Barry Katsof was a high-profile Montreal businessman, who ran a globally recognized company for 34 years. One morning in early 2000, he awoke to find blood in his urine. His doctors sent him for a large battery of tests, but they found nothing. Barry knew something was still wrong, so he persisted through two additional rounds of testing. Two years later, he received a diagnosis for a rare disease: paroxysmal nocturnal haemoglobinuria (PNH).
PNH is a very rare and debilitating disease of the bone marrow that affects the blood and major organs. It affects 1 to 1.5 people per million. A rare disease is generally defined as one that occurs in less than 1 in 2,000 people.
Facing a deadly diagnosis alone
When Barry was diagnosed in 2003, he was basically alone, facing his own death. There was no treatment for PNH. In the pre-Internet era, it was almost impossible to find information and support. He knew that his life expectancy without treatment was about 4 to 5 years. He had already lost two years in his search for a diagnosis. The clock was ticking.
Instead of seeing his situation as a curse, Barry took it as a challenge. He started to read everything he could find about PNH. His search led him to a London-based researcher, whom he arranged to meet. The scientist made Barry aware of a new treatment in development called eculizumab (Soliris®; Alexion Pharmaceuticals). But it was a long way from Health Canada approval and availability.
“I will always remember a time when I was walking through airport security. The lady who was working there told me, ‘You look so sick! Go get checked out by your doctor’.”- Barry Katsof
What doesn’t kill you makes you stronger
Sometimes when you don’t give up, you get a lucky break. That’s exactly what happened to Barry. He received the life-saving medication Soliris®. A pharmaceutical manufacturer granted access on a compassionate basis, four years before it received Health Canada approval. This medication saves patients with PNH from certain death. It returns their life expectancy to normal for their sex and age.
Treatment gave Barry his life back. Today, at 72 years of age, he is no longer short of breath. He can walk across a room. He cycles for miles and trains twice weekly.
Somebody saved my life. I am not a religious person, but I am a believer. And I run my life as ‘do unto others as you would have them do unto you’.”- Barry Katsof
Living by the golden rule
Since Barry got his second chance at life, he’s kept his word. He founded the Canadian Association of PNH Patients. He and his team advocated for the drug approval of Soliris® in Canada. In July 2011, Barry and his team were the first medication to go through the pan-Canadian Pricing Alliance (PCPA).
PNH Canada educates and supports patients and their community of friends, families, and caregivers. It provides them with much needed support during the COVID-19 pandemic. Beyond PNH, it advocates on behalf of all patients with rare diseases.
“I got this rare disease for a reason, so that I can help others. It sounds hookey pookey, but I bring to the table a skill set that other advocates don’t have.”
Advocating for change
According to Barry, advocacy for rare diseases must become even louder and more significant. He and his team have endorsed our petition for a national strategy for rare diseases.
But another crisis is underway in the rare diseases community: the impact of recently redrafted Patented Medicine Prices Review Board (PMPRB) guidelines. These guidelines may negatively influence the development and availability of new drugs in Canada. Barry and his team are active and providing tools to help the community to understand the situation and take action. We invite you to reach out to learn more about the implications for access to treatment.
“Get involved! We can’t stop. Everything will help! ”- Barry Katsof