opinion: suffer from a rare disorder in canada?

this month marks the 20th anniversary of the u.s. rare diseases act . the act established an office within the national institutes of health (nih) to recommend and coordinate a national research agenda on rare disorders and their treatment and to support educational activities. the office was recently upgraded to a division of rare diseases research innovation (drdri).

drdri maintains a database on nih research and promotes research into diseases and treatments. it also runs a rare diseases clinical research network , which has 20 consortia, a few involving canadian sites, that investigate many disorders. and it supports a website providing up-to-date, accurate information frequently requested by patients, caregivers, health professionals and researchers.

what about canada? we have neither a rare diseases act nor an organization like drdri. nor do we have a national rare disorder plan, nor or even a national definition of what constitutes a rare disorder, unlike quebec or many developed countries . the canadian institutes of health research does have its own definition of rare but no dedicated rare disease institute.

we do have patient advocacy associations, including the canadian organization for rare disorders (cord), which provides links to some sources of information. but cord doesn’t directly sponsor or undertake scientific research.

in 2015 it proposed a strategy to improve early detection of rare disorders, promote innovative research and provide timely, evidence-based care, as well as sustainable access to promising therapies. but the proposal didn’t gain traction with policy-makers. cord revived its efforts following a 2019 federal budget promise for a rare disease strategy (since unfulfilled ) and a discussion paper from health canada on “principles and pillars.” in sum, no canadian patient has yet benefited from ottawa’s still-being discussed plan.

on the goal of sustainable access to promising therapies the federal government has in fact moved backwards. over the past five years, it tried to cut drug prices drastically, seemingly without realizing or caring that doing so would delay or deny access to new medicines, including therapies for rare disorders, even more than before . this disregard of likely harms to patients sent a chilling message around the world of therapeutic innovation. true, most of the intended price cuts were cancelled after courts ruled them illegal but the liberals and the patented medicine prices review board, their rogue regulator , haven’t given up on the goal of reducing drug prices, as pmprb’s recently-released draft guidelines demonstrate.

price reductions imposed by fiat, not negotiation, will add yet another hurdle for drug developers to clear in bringing new medicines to canadians. existing ones include: poorer patent and data protection than in comparable countries; unaccountable, non-independent and non-transparent health technology assessments performed by organizations making recommendations to the federal, provincial and territorial governments that manage and fund them; and an unaccountable and non-transparent price negotiation organization responsible to the same governments.

in most cases, it takes years for a new drug for a rare disorder to get to canadian patients. and when the costs of such drugs are covered by provincial plans, restrictive patient access criteria are imposed to contain costs, not improve health outcomes .

at the moment 11,000 rare disorders afflict over three million canadians. unmet needs are huge. so far only 500 disorders have any treatment. canadians with such disorders have extremely limited resources from which to obtain diagnoses, services or therapies and are often on their own in trying to get health care . many end up going abroad.

medicines can improve patient health, reducing the need for care that is more expensive and, in canada, frequently difficult to access. instead of encouraging scientists and investors to make large bets of time and resources on small markets represented by people living with rare and ultra-rare disorders, canada takes a course of indifference or even outright hostility. innovation will remain at risk as long as policy-makers see only the high prices of new medicines and not the benefits they can bring to patients, the health care system and society.

canadians with rare diseases don’t need virtue-signalling, theoretical principles and pillars or policies that restrict access to innovative medicines. they need a law, like the u.s. rare diseases act, to provide resources to motivate research and development in diseases and therapies and to ensure fair and timely access to innovative treatments, many just on the research horizon, that can reduce suffering and improve and even save lives.

share story

share this story

opinion: suffer from a rare disorder in canada?

email

facebook

x

linkedin

copy story link