Health Canada launches pilot for select academic-led pediatric standard of care clinical trials: Notice

October 14, 2026
Our file number: 26-106002-437

Why we're launching the pilot

Many drugs used to treat children in Canada are prescribed "off-label" because they have not been formally authorized for pediatric use. As a result, clinicians often have limited evidence to guide treatment decisions.

To address this gap, researchers conduct pediatric standard-of-care studies, such as those that compare existing treatments or optimize dosing. These studies are conducted to generate evidence that can improve clinical practice.

As many drugs are used off-label, most pediatric studies require sponsors to file a clinical trial application (CTA) with Health Canada. This is the case even when the treatments being studied are commonly used in practice.

Academics have been advocating to reduce barriers to conducting pediatric research in Canada.

We're launching a pilot project to help address these challenges.

We want to make it easier for academics to conduct pediatric standard-of-care clinical trials yet maintain appropriate safeguards for participants. We also hope this will generate better evidence to support care for children in Canada. Our position is that certain pediatric standard-of-care clinical trials can proceed despite operating outside current regulatory requirements. As a result, the scope of this pilot will be limited.

We will re-evaluate this scope later on to determine if the scope should be expanded.

Eligibility, registering a trial and reporting results

Instead of filing a clinical trial application (CTA), you may apply for the pilot and qualify for reduced requirements if you're accepted.

Drug eligibility criteria

The drug must be a pharmaceutical (which includes prescription and non-prescription drugs) and being used in a standard of care manner. For the pilot, standard of care is where the drug and conditions of use for pediatric patients are included in either:

national or international consensus guidelines or
a Canadian or local institutional guideline

The drug must also be either:

approved in Canada for that indication ("on label") for adults or
on the Canadian market for 10 years or
approved for that condition of use in the target pediatric population by the U.S. Food and Drug Administration or European Medicines Agency

It must be sourced from the Canadian market. This requirement will also ensure compliance with good manufacturing practices (GMP). Thus, you will be asked to provide the product's drug identification number (DIN) in your application.

Recognizing the absence of available oral pediatric formulations, compounding of marketed drugs will be allowed if they meet all of the following:

drug subject to compounding is marketed in Canada and has a DIN
drug is administered orally
compliance with either the:
- National Association of Pharmacy Regulatory Authorities (NAPRA) model standards for pharmacy compounding of non-sterile preparations or
- U.S. Pharmacopeia (USP) 795
uses a standardized compounding formula (for example, one already on the formulary at a Canadian tertiary care pediatric hospital pharmacy)
formula is supported by data or literature references demonstrating the compound's stability
same standardized formula used for all clinical trial sites, for consistency

Note: Compounding doesn't include mixing, reconstituting or any other manipulation that's performed in accordance with the directions for use on an approved drug's labelling material.

You may include patients receiving controlled substances for supportive care prescribed as part of clinical practice. However, the clinical trial protocol may not direct the use of controlled substances (including narcotics).

Trial eligibility criteria

Clinical trial participants must be under the age of 18 years at the time of the initial signing of the informed consent form.

The trial must:

be led by a qualified investigator who is an academic holding a faculty appointment in a department of pediatrics at a Canadian faculty of medicine
follow good clinical practices (GCP), as outlined in ICH E6 (R3)
obtain informed consent and provide the informed consent forms in the application
be approved by a research ethics board (REB) associated with the relevant faculty of medicine
- REB members must be independent from those conducting the study

In most cases, you may obtain REB approval after we've notified you that your trial is eligible for the pilot. However, if deferred consent or waiver of consent are part of the trial, you must obtain REB approval first and provide the supporting documentation with your application.

Note: We won't be conducting a complete review of the informed consent forms. We also won't be reviewing REB approval, even though it's required.

If you discontinue the trial or trial site for safety reasons, you must notify us within 15 days.

Your trial isn't eligible for the pilot if you have already submitted a CTA and received a "no objection letter" (NOL).

Registering your trial

You are expected to:

register your trial before you enrol participants, but no later than 21 calendar days after the first participant has enrolled
use a clinical trial registry that complies with the international standards set by the World Health Organization (WHO)
- U.S. Clinical trials registry or
- U.K. Clinical study registry (ISRCTN)

Reporting the results of your trial

We encourage you to report all your results, both positive and negative, for example through publication or reporting results in a clinical trial registry.

For transparency, all clinical trials filed under the pilot will be posted on our Clinical Trial Search Portal.

Learn more:

How to apply

Academics interested in applying under the pilot program should submit the following:

cover letter
application form for this pilot
copies of listed references
approved product monograph
study protocol or protocols
informed consent forms
Canadian REB approvals and refusals, as applicable
international information on refusals by regulatory authorities outside Canada and foreign REBs, if applicable

For information on how to apply for the pilot and to request the application form, email the Office of Clinical Trials at oct.enquiries-requetes.bec@hc-sc.gc.ca.

After you apply

First, we will assess your application to ensure the proposed clinical trial meets the criteria for the pilot. We aim to verify eligibility within 30 days. However, there's no default date (that is, to presume Health Canada has no objection to the application) for a trial submitted under the pilot.

We will let you know if your clinical trial is eligible for the pilot. If accepted, you aren't expected to file a CTA or wait for us to issue an NOL to start the trial. However, a clinical trial can't start until we've notified you that it's been accepted under the pilot.

If we don't accept your application for the pilot, you may submit a CTA instead.

Making changes after you have been accepted under the pilot

Since clinical trials under the pilot won't undergo a full CTA review, any changes made can't be submitted for review as amendments or notifications. However, some changes to the protocol may require a re-application to the pilot.

A new application is needed if the changes could impact trial eligibility for this pilot.

Examples that would require a new application include:

changing the dosing schedule
expanding the patient population
adding a new drug to the protocol
a change in the standard of care treatment for the population under investigation
making changes that affect any of the eligibility criteria for the pilot

If you need to make changes to avoid endangering the health of a participant or another person, you may make those changes immediately. You must then file a new application with us within 15 days of making the changes.

We will assess your new application to ensure your trial is still eligible.

Your role as a sponsor

For clinical trials under the pilot, sponsors aren't expected to file a CTA, amendments (CTA–A) or clinical trial notification.

You aren't expected to comply with the following requirements set out in the Food and Drug Regulations:

labelling requirements in section C.05.011
record requirements in section C.05.12 (3)(a), (b) and (e)

As with all clinical trials in Canada, sponsors are responsible for ensuring the proper conduct of a clinical trial involving drugs in human subjects.

For trials accepted under this pilot, you are responsible for ensuring your clinical trial:

meets the criteria for the pilot at all times
has obtained research ethics board (REB) approval and informed consent from participants

Despite these flexibilities, you must comply with the following requirements set out in the regulations:

good clinical practice (GCP) principles in section C.05.010 (a) to (i)
- described in the ICH Guideline for Good Clinical Practice E6(R3), which we adopted on April 1, 2026
- visit the section on GCP in the GUI-0100 guidance
records requirements in section C.05.012 (with the exception of C.05.012(3)(a), (b) and (e))
- visit the section on records in the GUI-0100 guidance
criteria for submitting information and samples in section C.05.013
- visit the section on submission of information and samples in the GUI-0100 guidance
requirements for serious unexpected adverse drug reaction reporting in section C.05.014 (1) and (2)
- visit the section on serious unexpected adverse drug reaction reporting in the GUI-0100 guidance

For your reference, section C.05.014 (1) and (2) states:

(1) During the course of a clinical trial, the sponsor shall inform the Minister of any serious unexpected adverse drug reaction in respect of the drug that has occurred inside or outside Canada as follows:
- (a) if it is neither fatal nor life threatening, within 15 days after becoming aware of the information and
- (b) if it is fatal or life threatening, within 7 days after becoming aware of the information
(2) The sponsor shall, within eight days after having informed the Minister under paragraph (1)(b), submit to the Minister a complete report in respect of that information that includes an assessment of the importance and implication of any findings made

Learn more:

Health Canada oversight

We may conduct inspections of clinical trials included in this pilot to verify compliance with the GCP principles. These inspections also offer an opportunity to assess the:

validity and integrity of data generated during the clinical trial
extent to which the rights, safety and well-being of participants are protected

Inspections for clinical trials under the pilot will align with our approach for phase IV clinical trials. They may be subject to inspection, but in general, we don't focus inspection activities on phase IV trials, as outlined in the GUI-0100 guidance.

In the event that compliance actions are required, we will follow the current processes for enforcing compliance.

Implementing the pilot

We will begin accepting applications under the pilot on October 30, 2026.

We plan to start the first evaluation 6 months after launching the pilot. This pilot may inform potential future policy or regulatory changes.

We will assess the pilot from time to time to determine if we need to make any modifications and post updates if required.

Contact us

For questions about good clinical practices and inspections, email the Regulatory Operations and Enforcement Branch at GCP-BPC@hc-sc.gc.ca.

For questions or comments about the pilot, including changes that may require filing a new application, email the Office of Clinical Trials at oct.enquiries-requetes.bec@hc-sc.gc.ca.

Page details

2026-10-14