Learning Center

Group of diversity kids boys and girls conductive experiments with periodic table on background

Learning Center

IACT4C ensures that clinical sites remain at the forefront of quality and trial readiness by providing continuous education and training.

This proactive approach is crucial in the fast-paced realm of medical advancements, where timely access to innovative treatments can make a significant difference in the lives of children. The comprehensive strategy that includes regular calls with all sites featuring guest speakers, Educational Webinars, and Pediatric Research Innovation Forums, among others, fosters an environment of shared knowledge and continuous improvement. The collaboration with the Pediatric Research Site Network (PRSN) to drive quality improvement initiatives is a strategic move to accelerate the delivery of new medicines and devices to sick children of all ages.

Pediatric Forums

Exchange of ideas on cutting-edge research & innovation in pediatric drug development.

Best Practice

Identifying & sharing best practices in the conduct of pediatric clinical research.

Mentorship Program

Aimed at supporting & expanding the next generation of pediatric researchers.

Educational Webinars

Focused on the latest in pediatric clinical trials innovation & research.

Quality Improvement

Continuous actions to improve the conduct of pediatric clinical trials.

Educational Resources

Webinars are supported through an Educational Grant from Gilead Science.

Educational Webinars

Pediatric Cell and Gene Clinical Research

Consenting & Assenting in Pediatric Clinical Research

Strategies for Improving Study Start-up Timelines

Building a Culture of Clinical Research

Why Pediatric Trials Need to Grow Up

Enhancing Site and Sponsor Relationships

Kids are Not Small Adults

Decentralized Clinical Trials

Remote Monitoring

Pediatric Clinical Trial Budgets

Coverage Analysis

Electronic Informed Consent

Recruitment and Retention Clinical Research Staff

Research Compliance No Tears Approach

Fair Market Value - Impact on Pediatric Clinical Trial Budgets

Guest Speakers

Publications

I-ACT for Children: helping close the gap in drug approval for adults and children.

Emergency Use Authorization for Remdesivir: A Pediatric Perspective

Inclusion of Adolescents in Adult Clinical Trials: Report of the Institute for Advanced Clinical Trials for Children's Pediatric Innovation Research Forum.

Why Pediatric Clinical Trials Need to Grow Up

Harmonizing quality improvement metrics across global trial networks to advance paediatric clinical trials delivery, Therapeutic Innovation & Regulatory Science

Quality management with QA (assurance), QC (control) and improvement. Standardization and certification concept. Compliance to regulations and standards. Concept with manager or auditor.

Pediatric Regulations

Overview of Pediatric Regulations

Two pediatric regulations, the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children’s Act (BPCA) have resulted in over nine hundred labeling changes since their implementation. These two regulations mirror the stick-and-carrot approach to encouraging sponsors to include pediatric studies in their drug development.

PREA (“the stick”) requires certain new drugs and biologic applications to contain a pediatric assessment - the data necessary to label the product in pediatric patients - unless the requirement for conducting pediatric studies is waived or deferred. PREA is triggered by applications with a new active ingredient, formulation, dosage form, dosing regimen or route of administration. PREA requirements apply only to the indication that is the subject of the application. Except for oncologics, orphan products are exempt from PREA. In order to reach agreement with FDA on any planned waivers or deferrals, as well as the type of studies and age groups required to complete the pediatric assessment, an initial Pediatric Study Plan (iPSP) must be submitted no later than 60 days after an end-of-phase 2 meeting. For life-threatening conditions, an iPSP should be submitted earlier. Agreement on the iPSP must be reached before an NDA or BLA is submitted.

Guidance for the content of an iPSP can be found at: https://www.fda.gov/media/86340/download.

BPCA (“the carrot”) permits sponsors to potentially obtain 6 months of marketing exclusivity (“pediatric exclusivity”) for voluntarily performing pediatric studies. The scope of BPCA is broader than PREA. For that reason, studies can be requested for any indication for which the drug or biologic confers a public health benefit and not solely the indication which may be the focus of the NDA or BLA. In addition, studies for orphan products and/or rare diseases may be part of studies requested by FDA. To qualify for pediatric exclusivity, a sponsor must “fairly respond” to study requirements delineated in a Written Request (WR) issued by FDA. The sponsor can submit a Proposed Pediatric Study Request (PPSR) to FDA that outlines the indications to be addressed and studies that they intend to conduct in order to receive a WR. FDA will respond to a PPSR by either issuing a WR or an inadequate letter that outlines any deficiencies in the program.

If adult efficacy has been established, a “stand-alone” pediatric program may not be required to support a pediatric indication. Efficacy may be extrapolated from adequate and well-controlled studies in adults (or an older pediatric population) if the disease course and pathophysiology, along with response to treatment, is sufficiently similar between the two populations. The type of evidence to support extrapolation depends on the certainty that if a drug works in adults, it is likely to work in children. Neither dosing nor safety can be fully extrapolated; therefore, typically pharmacologic and safety data in pediatric patients is necessary to establish dosing and safety. However, data from other populations can be leveraged. The approach to pediatric extrapolation is evolving as more pediatric data in different conditions and drug classes are available.

Pediatric Regulations and Guidance

**Link to additional FDA Guidance Documents and other regulatory guidance:

http://lists all official FDA Guidance Documents and other regulatory guidance.

Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act - MAY 2023

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-drug-development-regulatory-considerations-complying-pediatric-research-equity-act-and

FDA Guidance - Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations - MAY 2023

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-drug-development-under-pediatric-research-equity-act-and-best-pharmaceuticals-children-act

FDA Guidance - Pediatric Study Plans: Content of and Process for Submitting Initial Pediatric Study Plans and Amended Initial Pediatric Study Plans - JULY 2020

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-study-plans-content-and-process-submitting-initial-pediatric-study-plans-and-amended

FDA Guidance - General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products - SEPTEMBER 2022

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/general-clinical-pharmacology-considerations-pediatric-studies-drugs-including-biological-products

FDA Draft Guidance - Ethical Considerations for Clinical Investigations of Medical Products Involving Children - Draft Guidance for Industry, Sponsors, and IRBs - SEPTEMBER 2022

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/ethical-considerations-clinical-investigations-medical-products-involving-children

FDA Guidance - Informed Consent Guidance for IRBs, Clinical Investigators, and Sponsor - AUGUST 2023

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/informed-consent

U.S. Department of Health and Human Services -

Informed Consent FAQs

https://www.hhs.gov/ohrp/regulations-and-policy/guidance/faq/informed-consent/index.html

FDA Guidance -E11A Pediatric Extrapolation - AUGUST 2022

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/e11a-pediatric-extrapolation

FDA Guidance -Cancer Clinical Trial Eligibility Criteria: Minimum Age Considerations for Inclusion of Pediatric Patients - JULY 2020

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/cancer-clinical-trial-eligibility-criteria-minimum-age-considerations-inclusion-pediatric-patients

FDA Guidance - FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act - Guidance for Industry - MAY 2021

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/fdara-implementation-guidance-pediatric-studies-molecularly-targeted-oncology-drugs-amendments-sec

Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies
Draft Guidance for Industry - JUNE 2024

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/diversity-action-plans-improve-enrollment-participants-underrepresented-populations-clinical-studies?utm_medium=email&utm_source=govdelivery

FDA Guidance - Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products - Guidance for Industry - AUGUST 2023

(also applies to pediatrics)

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug