Cystic Fibrosis (CF) Warropr Madi Vanstone and her mother Beth, along with the rest of the CF Community is appealing to the government for their support in getting the life-saving CF drug Trikafta approved and negotiated on both provincial and federal levels of government.
Just last month, pharmaceutical company Vertex, the manufacturer of Trikafta made the decision to bring new CF medications to Canada.
According to a press release on the CF Canada website, in a recent meeting with Cystic Fibrosis Canada, the Federal Minister of Health committed to fast track Trikafta through Health Canada for approval and for pricing review. Once a submission is completed and Trikafta is approved by Health Canada, it goes to the pan-Canadian Pharmaceutical Alliance (pCPA), through which the public drug programs negotiate a price with the manufacturer.
"As you can imagine this is huge for our community, although we are fully aware that the PMPRB (Patented Medicine Prices Review Board) regulations are most certainly going to continue to substantially reduce the number of new and innovative therapies to Canada," said Beth. "That is indeed a challenge we will continue to deal with, but for now we will deal with the issue at hand."
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 the coverage by OHIP, her family could have spent 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.
"I can tell you this is not an easy feat as we have seen with previous modulators, Kalydeco and Orkambi which remain in negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA)," Beth said about Trikafta. "While negotiations have dragged on Canadian CF patients have continued to decline."
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 recent history, we have witnessed government both federal and provincial putting aside politics in order to work together to keep Canadians safe during the pandemic," said Beth. "It's incredible to see how effective a government united in a cause can be when dealing with a life and death crisis. We are hopeful that with the same effort we will see Trikafta glide through the messy red tape that is our drug approval system."
It was just over a year ago that Madi and Beth met with Ontario's 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. They also discussed the Trikafta drug, which had already been approved in the U.S.
"She (Elliott) expressed her desire to support our efforts in getting the current modulators into negotiations and that she would work to get Trikafta to patients as it was a 'no brainer'," explained Beth.
Kalydeco and Orkambi finally made it to the pCPA in June but have yet to be approved.
"Our fear is that Trikafta will find the same fate. Our hope is that Ontario will begin the process to ensure Trikafta does not come to the same fate as previous modulators. Our community is expecting that Trikafta will be made available to all CF patients that will benefit from it, despite their health status or any other discriminating qualifiers that may be attached to it that could impact patient access," said Beth.
The Vanstones have written to Elliott in hopes of a follow-up meeting to discuss expediting access to the drug at a provincial level but have yet to hear back.
Recently, MPP Jim Wilson raised the question at Queens Park and Beth says the ministers' response sounded promising.
"MP Terry Dowdall has also been working with us at the federal level and we are hoping that perhaps both Jim and Terry may find a way to put their heads together to help us push this through," said Beth.
When contacted by BradfordToday, a spokesperson for Minister Elliott noted that pCPA and Vertex are in ongoing dialogue and active negotiation with respect to CF treatments and details regarding the ongoing discussions and negotiations between the pCPA and manufacturers are strictly confidential.
"Provinces are encouraged to hear that Vertex is submitting Trikafta to Health Canada," read the response.
"This is an important step in the drug funding review process. As with all new drugs, the next step in the drug funding process would be health technology assessment, followed by negotiation through the pan-Canadian Pharmaceutical Alliance (pCPA). The CADTH health technology assessment is being conducted in parallel to Health Canada’s review to streamline and reduce timelines."
"We eagerly await health technology assessment and review of Trikafta. This is a complex negotiation involving multiple drugs. All negotiating parties play an important role in reaching agreement and achieving timely outcomes and both pCPA and Vertex Pharmaceuticals continue these efforts."
The Vanstones are proud of the efforts made by the CF Community so far, including their e-mail campaign which garnered over 5,500 letters sent to 99 percent of MPs in the country asking for the life-saving medications 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.
For more information on the fight for lifesaving medications, visit the CF Get Loud website here.
