Guidance on submitting pediatric development plans and pediatric studies: When to submit plans and studies

Pediatric studies, data and evaluations are referred to as "pediatric studies" throughout this guidance document, unless otherwise indicated.

Sponsors are asked to include the studies with the new drug submission (NDS) or supplement to a new drug submission (SNDS) for the adult indication if a:

• pediatric study plan has been completed or
• pediatric study package is ready for regulatory review

Current guidelines and requirements for drug submissions continue to apply as outlined in the Food and Drug Regulations (FDR). Assessment of the studies will be conducted based on Health Canada's established approach for assessing drug safety, efficacy and quality in a pediatric population.

For information on the data protection provisions of the FDR or on the Patented Medicines (Notice of Compliance) Regulations, manufacturers should consult the following guidance documents:

• Data protection under C.08.004.1 of the Food and Drug Regulations
• Patented Medicines (Notice of Compliance) Regulations

Submission of pediatric studies with an NDS or SNDS will not constitute participation in the pilot, as this will continue to follow the established process.

If the pediatric studies are incomplete when the NDS or SNDS are filed, sponsors who wish to participate in the pilot should include a pediatric development plan (PDP) with their submission. This will notify Health Canada that you intend to provide the studies at a later date.

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

• 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 address:

• existing and proposed indications
• new dosage forms (if applicable)
• new routes of administration (if applicable)

Sponsors may also choose to develop pediatric-specific indications based on the active ingredient of the drug. For more information, please refer to the format and content section of the Canadian plans web page.

For more information on submitting a PDP, please refer to the following pages on:

• plans approved by a foreign authority
• Canadian pediatric development plans

If investigation of the drug in all or part of the pediatric population may not be appropriate or practical, sponsors who wish to participate in the pilot should include a rationale in the PDP.

The rationale must:

• explain in detail why the studies will not be undertaken
• contain the type of information included in a waiver request to the US Food and Drug Administration (US FDA) or European Medicines Agency (EMA)

For more information, please refer to the following section:

• Rationale for not conducting studies

Sponsors may submit 1 of 2 types of PDPs to Health Canada:

1. Foreign PDP:
   • may include an agreed US FDA initial Pediatric Study Plan (iPSP) or an approved EMA Pediatric Investigation Plan (EU-PIP)
     • guidance on how to complete a U.S. FDA iPSP
     • guidance on how to complete an EMA PIP (PDF)
   • may add an optional Canadian addendum to an approved foreign plan at the discretion of the sponsor if there is additional Canadian-specific context or new relevant information for Canada to consider
   • Health Canada is not accepting pediatric plans from regulators other than the US FDA or EMA as part of the pilot
2. Canadian-specific PDP:
   • may prepare and include a Canadian-specific PDP (C-PDP) if no appropriate foreign plan is available from the US FDA or EMA

Sponsors may ask for a pre-submission meeting to discuss all aspects of their submissions, including PDPs. Health Canada can advise you on:

• the most appropriate approach to take when submitting pediatric studies and/or PDPs
• any unique circumstances that are not directly addressed in this guidance document

Health Canada encourages all NDS and SNDS applications within the scope of this guidance to participate in the policy pilot. While the pilot is taking place, we will be asking all sponsors submitting an NDS or SNDS to answer a brief 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 scope.

Sponsors who choose to not participate will be asked to explain why. A decision to not participate in the pilot and the reasons given will not affect our review of their NDS or SNDS.

When filing the drug submission, sponsors are asked to specify in the note to reviewer if they are including pediatric studies or a pediatric development plan.

Documents should be submitted in the electronic common technical document (eCTD) format as follows:

Documents submitted in the eCTD format

Document type	eCTD module	eCTD document leaf title
Survey	1.0.7 General note to reviewer	Survey
Foreign agreed/approved PDP(s)	1.2.7 International information	FDA iPSP or EMA PIP
Canadian-specific PDP	1.0.7 General note to reviewer	Canadian-specific PDP
Canadian addendum to PDP	1.0.7 General note to reviewer	Canadian addendum to PDP

Any supporting quality, nonclinical or clinical studies should be submitted in their respective eCTD modules and be organized in accordance with the current electronic specifications in the following guidance document:

• Preparation of drug regulatory activities in Electronic Common Technical Document (eCTD) format

For more information on how to file submissions electronically:

• Filing submissions electronically

For information on the pre-submission meeting, screening process, clarification requests, filing a submission and timelines/performance standards, consult:

• The management of drug submissions and applications