CF Families in Ontario Send Video Plea for Access to Vertex Therapies

Parents of children and young adults with cystic fibrosis (CF) in Ontario sent a video appeal to the head of their province’s government, asking the premier to quickly expand prescription criteria that limits their access to key therapies by Vertex Pharmaceuticals.

Two of the three gene modulating treatments noted — Orkambi (lumacaftor/ivacaftor), Kalydeco (ivacaftor) — are approved for use by CF patients in Canada. The newest, Trikafta (elexacaftor/tezacaftor/ivacaftor), is currently under review at Health Canada.

The video sent to Premier Doug Ford features testimonies by some of the nearly 1,500 people living with CF in Ontario. Shared by CF Get Loud — Canada’s largest grassroots movement for CF — on the group’s social media networks, it has been seen over 11,000 times, according to a press release.

Orkambi was first approved by Health Canada, the country’s regulatory agency for medicines, in 2016, and Kalydeco in 2012. But whether and how they are added to the public health plans of each Canadian province and territory, which opens them to patients at low or no cost, is at the discretion of each region’s government.

CF Get Loud has been advocating for greater access to these treatments. The group states in its release that it is urging Ford and the Ontario health ministry to press the pan-Canadian Pharmaceutical Alliance — which works on a nationwide scale to expand and manage treatment access — to quickly finish negotiations with Vertex and list Orkambi and Kalydeco on the province’s “formulary,” so eligible patients can use them.

“Despite Orkambi and Kalydeco being approved for use in Canada … they are nearly impossible to access,” the group states.

It also asks that Trikafta, which the group calls a “breakthough” medication, to be listed as soon as it is approved. Trikafta is designed to treat patients with at least one F508del mutation — the most common CF-causing mutation, estimated to be in 90% of this patient population.

Trikafta given to a child born with CF today would add 9.2 years to life expectancy, CF Get Loud reports. It also notes that one person with CF, typically under age 33, dies in Canada each week. An estimated 4,440 Canadians are living with the disease.

A Health Canada decision on Trikafta is expected in late June, the group reports on a webpage. It also describes the drug approval and listing process, and where Trikafta stands in it.

CF Get Loud urges CF patients and their families to take “direct action” on therapy access by becoming more educated about these treatments, and by petitioning their provincial government. For more information, please visit this link.