A new life-saving drug to combat cystic fibrosis is now available and one Saskatchewan girl wants the province to act quickly to access the medicine.
Cassidy Evans, 12, formerly of Moose Jaw and now living in Saskatoon, encouraged the provincial government — specifically, Premier Scott Moe and Health Minister Jim Reiter — to work with Vertex Pharmaceuticals to acquire Orkambi and Trikafta, during a news conference in Regina on March 16 that the Canadian Cystic Fibrosis Treatment Society (CCFTS) organized. These are life-saving gene modulator medicines that the United States’ Food and Drug Administration (FDA) department has already approved.
Evans is known for raising awareness about cystic fibrosis (CF). She originally started a lemonade stand to fundraise for CF, but in 2017, she expanded that to include a mobile lemonade truck.
The new medicines are showing life-changing results for people who have received a clinical trial or have taken the drugs, Evans said. Even medical professionals recognize that these medicines are miracle drugs. However, Canada has not yet met with Vertex to negotiate a price to acquire the drugs.
The company said recently there is an offer available for a portfolio deal of all its medicines for one low price, but it is waiting for governments in Canada to negotiate a deal, according to CCFTS.
Canada is an outlier since only citizens with private insurance plans can access Orkambi, Evans continued. People who rely on public programs, including pharmacare or the Saskatchewan Drug Plan, are not allowed to have a treatment that could prolong and improve their lives.
“People living with CF are the most vulnerable to viruses like COVID-19, (so) the Government of Saskatchewan needs to take immediate steps to ensure this vulnerable population stays out of hospitals during this crisis,” said Chris MacLeod, founder and chair of the CCFTS. “COVID-19 is a real and pressing concern that cannot be ignored.”
While everyone fears catching the coronavirus, CF patients already experience breathing problems whenever they catch the common cold, Evans said. She coughs uncontrollably, experiences shortness of breath, acquires fevers and requires urgent health care. She must also isolate herself.
“If you (Premier Moe) believe that the health of Saskatchewan residents does come first, please give CF patients access to gene modulators, like Orkambi and Trikafta, so they can have a chance at beating COVID-19 or any other virus they encounter,” Evans said. “Without gene modulators, CF is 100-per-cent fatal.”
She challenged Moe to negotiate with Vertex for the medicines. She also expressed her gratitude to Moe for sharing her story with the province.
“This is not a difficult decision to make. It’s been made by politicians and government leaders in other major countries around the world, and it saves lives,” added Evans. “It’s time for Canada, and time for Saskatchewan, to make this life-saving drug available to CF patients in our country.”
Government response
New drugs are added to the Saskatchewan Formulary based on the advice from the national and provincial drug review processes in conjunction with pan-Canadian Pharmaceutical Alliance (pCPA) negotiations. The decision to list a medication on the formulary is based on both the clinical evidence and the cost-effectiveness of the drug, according to a statement from the Ministry of Health. This rigour is necessary to provide access to the best care while sustaining a publicly-funded drug plan for future generations.
Trikafta, a new cystic fibrosis medication that consists of three drugs (elexacaftor, tezacaftor and ivacaftor), is not yet available in Canada. To receive market approval for sale in Canada, the manufacturer has to submit information to Health Canada for review; however, this has not yet happened, the statement continued. Additionally, the manufacturer has not yet submitted clinical and cost information for review by the national health technology agency. As a result, Trikafta is not an eligible benefit under the drug plan at this time.
The national Common Drug Review reviewed Orkambi (lumacaftor-ivacaftor) and all the evidence submitted by the manufacturer was assessed. The review found there was uncertain clinical benefit with this medication, the statement said. However, Saskatchewan and select jurisdictions considered Orkambi further and have agreed on case-by-case criteria for Orkambi for children with cystic fibrosis who are rapidly deteriorating.
Broad coverage of Orkambi is not being considered at this time and the funding status will be monitored and re-evaluated if appropriate.
“The Ministry of Health recognizes and understands that drugs for rare diseases present many challenges for patients and their families, as well as the health care system,” the statement added. “New treatments and technologies are continually being considered and Saskatchewan will continue to collaborate with other provinces and territories on this complex topic.”
