Nigel Rawson and Beth Vanstone | CHP Opinions | 9 Sept 2021
Photo: Beth and Madi.
In an article in the Hamilton Spectator, we wrote that patients access to innovative medicines should be an election issue. Canadians have heard little to nothing from any political party in the campaign about how they will improve access to innovative medicines that can provide effective therapy for previously untreatable diseases but whose cost is frequently beyond the average Canadian’s ability to pay.
Some politicians and academics believe that a national pharmacare program relying on federal regulations to drastically reduce drug prices will resolve patient access issues. It won’t.
Beth’s daughter, Madi, has cystic fibrosis. Her urgent need, and that of many CF sufferers, for access to Trikafta is being ignored by politicians and their officials while CADTH considers its final recommendation for the drug (its draft recommendation restricts Trikafta’s use to only a few CF sufferers) and the national price negotiator eventually accepts the drug for negotiation. CF sufferers will die as these cumbersome processes grind on.
We wanted to include Madi’s own comments in our Spectator article, but it was not possible. However, it is crucial that politicians, bureaucrats and the public understand the human toll of devastating diseases like CF, so let’s hear from Madi.
I have cystic fibrosis. It is a terminal disease that mainly attacks the lungs, but affects the entire body. When I was diagnosed, my parents were told I would be lucky to make it to 25 years old. At the age of 10, my health started declining rapidly, so we started preparing for the worst. As hard as it is to admit, I gave up. I was tired of fighting just to be alive.
But just as time was running out, a medication called Kalydeco saved my life. Kalydeco has given me 9 years of life that I couldn’t be more thankful for. I have got to experience the world, graduate high school and start college, fall in and out of love, meet the most incredible people and make the best memories. I’ve got to live.
Unfortunately, as I’ve got older, my disease has been making a slow comeback. This past year in particular has been a struggle. I feel myself slipping back, and it’s awful. Recently my lung function dropped to 53 percent – the lowest it’s been since I was 5 years old. My doctors have recently expressed that it’s crucial I switch to a new medication called Trikafta before my condition worsens. This medication corrects both defective genes in my body (Kalydeco only corrects one), but isn’t funded for all Canadian CF patients that could benefit.
Gaining access to Trikafta is a huge fight for Canadians. Families and patients have been working non-stop to get this medication to those who need it. As I have been struggling silently, I’ve been watching the CF community come together and fight for what is right.
I just want to thank everyone who has contributed to this fight for Trikafta. You are all absolutely amazing and I can’t thank you enough for the work you have been doing to save not only my life but my friends’ lives. To my fellow CF’ers fighting, you ROCK. It’s exhausting fighting for your life and fighting the government at the same time. You are all amazing!
We are close to gaining Trikafta. I can’t wait to celebrate with all of you when this fight is finally over. I promise to do what I can to help finish this once and for all. Stay strong and keep fighting!
Unless you have ice in your veins, it is impossible to read Madi’s words without being moved, firstly, by her strength to keep fighting both CF and the system and, secondly, by the suffering caused by a disease that could be helped by access to an innovative medicine.
The suffering is not confined to the patient but extends to their family and friends. Her mother describes the impact and the gruelling fight against both CF and governments eloquently.
Fighting chronic illness is a life-changing experience not only for the patient but the entire family. The daily challenges of appointments, therapies and hospitalizations, along with investing time and energy into supporting efforts to raise money and participate in research to improve treatments and search for cures, is more than many patients and families can endure.
To invest personal resources in an effort for better outcomes for patients and then have to battle the government to access these life-changing, life-saving medications is inhumane. We have spent Madi’s entire life doing therapy, attending clinics, and enduring hospitalizations trying to save her.
The first CF modulator (Kalydeco) was the culmination of decades of fundraising and research, and it represented hope for CF patients. We had no idea that it would unlock the door to an unforeseen battle with our own government. We have been advocating for nearly a decade now, over half of Madi’s life, to access these medications. During the past decade, we have only seen changes that make the process nearly impossible for Canadian patients trying to access these medications.
As we watch countries around the world create solutions to make these innovative medications available for patients, Canada has thrown up additional roadblocks to ultimately prevent access to the majority of these medicines. As a parent and advocate, I can only see a future where, rather than being able to celebrate innovation and the benefits to patients, we will forever be embroiled in a battle for new and improved therapies.
It shouldn’t be this hard. Canada needs to invest resources in finding a fast and effective pathway to these medications.
When politicians and academics suggest that national pharmacare will improve access, you should realize that they haven’t a clue about the real toll of their obstacles to drug access on human health. A pharmacare program covering only a small number of medicines for common disorders, with a vague promise to phase in other medications over an unspecified number of years subject to regulations forcing severely reduced drug prices to make the program affordable, is not going to help individuals like Madi – it’s just false hope.
Patients whose health could be improved by innovative medicines will continue to suffer and die prematurely if Canada’s hostile attitude towards drug developers is maintained. Canadians need a federal government that adopts a collaborative relationship with the biopharmaceutical industry to ensure patients have access to new therapeutic advances now – and into the future.
Nigel Rawson is an independent researcher and an affiliate scholar with the Canadian Health Policy Institute. Beth Vanstone is a long-time advocate for Canadians with cystic fibrosis and a director with CF Get Loud. The views expressed are the authors’ own and do not necessarily represent those of organizations with which they collaborate.