Rare lung disease patients fear lengthy negotiation trend will delay access to another new therapy, putting lives at risk

Rare lung disease patients fear lengthy negotiation trend will delay access to another new therapy, putting lives at risk


PAH physicians, patients and caregivers urge governments across Canada to uphold funding recommendation and grant swift access to life-extending treatment option

VANCOUVER, BC and HAMILTON, ON – November 17, 2016
– Canadians affected by pulmonary arterial hypertension (PAH), including those living with scleroderma, are pleased with the recent Common Drug Review (CDR) recommendation to reimburse Uptravi (selexipag). However, the community fears access to this treatment option – the third new PAH therapy approved by Health Canada in recent years – will be delayed due to a lengthy negotiation process through the pan-Canadian Pharmaceutical Alliance (pCPA). Another Health Canada-approved PAH treatment, Opsumit (macitentan), was recommended for public funding by the CDR in January 2015, yet still remains out of reach due to unsuccessful pCPA negotiations, giving patients reason to be concerned that Uptravi may follow in its path.

In January 2016, Health Canada approved Uptravi for the long-term treatment of idiopathic PAH, heritable PAH, PAH associated with connective tissue disorders (including scleroderma), and PAH associated with congenital heart disease, to delay disease progression in adult patients with Functional Class II or III. In October 2016, the CDR recommended that Uptravi be publicly funded for patients whose disease is not being adequately controlled with a first- and second-line therapy. The hope is that publicly-funded access to Uptravi will be successfully negotiated through pCPA, but physicians, patients and caregivers are concerned that any delay will impact the prognosis of those who require this treatment option. 

“PAH patients have high hopes for Uptravi as a new treatment option to slow the progression of this rare but very complex and serious lung disease that significantly limits life expectancy,” says Maureen Worron-Sauvé, Vice President of Advocacy, Scleroderma Canada. “Our fear is that if we are faced with another lengthy negotiation process, as with other PAH drugs, patients will be denied access to a treatment option that may be urgently needed by some, and seen as a last resort by others.”

To date, none of the recently approved treatments for PAH are accessible to patients in Canada through public funding, with the exception of Opsumit, which is only publicly funded in Quebec. This delay in access to treatment restricts PAH physicians’ ability to exercise the individual clinical judgement that is essential to the optimal management and long-term health of those living with this complex disease. As the first oral prostacyclin receptor agonist, Uptravi is a new form of treatment that can address patients’ diminished quality of life and potentially slow the progression of their disease. For patients who are unable to be treated with an intravenous prostacyclin, Uptravi may be their last hope. 

“When I was diagnosed with PAH in 2009, I was told I only had three months to live. My initial treatment regime prescribed by my PAH specialists moderately improved my symptoms, but it wasn’t until Uptravi was added to my combination therapy that I really felt I had an effective course of treatment that controlled and reduced my PAH symptoms,” explains Joan Nemeth from Vernon, B.C. “Prior to Uptravi, I couldn’t fully get through my days and had to prioritize my activities. I couldn’t work two days in a row, or make the bed and do the vacuuming on the same day. Now, I am able to lead a more normal life, and I would hate to think what would happen to me or other PAH patients if we were deprived of this life-changing medication.”

While the prognosis for PAH patients has improved somewhat in recent years, it remains poor despite currently available treatments. Many patients remain significantly ill with moderate-to-severe symptoms and progressive right-ventricular heart failure. The average survival in adult PAH patients following diagnosis is estimated at only five to seven years, which means they do not have time to wait for the necessary treatment options to be made accessible to extend their already limited life expectancy.

“It is unacceptable that PAH patients who rely on public drug funding are prevented from accessing Health Canada-approved treatments and, as such, we as PAH specialists are unable to exercise the individual clinical judgment that is essential for the optimal treatment of this serious and fatal 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 concerned that the trend to delay and deny public funding of the newest, most effective PAH treatments is taking treatment decisions out of the experienced hands of PAH medical experts. We strongly urge governments across Canada to immediately fund Uptravi and ensure all PAH patients have timely access to optimal therapy to improve and extend their lives.”

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 only two to three years.

Please visit www.phacanada.ca or www.scleroderma.ca for more information.

For more information, please contact:

Jamie Myrah
PHA Canada
604-682-1036 ext. 101 
Anna McCusker
Scleroderma Canada
 PHA-Logo-PMS271.png  Scleroderma-Society-Canada-Societe-Canadienne-de-la-Sclerodermie.gif


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