Canadians with rare disorders will be even worse off if NDP’s parliamentary blackmail works
Last month the federal NDP convention in Hamilton voted unanimously to force the Liberals to introduce a single-payer universal pharmacare program or see the current “confidence-and-supply” deal canceled. Will universal government-run pharmacare benefit Canadians with rare disorders? We fear not.
Canadians with such disorders are already disadvantaged compared with sufferers in other countries. Fewer specialized drugs are launched in Canada than in the United States and Europe. Those that are get approval for marketing about a year, on average, after they do there.
That’s not because Health Canada takes longer to review new medicines. The process takes about the same time in the three places. Rather, delayed approval is likely due to manufacturers submitting later to Health Canada because federal, provincial and territorial hostility towards the industry has made our biopharmaceutical market less attractive.
Approval doesn’t mean government drug plans will pay for a drug, however. Further government-created barriers impact all Canadians, but particularly those with rare disorders who want access to novel drugs for their unmet or poorly met health needs. As a consequence, what gets listed in government drug plans varies widely, leading to a postal code lottery.
In a set of articles published over the summer by the Macdonald-Laurier Institute, we discuss the several obstacles patients and their families face as they try to gain access to new or expensive innovative therapies. They include: the lack of federal incentives for developers to submit new medicines to Health Canada; health technology assessment that is neither accountable, independent nor transparent and makes recommendations about which drugs to cover in public drug plans to governments; and price negotiations between government drug plans and manufacturers.
Even when drug developers clear these government-created barriers, public drug plans are under no obligation to add the approved medicines to their benefit lists. Too often governments focus only on drug costs and ignore the broader benefits effective drugs can bring, not only to the health and well-being of patients and their families, but also to other parts of the health system, to the economy and to society at large. If a new drug reduces doctor or emergency visits or hospitalizations or helps a person get back to work, those benefits typically are ignored by our drug assessment system.
The federal government made matters worse over the past six years by planning to drastically reduce drug prices by regulatory order, not negotiation. This caused considerable uncertainty among developers, resulting in even fewer new drugs being submitted for marketing approval here than in the U.S. and EU.
Proponents plainly want a lowest-common-denominator government-run public plan that would crowd out private plans, which over two-thirds of Canadians currently rely on for drug access.
Despite the federal government committing $1.5 billion over three years to “increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada,” its initiative is not comprehensive. So far, Canada has neither a government-endorsed national rare disorder strategy nor an Orphan Drug Act providing incentives to developers to launch orphan medicines in Canada. Most other developed countries have both.
Patients’ organizations have stepped in where governments have failed to act and proposed a Canadian strategy that would include incentives and funding to encourage developers to launch drugs in this country and cut through the barriers we have described to provide timely access to the many innovative treatments on the research horizon. For example, access to breakthrough drugs could be allowed as soon as Health Canada says they are safe and effective, even as other administrative boxes are checked and prices negotiated. Other countries use this approach.
Canadians afflicted with any of the 11,000 or so known rare disorders have significant unmet needs. Fewer than five per cent have any treatment beyond symptom relief or palliative care. The last thing these people need is for governments to ration innovative drugs even more than they already do or to force even deeper price cuts from drug developers in order to pay for universal pharmacare that covers only basic medicines.
Canadians with rare disorders almost certainly will be even worse off if the NDP’s parliamentary blackmail works.
Nigel Rawson is an affiliate scholar with the Canadian Health Policy Institute and a senior fellow with the Macdonald-Laurier Institute, as is John Adams, co-founder and CEO of Canadian PKU and Allied Disorders Inc.
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The FDA old guard criticized the new leadership in a Dec. 3 New England Journal of Medicine (NEJM) letter over a higher regulatory bar for vaccines, namely the expectation that most new vaccine approvals will require randomized clinical trials, arguing it could hamper the market.
“Insisting on long, expensive outcomes studies for every updated formulation would delay the arrival of better-matched vaccines when new outbreaks emerge or when additional groups of patients could benefit,” the former commissioners wrote. “Abandoning the existing methods won’t ‘elevate vaccine science’ … It will subject vaccines to a substantially higher and more subjective approval bar.”
But while the former commissioners disclosed their conflicts of interest to the medical journal — per standard practice in scientific publishing — reporters didn’t relay them to the broader public in reports in the Washington Post, STAT News and CNN.
The headlines about a bipartisan rebuke from former occupants of FDA’s highest office give the impression that the Trump administration is contravening established science, but closer inspection reveals a revolving door between pharmaceutical corporations and the agencies overseeing them.
Three of the signatories have received payments totaling $6 million from manufacturers or former manufacturers of COVID vaccines.
Scott Gottlieb has received $2.1 million in cash and stock from his position on the Pfizer board of directors, where he has advised on ethics and regulatory compliance since 2019, according to company filings to the Securities and Exchange Commission. Stephen Ostroff has received $752,310 from Pfizer in consulting fees since 2020, according to OpenPayments.
Gottlieb and McClellan did not respond to requests for comment. Ostroff could not be reached for comment.
FDA Center for Biologics Evaluation and Research Director Vinay Prasad outlined the higher standards and shared the results of an internal analysis validating 10 reports of children’s deaths following the COVID-19 vaccine in a Nov. 28 memo to staff. He called for introspection and reform at the agency.
The NEJM letter criticizes Prasad for cracking down on a practice called “immunobridging” that infers vaccine efficacy from laboratory tests rather than assessing it through real-world reductions in disease or death. The FDA under the Biden administration expanded COVID vaccines to children using this “immunobridging” technique, extrapolating vaccine efficacy from adults to children based on antibody levels.
Norman Sharpless — who in addition to previously serving as acting FDA commissioner also served as the head of the National Institutes of Health’s National Cancer Institute — consults for Tempus, a company that collaborates with COVID vaccine maker BioNTech. He has helped steer $70 million in investments in biotech through a venture capital firm he founded in November 2024. Sharpless also disclosed $26,180 in payments in 2024 from Chugai Pharmaceutical, a Japanese pharmaceutical company that markets mRNA technology among other drugs, on OpenPayments.
“I was grateful for the opportunity to serve as NCI Director and Acting FDA Commissioner in the first Trump Administration, and strongly support many of the things President Trump is trying to do in the current Administration,” Sharpless said in an email.
Margaret Hamburg, another former FDA commissioner and signatory of the NEJM letter, has since 2020 earned $2.8 million as a member of the board of Alnylam Pharmaceuticals, which markets RNA interference (RNAi) technology.
Hamburg did not respond to a message on LinkedIn.
Most signatories disclosed income from biotech companies testing experimental cancer treatments. These products could face tighter scrutiny under Prasad, a hematologist-oncologist long wary of rubberstamping pricey oncology drugs — which Prasad points out often cause some toxicity — without plausible evidence of an improvement in quality of life or survival.
The former FDA commissioners disclosed ties to Sermonix Pharmaceuticals Inc.; OncoNano Medicine; incyclix; Nucleus Radiopharma; and N-Power, a contractor that runs oncology clinical trials.
Andrew von Eschenbach, who like Sharpless formerly served both as FDA commissioner and the head of the National Cancer Institute, disclosed stock in HistoSonics, a company with investments from Bezos Expeditions and Thiel Bio seeking FDA approval for ultrasound technology targeted at tumors.
Some FDA commissioners who signed onto the letter opposing changes to vaccine approvals have ties to biotechnology investment firms, namely McClellan, who consults Arsenal Capital; Janet Woodcock, who consults RA Capital Management; and Robert Califf, who owns stock in Population Health Partners.
Califf did not respond to an email requesting comment. Woodcock did not respond to requests for comment sent to two medical research advocacy groups with Woodcock on the board. Eschenbach did not respond to a LinkedIn message.
The two signatories without pharmaceutical ties may find their judgement challenged by the FDA investigation into COVID-19 vaccine deaths, having either implemented or formally defended the Biden administration’s headlong expansion of vaccines and boosters to healthy adults and children.
David Kessler executed Biden’s vaccination policy as chief science officer at the Department of Health and Human Services, helping to secure deals for shotswith Pfizer and Moderna.
Meanwhile Jane Henney chaired a National Academies of Sciences, Engineering, and Medicine report published in October 2025 that praised the performance of FDA and Centers for Disease Control and Prevention (CDC) vaccine surveillance during the pandemic — underwritten with CDC funding.
That assessment clashes with that of a Senate report, citing internal documents from FDA, finding that CDC never updated its vaccine surveillance tool “V-Safe” to include cardiac symptoms, despite naming myocarditis as a potential adverse event by October 2020, and that top officials in the Biden administration delayed warning pediatricians and other providers about the risk of myocarditis after their approval in some children in May 2021, months after Israeli health officials first detected it in February 2021. The Senate investigation named Woodcock, a signatory of the NEJM letter, as one of the FDA officials who slow-walked the warning.
HHS is also working to “reverse the Biden administration’s attempt” to classify gender dysphoria as a type of disability.
The U.S. Department of Health and Human Services unveiled a multi-pronged regulatory effort Thursday to curtail gender-affirming care for minors, including gender transition procedures at hospitals.
The Centers for Medicare & Medicaid Services has drafted a rule that would prohibit pharmaceutical or surgical gender reassignment procedures from receiving federal Medicaid or Children’s Health Insurance Program funding. It’s also proposing a rule that would allow it to withdraw Medicare and Medicaid funding from hospitals that perform such surgeries on minors. HHS is also working to “reverse the Biden administration’s attempt” to classify gender dysphoria as a type of disability. If gender dysphoria were to be defined as a disability, then health care providers who don’t want to perform what the department has dubbed “sex-rejecting” procedures could be in danger of violating anti-discrimination laws.
Health and Human Services Secretary Robert Kennedy, Jr., described gender affirming procedures as “unsafe” and “irreversible,” and framed the administration’s actions as “[protecting] America’s most vulnerable.”
“Our children deserve better – and we are delivering on that promise,” Kennedy told reporters Thursday.
The department is acting on directives from an executive order from President Donald Trump’s first few weeks in office. The Jan. 28 order called on government agencies to “[defund] chemical and surgical mutilation” of children, seemingly in the manner that HHS has proposed, as well as “rescind or amend all policies” relying on guidance from the World Professional Association for Transgender Health.
The Food and Drug Administration is also taking regulatory action against some organizations that market breast binders to minors.
“Illegal marketing of these products for children is alarming, and the FDA will take further enforcement action such as import alerts, seizures, and injunctions if it continues,” said Food and Drug Commissioner Marty Makary.
Kennedy signed a declaration Thursday that gender affirming procedures for minors “do not meet professionally recognized standards of health care” and the Assistant Secretary for Health and Head of the United States Public Health Service Commissioned Corps, Admiral Brian Christine, signed a public health message stating the same.
“Evidence shows sex-rejecting puberty blockers, cross-sex hormones, and surgeries are dangerous. Providers have an obligation to offer care grounded in evidence and to avoid interventions that expose young people to a lifetime of harm,” Christine said.
The House of Representatives passed a bill Wednesday that would criminalize the act of providing gender affirming care to minors.
