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Opinion: For drug access, Canadians with rare disorders are 40 years behind Americans

FILE PHOTO: A pharmacist counts prescription drugs at the at the CentreTown Pharmacy in Ottawa
FILE PHOTO: A pharmacist counts prescription drugs at the at the CentreTown Pharmacy in Ottawa

By Nigel Rawson and John Adams

Forty years ago this month, the United States passed the Orphan Drug Act (ODA) to facilitate the development of drugs for rare disorders. This was an important political issue in the late 1970s and early 1980s because, although individual disorders impacted small numbers of patients, overall rare diseases affected 20-25 million people and most disorders had no treatment.

Rare means patient numbers are small, ranging from less than 100 to several thousand. Such numbers don’t make much of a business case for developing medicines. The ODA introduced incentives to stimulate research and development of small-market medicines, including tax credits for qualifying clinical trials, exemption from regulatory fees, and a potential seven years of market exclusivity after approval, regardless of patent or data protections.

The ODA has been successful in its objective. In 1983, only 38 drugs were approved in the United States specifically to treat orphan diseases. But between 1983 and 2019, more than 5,000 medicines were, with the number more than quadrupling between the 1990s and 2010s. In the last decade, the number of drugs available to treat rare diseases in children has increased markedly.

Canada has no orphan drug act nor any incentives to encourage drug developers to launch medicines for small numbers of patients. There is no national strategy to treat rare disorders, while at the provincial level, only Quebec has a plan for the care of individuals with rare diseases — which it introduced only last year. Other provinces have no plan — just piecemeal efforts for some patients to access some drugs. When a new costly rare-disorder drug is approved in Canada, public insurers’ first thought seems to be how to avoid paying for it.

Patient advocates, including the Canadian Organization for Rare Disorders (CORD), have attempted to get the federal government to implement a national plan. CORD proposed a strategy that included promoting innovative research and providing timely, evidence-based, sustainable access to promising therapies. But policy-makers failed to get on board.

In the 2019 federal budget, the Liberals did say they would introduce a strategy and in their 2021 election platform they promised $500 million per year to fund costly drugs for rare diseases. But the only action so far has been a Health Canada discussion paper. Canadian patients have yet to benefit from any funding.

What the federal government has done in the general area of therapeutic drugs is move backwards on sustainable access to promising therapies. Over the past seven years, it has tried to drastically cut drug prices — apparently without either realizing or caring that doing so would delay or deny access to new medicines. This disregard of likely harms to patients has sent a chilling message about Canada’s dismissive attitude to therapeutic innovation.

The result? Between 2006 and 2014, 80 per cent of medicines submitted for regulatory approval in the United States or the European Union were also submitted for regulatory approval in Canada but, by 2020, that number had decreased to just 44 percent. Corresponding figures for orphan drugs were similar: 79 per cent on average between 2006 and 2014 down to 39 per cent in 2020 When developers are discouraged from submitting new medicines for marketing authorization in Canada, the drugs won’t be available to any Canadian.

Marketing approval is just the first step in getting medicines to patients. Once approved, developers face more obstacles in Canada: unaccountable, non-independent and non-transparent health technology assessments performed by organizations making recommendations to the same governments that fund and manage them; an unaccountable and non-transparent price negotiation organization also responsible to the same governments; and officials working for these governments who have no motivation to add new drugs to public drug plan formularies.

Overcoming these hurdles usually takes years. Even when the costs of drugs are covered by government plans, restrictive patient access requirements are imposed to contain these costs, not improve health outcomes.

People suffering rare disorders are like all other Canadians — they want affordable access to medicines for their unmet health needs. But at the same time they don’t want access denied by Ottawa’s plans to force down drug prices to levels that are unsustainable for developers, who then decide the Canadian market isn’t worth bothering with. Canadians won’t have first-class access to biopharmaceutical innovation if their government only pays for a third-class ticket.

Four decades after the ODA was enacted, Canadians with rare disorders continue to suffer from a lack of fair and timely access to innovative therapies. They need many things but they especially need a law like the ODA to incentivize developers to launch rare-disorder medicines in Canada and provide early access to the many innovative treatments on the research horizon that can reduce suffering and improve and even save lives.

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.