Guidance on submitting pediatric development plans and pediatric studies: Canadian plans
On this page
- Format and content
- Reviewing a Canadian PDP
- Amending an agreed-to Canadian PDP
- Canadian PDP template
Format and content
Sponsors who do not have an initial pediatric study plan (iPSP) or pediatric investigation plan (EU-PIP) can participate in the policy pilot by including a Canadian pediatric development plan (C-PDP) with submissions that are within the scope of this guidance.
C-PDPs will undergo scientific review to determine whether the proposed plan is adequate to study the efficacy, safety and quality of the drug in the pediatric population(s). To be accepted into the pilot, the plan should follow the criteria outlined in this guidance. A C-PDP template is included at the end of this page.
Sponsors are encouraged 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. Citations can include references that appear in other submission modules.
Sponsors are encouraged to review the applicable guidelines issued by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). Foreign regulators also publish relevant guidelines that can assist when developing your pediatric plans, such as:
- S11: Nonclinical safety testing in support of development of paediatric pharmaceuticals
- E11: Addendum to ICH E11: Clinical investigation of medicinal products in the pediatric population
- Pediatric study plans: Content of and process for submitting initial pediatric study plans and amended initial pediatric study plans (US FDA)
- Pediatric investigation plans (EMA)
Submissions for drugs associated with more than 1 indication may address all indications in a single pediatric plan or in separate pediatric plans.
Title page
Sponsors should include the following information about the drug in the title page of the PDP:
- drug brand name
- drug proper or common name
- dosage form
- therapeutic, diagnostic or pharmacological classification and code
- in accordance with the World Health Organization's Anatomical Therapeutic Chemical (ATC) index
- approved indication(s) (if applicable)
- proposed indication(s)
- key elements of proposed plan:
- age groups for which a rationale for not conducting studies is provided
- age groups for which studies will be undertaken
- identification of the study types
- control numbers for any previous submissions (including amendments) for this drug relevant to the C-PDP
Overview of the disease or condition in the pediatric population
This section should briefly summarize the available information on:
- pathophysiology of the disease/condition
- incidence and prevalence in both the adult and the pediatric population
- include information on specific age subgroups where available and/or appropriate
- method of diagnosis in both the adult and the pediatric population
- discussion of the available evidence supporting the similarities and differences between the disease/conditions in the adult and pediatric populations, as required
- currently available treatments and/or prevention strategies in the pediatric population
Sponsors may cross-reference to Module 2 of the NDS/SNDS as applicable.
Proposed pediatric development plan
In keeping with the Scope and application section of this guidance, the C-PDP should address:
- existing and proposed adult indications
- new dosage forms (if applicable)
- new routes of administration (if applicable)
If it is not appropriate or practical to investigate the drug in all or part of the pediatric population, sponsors who wish to take part in the pilot should include a rationale within the C-PDP.
The rationale should:
- explain in detail why the studies will not be undertaken
- contain the type of information included in a waiver request to the US FDA or EMA
For more information on rationales, refer to the following sections:
- Rationale for not conducting studies
- Rationale for not conducting studies based on an adult-related condition that rarely or never occurs in children or is on the MA class waiver
A C-PDP is based on the knowledge of the drug and disease/condition at the time the NDS or SNDS is submitted to Health Canada. During the implementation of the plan, additional data may become available that may affect aspects of the C-PDP. Sponsors may submit amendments to an agreed C-PDP according to the process outlined in the section on Amending an agreed-to Canadian PDP.
Overview of planned extrapolation to specific pediatric populations
It may be appropriate to extrapolate the efficacy of a drug if the course of the disease or condition and the expected response to the product between the pediatric and adult populations are similar. In some cases, it may also be appropriate to do so from 1 pediatric age group to another.
The C-PDP should provide details on any planned extrapolation of efficacy for the proposed product.
Sponsors should also justify the use of extrapolation. This justification should include:
- information on the similarities and differences between the disease in adults and children, or between 1 pediatric age group and another, that would support the extrapolation
- for example, disease pathogenesis, disease progression, disease characteristics
- supportive data from all available sources
- for example, sponsor-generated data, published literature, expert panels or workshops
Extrapolation of efficacy for other drugs in the same class, if previously accepted by Health Canada, the US FDA or the EMA, may also be considered.
If appropriate, the sponsor should also discuss the:
- similarity of the efficacy-exposure-response relationships between adults and pediatrics
- based on experience with drugs in the same class or other drugs approved for use in the same disease/disorder
- use of modelling and simulation studies to support extrapolation
In some cases, it may be possible to use existing safety and dosing information in adults or other pediatric populations to draw inferences about the safety of the drug in pediatric population(s).
Sponsors proposing to use extrapolation as a tool to support inferences about safety should clearly:
- give details on how the available knowledge of the known and/or potential safety issues in the reference population apply to the target population
- justify the use of safety extrapolations from each available source
- identify potential gaps and ensure that they are addressed elsewhere in the C-PDP, such as through clinical studies
Rationale for not conducting studies
Sponsors should provide a rationale to Health Canada when there is a reason for not conducting studies in all or part of the pediatric population.
This rationale should include:
- a summary of the supporting data for each age group for which studies will not be taking place
- supporting information from all available sources such as:
- data from nonclinical safety assessments, findings from adult clinical trials, published literature and other applicable evidence
- full or partial waivers previously granted to other drugs in the same class by Health Canada, the US FDA or the EMA
In line with US FDA and EMA standards for pediatric study waiver requests, this rationale should indicate that studies will not be taking place for any of the following reasons:
- The necessary studies are impossible or highly impracticable:
- justification may be based on the incidence or prevalence of the condition in all or part of the pediatric population
- The evidence strongly suggests that the drug would be ineffective and/or unsafe in relevant pediatric populations
- justification may be based on a pharmaceutical rationale or (preliminary) data suggesting lack of efficacy or safety in the pediatric population
- justification may be based on the lack of efficacy in the pediatric population as a whole or in subsets, where a rationale may be provided to apply a lack of efficacy in a pediatric subset to the pediatric population as a whole
- The drug does not represent a significant therapeutic benefit over existing treatments for pediatric patients
- justification should be based on a detailed discussion of the existing authorized and marketed treatment methods
- Rationales related to not undertaking development of pediatric formulations for specific age groups
- justification should be based on the applicant demonstrating that reasonable attempts to produce the required pediatric formulation have failed or are not feasible
Sponsors who provide a rationale for not conducting any pediatric studies are to complete only the following sections (numbering according to the C-PDP template):
- Title page
- 1 Overview of the disease or condition in the pediatric population
- 2.2 Rationale for not conducting studies
- 3 Agreements for pediatric studies with other regulatory authorities (if applicable)
Rationales for not conducting studies based on a drug being ineffective or unsafe in pediatric populations may need to be reflected in the product labelling. Appropriate labelling statements will be determined based on the data submitted and in collaboration with Health Canada.
Rationale for not conducting studies based on an adult-related condition that rarely or never occurs in children or is on the EMA class waiver
A shorter C-PDP is requested for sponsors who are unable to undertake studies on any part of the pediatric population, as the condition is on the:
- US FDA list of adult-related conditions that qualify for a waiver because they rarely or never occur in pediatrics
- EMA class waiver list
In these circumstances, sponsors should include the following in their C-PDP:
- Title page
- 2.2 Rationale for not conducting studies
- identify the condition on the US FDA or EMA list that the product is intended to treat
- if available, include an EMA-approved request for confirmation of the applicability of the agency's decision on class waivers
- include a statement indicating the sponsor is unable to undertake pediatric studies
Adult cancer drugs determined to be substantially relevant to the growth or progression of a pediatric cancer
An adult cancer drug may be substantially relevant to the treatment of a different pediatric cancer. For these products, it may not be sufficient to submit a rationale for not conducting studies solely on the grounds that the condition does not occur in pediatric populations.
To develop C-PDPs for these types of drugs, sponsors are encouraged review the:
For molecularly targeted cancer drugs, sponsors are encouraged to review the US FDA's guidance on pediatric studies of molecularly targeted oncology drugs (PDF).
The US FDA also publishes 2 regularly updated lists to support pediatric development for molecularly targeted cancer drugs:
- molecular targets that are substantially relevant to the growth and progression of pediatric cancers (PDF)
- sponsors of drugs for these molecular targets are asked to:
- submit a C-PDP outlining the planned development program
- explain if studies are not possible based on reasons identified in Rationale for not conducting studies in section 2 of the C-PDP
- non-relevant molecular targets that will be automatically considered for a waiver (Excel document)
- the C-PDP for these drugs should only include the:
- Title page
- 2.2 Rationale for not conducting studies
- identify the molecular target
- include a statement indicating the sponsor is providing a rationale for not conducting any pediatric studies
- this C-PDP should not to exceed 1 page
- the C-PDP for these drugs should only include the:
Tabular summary of planned nonclinical and clinical development
The summary should be provided in a table that:
- lists each individual study included in the proposed C-PDP, along with each study's current status
- organizes the studies in the same order that they are discussed in their respective sections of the C-PDP
- includes any age group for which the sponsor provides a rationale for not conducting studies
The following table is an example of the recommended format for the summary table. Sponsors may use shorthand to describe the proposed studies, provided they give an explanatory note at the end of the table (for example, R = randomized, PC = placebo-controlled).
| Species/age group/study ID (if known) List all groups, including those for which a rationale for not conducting studies has been provided |
Type of study (if known, include dosing/treatment duration) |
Comments (may include study purpose/objective) |
Study status and estimated initiation/ completion date (MM/YYYY) |
|---|---|---|---|
| Nonclinical studies | |||
| Rat Approximately PND 35 Study #: ABC123 |
Dose range finding juvenile toxicology 3 weeks |
Study submitted with NDS application | Completed |
| Clinical studies | |||
| Pediatric PK or PK/PD studies | |||
| 2 to < 12 years | Open-label, single-dose study to evaluate safety, tolerability and PK |
|
Estimated start: 06/2025 Estimated end: 04/2026 |
| Clinical safety and efficacy evaluation | |||
| 0 to < 2 years |
|
|
n/a |
| 2 to < 12 years |
|
|
Enrollment not started Estimated start: 12/2026 Estimated end: 03/2029 |
| 12 to <18 years Study #: XYZ123 |
|
|
Completed |
| Target date for submission to Health Canada | 10/2029 | ||
n/a: not applicable |
|||
Development of age-appropriate formulation
If the current formulation of the drug is not suitable for all pediatric age groups, sponsors should explain how an age-appropriate formulation will be developed for the relevant age groups.
In this section, sponsors would provide information on the:
- proposed pharmaceutical form
- for example, liquid, capsules, tablets, ointment
- route of administration
- strength
- formulation
- appropriateness of the formulation
- for example, discussion of its excipients and ongoing studies in the development of the formulation
Nonclinical studies
In this section, sponsors should:
- provide a brief summary of any completed, ongoing and planned nonclinical studies that support the use of the drug in the proposed pediatric age groups
- include evidence from these studies that supports the maximum dose and duration of treatment to be used in the planned clinical studies (if applicable)
If further nonclinical studies are not required, the sponsor should provide a clear rationale for this conclusion.
If further nonclinical studies are required before starting the proposed clinical trials, sponsors should provide a brief description of study. This description would include, at a minimum:
- study type
- study objectives
- species being studied
- age of animals at start of dosing
- dosing duration
- route of administration
Clinical data to support design and/or initiation of studies in pediatric patients
In this section, sponsors should:
- provide a concise overview of any existing clinical data that has been used to support the design or initiation of pediatric studies
- include results of clinical studies of the drugs conducted to support other indications (if applicable)
- include evidence from studies that support the dose(s) and duration of treatment in the clinical studies
Ongoing and planned pediatric studies
Pediatric pharmacokinetic or pharmacokinetic/pharmacodynamic studies
In this section, sponsors should:
- include a description of each of the ongoing and planned pediatric pharmacokinetic (PK)/pharmacodynamic (PD) studies included in the tabular study summary
- follow the order that they appear in the tabular study summary table
- address the following design aspects for each study:
- study type
- study design and control
- study objectives
- age group and population in which the study will be conducted
- pediatric formulation(s) to be used in the study
- dose ranges to be used in the PK studies
- endpoints and justification (PK parameters, PD biomarkers)
- existing or planned modelling and simulation to support dose selection and/or study design, data analysis and interpretation for planned pediatric studies, including any planned extrapolation
- planned pharmacogenomic analyses
- sample size justification
Dedicated PK studies may not be needed for every age group in a C-PDP. In some cases, it may be appropriate for a sponsor to use alternative studies, such as appropriate dose scaling from adult dosing and/or confirmatory population PK studies, in place of a dedicated PK study. Sponsors should clearly and concisely justify the extrapolation study design.
Clinical efficacy and safety studies
In this section, sponsors should:
- provide a brief outline of each ongoing and planned clinical efficacy and safety study included in the tabular summary
- follow the order that they appear in the table
- address the following design aspects for each study:
- study type and design
- study objectives
- age group and population in which the study will be conducted
- key inclusion and exclusion criteria
- primary and key secondary efficacy endpoints
- timing of endpoint assessments
- safety assessments (including timing and length of follow-up)
- statistical approach
- modelling and simulation to be used to optimize the design of planned pediatric studies, when applicable
A detailed statistical analysis plan is not needed for the PDP.
Agreements for pediatric studies with other regulatory authorities
Sponsors with an agreed-upon foreign pediatric development plan(s) from jurisdictions other than the US FDA and EMA should include a brief summary of the plan(s) in this section. This will help Health Canada align the C-PDP with pediatric plans of other jurisdictions (where possible).
Sponsors should include:
- a copy of the most recent agreed-to or approved version of the foreign plan
- a copy of the most recent draft of the new plan or a brief summary of the changes being made to an existing plan (if negotiations are ongoing for a new or existing plan)
- any differences between the proposed C-PDP and foreign pediatric plan
Reviewing a Canadian PDP
Health Canada reviews, consults with appropriate subject matter experts and makes decisions on all C-PDPs.
Reaching an agreement on a C-PDP is a collaborative process that may require discussion between Health Canada and the sponsor. We may:
- communicate with the sponsor if, during our review, we have questions about the pediatric plan
- request a meeting to discuss elements of the plan in order to reach an agreement
Any communications concerning pediatric plans should be appropriately documented by both stakeholders and Health Canada. The processes for documenting communications, including filing any solicited and unsolicited information during the review of a pediatric plan, are the same as those for drug submissions.
For more information, please consult the following guidance document:
The review period for C-PDPs begins after the screening acceptance letter (SAL) is issued. Health Canada will conduct the pediatric plan review at the same time as the pre-market review. The service standard for reviewing the C-PDP is the same as the service standard of the submission that included the pediatric plan.
If the sponsor and Health Canada cannot agree on the content of the C-PDP, the sponsor may withdraw from the pilot. This will not affect our review decision for the associated NDS or SNDS.
Health Canada will provide the outcome of the assessment through a letter that will be sent at the time of the notice of compliance (NOC). The letter will identify the timeframe within which completed study reports should be submitted to us. All study reports identified in the C-PDP are expected to be submitted within 6 months after the final study has been completed, unless otherwise agreed to by the sponsor and Health Canada.
If:
- the review decision on the submission is negative, no decision will be made on the C-PDP
- a drug authorized for sale in Canada becomes cancelled pre- or post-market, completion of the studies in the C-PDP for that drug will no longer be expected
Health Canada's agreement to a C-PDP is not indicative of a future positive decision on the pediatric studies submitted as a result of the C-PDP for the purpose of obtaining approval of a pediatric indication, formulation or labelling change. We will conduct a full and independent review of all studies included in subsequent drug submissions based on the C-PDP.
Likewise, agreement to a C-PDP does not constitute approval of the clinical trials described in the plan. Sponsors are responsible for seeking approvals for clinical trials as required by the jurisdictions in which the trials take place.
For Canadian clinical trials, sponsors should communicate with the relevant Office of Clinical Trials, in accordance with the:
Amending an agreed-to Canadian PDP
Circumstances may arise during the course of an investigation that require a change to a C-PDP after it has been agreed to by Health Canada. If this is the case, sponsors may request an amendment to the plan.
Examples of such situations may include:
- significant delays in study completion
- for example, over 1 year
- changes to rationales for not conducting studies
- modifications in the types of studies being conducted
All changes to a C-PDP that are outside of the scope of an amendment should be included as part of the annual report.
Sponsors may ask for an amendment to an agreed-to C-PDP at any time. Proposed changes to the C-PDP should be communicated as early as possible to allow for their review.
Amendments should be filed through the Common Electronic Submissions Gateway (CESG) under the regulatory activity type Pediatric Drug Plan – Amendments (PDPAM) in the electronic common technical document (eCTD) format as follows:
| Document type | eCTD module | eCTD document leaf title |
|---|---|---|
| Amended Canadian-specific PDP | 1.0.7 General note to reviewer | Annotated C-PDP Non-annotated C-PDP |
Any supporting quality, nonclinical or clinical studies should be submitted in their respective eCTD modules and organized in accordance with the current electronic specifications in the following guidance document:
For more information on how to file submissions electronically, refer to:
A request for an amendment should include the following:
- note to reviewer, identifying the requested change(s) and justification
- include documentation to support the request (as appropriate)
- track changes (annotated) copy of the agreed-upon C-PDP in Word
- clean version (non-annotated) of the proposed amended C-PDP in Word
Amendments will be subject to a 10-calendar day processing period, a 15-calendar day screening period and a 90-calendar day review service standard.
Amendments to a C-PDP will not be considered agreed-upon until Health Canada notifies the sponsor that it has accepted the amendments.
Canadian PDP template
For guidance on information to include in each section, refer to the Format and content section of this page.
- Title page
- 1. Overview of the disease or condition in the pediatric population
- 2. Proposed pediatric development plan
- 2.1 Overview of planned extrapolation to specific pediatric populations
- 2.2 Rationale for not conducting studies
- 2.3 Tabular summary of planned nonclinical and clinical development
- 2.4 Development of age-appropriate formulation
- 2.5 Nonclinical studies
- 2.6 Clinical data to support design and/or initiation of studies in pediatric patients
- 2.7 Ongoing and planned pediatric studies
- 2.7.1 Pediatric pharmacokinetic or pharmacokinetic / pharmacodynamic studies
- 2.7.2 Clinical efficacy and safety studies
- 3. Agreements for pediatric studies with other regulatory authorities
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