Navigating Pediatric Drug Development (PDP) in Canada
A comprehensive guide part 2
In the previous blog of this series, we introduced the pilot PDP initiated by Health Canada including the types of PDP that can be submitted and the content and format of a C-PDP. This blog discusses the review of PDPs by Health Canada, amending agreed-to PDPs and annual reporting requirements.
Review of a foreign-approved PDP or a Canadian PDP by Health Canada
Verifying foreign-approved PDP:
Verification of a foreign-approved pediatric plan will consist of confirming that the iPSP or PIP is complete and is in line with C-PDP recommended content. During the verification, Health Canada may seek clarification on parts of the plans. Once Health Canada verifies that the foreign-approved plan meets the C-PDP content recommendations, then it will become identified as the PDP without changes to the plan1.
Review of a C-PDP:
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 specified pediatric population. Health Canada will review, consult with appropriate subject matter experts and make 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. Health Canada may communicate with the sponsor if, during review, they have questions about the pediatric plan or request a meeting to discuss elements of the plan in order to reach an agreement1.
Review process:
The review period for a foreign-approved PDP or a C-PDP will begin after the screening acceptance letter (SAL) is issued for the NDS or SNDS. Health Canada will conduct the pediatric plan review at the same time as the pre-market review. The service standard for reviewing the PDP is the same as the service standard of the submission that included the pediatric plan1.
If the sponsor and Health Canada cannot agree on the content of the foreign-approved PDP or C‑PDP, the sponsor may withdraw from the pilot. This will not affect the review decision for the associated NDS or SNDS1.
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 the regulatory authority. 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 Canada1.
In case1:
- The review decision on the submission is negative, no decision will be made on the foreign-approved PDP or C-PDP
- A drug authorised for sale in Canada becomes cancelled pre- or post-market, completion of the studies in the foreign-approved PDP or C-PDP for that drug will no longer be expected
Health Canada's agreement to a foreign-approved PDP or C-PDP is not indicative of a future positive decision on the pediatric studies submitted under the PDP for the purpose of obtaining approval of a pediatric indication, formulation or labelling change.
Similarly, agreement to a foreign-approved PDP or C-PDP does not constitute approval of the clinical trials described in the plan. Sponsors will be responsible for seeking approvals for conducting clinical trials as required by the jurisdictions in which the trials take place1.
Amending an agreed-to PDP
Amendment to a foreign-approved PDP:
Health Canada requests sponsors to inform them of all amendments once approved by the US FDA or EMA. If an iPSP or EU-PIP is amended with the foreign regulator, the approved amendment may be submitted to Health Canada in the same format in which it has been submitted to the US FDA or EMA1.
Amendment to a C-PDP:
Sponsors can request an amendment to a C-PDP according to conditions and requirements described in Health Canada Guidance: Canadian pediatric development plans: Guidance - Canada.ca. 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. All changes to a C-PDP that are outside of the scope of an amendment should be included as part of the annual report1.
Annual reports
To assist Health Canada to track the progress of pediatric plans within the pilot, sponsors are requested to submit annual reports detailing the status of the studies identified in their PDPs or C‑PDPs.
Sponsors should submit a single annual report for all open PDPs and C-PDPs on the anniversary date of the NOC for a drug with which the first pediatric plan was attached1. Annual reports should include a note to reviewer and the annual report document.
Changes to the PDP identified in an annual report will be noted by Health Canada. An approval letter will not be issued.
Transparency
PDPs submitted during the pilot will be subject to Health Canada’s transparency initiatives. Health Canada’s Notice of Compliance (NOC) website and Drug Product Database website will report the outcome of Health Canada’s regulatory review, but will not contain a direct reference to PDPs. The Drug Health and Product Portal (regulatory decision summaries and summary basis of decision) may refer to the PDPs submitted during the pilot. The summaries may include statements about a submission having a PDP and whether it was based on a foreign PDP or was a C-PDP or the submission contained rationales for not conducting studies in all or part of the pediatric population.
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 is intended 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. As the pilot progresses, the feedback and expertise gained will be instrumental in shaping future policies and enhancing Health Canada's approach to pediatric drug development. Ultimately, this program underscores the commitment to improving pediatric healthcare and ensuring that children receive the best possible treatments based on robust scientific evidence.
To read part 1 of this blog series, click here.
Author
Pratibha Duggal
Regulatory Affairs Manager,
Regulatory Consulting Solutions Canada.
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