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Navigating Pediatric Drug Development (PDP) in Canada

A comprehensive guide part 1

Many medications that are prescribed for children are prescribed off-label. Information and data from clinical trials of drugs in children are essential to support evidence-based pediatric therapies. Without this information, health care providers rely on other sources of clinical and scientific evidence to assess if the benefits of a medication outweigh the risks for a specific patient. Health Canada is taking steps to increase the availability of data that will help health care providers make these important decisions. International regulators such as the United States Food and Drug Administration (US FDA) and the European Medicines Agency (EMA) have developed legislative and regulatory tools to encourage the:

  • Study of the safety and efficacy of drugs in pediatric populations and
  • Submission of findings from those studies to the regulatory agencies

Pediatric drug development is a critical aspect of pharmaceutical research, ensuring that medications are safe and effective for children. To increase access to safety, efficacy and quality information in pediatric populations and to align with international standards, Health Canada is conducting a pilot of the pediatric study submission policy which includes the submission of a pediatric development plan (PDP) by the sponsor should they wish to participate in the pilot.1

Pediatric Study Submission Policy

Under the pilot for this policy which took effect on February 26, 2024, and runs for at least 2 years, Health Canada is asking sponsors who file a new drug submission (NDS) and certain supplements to a new drug submission (SNDS – for new indications, new dosage forms and new routes of administration) to include a PDP with their submission1,2. The PDP1:

  • Should provide details of any ongoing or planned pediatric studies
  • May also include a rationale for not studying the drug in all or part of the pediatric population

All studies within the PDP will be expected to be submitted to Health Canada. 

According to Health Canada Guidance Submitting pediatric development plans and studies: Guidance - Canada.ca, the following classes of “drugs” will be under the scope of this policy1:

  • Pharmaceutical drugs (prescription and non-prescription)
  • Biologics, including biotechnology products, vaccines and fractionated blood products
  • Radiopharmaceutical drugs 

Medical devices, natural health products, biosimilar biologics, generics and disinfectants are out of scope. Pediatric populations for the purposes of this policy will include individuals aged from birth to under 18 years1.

Submissions relying on third-party data and administrative submissions are out of scope1.

Objectives of this policy1, 2: 

  • To encourage sponsors to submit, in a timely manner, safety and efficacy information for drugs expected to be used in pediatric populations
  • To provide more information on the safety, efficacy and dosage of drugs used in pediatric populations to health care providers, patients and their families

When to submit plans and studies

Participation in the pilot is voluntary and will not affect the outcome or timeline of the review of NDS or SNDS2.

Health Canada asks sponsors to include the pediatric studies with an NDS or SNDS for the adult indication if a pediatric study plan has been completed or a pediatric study package is ready for regulatory review1. Health Canada will assess the pediatric studies based on Health Canada's established approach for assessing drug safety, efficacy and quality in a pediatric population1. 

Submission of pediatric studies with an NDS or SNDS will not constitute participation in the pilot. However, if the pediatric studies are incomplete when the NDS or SNDS are filed, sponsors wishing to participate in the pilot should include a PDP with their submission in order to notify Health Canada of their intention to provide the studies at a later date. Sponsors will also need to complete a Pediatric Pilot Survey and include it in their submission1. 

When filing the drug submission, Health Canada asks sponsors to specify in a note to reviewer if they are including pediatric studies or a pediatric development plan1.

Content of a Pediatric Development Plan

PDPs detail the research and development that a sponsor intends to undertake to generate information that may support the authorised use of a drug in a pediatric population. The plans may involve developing a1:

  • Pediatric formulation
  • pediatric extension of an existing indication
  • New pediatric indication based on the active ingredient of the drug

To participate in the pilot, pediatric studies and/or the PDP must address1:

  • Existing and proposed indications
  • New dosage forms (if applicable)
  • New routes of administration (if applicable)

During the pilot, Health Canada will ask all sponsors submitting an NDS or SNDS to complete a Pediatric Pilot Survey at the time of filing, for data collection purposes. The Survey will prompt sponsors to consider taking part in the pilot if their submissions are within scope1. Sponsors who choose to not participate will need to provide a rationale. A decision to not participate in the pilot and the reasons given will not affect Health Canada’s review of the NDS or SNDS1.

Under the pilot, sponsors may submit either an agreed US FDA initial Pediatric Study Plan (iPSP) or an approved EMA Pediatric Investigation Plan (EU-PIP). If no appropriate foreign plan is available from the US FDA or EMA, sponsors may prepare and include a Canadian-specific PDP (C-PDP).

Foreign-approved Plans

Sponsors who have both an iPSP and EU-PIP may select which plan to submit to participate in the pilot. Health Canada will accept the submitted plan as the PDP1.

Sponsors may choose to complete a supplementary annex (Canadian addendum) if they have additional information that may help to clarify aspects of their foreign plan. This addendum may include details such as1:

  • Communications between sponsors and foreign regulators
  • Special considerations to factors that are specific to the Canadian population (for example, genetic or extrinsic factors)
  • Information that reflects the authorised indication(s) in Canada for drugs where the Canadian-authorised indication varies from that of the US FDA and/or EMA
  • Relevant differences in the authorised and marketed therapies for the condition in Canada, compared to the therapies authorised and marketed in the jurisdiction for which the plan was developed
  • Other information that the sponsor believes is relevant to the Canadian context

Canadian PDP

A C-PDP should include the following information1:

  • Title page
  • Section 1: Overview of the disease or condition in the pediatric population
  • Section 2: Proposed pediatric development plan
    • Section 2.1: Overview of planned extrapolation to specific pediatric populations
    • Section 2.2: Rationale for not conducting studies
    • Section 2.3: Tabular summary of planned nonclinical and clinical development
    • Section 2.4: Development of age-appropriate formulation
    • Section 2.5: Nonclinical studies
    • Section 2.6: Clinical data to support design and/or initiation of studies in pediatric patients
    • Section 2.7: Ongoing and planned pediatric studies
      • Section 2.7.1: Pediatric pharmacokinetic or pharmacokinetic / pharmacodynamic studies
      • Section 2.7.2: Clinical efficacy and safety studies
  • Section 3: Agreements for pediatric studies with other regulatory authorities

Refer to Health Canada page: Canadian pediatric development plans: Guidance - Canada.ca for further information on format and content of a C-PDP1.

Health Canada encourages sponsors to consider the needs of all pediatric populations, including neonates, where possible, when developing their C-PDP. Sponsors may also propose studying the potential therapeutic benefits of the active ingredient of the drug in the relevant pediatric population for indications that go beyond those that complement existing and proposed adult indications. All data sources should be supported by references included with the submission.

The Survey, Canadian addendum to PDP and Canadian-specific PDP should be included in Module 1.0.7 General note to reviewer and a foreign agreed/approved PDP should be included in Module 1.2.7 International information1.

Conclusion

Health Canada's pilot program for PDPs represents a significant step forward in ensuring the safety and efficacy of medications for pediatric populations. By encouraging the submission of comprehensive PDPs alongside NDS and certain SNDSs, Health Canada aims to align with international standards and increase the availability of critical data. For this, Health Canada has issued the Submitting pediatric development plans and studies: Guidance - Canada.ca which outlines the agency’s policy on submitting a pediatric study. The guidance intends to assist sponsors to align their submissions with the policy by including pediatric studies and a PDP when filing an NDS or an SNDS that meets certain criteria. Submission of these data will increase the availability of safety, efficacy and quality data that will help health care providers make important decisions when treating pediatric populations. Specifically, the ongoing pilot program allows Health Canada to gain expertise around pediatric plans which is needed to provide important feedback for future policy decisions around Health Canada’s approach to pediatrics.

Part 2 of this blog series will cover the review of PDPs by Health Canada, amending agreed-to PDPs and annual reporting.

 

References:

1Health Canada. Guidance on submitting pediatric development plans and pediatric studies: Modified on May 01, 2024. Accessed February 24, 2025 at: https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/pediatrics/guidance-submitting-development-plans-studies.html

2Health Canada. Pilot on pediatric development plans and studies. Modified on April 19, 2024.  Accessed February 24, 2025 at: https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/pediatrics/pilot-development-plans-studies.html 

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