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Health Canada is proceeding with plans to add more flexibility to processes for approving medical treatments through an approach known as "agile licensing." The plans have raised hopes for speedier approvals and concerns about the potential for increased risk to patients.

Health Canada intends to finalize the agile licensing proposal later this year. Regulations will be implemented in a phased approach, Anne Génier, a Health Canada spokesperson, told Medscape Medical News.

The federal health department has been working on this project for several years. In December 2019, the Parliament passed an omnibus budget bill that mandated an update of the rules for approving medical products.

In December 2022, Health Canada unveiled its proposed regulations and accompanying guidance documents. The wide-ranging package included proposals regarding the manufacture and storage of drugs, as well as the approval of new medicines. More than 760 individuals, organizations, and companies offered public comments on the proposals.

Reaction to the agile licensing proposals was divided. Drugmakers such as Johnson & Johnson and Roche generally supported efforts to quicken the approval process.

In its comment, Innovative Medicines Canada (IMC), a pharmaceutical trade group, asked regulators to keep in mind delays that Canadians face in accessing new medicines. Health Canada's approval is only a starting point in a process that also involves other federal and provincial officials. Hence, Canadian patients may wait 732 days, which is twice as long as the wait time experienced by patients in most peer countries, for public access to new medicines following Health Canada's approval, according to IMC.

The Vasculitis Foundation Canada, which represents patients with rare diseases that cause inflammation of the blood vessel wall, said that delays in accessing new medicines can be especially frustrating for patients with uncommon conditions.

"There are few approved therapies for vasculitis, so we know the importance of being able to access new therapies in a timely manner," the group said in its comment. "Canada has a chronic problem of slow approvals for new therapies, compared with other jurisdictions" such as the United States.

Many comments on the agile licensing proposals were anonymous. Some expressed concern that the proposed changes in approval processes would weaken standards for evidence.

Researchers also questioned Health Canada's broadly stated goal of setting terms and conditions (T&Cs) for requiring the continued study and monitoring of approved products. Studies of Canadian and international drug approvals show that regulators are increasingly willing to approve drugs based on early trial results that suggest a benefit, instead of waiting for the results of more advanced studies, wrote the Canadian Network for Learning Healthcare Systems and Cost-Effective Omics Innovation (CLEO) in its comment.

"We are concerned that the imposition of T&Cs at the time of authorization may further increase regulators' tolerance for risk and uncertainty in a way that erodes the signal of confidence that a market authorization sends to Canadians," wrote CLEO.

CLEO is a multidisciplinary team housed at the British Columbia Cancer Research Institute in Vancouver, British Columbia, Canada. Its members include researchers in precision oncology, health economics, health informatics, and health policy, as well as clinicians and patients who have undergone cancer treatment.

Like other regulatory bodies, Health Canada has long been willing to accept higher risks for new products that are considered likely to help people with serious illnesses.

In 1998, it introduced the notice of compliance with conditions (NOC/c) policy that allows drug approvals based on promising initial evidence and companies' commitment to conduct the research needed to confirm effectiveness and monitor safety. This approach resembles the US Food and Drug Administration's accelerated approval process for medicines. Several of the commenters asked Health Canada for more clarity about how it will implement the T&C pathway and how it would mesh with the existing NOC/c program.

Among those with doubts and questions about the T&C pathway is Joel Lexchin, MD, professor emeritus of health policy and management at York University in Toronto, Ontario, Canada, and a longtime pharmaceutical industry watchdog.

The agile licensing proposals prompted Lexchin to examine the track record for NOC/c approvals. This pathway allows drugmakers to introduce medicines based on surrogate endpoints after committing to further evaluation of whether the medicines help patients live longer or better.

In a 2020 paper published in Healthcare Policy, Lexchin found that follow-on research requirements were fulfilled for 50 NOC/cs (56.2%) between 1998 and 2017. Thirty-one (34.8%) commitments had not yet been fulfilled. In eight (9.0%) cases, either the indication or the drug was withdrawn.

The agile licensing proposal might lower the bar for the evidence needed for initial clearances of products, said Lexchin. "I guess we'll have more drugs that we know less about on the market," he said.

Many physicians are unaware that some of the drugs that they prescribe were approved through the NOC/c pathway, Lexchin added. Most physicians also are unaware that Health Canada is considering further changes to the approval standards. "I'd be surprised if you'd find more than a handful of doctors who actually know that these things are happening," said Lexchin.

Rolling Review Scrapped? 

The finalized version of the agile licensing plan will likely disappoint those who had hoped for a formal rolling review process, Declan Hamill, vice president of policy, legal, and regulatory affairs for IMC told Medscape Medical News. Health Canada officials told IMC as much in June, citing a lack of operational capacity or staffing to manage this process.

"I'm not going to question that, but I would say that if something is valuable for patients and so valuable for the industry and the regulator, there should be a way of working this out," said Hamill. "There should be some way of doing this efficiently."

Health Canada has used rolling review to speed approvals of drug and vaccine applications during the COVID pandemic. Many companies had hoped that the department would establish rolling review procedures for other products. The idea is to allow companies to start their applications when they have some of, but not all, the critical information ready for regulators.

Health Canada also uses rolling review for annual updates of seasonal influenza vaccines and for simultaneous review of veterinary drugs with the United States under the Canada–United States Regulatory Cooperation Council.

Asked to respond to Hamill's comments, Health Canada said it is considering proposing that rolling reviews only be used for submissions for public health emergency drugs. "This [approach] will help ensure that drugs that are needed to respond to public health emergencies are made available to Canadians in a timely way without compromising on the safety, efficacy, and quality data needed to support regulatory approval," Anna Maddison, a Health Canada spokesperson, told Medscape Medical News.

Maddison added that Health Canada will also "continue to work with stakeholders to find other ways to build upon existing expedited review mechanisms. Facilitating access to much-needed drugs by ensuring that Canada's drug approval process remains agile is critical to protecting the health and safety of Canadians." 

But such changes could take years to enact if they are not included in the pending agile licensing proposal, said Hamill. Canadian drug regulations are not often modified on this scale, he said.

"While Health Canada may say, 'We'll do more later. There are future phases of agile licensing,' this is a very slow-moving process," said Hamill. "They should be trying to capture momentum and learn from what was done in the pandemic to improve processes now, rather than suggesting that there will be future iterations. That will take a lot of time — in all likelihood, years."

Between 2021 and 2024, Lexchin received payments from the law firm Koskie Minsky for writing a legal brief on the role of promotion in generating prescriptions, from Strategy Institute for being on a panel about pharmacare, and from BMJ Publishing Group for cowriting an article for a peer-reviewed medical journal.

Kerry Dooley Young is a freelance journalist based in Washington, DC. Follow her on threads at @kerrydooleyyoung.