This article is the fourth part of an 8-part series from the authors. The series, Waiting for new drugs for rare disorders in Canada, is an in-depth look at the disadvantages faced by Canadians with rare disorders in accessing needed, innovative drugs.
By Nigel Rawson and John Adams, August 2, 2023
So far in our article series, we have explained that medicines are submitted to Health Canada for regulatory approval later than in the United States and Europe and that access delays are caused by health technology assessment (HTA) and collective price negotiation instituted by government drug plans.
The time taken by drug plan officials to decide whether to list a new rare disorder drug further extends the wait for patients trying to get access to it. Listing means physicians can write prescriptions and eligible patients get them filled and paid for.
Government drug plans are not mandated to list medicines that successfully pass through government-funded and managed HTA and price negotiation steps. They can negotiate further price concessions with the developer before deciding whether or not to list a drug.
Other than recognizing a need for a medicine among their covered population, drug plan officials have no motivation to list when their government employers want to contain drug costs in their siloed accounting systems (they rarely evaluate benefits of medicines to the health system as a whole or the economy). The result is the listing of drugs for rare disorders varies substantially across Canada.
Of 43 rare disorder drugs given regulatory approval in Canada between 2015 and 2022 with completed HTAs and price negotiations, seven provinces (British Columbia, Alberta, Saskatchewan, Manitoba, Ontario, New Brunswick and Nova Scotia) list 65 percent or more. The percentage is considerably lower in the other provinces, especially Prince Edward Island (33 percent) and Newfoundland and Labrador (15 percent).
Although 65 percent in a majority of provinces may appear reasonable, just 22 (42 percent) of the 52 rare disorder drugs approved by Health Canada between 2015 and 2020 have been listed in seven or more government plans, despite an average of four years elapsing since regulatory approval.
Listing of drugs for rare disorders in government plans is closely aligned with outcomes of the HTA and price negotiation processes. Medicines with an unsuccessful price negotiation typically have a negative HTA and usually are not listed. However, not all drugs with a successful price negotiation are listed. In other words, a negative HTA generally means “no” listing, while a positive HTA means only “maybe.”
Furthermore, listing does not necessarily mean all patients can access a rare disorder drug. In most government plans, they are only available through special access programs subject to patients satisfying defined criteria, usually copied from clinical trials. Patient experiences indicate that drugs for rare disorders are often only accessible through government plans on a case-by-case basis, but this is not widely known until patients actually try to access them.
Access criteria recommended by HTA agencies and implemented by government drug plans have become more detailed and more stringent in recent years. Some criteria limit access to children, literally cutting off their access after a certain age. Other criteria deny access to patients in the first stages of a disorder who might benefit most, but provide access to patients at a much later stage in disease progression when patients are much sicker and may not benefit as much.
Access criteria can lead to patients taking perilous action to maximize their opportunity of meeting the requirements. For example, four drugs that treat life-threatening cystic fibrosis caused by specific rare gene mutations have been launched over the past decade – the first was Kalydeco and the most recent and more effective is Trikafta. HTA-defined clinical criteria for accessing Trikafta require untreated lung function measurements to be below a certain level, but this ignores the fact that many cystic fibrosis sufferers are taking Kalydeco and don’t have “untreated” measurements. Kalydeco patients wanting to change to Trikafta face a choice: continue on Kalydeco and risk having lung function measurements that don’t comply with Trikafta access criteria, or cease Kalydeco for a washout period and suffer a decline in health and lung capacity to ensure their lung function level fulfills the Trikafta criteria. This is unnecessary suffering simply to satisfy poorly-devised bureaucratic requirements copied from clinical trial criteria.
Canadians with rare disorders can wait years for a medicine to be listed in government plans and still may not be able to access it due to restrictive access criteria. In contrast, Germany has a regime for rare disorder drugs in which patients get a drug as soon as the country’s regulator approves it. Once HTAs and price negotiations are complete, the outcome is made retroactive to the date of first regulatory approval as long as the budget impact of the drug across all German payers does not exceed 50 million euros. This puts patient needs ahead of HTAs and price negotiations.
It is high time Canadian governments put the needs of patients with rare disorders before bureaucratic overlapping processes and real or feigned shock at sticker prices.
Nigel Rawson is a Senior Fellow with the Macdonald-Laurier Institute, an Affiliate Scholar with the Canadian Health Policy Institute and a Senior Fellow with the Fraser Institute. John Adams is cofounder and CEO of Canadian PKU and Allied Disorders Inc., a Senior Fellow with the Macdonald-Laurier Institute and volunteer board chair of Best Medicines Coalition.
The views expressed are the authors’ own and do not necessarily represent those of organizations with which they collaborate.