Part I: Access to Opsumit in Canada
In March 2015, the Canadian Agency for Drugs and Technologies in Health (CADTH) suggested that all newly diagnosed people with Pulmonary Arterial Hypertension (PAH) in functional class II or III receive the same mono-therapy approach for their treatment. This approach would take the decisions for treatment out of the hands of PH experts and place it in the hands of government bureaucrats. I asked PH specialist Dr. Sanjay Mehta* to answer some questions I have regarding the accessibility of PH therapies in Canada.
Serena: I know that you are very involved and passionate about the PH community, and that you have been working very hard to ensure that all newly diagnosed people have access to the therapy that is best suited to their needs.Could you briefly explain why it is so important that PH specialists, like yourself, are able to treat PH patients on an individual basis and recommend a therapy suited to their needs instead of the stepwise, rigidtherapy approach suggested by the CADTH?
Dr. Mehta: It is one of the most basic principles of medicine: that each patient is an individual, and needs to be understood, respected, and treated as an individual. This includes doctors making decisions about the best medical treatment for a patient’s illness. Although many patients may have the same illness, as in the case of PH, they are all still unique individuals. As such, there is no reason to expect that they respond similarly to PH medications, or that the same medication is the best one for each patient. Expert PH physicians need to consider many factors in deciding on the best initial and subsequent medical treatment for each PH patient. The decision is based on having expertise and experience in PH, understanding the patient and their other conditions, and the specific risks of each treatment. Even in clinical studies of PH medications, all patient participants don’t respond similarly and, while some may respond well, others may not. Importantly, in dealing with a serious, progressive illness like PH, a patient can’t afford to waste precious time “trying” anything other than the most effective, best treatment for them.
Serena: The newest Health Canada-approved oral medication for PAH, macitentan (Opsumit) is not available to most Canadian PAH patients, as per the recent pan-Canadian Pharmaceutical Alliance (pCPA) decision to close negotiations with the manufacturer, with the impact that this drug is not approved for funding in most of Canada.
I understand that Opsumit is generally well tolerated, and that it was tested in one of the largest and longest clinical studies of any approved PH treatment. Can you discuss some of the benefits of Opsumit? If the government continues to deny funding for Opsumit, what is the future for this medication in Canada?
Dr. Mehta: In 2013, the Seraphin study reported on the benefits of macitentan (Opsumit) in what was at the time the largest, longest study ever in PAH patients. It showed that macitentan reduced the morbidity (severity of illness) in PAH patients, specifically reducing the risk of progressive worsening of PAH by 45% and reducing the risk of hospitalization by 50% over 3 years. Moreover, treatment with macitentan significantly improved symptoms, quality of life, and exercise capacity in PAH patients. This is a unique study that showed for the first time the long-term benefits of treatment with a PAH medication, compared to all other PAH studies which only looked at benefits over 3-6 months. Clearly, PAH patients don’t want to just improve over the short-term, but hopefully remain well for many years!
As a result, treatment with macitentan has been strongly recommended by the most recent 2014 PH Clinical Practice Guidelines jointly published by the European Society of Cardiology and the European Respiratory Society. Moreover, macitentan has been approved for funding in the US, and in many European countries, and many PAH patients are currently being treated with macitentan. Notably in Canada, Quebec approved public funding of macitentan in October of 2013, such that Quebec PAH patients have complete access to macitentan as a treatment for their PAH, should their expert PH physician decide it is the best treatment for them. Similarly, several large private insurance companies across Canada have approved macitentan coverage for their clients. However, the pCPA recently denied funding for macitentan for the rest of Canadians (PAH patients living in all the other provinces and territories who do not have access to private health care), and indeed, has broken off negotiations with the pharmaceutical manufacturer, Actelion.
This is a very concerning development for Canadian PAH patients, most of whom have been denied access to public funding for treatment with macitentan. Moreover, this establishes a dangerous precedent whereby any and all future new PAH therapies (for example, the new oral **selexipag or Uptravi, which was just approved by the FDA in the US) may similarly not get approval for funding for Canadian PAH patients. It would appear that Canadian and provincial governments are saying that PAH patients are doing just fine with the therapies they have available today! Clearly, PAH patients and their physicians know otherwise; despite treatment with the many PAH medications we have, many PAH patients remain seriously ill, limited in everyday life, and their disease continues to progress until it takes their lives, on average 7-10 years after diagnosis. Is it reasonable to accept that? Should we not try to further improve the health and lives of PAH patients? Most definitely, all Canadian PAH patients and their caregivers would want us to continue to develop, test, approve and make available newer and better PAH treatments. Lack of government understanding of this critical issue is already leading to less than optimal treatment of Canadian PAH patients, as currently demonstrated by lack of access to macitentan, and likely to reoccur with lack of availability of future new PAH treatments.
Serena: Thank you for sharing your thoughts on this complex issue. Is there anything that you would like to share going forward?
Dr. Mehta: All Canadian PAH patients and their caregivers should be heartened by the incredible progress we’ve made in the treatment of PAH, since the 1st medication, intravenous epoprostenol (Flolan) became available in 1997. Currently, 9 different medications are approved and generally available for the treatment of PAH in Canada. As a result, the quality of life and survival of most PAH patients have significantly improved. This is important to keep in mind and we should remain hopeful that, thanks to the joint efforts of dedicated advocates, members of the medical community, and PHA Canada, new treatments such as Opsumit will be made available to all PH patients in Canada. This is what we must continue to strive for.
** selexipag/Uptravi received approval by Health Canada on Tuesday, January 26th, 2016 for the treatment of PH.
The PHight or Flight Project would like to thank Dr. Sanjay Mehta and The Pulmonary Hypertension Association of Canada for their assistance on the Accessibility of PAH Therapies in Canada Series.