Trikafta is a new and innovative drug parented by American company, Vertex Pharmaceutical. A triple combination precision medicine, Trikafta treats the genetic F508del mutation which is the most common cystic fibrosis mutation accounting for approximately 90% of patients. Use of the medication has lead to an average 14% increase in lung function in patients compared to those not being treated.
Trikafta has been acknowledged by leading medical professionals as a disease-modifying “miracle” drug that dramatically reduces the number and severity of pulmonary exacerbations. It effectively leads to less hospitalizations and an overall improved lung function for patients with cystic fibrosis, which has a cascading effect on a patients physical and mental well-being. It is the single biggest advancement in treating cystic fibrosis in the history of the disease.
For more information, visit the Cystic Fibrosis Canada webpage on Trikafta.
This July, both the United Kingdom and the European Union struck deals with Vertex Pharmaceuticals to make Trikafta accessible to citizens across Europe. In fact, Canada is the only G7 country that has yet to take the necessary steps to allow its citizens to access the medication. It was even given priority status in the United States in August 2019 once it was declared a breakthrough therapy by the FDA and was approved for use a short time after. Trikafta's inaccessibility in Canada is due to the manufacturer's fear of new regulations that Canada is poised to make under the PMPRB (Patented Medicine Prices Review Board). Due to COVID-19, the PMPRB has extended the deadline to implement changes that would have an impact on the accessibility of Trikafta. The new date is January 1st, 2021, allotting some time for bargaining with stakeholders.
According to Cystic Fibrosis Canada, the PMPRB determines the maximum price a company can charge for a new patented medicine sold in Canada. The board has already made recent regulatory changes to its mandate, ultimately preventing medications like Trikafta from qualifying for approval. These changes are to officially come into effect in January, but the board has already exercised its new regulations when considering new therapies in Canada. This is essentially the reason that Trikafta has yet to be brought forward to Health Canada for approval as the likelihood of its marketability in the country is slim. The changes implemented by the board is the first challenge among m
Following approval, prices are then negotiated between the manufacturer, private, and public insurers. The pCPA (pan-Canadian Pharmaceutical Alliance) negotiates on behalf of the provincial, territorial and federal programs to determine the price that will be paid.
On May 6 2020, access to Trikafta was raised by an MP at the fourth meeting of the Special Committee on the COVID-19 Pandemic. Rather than address the issue with a meaningful response, Health Minister Patty Hajdu suggested that cystic fibrosis patients continue to apply for compassionate care from Vertex. However, this is not an easy process and many of these patients have since tragically passed away while waiting for government action, never being given the chance to experience the benefit of a life-changing medication.
As of May 20th, 2020, over 17,000 Canadians signed a petition calling for the PMPRB to revoke the changes it has made toward access to medications in Canada that will be adopted in July. These changes will restrict access to Trikafta for cystic fibrosis patients and the results of the petition have yet to be addressed in Parliament.
Many MPs have since joined the fight alongside cystic fibrosis patients to gain access to Trikafta, but there is still work to be done. Both the federal and provincial governments need to put pressure on health ministers to fast-track the bureaucratic process of drug approval. We support Cystic Fibrosis Canada in its advocacy for a drug pricing system that enables faster access to Trikafta, and broader, less restrictive access to other cystic fibrosis treatments.
Thanks to the help of our generous friends and family, we were fortunate enough to raise over $2,500 this year towards the work of Cystic Fibrosis Canada and the Walk to Make Cystic Fibrosis History!
Along with his family and friends, Aaron has been active on keeping up-to-date on developments regarding Trikafta. We have been contacting Members of government to raise awareness and will continue to pressure them to promote access to this important medication. We have requested meetings and have been in touch with the media to try and bring further attention to this issue. We strongly believe that with public pressure on this issue though increased written correspondence and petition signatures, the government will be unable to ignore the demands of the cystic fibrosis community.
We will continue to post our progress and updates on Trikafta.
Below you will find articles and sources related to media surrounding Trikafta. We will be updating these sources regularly.
’Miracle’ cystic fibrosis drug approved in U.S., Canadian trials underway (October 29, 2019)