Drug Approval Process
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Trikafta was submitted to Health Canada for priority review on Dec 4th, 2020.
It is imperative that Trikafta be approved in the broadest possible indication so that EVERY patient in Canada that can possibly benefit from this medication has access.
We cannot have restrictive prescribing criteria on this medication. We are working to make sure Trikafta is not viewed as a medication to be used reactively or as a last resort, but rather proactively as part of a standards of care treatment plan by our CF physicians.
Our goal is to make sure that patients with any mutation that is responsive to the medication (at least one f508del mutation) get access to Trikafta regardless of second mutation.
CADTH and INESSS in Quebec have confirmed as of December 17, 2020 that they are reviewing Trikafta and completing the health technology assessment (HTA). We have been advocating for an aligned review so that these processes run simultaneously in order to shorten as much as possible the amount of time patients will have to wait before having access.
The same considerations listed above regarding approval and prescribing criteria apply to the CADTH/INESSS review: Trikafta for ALL.
The pan-Canadian Pharmaceutical Alliance (pCPA) is currently in negotiations with Vertex for Kalydeco and Orkambi, the first and second generation of Vertex’s gene modulating drugs that lef to the development of Trikafta. This is great news, but also an example of the underlying challenges. Kalydeco was first approved by Health Canada in 2014 and some patients that can benefit from this almost decade-old modulator (FDA approved Kalydeco on January, 2012) still have barriers to access due to restrictive prescribing criteria.
We are working on making sure all provincial ministers of health commit to listing Trikafta, along with the other modulators, in order to allow our CF physicians to prescribe what they feel would be appropriate. We have an excellent CF registry and there is no risk to overprescribe due to the genetic data being readily available.
Our goal is to work within the current negotiation framework to have the pCPA negotiate a price for Trikafta ahead of regulatory approval so there is no wait time between approval and access.
Regardless of pCPA negotiation status, each individual province/territory ultimately has to make the decision to negotiate one final time with Vertex and enter into a Product Listing Agreement (PLA). It is imperative we continue working with our MLAs/MPPs to raise this issue provincially and pressure each Minister of Health to list this drug in the public formulary.
Useful Links
Patented Medicine Prices Review Board (PMRPB)
Canadian Agency for Drugs and Technologies in Health (CADTH)
Institut National d'Excellence en Santé et Services Sociaux (INESSS)
pan-Canadian Pharmaceutical Alliance (PCPA)