• Biospecimen SolutionsPrecision Value & HealthCareers
    P_ForMedicine_no_tag_RGBP_ForMedicine_no_tag_RGBP_ForMedicine_no_tag_RGBP_ForMedicine_no_tag_RGB
    • Clinical
      Trial Services
        • Overview
            • Oncology Clinical TrialsImage

              Oncology Clinical Trials


              Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

              • Rare & Orphan DiseaseImage

                Rare & Orphan Disease


                With experience built on 150+ orphan disease projects covering 80+ rare diseases, we know how to anticipate logistical and regulatory obstacles and craft bold solutions that drive rare disease development programs.

        • Global Clinical Trial Support
            • North & South America
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Europe
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • APAC
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

        • Clinical Trial Management
            • Study Start-up & Feasibility
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Project Management
            • Patient Recruitment
            • Personalized Clinical Trial Operations
            • Biometrics
            • Medical & Safety Monitoring
            • Clinical Data Management
            • Medical Writing
        • Clinical Development Strategy
            • Clinical Development Planning
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Biomarker Strategies
            • Cell & Gene Therapy Strategies
            • CDx Regulatory and Market Access
        • Clinical Trial Design
            • Basket & Umbrella Trials
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Biomarker Clinical Trials
            • Master Protocol Trials
            • Decentralized Clinical Trials
        • Biostatistics
            • Statistical Consulting
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Study Design
            • Data Monitoring Committees
            • CDISC Programming and Documentation
            • eCTD Submissions
        • Central Lab Services
            • Clinical Specimen Kitting
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Sample Processing
            • Global Specimen Logistics and Biostorage
            • Biospecimen Data Services
            • Precision Lab e-Portal
            • Virtual Sample Inventory Management
        • Training
            • Oncology Clinical TrialsImage

              Oncology Clinical Trials


              Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

              • Rare & Orphan DiseaseImage

                Rare & Orphan Disease


                With experience built on 150+ orphan disease projects covering 80+ rare diseases, we know how to anticipate logistical and regulatory obstacles and craft bold solutions that drive rare disease development programs.

    • Lab
      Services
        • Central Lab Services
            • Clinical Specimen Kitting
              • Oncology Clinical TrialsImage

                Oncology Clinical Trials


                Through 20+ years running successful oncology trials, we’ve developed a unique blend of proprietary technologies, flexible processes, and creative problem solving abilities that advance even the most challenging clinical development programs.

            • Sample Processing
            • Global Specimen Logistics and Biostorage
            • Biospecimen Data Services
            • Precision Lab e-Portal
            • Virtual Sample Inventory Management
        • Specialty Lab Services Overview
            • Immune MonitoringImage

              Immune Monitoring


              Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

              • Tissue & Liquid BiopsyImage

                Tissue and Liquid Biopsy


                Obtain a detailed molecular view into patient biology with a range of tissue and liquid biopsy technologies, including our proprietary ApoStream platform which can isolate and enrich circulating tumor cells for downstream analysis.

        • EU Contract Research Organization
        • Immune Monitoring
            • Immune Monitoring by Flow Cytometry
              • Immune MonitoringImage

                Immune Monitoring


                Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

            • Immune Cell Phenotyping by Epiontis ID
            • Cytokine Profiling
            • Gene Expression Profiling
            • ELISpot and FluoroSpot
            • Custom Assays
        • Flow Cytometry
            • Receptor Occupancy Assays by Flow Cytometry
              • Immune MonitoringImage

                Immune Monitoring


                Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

        • Genomics
            • NanoString Assays
              • Immune MonitoringImage

                Immune Monitoring


                Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

            • ddPCR
            • qPCR
            • NGS
            • Fluorescence In Situ Hybridisation (FISH) / ISH
        • Tissue & Liquid Biopsy
            • ApoStream – CTC and Rare Cell Isolation for Liquid Biopsy
              • Immune MonitoringImage

                Immune Monitoring


                Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

            • CTC / cfDNA / Exosome Analysis
            • Multiplex Immunofluorescence
            • Immunohistochemistry
            • Fluorescence In Situ Hybridisation (FISH) / ISH
            • Pathology Services
            • NanoString Assays
        • Bioanalytical Testing
            • Immunogenicity Testing
              • Immune MonitoringImage

                Immune Monitoring


                Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

            • PK Assays
            • ADA Assays
            • MesoScale Discovery (MSD) Assays
            • Quanterix SIMOA
            • ELISA
        • Protein & Cytokine Analysis
            • ELISA
              • Immune MonitoringImage

                Immune Monitoring


                Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

            • MesoScale Discovery (MSD) Assays
            • Luminex
            • Quanterix SIMOA
            • Automated Western Blotting
        • Preclinical Target and Biomarker Validation
            • Cell Based Assays
            • Target Expression – XpressWay Profiles
            • Tissue Cross-Reactivity
            • Fluorescence In Situ Hybridisation (FISH) / ISH
            • Multiplex Immunofluorescence
            • Immunohistochemistry (IHC)
        • Biospecimens
            • Immune MonitoringImage

              Immune Monitoring


              Leverage sophisticated immune monitoring approaches, including flow cytometry, Epiontis ID immune cell phenotyping, and cytokine profiling, to characterize the immune cells in your samples and understand their impact on patient biology.

              • Tissue & Liquid BiopsyImage

                Tissue and Liquid Biopsy


                Obtain a detailed molecular view into patient biology with a range of tissue and liquid biopsy technologies, including our proprietary ApoStream platform which can isolate and enrich circulating tumor cells for downstream analysis.

    • Data
      Sciences
        • Overview
            • BiostatisticsImage

              Biostatistics


              Seasoned biostatisticians and statistical programmers support every phase of your trial, from study design for small, rare disease trials to regulatory submissions, all backed by meticulous documentation.

              • QuartzBioImage

                QuartzBio® Solutions


                Purpose-built SaaS solutions developed to address the complex, manual, and siloed processes that exist in sample and biomarker data management by delivering innovative data-enabling technologies in a single, scalable platform.

        • Biostatistics
        • Clinical Data Management
        • Biometrics
        • QuartzBio® Solutions
            • enterprise Biomarker Data Management (eBDM)
              • BiostatisticsImage

                Biostatistics


                Seasoned biostatisticians and statistical programmers support every phase of your trial, from study design for small, rare disease trials to regulatory submissions, all backed by meticulous documentation.

            • virtual Sample Inventory Management (vSIM)
            • Analytics, Information, Insights, and Reporting
    • Diagnostics
      & CDx
        • IVD and CDx Regulatory Affairs Consulting
          • spevialty_labs_slice-300×54Image

            Specialty Lab Services


            Our comprehensive approach starts with a foundation of expertise in key biomaker discovery and analysis methods such as immune monitoring, genomics, and bioanalysis.

        • COVID-19 & Viral Panel Assays
          • spevialty_labs_slice-300×54Image

            Specialty Lab Services


            Our comprehensive approach starts with a foundation of expertise in key biomaker discovery and analysis methods such as immune monitoring, genomics, and bioanalysis.

        • Biospecimens Solutions 
          • spevialty_labs_slice-300×54Image

            Specialty Lab Services


            Our comprehensive approach starts with a foundation of expertise in key biomaker discovery and analysis methods such as immune monitoring, genomics, and bioanalysis.

        • Diagnostic Market Access Services
          • spevialty_labs_slice-300×54Image

            Specialty Lab Services


            Our comprehensive approach starts with a foundation of expertise in key biomaker discovery and analysis methods such as immune monitoring, genomics, and bioanalysis.

        • Companion Diagnostics
    • Therapeutic
      Areas
        • Overview
            • By Specialty Area
              • Global-clinical-trial-support-2Image

                Global Clinical Trial Support


                Sample processing labs, clinical trial sites and offices in five continents provide the clinical reach and scale to manage complex global programs.

            • Oncology and Immuno-Oncology
              • Oncology Clinical Trials
            • Rare & Orphan Disease
              • Global-clinical-trial-support-2Image

                Global Clinical Trial Support


                Sample processing labs, clinical trial sites and offices in five continents provide the clinical reach and scale to manage complex global programs.

            • Cell & Gene Therapy
              • Cell Therapy
              • Gene Therapy
              • Cell and Gene Therapy Manufacturing
            • CNS & Neuroscience
              • Global-clinical-trial-support-2Image

                Global Clinical Trial Support


                Sample processing labs, clinical trial sites and offices in five continents provide the clinical reach and scale to manage complex global programs.

            • COVID-19
            • AutoImmune & Allergy
            • Pediatric
        • By Research Phase
        • Preclinical Development
        • Early Phase Trials (I-IIa)
        • Late Phase Trials (IIb-III)
    • News &
      Insights
        • Blog
            • iStock-945738094Image

              Blog


              Industry trends and best practices from Precision experts

              • shutterstock_1033743346Image

                Resources


                A searchable hub featuring the latest Precision case studies, white papers, capabilities brochures and more.

        • Resources
        • Thought Leadership
        • News
            • iStock-945738094Image

              Blog


              Industry trends and best practices from Precision experts

              • shutterstock_1033743346Image

                Resources


                A searchable hub featuring the latest Precision case studies, white papers, capabilities brochures and more.

        • Events
            • iStock-945738094Image

              Blog


              Industry trends and best practices from Precision experts

              • shutterstock_1033743346Image

                Resources


                A searchable hub featuring the latest Precision case studies, white papers, capabilities brochures and more.

    • About
      Us
        • Our Passion
            • Patient RecruitmentImage

              Our Passion


              What we do, why we do it and the principles that guide us every day.

              • meet-our-peopleImage

                Meet Our People


                We are devoted professionals with deep expertise in translational science, clinical trials and data sciences.

        • Meet Our People
        • Careers
        • Regulatory Information
            • Patient RecruitmentImage

              Our Passion


              What we do, why we do it and the principles that guide us every day.

              • meet-our-peopleImage

                Meet Our People


                We are devoted professionals with deep expertise in translational science, clinical trials and data sciences.

    • Contact
      Us
    ✕
    • Home
    • FDA Guidance on Pediatric Drug Development
    Blog Clinical Research Insights Translational & Lab Insights
    Article

    FDA Guidance on Pediatric Drug Development

    Written By Vann Parker, PhD
    July 25, 2023 • 5 min. read

    Pediatric Populations Require Special Considerations for Drug Development

    FDA generates guidance documents to convey their current thinking related to areas they regulate. Currently there are more than 2700 listed on their website. Often, guidance documents are produced that summarize and expand upon information included in prior postings. Several guidance documents have been issued this year summarizing considerations for the development of drugs in pediatric patients:

    • Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations, May 2023
    • 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
    • Research Involving Children as Subjects and Not Otherwise Approvable by an Institutional Review Board: Process for Referrals to Food and Drug Administration and Office for Human Research Protections: Draft Guidance for IRBs, Investigators, and Sponsors, March 2023

    Defining Pediatric Age Groups to Guide Clinical Trials

    FDA recognizes four distinct pediatric patient populations:

    • Neonates – birth through 27 days, corrected for gestational age
    • Infants – 28 days to 23 months
    • Children – 2 to 11 years
    • Adolescents – 12 to less than 17 years

    The Food and Drug Modernization Act of 1997 (FDAMA) established incentives for conducting trials in pediatric patients. Congress subsequently passed the Best Pharmaceuticals for Children Act (BCPA, 2002) and Pediatric Research Equity Act (PREA, 2003).

    In addition to codifying incentives for conducting studies, sponsors are obligated to submit a pediatric assessment to accompany an NDA or BLA submission. This is typically done through the filing of an initial Pediatric Study Plan (iPSP).

    Recommended Reading

    Considerations for Your Pre-IND Meeting With the FDA

    Timeline for Submitting and Confirming Pediatric Study Plans with FDA

    FDA issued a guidance document in 2020 detailing the process for submission of a pediatric study plan including expectations for content.

    • Pediatric Study Plans: Content of and Process for Submitting Initial Pediatric Study Plans and Amended Initial Pediatric Study Plans, July 2020

    The review period for an iPSP can be up to 210 days, incorporating FDA review and comments, revisions by the sponsor and resubmission, and FDA secondary review and plan confirmation. To ensure the plan is in place by the time of NDA submission, the initial plan should be submitted within 60 days of the end-of-phase 2 meeting.  

    The iPSP needs to describe plans for each of the pediatric populations and the timing for when studies will be conducted. Requests for a deferral, typically until after pivotal studies in adults are completed or after drug approval, can be made and justified in the plan. Likewise, a waiver may be requested if studies are impractical due to the nature of the disease, or an unfavorable benefit-risk exists. Note: applications for drugs that have orphan designation are exempt from having to submit an iPSP.

    However, the FDA Reauthorization Act of 2017 (FDARA) amended PREA for targeted oncology drugs. Applications for targeted oncology drug approval now require an iPSP evaluating pediatric cancer indications irrespective of orphan drug status. For instance, application for approval of a drug for an orphan oncologic disease that only occurs in adults such as a BRAF inhibitor for melanoma would require an iPSP for glioma in children.

    FDA maintains lists of those molecular targets that do or do not require an iPSP.

    Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act

    In March 2019 FDA issued a guidance document discussing how to incorporate pediatric dosing information into product labeling. The document describes consistent placement of information to assist health care providers in the safe use of drugs in pediatric patients.

    In general, whatever is known about use in pediatric populations should be placed in the subsection 8.4 “Use in Specific Populations: Pediatric Use”. If studies have been conducted that FDA has concluded support use of the drug, appropriate information should also appear in sections describing the data derived from studies.

    Negative or incomplete studies should not appear elsewhere in labeling unless there is a contraindication for use, which could be placed in the Contraindication, Boxed Warning, and Warnings and Precautions sections.

    A separate guidance specific to clinical pharmacology was issued in 2022 which emphasizes the need to understand drug disposition in individuals with the disease being treated.

    • General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products, Sept. 2022

    Ethical Requirements for Clinical Trials Involving Children

    The practical and ethical considerations for inclusion of pediatric patients in clinical trials are spelled out in 21 CFR part 50, Subpart D and are included in the draft guidance issued by FDA in 2022.

    • Ethical Considerations for Clinical Investigations of Medical Products Involving Children Guidance for Industry, Sponsors, and IRBs, Sept 2022

    Perhaps the most important element of that is the condition that any investigation involving greater than minimal risk requires the intervention to provide the “prospect of direct benefit” to participants.

    This presents unique challenges of balancing what is likely to be a safe dose in pediatric patients with what could possibly be an effective dose. Strategies to identify an appropriate dose commonly include prior testing in adults. When testing in adults is not feasible (e.g., a gene therapy for a life-threatening pediatric disease) support needs to come from preclinical evaluations or related clinical trials.

    Incentives for Conducting Pediatric Studies: Extensions, Waivers, and Vouchers

    In addition to requirements for conducting clinical trials in pediatric patients, congress has enacted several incentives for doing so. A six-month extension of product exclusivity can be granted if pediatric studies are completed that comply with a written request for studies from the FDA. Note: this is not the same agreement contained in an iPSP.

    If the indication is eligible for Orphan Drug designation, that is there are less than 200,000 patients with the condition in the US, incentives include a waiver of PDUFA filing fees for the NDA/BLA (currently more than $3,200,000) and seven years of market exclusivity.

    If the orphan indication is specific to pediatric patients, approval can be accompanied by the granting of a Rare Pediatric Disease Priority Review Voucher (PRV). These allow a company to request Priority Review of a submission to potentially accelerate FDA review and approval of a marketing application. Most PRVs have been sold at a price in excess of $100,000,000.

    Recommended Reading

    Rare Disease Clinical Trials in Pediatric Populations: Lessons Learned

    Key takeaway

    Recognition of the need for more studies to understand the safety and effectiveness of drugs in the pediatric population has led to an increased focus from the FDA and congress for clinical trials that include pediatric subjects.

    Understanding the requirements and incentives for pediatric drug development are important for all drug developers. Ultimately, these efforts are designed to enhance the health and medical care of children.

    Learn about the regulatory pathway that might lie ahead for your next study.

    Speak with an expert >


    • Vann Parker, PhD

      Vann Parker is the Head of Regulatory Consultancy (North America) for Precision for Medicine. He received his PhD in Molecular Biology from The California Institute of Technology and spent the past 30 years supporting biopharmaceutical and drug development. He currently focuses on strategies for clinical development and interactions with FDA and other regulatory agencies. In addition to support of pre-IND activities, Vann has supported sponsors throughout the drug development process including applications for Fast Track, Breakthrough Therapy and Orphan Designations, formal meetings with FDA and full NDA/BLA submissions.

    Related Posts

    FDA Accelerated Approval for Oncology Therapeutics: Clinical Trial Considerations

    Vann Parker, PhD

    Jun 19, '23 · 3 min. read

    FDA Procedural Guidance: Expansion Cohorts in FIH Oncology Clinical Trials

    Vann Parker, PhD

    Jan 26, '23 · 5 min. read

    COVID-19 Home Testing: Looking Back at the Journey to Authorization

    Jo-Ann Fabila Gonzales

    Jun 07, '22 · 5 min. read

    IVD Manufacturers & EU IVD Regulation Changes (May 2022)

    Jo-Ann Fabila Gonzales

    Mar 16, '22 · 6 min. read

    Precision for Medicine is part of the Precision Medicine Group, an integrated team of experts that extends Precision for Medicine’s therapeutic development capabilities beyond approval and into launch strategies, marketing communication, and payer insights. As one company, the Precision Medicine Group helps pharmaceutical and life-sciences clients conquer product development and commercialization challenges in a rapidly evolving environment.

    Visit the website
    Clinical Trial Services
    • Overview
    • Global Clinical Trial Support
    • Clinical Trial Management
    • Clinical Development Strategy
    • Clinical Trial Design
    • Biostatistics
    • Clinical Sample Management
    • Clinical Research Organization
    Specialty Lab Services
    • Overview
    • Immune Monitoring
    • Flow Cytometry
    • Genomics
    • Tissue & Liquid Biopsy
    • Bioanalytical Testing
    • Cytokine Analysis
    • Biospecimens
    • Sample Processing
    • Global Specimen Transport & Biostorage
    • Contract Research Organization
    • Bioanalysis
    • Neutralizing Antibody (NAb) Assays
    • PBMC Processing
    Data Sciences
    • Overview
    • Biostatistics
    • Clinical Data Management
    • Biometrics
    • QuartzBio
    • Virtual Sample Inventory Management
    Therapeutic Expertise
    • Overview
    • Preclinical Development
    • Early Phase Trials (I-IIa)
    • Late Phase Trials (IIb-III)
    • Oncology and Immuno-Oncology
    • Rare & Orphan Disease
    • Cell & Gene Therapy
    • Neurology
    • COVID-19
    • Autoimmune & Allergy
    • Pediatric
    • Companion Diagnostics
    About Us
    • Our Passion
    • Meet Our People
    • Careers
    • ESG Policy
    • Regulatory Information

    News & Insights
    • Publications
    • Case Studies
    • Thought Leadership
    • News
    • Events
    © 2023 Precision Medicine Group, LLC. All rights reserved. Privacy Policy    GDPR