CADTH DRAFT Review Released
On July 8th, CADTH posted their draft recommendation on their website.
What impact does the CADTH review have?
“The objectives of CADTH’s reimbursement review processes are to reduce duplication across jurisdictions, maximize the use of limited resources, and enhance the consistency of drug reviews. CADTH undertakes reviews of drugs and issues reimbursement recommendation and/or review reports to all federal, provincial, and territorial drug programs and cancer agencies that participate in CADTH’s review processes and Canadian Blood Services (together hereafter referred to as “drug programs”). It is important to note that CADTH’s recommendations are non-binding to the drug programs.
Each drug program makes its own reimbursement decisions based on the CADTH’s recommendation, in addition to other factors, including the plan’s mandate, jurisdictional priorities, and financial resources.”
It’s important to note that although each province will take the CADTH recommendations into consideration, they CAN move forward and go against these recommendations and say YES to Trikafta without these recommended conditions. Each province will need our community to GET LOUD and educate our elected officials on why it should be our doctors alone that decide whether a patient can and should benefit from Trikafta.
Conditions for Reimbursement
1. You need a lung function (FEV1) lower than 90% to be able to access Trikafta
The draft criteria demonstrates that the organization does not fully understand the complex nature of cystic fibrosis. CF is not simply a lung disease – it’s a genetic disease that impacts multiple organs, the digestive and reproductive systems, and affects bone density, hearing, and sinuses.
Cystic fibrosis is a disease that greatly varies in severity from person to person. Many patients have ‘normal’ lung function but suffer from sever debilitating digestive issues including life-threatening bowel obstructions. Trikafta has significantly reduced digestive issues in CF patients. In fact, 1 in 5 babies are born with a bowel obstruction in the small intestine called, Meconium Ileus.
Cystic fibrosis is a progressive disease. Most patients begin life with healthy lungs and pulmonary function levels of 120%. The current CADTH criteria require these patients to lose 30% of their lung function before they can benefit from a gene modulator that helps all aspects of the disease. Instead of patients staying healthy and using a preventative approach, they are being asked to allow CF to permanently scar their lungs and succumb to the devastation of structural changes to their lungs from the disease before being considered for Trikafta. Lung tissue is extremely delicate and often lost lung function cannot be recovered.
Pulmonary Function Tests are not our CF care team’s most accurate way to evaluate lung damage. Even in patients with a high FEV1, a CT scan will show extensive damage to the airways.
QUOTES FROM CF FAMILIES:“My lung function used to be over 90%, so I would not have qualified for Trikafta, given the new recommendations by CADTH. While my lung function was great, my GI issues were severe; so severe, I ended up sedentary for nearly two years, after which my PFT dropped by 40%. Five years later, I have only been able to gain 20% back. To disqualify someone based on lung function is so blatantly discriminatory, it is mind boggling to think this recommendation was actually made public.”
- Courtney Masters, CF patient“Born in 1983 with meconium ileus, I have had severe digestive issue for 36 years. Prior to being prescribed Trikafta, I would have monthly bowel obstructions and frequent emergency hospital admissions. One bowel obstruction nearly took my life in 2015. My life was focused around pain management, special diets, feeding tubes and hospital admissions. Since starting Trikafta a 18 months ago, I no longer experience severe digestive issues. For the first time in my life, I digest fatty foods and I am have a normal BMI. My liver levels and my bone density have improved due to proper digestion and I no longer need pain management.”
-Stephanie Stavros, CF patient
“My son has decent lung function baseline 94% but horrible GI effects, extreme difficulty gaining weight and CF Related Diabetes. We have been dreaming of Trikafta hoping for some relief but am discouraged that his access may be contingent on his lung function declining. So if he falls below 90 and increases to above 90, will he lose access?”
– Cheryl Ann Smith, CF Parent
”My teenage son is already threatening to stop doing his treatments so that he will be able to qualify for Trikafta and it’s breaking my heart.
- CF mother
2. Renewal Criteria: sustained improvement of lung function (5%) after the first year, and every year going forward
A 5% decline in lung function can be caused by existing damage in the lungs. Studies show Trikafta reduces lung infections by 60%. Patients will still experience life-altering infections due to the aggressive nature of the disease that will deteriorate lung function.
Adults with cystic fibrosis culture antibiotic-resistant bacteria and have structural changes to their airways. Many CF patients experience collapsed lungs due to extreme damage. A gene modulator cannot change the permanent impact of the disease that happened prior to the release of Trikafta. CF patients that have damaged lungs will unfortunately still experience disease progression, thankfully at a much slower rate thanks to Trikafta.
A 5% decline in lung function can be caused by reasons outside of the CF diagnosis: aging, a cold, COVID, allergies, a collapsed lung due to impact, pregnancy, other disease etc. Trikafta cannot protect against external factors that will impact lung function. Unfortunately, CF does not happen in a vacuum.
A very important criteria to be noted: there is currently NO transplant patient access recommended by CADTH.
A double lung transplant is a treatment for end-stage cystic fibrosis but it is not a cure. CF patients that have received double lung transplants suffer from digestive issues, CF related diabetes, low bone density, low BMI and sinus infections. In some cases, sinus infections can travel down to the new lungs and begin to negatively impact lung function. We need our doctors to be able to decide who will benefit from Trikafta.
“Since having my transplant, I have had multiple, painful sinus surgeries. During my last appointment, we discussed removing more bone in my face to ease the pressure. I asked my doctor about the possibility of Trikafta if it comes to Canada and he gushed about how he’s seen all the proof of complete sinus improvement with it. So needless to say I have some renewed motivation to fight to change the restrictions around its use in Canada.”
- CF patient and Double Lung Transplant Recipient
What does this mean for access?
Private Insurance:
It is our understanding that it is highly unlikely this will affect private coverage, but we are certainly tracking it and look to you, the community, for updates in your own conversations with your insurers. Please share all that you learn along the process so that we can help amplify it in order to help others along this path.
Public (Provincial) Plans:
This is only a draft recommendation, so stakeholders like CF Get Loud, CF Canada and CF Treatment Society, as well as Vertex, have the ability to push back. End of the stakeholder feedback period is July 22, 2021. We will plan a comprehensive response and look to you to continue working with us in our advocacy efforts at all levels and social media platforms. We are in communication with the other CF organizations and will be pushing back privately and publicly, so stay tuned for future initiatives (Social Media, Twitterstorms, etc).
The manufacturer will ask for a reconsideration so this mean the timeline for public access will be slightly delayed by an additional month or so. In speaking with the manufacturer, they said they intend to get it right at the CADTH level so that our community does not have to fight these restrictions at the individual provincial level.
It is our understanding that they have successfully pushed back on similar conditions in Europe and have enough data to support overturning these conditions.