Mr. Chair and members of the committee, thank you for the opportunity to appear before you to share my personal views about the Patented Medicine Prices Review Board and my personal experiences as a rare disease advocate.
My name is Erin Little. I am the president and co-founder of Liv-A-Little Foundation.
Liv-A-Little Foundation was founded in 2013, two years after our daughter, Olivia, was diagnosed with the rare disease cystinosis. We are a volunteer-run organization committed to supporting the advancement of treatments and, ultimately, a cure for cystinosis.
Procysbi is an excessively-priced drug that was flagged by the PMPRB in 2017. It's a perfect example of how the PMPRB protected patients. I have provided you with the PMPRB statement of allegation regarding Procysbi.
The Cystinosis Research Foundation funded every bench study and early clinical trials at UCSD, which resulted in the development of a slow-release form of cysteamine. In 2016, Raptor Pharmaceutical was bought by Horizon for $800 million. Procysbi is the result of a business deal, not R and D. How many other companies are using this same business model to build their portfolios?
As members of Parliament argue about the PMPRB spending $56,000 to invest in an effective communication plan, I would recommend they be more concerned that in 2020, Horizon's CEO raked in $21.63 million U.S., which is more than the PMPRB's annual budget. Let us be honest, the PMPRB can use all the help it can get when it comes to educating patients and Canadians on what and why it does what it does.
In 2019, Recordati brought the new, non-patented drug Cystadrops to the Canadian market with a sticker price of $120,000 a year. No patent meant no protection from the PMPRB review board. Cystadrops is another example of taking an old drug, tweaking the delivery mechanism and marking it up by 4,000%.
Recordati's GM and I were discussing the price. He admitted the drops would be expensive and then proceeded to tell me that all they're asking for is a little bit more from each taxpayer to cover the cost of a small population of patients. I sat there in dismay for two reasons. First, I was in shock that pharma prices drugs based on what the market will bear and not on what it cost to make them. The second was that I am one of those taxpayers.
What are we going to do about this? Are we printing money to continually pay for high-cost drugs? If we are, then let's print it for clean water, for the 215 bodies just found, for the homeless, for the kids who go to school hungry, for mental health services and for LTC homes. When we pay for excessively priced drugs, the money needs to come from someplace, which means someone else goes without. If and when we hold big pharma accountable to charge what the drug is worth instead of what the market will bear, we all win.
We are all familiar with the term “grooming” when it's referencing sex trafficking children, but no one is yet talking about how pharma is grooming advocates. Grooming is when someone builds a relationship, trust and emotional connection with someone so they can manipulate, exploit and abuse them.
In November 2017, Horizon Pharma invited us and a select few Canadians to a round table meeting. The meeting wasn't about our concerns; it was about building a common advocacy voice shaped by Horizon. I have provided you with the agenda from that meeting along with a letter of intent. That meeting is an example of grooming patients.
Horizon has also created the platform RAREis on Instagram. RAREis gives patients, advocates and families a place to share their stories and be heard. This is a perfect example of building trust and emotional connection with vulnerable advocates, which leads to manipulation and abuse. The repercussions of these relationships mean that advocates turn against our government.
In October 2018, I sat at this table and heard what it would cost to keep Olivia alive based on the average life expectancy at that time. It was hard to hear this, as a mother who wants nothing more than to outlive her child. I also understand why she had to bring that cost to the committee's attention: High-cost drugs are not sustainable in our health system.
I want to share with you the current cost faced by two different families living with cystinosis. Family A has a Procysbi cost of $56,000 a year and Cystadrops is $120,000. For family B, Cystagon costs roughly $18,000 a year and compounded drops are just under $3,000. All four of these drugs are made with cysteamine.
The first evidence regarding the therapeutic effect of cysteamine on cystinosis dates back to the 1950s. The delivery is changing, but not the ingredients. Is the change in the the delivery mechanism worth a 4,000% increase?
In conclusion, I support the implementation of the new guidelines and I strongly suggest we look hard at advocacy groups that are funded by industry and question whether they are being groomed by the industry. I strongly believe that every patient deserves to access drugs that are safe, effective and affordable.
Health Canada needs to do a better of job of listening to the patients, caregivers, and organizations not funded by pharma. We once trusted our child's life in the hands of a Canadian organization only to be betrayed, as their agenda was to support the industry. This organization happens to receive funding from both pharma companies treating cystinosis patients in Canada.
I will end with this. I am not against for-profit companies, but I am against the greed, manipulation and control they hold over the lives of every Canadian. Our child like many others would die without access to these drugs. I remember the days when I feared if Olivia would live. Now I fear if she will be able to afford to stay alive.
Thank you, and I'm willing to answer questions.