Complex patients in a complex world
October 16, 2019
How much difference a single year can make. In 2019 alone, policy changes have torn through the fabric of specialty medicine, keeping stakeholders on their toes all year long.
For starters, the federal government’s 2019 budget included steps toward a national Pharmacare program, including a national drug agency, a national formulary of evidenced-based drugs, and a strategy for high-cost drugs for rare diseases.1 Along similar lines, the Patented Medicine Prices Review Board (PMPRB) announced a series of reforms to make drug pricing more transparent, enabling the agency to set price ceilings and make specialty medicines more affordable to Canadians.2
As expected, not all manufacturers are on board with these changes. Indeed, a group of manufacturers plans to take the federal government to court over the PMPRB reforms, maintaining that Ottawa lacks the constitutional authority to set price ceilings.3
Changes have also rattled the world of biologic medications, spearheaded by the BC government’s expanded use of biosimilars—functionally equivalent versions of originator biologics that cost up to 50 percent less—within its provincial formulary.4
These upheavals have also nudged patients into new territory. As they brave increasingly complex treatment regimens and stick-handle around access hurdles, they are finding their voices and seizing opportunities to speak up. So how are they handling it all? How much sway do they have in their own treatment? Are their voices landing in the right ears?
As the science and business of specialty medicine charges ahead, stakeholders need to keep an eye on the patient perspective—to shine a light on what patients want, lack and need, and what drives them to push forward.
Engaged to the max
The term “patient engagement” may have buzzword status, but the phenomenon is real. Today’s patients not only have professional-level knowledge about their disease, but also keep up with policy changes—and speak up when they disagree.
When the PMPRB announced its pricing reforms, the patient-led Canadian Organization for Rare Disorders (CORD) responded with a flurry of news releases and marketing campaigns to communicate their dismay. They didn’t mince words, either. “Either the federal government can’t understand the disastrous impact on patients with high needs or they simply don’t care,” said CORD president Durhane Wong-Rieger in a press release.6 Concerned the price drops required by the new regulations would dissuade pharma companies from playing in the Canadian market, Wong-Rieger predicted the changes would “compound our existing drug shortages”—and more importantly, hurt patients.6
In response to the outcry, the PMPRB assured stakeholders that it will be “more forgiving in the ceilings that we apply to [rare disease] drugs, because we want to be consistent with the government’s broader commitment to trying to facilitate access.”7 While the chips have yet to fall, the ongoing dialogue exemplifies patients’ burgeoning role in shaping policy.
The sea change goes both ways: more patients want to speak up, and more policymakers want to hear from them. Following a call for nominations, the Committee for the Assessment of Devices and Technologies in Health (CADTH) assembled a new Patient and Community Advisory Committee composed of 12 patients and caregivers of diverse backgrounds.8 According to a CADTH press release, the committee will “advise CADTH on all areas of the agency’s work”—an ambitious vision that, if all goes well, will help steer CADTH toward more equitable and appropriate recommendations.8
A full-time job
With all the opportunities to weigh in on research, access and policy, people with complex diseases can make a career of being a patient—and some of them do just that. Take Lisa Machado, for example. About a decade ago, Machado developed Chronic Myelogenous Leukemia (CML), a type of leukemia diagnosed in 500 Canadians every year.9 Finding no group dedicated to the disease, Machado created one of her own. Called the Canadian CML Network, the group is working on putting together a Task Force on Rare Cancers, working on a peer-to-peer training program, and joining forces with global oncology organizations on advocacy initiatives.10
Machado herself has launched Canada’s sole educational conference on CML, created and deployed a Novartis Oncology-backed social media campaign on leukemia, and written an award-winning CML educational resource. Along the way, she has also become a sought-after public speaker, invited by such leading broadcasters as CBC and CTV to share her perspective as a patient.10
Machado’s passion comes through in every word she says and writes about the patient experience. From an article titled Lives Depend on Patient Engagement:11 “When I laud an institution for attempting patient engagement, it’s because I have seen the other side of the bed and know without a doubt that involving patients is critical to a system that works and achieves better outcomes.”
As if advocacy leader, public speaker, and medical journalist weren’t enough, some patients and their families reach into the science of medicine itself. After learning there was no effective treatment for his young daughter’s ultra-rare neurologic disease, alternating hemiplegia of childhood, a US real estate developer called Simon Frost—with no background in science—set about developing a gene therapy for the disorder.12 Now an inventor with international patent applications, Frost has also become a project manager who coordinates the research.12 As Frost’s trajectory exemplifies, today’s patients have limitless means to make change—and many devote their lives to the effort.
It gets complicated
Hope is what drives patients to get involved, and new specialty medicines offer hope to the most treatment-resistant patients. But this promise comes with a high level of complexity, making it critical to set things up so patients can benefit from the innovations without undue strain on their time, energy, and mental reserves.
Complexity may crop up before treatment even begins. Eligibility for the recently-approved anti-cancer drug Vitrakvi, for instance, rests on confirmation of a very specific gene mutation, requiring patients to undergo genetic testing to find out if they qualify.13 With similar treatments multiplying in the pipeline, hospitals and clinics will need to expand their genetic testing infrastructure to meet the increasing demand.
Eligibility hardly guarantees access, of course. In a striking illustration of the difference, André and Joshua Larocque, two young brothers living with cystic fibrosis (CF) in Tottenham, Ont., have found themselves on very different treatment paths. André, 8, was selected to enter a clinical trial of a promising new CF medication called Symdeko. Health Canada has already approved a similar drug, Orkambi, but the province won’t pay the $250,000 annual tab for six-year-old Joshua to take it, based on a negative recommendation from CADTH.14 The decision doesn’t sit well with the boys’ parents, who have been lobbying the province to fund the medication.15 For better or worse, the realities of specialty medicine have thrust the Larocques into challenging new roles.
As specialty medicines keep tumbling into the market and policies continue to shape-shift, patients will raise their voices still further, blurring the lines between healthcare user and advisor. As Machado put it in a recent online article, “Patients and caregivers are experts in their field, like any consultant. Why are we still debating whether or not to compensate this expertise?”16 One thing we know for sure: the patient voice is here to stay, and stakeholders need to listen more closely than ever.
References
Moving forward on implementing National Pharmacare. Government of Canada Budget 2019, last updated March 19, 2019. https://www.budget.gc.ca/2019/docs/themes/pharmacare-assurance-medicaments-en.html
Government of Canada Announces Changes to Lower Drug Prices and Lay the Foundation for National Pharmacare. Government of Canada, last updated August 9, 2019. https://www.canada.ca/en/health-canada/news/2019/08/government-of-canada-announces-changes-to-lower-drug-prices-and-lay-the-foundation-for-national-pharmacare.html
B.C. expands use of biosimilars to offer coverage for more treatment options. BC government news release, May 27, 2019. https://news.gov.bc.ca/releases/2019HLTH0080-001072
Grant K. Pharmaceutical companies file constitutional challenge to Canada’s new drug price regulations. The Globe and Mail, August 23, 2019. https://www.theglobeandmail.com/canada/article-pharmaceutical-companies-file-constitutional-challenge-to-canadas-new/
Canadian Association for Rare Disorders (CORD). https://www.raredisorders.ca/
New federal drug pricing regulations mean disaster for Canadians with rare diseases. CORD media release, August 9, 2019. https://mailchi.mp/69ff2dfd9678/new-federal-drug-pricing-regulations-mean-disaster-for-canadians-with-rare-diseases?e=[UNIQID]
Martell A. Canadian drug price regulator may be flexible on rare diseases. The Globe and Mail, August 13, 2019. https://www.theglobeandmail.com/business/article-canadian-drug-price-regulator-may-be-flexible-on-rare-diseases/?cmpid=rss
New committee brings diverse patient, family, and caregiver voices to CADTH. CADTH e-alert, July 10, 2019. https://mailchi.mp/cadth.ca/e-alert-mittmann-932637?e=490d3d5e27
Understanding CML. The Canadian CML network. https://cmlnetwork.ca/i-have-cml-now-what/#understandingcml
Machado L. LinkedIn profile. https://www.linkedin.com/in/lisa-machado/
Machado L. Lives depend on true engagement. LinkedIn, August 5, 2019. https://www.linkedin.com/pulse/lives-depend-true-engagement-lisa-machado/?trackingId=Uq2N8AcJjDXZAe5VDhC0hg%3D%3D
Broder J. His daughter Annabel has a rare disorder. He’s developing a novel gene therapy. The Washington Post, September 3, 2019. https://www.washingtonpost.com/health/his-daughter-annabel-has-a-rare-disorder-hes-developing-a-novel-gene-therapy/2019/08/30/e47a1678-c80e-11e9-be05-f76ac4ec618c_story.html
Vitrakvi product monograph (Bayer): https://www.bayer.ca/omr/online/vitrakvi-pm-en.pdf
Crowe K. 2 young Canadian brothers, a life-threatening disease — and the harsh reality of drug prices. CBC news, May 11, 2019. https://www.cbc.ca/news/health/cystic-fibrosis-vertex-cadth-orphan-drugs-prices-health-technology-assessment-1.5132294
Pritchard B. Parents of Tottenham boys with cystic fibrosis pleading with province to fund high-priced drug. Simcoe.com, April 1, 2019. https://www.simcoe.com/news-story/9245706-parents-of-tottenham-boys-with-cystic-fibrosis-pleading-with-province-to-fund-high-priced-drug/
Machado L. LinkedIn commentary. August 2019. https://www.linkedin.com/posts/lisa-machado_should-money-come-into-it-the-decision-activity-6564864266598522880-fcDM