Update: In October, the US Food and Drug Administration also announced new draft guidance, focused on therapeutic protein products. The draft guidance recommends biosimilar sponsors to consider a ‘streamlined approach’ without a comparative clinical efficacy study in situations where a comparative analytical assessment establishes the proposed biosimilar is highly similar to the reference product, and a human pharmacokinetic similarity study and immunogenicity study are sufficient to assess any clinically meaningful differences.
On June 10, 2025, Health Canada launched a consultation on its Draft Guidance Document: Information and Submission Requirements for Biosimilar Biologic Drugs which includes numerous proposed revisions to the current guidance document.
The most notable revision is the removal of the requirement that the biosimilar submission include comparative clinical efficacy trials in most cases:
Current guidance: Clinical efficacy trial(s) In most cases, a comparative clinical trial(s) is important to rule out clinically meaningful differences in efficacy and safety between the biosimilar and the reference biologic drug. A clinical efficacy trial may not always be necessary, e.g. where there is a clinically relevant PD [pharmacodynamic] endpoint. In such cases, a scientific justification is needed and safety as well as comparative immunogenicity data are still required.
Proposed guidance: Comparative clinical efficacy and safety trial(s) In most cases, a comparative clinical efficacy and safety trial(s) is not required. Safety and comparative immunogenicity data are still required and should be collected within the comparative clinical pharmacology studies but could be supplemented with data collected using other trial designs (e.g., studies designed to specifically focus on safety and/or immunogenicity). If a comparative clinical efficacy and safety trial(s) is deemed necessary, sponsors should provide a rationale to explain the purpose of the trial(s) in the context of a biosimilar submission.
Health Canada’s removal of a requirement for comparative clinical efficacy studies aligns with a recent proposal from the European Medical Agency (Streamlining development and assessment of biosimilar medicines) and a recently introduced bill in the United States (Expedited Access to Biosimilars Act).
Rather than comparative clinical efficacy trials, the draft guidance suggests that the clinical program should primarily include a comparative pharmacokinetic study, and if feasible, a comparative evaluation of pharmacodynamics, and only in “exceptional circumstances” would an additional clinical trial be required. In addition to the clinical program, the draft guidance outlines the requirements for a biosimilar submission to include extensive comparative quality studies to demonstrate a high degree of similarity, including physicochemical characteristics, functional properties and stability profile.
With respect to indications, the draft guidance removes the previous language requiring a “detailed rationale that scientifically justifies authorization of the biosimilar in each indication”. Instead, the draft guidance states that “[w]here a high degree of similarity to the CRBD [Canadian reference biologic drug] has been established, indications may be granted for the biosimilar candidate based on evidence of the safety and efficacy of the CRBD gathered in both the pre- and post-market settings.”
The draft guidance is consistent with a 2023 change in policy regarding biosimilar product monographs, as reported in our March 2024 Biosimilars Update, which removed the requirement for inclusion of comparative clinical data in such product monographs.
The consultation is open until September 8, 2025, and comments can be provided to brdd-cppic_brdd-cppci@hc-sc.gc.ca.
Should you have any questions, please do not hesitate to contact a member of the Life Sciences Regulatory & Compliance Group.
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