PNH patients urge government to provide access to life-saving treatment

With so few people affected by rare diseases, how does a group of less than 90 patients across Canada make their voices heardon a life-and-death issue such as access to treatment? Barry Katsof, President of the Canadian Association of PNH Patients, says his group is turning to social media to ask others to take a stand with them in the fight for their lives.

"Today, patients living with Paroxysmal Nocturnal Haemoglobinuria (PNH) and their loved ones will mark Rare Disease Day in Canada by turning up the volume on an issue that is threatening our lives - our immediate need for publicly-funded access to treatment with Soliris," says Katsof. "We had the government's attention for a while, but they have stopped listening. Through this campaign, we hope to rallywide support for access to Soliris, the first and only treatment proven effective in treating our ultra-rare and life-threatening disease."

In January, 2011, Lucas Maciesza's front-page battle with PNH spurred a change in a government policy which granted him only temporary access to Soliris, a treatment he requires for the rest of his life. This impromptu move by the Ontario government came at the eleventh hour for Maciesza who, after being pulled back from the brink of death by hospital funding for his life-saving infusions of Soliris, found himself again not knowing where funding for his treatment would come from.

"No one should have to beg for their life in that situation," says Lucas, 26, in a new video released today on YouTube by the Canadian Association of PNH Patients. "I barely had any energy to beg for my life in that situation."

While Lucas' Soliris treatments will likely be covered for a maximum of six months through this policy change, without a long-term solution he will again be without funding after this period. Other PNH patients in Ontario who could benefit from the treatment now are concerned they will have to suffer a catastrophic health event, or even face death, to qualify for only short-term coverage.

"I have to wait until I'm in danger of losing an organ, a limb or my life before the province will help me," says Hilary Handley, a PNH patient and mother of three from Toronto who also shares her story in the YouTube video. "It is like a death sentence - I can die tomorrow from a blood clot. Luck of the draw, I guess." 

Katsof stresses that the government's work is far from done with respect to accessibility to this life-saving treatment for PNH. "On Rare Disease Day, we strongly urge government to make an expeditious and permanent decision to fund Soliris and save PNH patients and their families from the nightmare Lucas has been living, and that Hilary continues to face," says Katsof.

The Canadian Association of PNH Patients has established a presence on social media platforms including YouTube, Facebook and Twitter, and supporters are urged to join the online conversation. By visiting pnhca.org, Canadians canlend their voice to the fight by contacting their provincial health ministers and premiersto tell them to fund Soliris for PNH.

"We are calling on all Canadians to take a stand on this issue, and ask their provincial government to provide the care that PNH patients clinically require and ethically deserve," says Katsof. "It is high time that Canada follows the lead of most other developed nations that already fund Soliris, and make the right choice for our patients - the choice for life."