hope for adult PAH patients and their physicians.2 In January 2015, the PAH community was further encouraged by the Common Drug Review (CDR) recommendation that Opsumit be publicly funded for the treatment of PAH to reduce morbidity (severity of illness).3

 However, despite the positive CDR funding recommendation, Ontarians affected by PAH still do not have publicly-funded access to Opsumit through the Ontario Public Drug Program (OPDP). Under this current paradigm, Ontario physicians are limited to prescribing Opsumit only to PAH patients with private health insurance, or the ability to pay out-of-pocket—despite their view that a patient who depends on public funding might benefit from receiving this treatment.

“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,” says 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 PHA Canada. “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.”

History set to repeat itself if Opsumit is not accessible

Unfortunately, delays in accessing much-needed therapies are all too familiar for the PAH community, and previous limitations, imposed with little specialist consultation, still haunt Ontario patients and families. Over the course of 2008 and 2009, the OPDP made a decision that restricted PAH patients’ treatment options to a stepped approach, requiring specialists to follow a set procedure and prescribe medications in a particular order, instead of allowing them to prescribe the medication(s) they felt would most benefit their patients with this serious lung disease.4

 PAH patients affected by this decision suffered greatly—and one Ontario mother died as a result of restricted access to treatment.5 This patient, whose story made front page news, was told by the province that a certain medication prescribed by her specialist would be covered only if she scaled back her treatment and stopped another effective drug. When she did, her condition deteriorated so rapidly that she passed away soon after. As the result of her death and advocacy efforts by the PAH community, the OPDP removed the restrictions.

“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 since the province has not yet listed Opsumit, we find ourselves in a similarly precarious position,” says Ruth Dolan, Director at PHA Canada and Co-Leader of the Toronto PH Support Group, and mother to 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.”

About Pulmonary Arterial Hypertension

In PAH, the arteries (blood vessels) of the lungs become narrowed by scar tissue, sometimes to the point of being completely closed. Those affected by PAH suffer from high blood pressure in the lungs, which results in strain and enlargement of the heart, which ultimately leads to heart failure and death. PAH is a common complication of scleroderma, a chronic hardening and contraction of the skin and connective tissue. PAH shares many symptoms with other diseases, which often leads to lengthy delays in diagnosis, and misdiagnosis. There is currently no cure for PAH, and if left untreated, patients live an average of two to three years. 


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 1 Pulido T, Adzerikho I, Channick RN, Delcroix M, Galiè N, Ghofrani H-A, et al (2013). Macitentan and Morbidity and Mortality in Pulmonary Arterial Hypertension. New England Journal of Medicine. 369(9). pp.809-818. Retrieved from Accessed on December 1, 2015.

2 Health Canada. Drugs and Health Products – Opsumit. Retrieved from Accessed on December 1, 2015.

3 Common Drug Review. CDEC Notice of Final Recommendation. Retrieved from Accessed on December 1, 2015.

4 Progress in Ontario for patients with uncommon but serious lung disorder, but more access to current treatments still needed [news release]. Toronto, ON: Pulmonary Hypertension Association of Canada; May 15, 2009. Accessed on December 1, 2015.

5 Leeder J. Funding for rare-disease therapy under the microscope. The Globe and Mail. November 2, 2009. Accessed on December 1, 2015.