Advocating Out Loud
July 16, 2021
Meet two cystic fibrosis (CF) patient advocates who have been vocal about what needs to change in the CF treatment landscape.
Stephanie Stavros, founder and co-director of the patient advocacy group CF Get Loud, has been fighting CF since birth. She made news in 2020 when she became the first person in Canada to be approved for compassionate care for Trikafta, after tirelessly lobbying to government and to the manufacturer of the drug. Now approved by Health Canada for selected CF patients, Trikafta costs about $420,000 per year.
Beth Vanstone, who shares the CF Get Loud director position with Stephanie, has been advocating for CF patients since her daughter Madi was born with the disease 19 years ago. After successfully lobbying for funding for Kalydeco, a $350,000/year medication targeted to people with Madi’s type of CF gene mutation, she continues to speak for CF patients facing treatment access barriers.
In this no-holds-barred chat, Stephanie and Beth talk about their triumphs, frustrations, and the fault lines in the Canadian access landscape.
On the journey to CF Get Loud
Stephanie: I spent the first 34 years of my life hiding my CF because I didn’t want it to limit me, but when I got to end-stage disease and found out my own country didn’t have my back, I decided to stop hiding. I’ve spent the last 2 years fighting for myself and the rest of the CF community. Launching CF Get Loud has been part of that process. The name of the group wasn’t chosen by accident: our community has to literally fight for the breath to speak up.
Beth: I’ll never forget when we entered Madi in a clinical trial of Kalydeco when she was 10 years old. Within two days I knew she was in the treatment group and within a month all her issues resolved. She told me, ‘Mummy, I can breathe through my nose.’ I joined CF Get Loud because I want other parents to experience moments like this.
On access to medications for advocates and fundraisers
Beth: You submit your child to test after test, you take trips to the hospital to do extra tests for research, you work to raise money. You do this for years. If we’re not going to get access to the drugs after all that, then why are we doing this? We have to help decision-makers understand that we’re part of this process and they have to meet us partway.
Stephanie: I’ve had a similar experience. From the age of 8 months to 10 years, I participated in clinical research every 3 months. I spent 37 years asking how I can help, doing fundraisers, galas, zoo walks, you name it. To be denied access to Trikafta after that felt like a kick in the gut. I’m obviously grateful to the manufacturer for coming through with compassionate care, but it breaks my heart to see other children and adults in hospital with deteriorating lung function.
On the PMPRB’s new pricing regulations, coming into effect January 2022
Stephanie: Two of our CF medications, if subjected to these regulations, would require a 99% price reduction to come into Canada. No manufacturer can possibly offer that, so what ends up happening is that the regulations prevent or delay the introduction of drugs like Trikafta into the country.
Beth: Unrealistic price limits aside, the PMPRB rules have created an extra layer of bureaucracy in a country that’s already hard for pharma manufacturers to do business with.
On the HTA assessment process in Canada
Stephanie: The average time from Health Canada approval to listing is 600 days. People don’t realize the layers of red tape involved. When patients new to the game learn of a drug submission and start celebrating, I secretly think, “if you only knew.”
On OBA-type agreements for rare disease drugs
Stephanie: Innovative agreements like OBAs definitely play a role. When a drug shows great promise but there is uncertainty around efficacy or price, it makes sense to provide the drug first, track the data, and then review and adjust access criteria and price accordingly. It’s a common-sense approach for people who need quick access.
Beth: If an OBA can get a baby with a neuromuscular disease on a drug more quickly, so they’ll still be able to move their legs by age 2, let’s do it! Right now the pharma companies are saving many lives through their compassionate care programs, but we need more equitable ways to share the risk and OBAs can do that. There is an opportunity to collect data on how patients are doing with these medications and use the data to drive access.
“If an OBA can get a baby on a drug quickly so they can still move their legs by age 2, let’s do it!”
On the role of patient advocates in shaping policy
Beth: I see our role as educating policymakers, who are often unaware of how this or that regulation affects patients. I’ve learned that you have to be both specific and persistent as an advocate. Know what you’re asking for and why—and don’t accept offers that make no sense. For example, at one point I was told Madi could access Kalydeco if she was in the hospital—but the whole point of the medication was to keep her out of the hospital.
Stephanie: You’re touching on something very important, which is that the metrics and cut-offs used in our current system don’t make sense for rare diseases. For example, the average gain of lung function on Trikafta is 14 percent. This may not seem like much to the average person or to regulators, but to someone like me it’s the difference between a transplant, supplemental oxygen, and normal life.
On the importance of patient-reported outcome measures (PROMs)
Stephanie: PROMs should be part of the conversation with assessors and payers because they reflect quality of life, and what’s more important than that? With CF you’re slowly drowning in your own lungs. If you can come up for air, all kinds of things improve in your life. PROMs capture that.
“Patient-reported outcome measures reflect quality of life, and what’s more important than that?”
On the drive to keep fighting for CF patients
Beth: We won the two-year battle with the provincial government and got funding for everyone with Madi’s gene type, but it didn’t feel right to stop there. Plus my in-box is flooded with questions from other parents: when, what, how… the least I can do is help make things fairer for this vulnerable community.
Stephanie: I feel like I’ve won the life lottery. I have a new shot at life, which is the biggest gift of all. I don’t want to waste this opportunity, so I plan to stay loud!