The Canadian Association of PNH Patients is a not-for-profit Canadian organization formed in 2009. The mission of the organization is to connect Canadians affected by Paroxysmal Nocturnal Haemoglobinuria and advocate for the best possible care for patients and ensure they are equipped with the most current tools and information to help them live well with the condition. The organization also provides support to caregivers, and works to increase awareness and understanding of PNH.
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare and debilitating disease of the bone marrow that affects the blood and major organs. It is a chronic, life-threatening illness and can have devastating effects upon a patient physically, mentally and emotionally. This website represents a growing community of patients, caregivers and members of the medical community who wish to support and improve the quality of life of those with PNH.
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On August 4, the Canadian Association of PNH Patients made a submission in response to the consultation currently underway regarding proposed amendments to the Regulations governing the Patented Medicine Prices Review Board (PMPRB). Despite the most recent changes, as reflected in the June 2020 Draft Guidelines, we remain concerned that the health of Canadians living with PNH continues to be put at risk by the uncertainty generated by the PMPRB reform process to date.
Read our full submission here
Some highlights from our submission:
- A single treatment will not be the solution for everyone with PNH, so options are needed – especially with a life-threatening disease like PNH. As such, we view any new or existing barriers to treatment as unacceptable to our patient community, and the uncertainty caused by the proposed PMPRB reforms continues to be the barrier of greatest concern.
- At last count, there were 18 molecules being studied in clinical trials around the world for the treatment of PNH. This is unprecedented, but largely meaningless to Canadian patients as only one of these clinical trials has sites in Canada. None of the companies conducting these trials – even those in Phase 3 – have confirmed if/when Canada will be included in their global launch plans.
- Elsewhere in the world, up to 70% of PNH patients have been able to switch a new treatment option (a reformulation of the only existing drug for PNH), which brings a very timely advance in delivery (given COVID-19) by reducing the frequency of infusions. Due to the uncertainty caused by the proposed PMPRB reforms, Canadian patients are still awaiting access – and needlessly being exposed to health risks from COVID-19.
- The processes in place at the pan-Canadian and provincial/territorial levels to ensure cost savings on drugs are far from ideal as they take a considerable amount of time, but at least result in most treatments eventually reaching patients – but PMPRB reform has disrupted the fine balance between cost and effectiveness of drugs, especially those for rare diseases.
- Almost half (42%) of new drug launches in Canada were delayed for longer than six months following Health Canada approval, or were not launched here at all (compared to 22.5% in 2018-2019), which means that PMPRB reform the is already having a quantifiable and detrimental, if not deadly, effect on patients’ lives.
The PNH community questions how those responsible for the delivery of healthcare across Canada (the provinces and territories) feel about the impact that the proposed changes to PMPRB will most certainly have on their ability to save and extend the lives of people with rare diseases?
Drugs for rare diseases still only represent a very, very small portion of public drug costs across Canada (approximately 2.5%). Until now, these medicines have been getting to patients who need them through public funding, but what happens when new life-saving and life-extending medicines stop coming to Canada because of the proposed changes to PMPRB? What will our provincial and territorial governments say to their constituents who put their lives in their hands?
Follow us for updates on our ongoing advocacy efforts around this important issue and how you can get involved!
Connect with us to receive the latest news and updates: http://www.pnhca.org/contact-us/
Read stories from patients within the Canadian PNH community.