CDR recommendation limits specialists' ability to prescribe new medication
VANCOUVER, March 5, 2015 /CNW/ - Canadians affected by pulmonary arterial hypertension (PAH) fear the threat of heart failure, or death, could soon be even more difficult to prevent given a recent recommendation to restrict patients' access to treatment. PAH is a rapidly-progressing and difficult-to-treat rare lung disease with no regard for race or age and is fatal within two to three years if left untreated.
If heeded by provincial and territorial public drug programs, a recommendation by the Common Drug Review (CDR) for Opsumit (macitentan) will limit newly-diagnosed patients' access to this new medication. It will also restrict PAH physicians' ability to exercise the individualized clinical judgement that is essential to the optimal management of this complex disease. Today, PAH patients living in Quebec, the only province currently not participating in CDR, are the only Canadians with publically-funded access to Opsumit.
Opsumit, the first therapy approved in Canada for the long-term treatment of PAH, reduces morbidity in patients' in Functional Class II or III PAHi. For these patients, living with PAH is already a significant burden, with any physical activity resulting in tiredness, rapid heartbeat and shortness of breath.
"By recommending that physicians follow a stepped approach in prescribing PAH treatments, including Opsumit, the CDR is suggesting that governments deny this small group of patients' immediate access to optimal, individualized treatment options that could have long-term, life-altering outcomes," says Angie Knott, National Manager, Pulmonary Hypertension Association of Canada. "As a result, newly diagnosed PAH patients could reach a more advanced stage of the disease, suffer from a worse quality of life, require a lung transplantation or die much earlier than those with access to all treatments."
Stepped approach to treatment will delay access, shorten lives
In January, 2015, the CDR recommended that Opsumit be publicly funded for the long-term treatment of PAH to reduce morbidity in patients in Functional Class II or III, but only after an initial trial with a phosphodiesterase type-5 (PDE-5) inhibitor has resulted in an inadequate responseii. This stepped approach to initial therapy also appeared in the Therapeutic Review Recommendation Report of Drugs for PAH, released in draft form by the Canadian Agency for Drugs and Technologies in Heath (CADTH) in November, 2014iii.
Opsumit has since become the first example of how the Therapeutic Review Report can be applied to restrict access to treatment for adult PAH patients.
Given the complexity of this fatal disease, the PAH community believes that treatment decisions should be made on a patient-by-patient basis by specialists in PAH. They worry that standardizing treatment at diagnosis through stepped therapy and delaying timely access to optimal treatment could shorten patients' lives and worsen clinical outcomes.
"PAH is not a one-size-fits-all disease. These recommendations could have ended my life," says Tarya Laviolette, a PAH patient. "When I was diagnosed, I was losing my breath with the smallest activity. I couldn't make the bed and I could barely pick up my infant son. A flight of stairs looked like a mountain to me. My specialist didn't think I had time to try one drug and then wait to determine what to do next. He wanted to prescribe the treatment he thought was right for me immediately in order to save my life. If these recommendations had been in place when I was diagnosed, I truly believe that I would not have the quality of life that I enjoy today."
Individual treatment plans improve patient outcomes
Since 1997, nine PH-specific treatments have been approved in Canada and thanks to these advancements, many patients are living longer and heathier livesiv. While therapy generally reduces the severity of PAH, treatment response greatly varies day-to-day and person-to-person. CADTH acknowledges that medical specialists working in pulmonary hypertension clinics are best suited to make prescribing decisions for adults with PAH given the individual nature of this diseaseiii. Yet, limiting access to Opsumit as a choice among all approved PAH treatment options contradicts this recognition of the important role that specialists play in responding to the individual specificities of the disease.
"This is a disease that can progress very quickly and we know that PAH patients can respond very differently to different treatments. Denying equal and consistent access to any and all effective medications, either alone or in combination may limit the successful treatment of our PAH patients which could be fatal," 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 the Pulmonary Hypertension Association of Canada. "As PAH physicians, we need the latitude to make individual prescribing decisions from among all Health Canada-approved therapies, to ensure the best outcomes for our patients."
Funding decision urged based on evidence and experience
People with PAH are living longer thanks to improvements in treatments and patient careiv. Currently, PAH specialists prescribe front-line therapeutic choices (including PDE-5 inhibitors) that they believe are in the best interest of the long-term health of their patientv. Patients and caregivers are concerned that tying the hands of specialists by denying access to Opsumit or other treatment options at diagnosis will have a negative impact on patient outcomes, especially considering how quickly an individual's health can decline.
"Each person suffering from this aggressive and ultimately fatal disease deserves the best possible quality of life," adds Ms. Knott. "While we are pleased that this recommendation notes that Opsumit is effective when used in combination therapy with PDE-5s, as well as monotherapy, we urge provincial and territorial governments to disregard the flawed CDR recommendation that limits access for Opsumit and does not allow our PAH experts to make treatment decisions – deemed necessary for patients' long-term stability and survival – based on both clinical evidence and clinical experience."
About Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension (PAH) – the most severe form of pulmonary hypertension – is a rare disease affecting the arteries of the lungs. When an individual has PAH, the arteries of their lungs become narrowed and scarred. This can result in almost complete closing of the arteries, which can lead to heart failure. Some symptoms of PAH are shortness of breath; bluish hands, feet and lips; swelling of hands and feet; light-headedness and dizziness; chest pain; exhaustion and fainting. Because it shares many symptoms with other diseases, PAH is often misdiagnosed. It is a fatal disease for which there is no known cure.
About the Pulmonary Hypertension Association of Canada (PHA Canada)
The Pulmonary Hypertension Association of Canada (PHA Canada) is a national, registered charity. Our mission is to empower the Canadian pulmonary hypertension community through awareness, advocacy, education, research and patient support. PHA Canada is working to end the invisibility and isolation often experienced by those living with this life-changing disease. To learn more about PHA Canada and pulmonary hypertension, please visit www.phacanada.ca.
i 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. Retrived from http://www.nejm.org/doi/pdf/10.1056/NEJMoa1213917.
ii Common Drug Review. CDEC Final Recommendation. Macitentan (Opsumit – Actelion Pharmaceuticals Canada Inc.) Indication: Pulmonary Arterial Hypertension. January 28, 2015. Retrieved from http://www.cadth.ca/media/cdr/complete/cdr_complete_SR0364_Opsumit_Jan-30-15.pdf.
iii Canadian Agency for Drugs and Technologies in Health (CADTH). Therapeutic Review Recommendation Report, Drugs for Pulmonary Arterial Hypertension: Comparative Efficacy, Safety and Cost Effectiveness [DRAFT]. November 2014. Retrieved from http://www.cadth.ca/media/pdf/TR0006_PAH_Draft_Recommendations.pdf.
iv Pulmonary Hypertension Association of Canada, Toronto Chapter. Retrieved from http://www.phatoronto.ca/about-ph/
v Pulmonary Hypertension Association of Canada. CADTH Therapeutic Area Review: Drugs for Pulmonary Arterial Hypertension Draft Recommendations Feedback. November 2014.
SOURCE Pulmonary Hypertension Association of Canada
For further information: Alyssa Acorn, Cohn & Wolfe, 647-259-3304, [email protected]