Local mother Beth Vanstone and members of the cystic fibrosis community have sent a video documentary to Premier Doug Ford in hopes of getting access to life-saving medication.
Vanstone, whose daughter Madi is a CF warrior who was diagnosed at nine months of age, is part of the CF Get Loud campaign that advocates for the approval of orphan drug use and coverage in Canada.
Seven years ago, Madi made headlines when she convinced then-premier Kathleen Wynne and the Ontario government to approve coverage for her life-saving drug, Kalydeco.
Without coverage by OHIP, her family would have to spend almost $350,000 a year for the drug, although insurance and the fact that she was part of a study reduced the cost to the Vanstones to about $60,000 per year.
And although modulators Orkambi and Kalydeco have been approved for use in Canada, the strict prescribing criteria needed to be met make it nearly impossible for patients to access. Trikafta has been submitted to Health Canada and is pending approval.
Cystic fibrosis is a fatal genetic disease that affects 4,400 Canadians, including 1,500 Ontarians.
The six-minute mini-documentary features the voices and stories of CF patients and their families across the country and the challenges they face daily due to their illness.
The video was produced by CF patient and journalism student Kaity Brady.
Brady is 28-years-old and lives in Toronto. She was diagnosed with CF at birth. Her family had to move to Toronto to be closer to Sick Kids Hospital because her older sister, Elizabeth, had a very extreme case of CF and needed to be close by for treatment. At eight years old, Elizabeth needed a lung transplant due to the disease.
In 1999, Elizabeth passed away because her lungs were so small and it was near impossible to find a donor. At the time, the transplant was the only option for treatment.
"Since losing my sister, I spent most of my life waiting for a breakthrough medicine. My hope is that the video will spark the urgency and the political will needed to finally get this done so that families in the video won't have to go through what my family went," she wrote on the CF Get Loud Facebook page.
One CF patient featured in the video is Ava, 6, who lives in Etobicoke. She had her first surgery at Sick Kids before she was 10 hours old and was only able to come home at eight months old. She has had five more surgeries after that, being admitted to Sick Kids six times.
"In the past two years, CF has taken everything from me," said Hailey, 26, from Toronto, who has been in the hospital for 12 weeks this year.
According to the video, in 2019, Canadians with CF have spent a total of 25,246 days as in-patients in hospital. Treatments for Canadian CF patients include inhaled IV and oral therapies, enzymes, insulin, physiotherapy and surgeries. The end-stage treatment option is a lung transplant.
The CF Get Loud online community, CF Treatment Society and CF Canada have joined forces to help expedite the process of getting modulators into the hands of patients before it's too late.
In 2019 Vanstone and Madi met with Ontario Minister of Health Christine Elliott to discuss modulators and the negotiations for Orkambi and Kalydeco. At the time, they had not been submitted to the pCPA (pan-Canadian Pharmaceutical Alliance). They also discussed the Trikafta drug, which had already been approved in the U.S. and can treat 90 per cent of CF patients.
The modulators Kalydeco and Orkambi finally made it to the pCPA in June but have yet to be approved.
Until Trikafta is approved, it is only available for emergency use under the special access program for patients who are seriously ill.
"Just knowing that there is this drug out there that I see people in the U.K. and the U.S., to see them have these opportunities to live their life...at 26 years old I shouldn't be thinking like this and needing to rely on medication I can't receive," said Hailey.
So far the video has 252 shares and 6.4K views on Facebook.
"We need your help to get better," and "Will you be our champion?" the patients ask Ford in the video.
The video was delivered to Ford on Wednesday with a press release from the CF Get Loud.
For more information on the fight for lifesaving medications, visit the CF Get Loud website here.
