Understanding drug effects in children

The evolution of the pediatric regulatory environment has increased the demand to develop a common scientific-based approach that is acceptable to global regulatory authorities.  It is crucial to create a thoughtful plan to support efficient pediatric drug discovery and development to ensure safe and effective medicines for children.  Typical challenges encountered in a pediatric program include:

  • Enrollment challenges due to small populations and multiple sponsors targeting the same population
  • Burden of research procedures
  • Limitation in use of placebo and lack of active comparators
  • Physiological differences across the entire pediatric population
  • Bridging of knowledge from adults to pediatrics

Certara has unique and extensive capabilities and experience to address these challenges and help set up your pediatric program for success.

With experience working on over 100 pediatric programs, we have accumulated a wealth of experience in pediatric drug development that is unparalleled, leveraging our industry-leading Simcyp Pediatric PBPK Simulator and our pediatric expertise spanning model-informed drug discovery and development.  Modeling and simulation, integrated with regulatory science and real world evidence, is the cornerstone of all pediatric development programs.  This integrated approach rooted in model-informed drug discovery and development enables:

  • Proper dose selection
  • Optimal clinical trial design and endpoint selection
  • Extrapolation of efficacy from adults to pediatrics

Our multi-disciplinary team of experts provides you with end-to-end, integrated solutions, supporting you throughout the entire pediatric drug discovery and development program.  Alternatively, we can supplement specific areas where you need additional support, including developing your pediatric investigational plan (PIP) and initial pediatric study plan (iPSP).

Unique capabilities in pediatric drug development

Recognizing the great importance of serving pediatrics balanced against the inherent hurdles, Certara Pediatrics offers comprehensive pediatric drug development services focused on:
• Strategic planning of pediatric development plans
• Clinical pharmacology strategy and stewardship of complex pediatric programs
• Pediatric clinical trial study design
• Real world evidence
• Juvenile toxicology
• Innovative modeling and simulation, characterizing PK, PD, disease progression, safety and efficacy
• Regulatory strategy and writing from IND through submissions and post-marketing
• Interactions with health authorities
• Pediatric formulations

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RACE for the Children Act went into effect on August 18, 2020

With the RACE for Children Act, the FDA is now authorized to require sponsors to submit an agreed initial Pediatric Study Plan (iPSP) with an original NDA or BLA oncology product that may target the growth or progression of a pediatric cancer. New cancer therapies with orphan-designated indications will no longer be exempt from PREA.

The iPSP must include an outline of the planned pediatric investigation(s) and any planned request for deferral or waiver with supporting documentation.  According to the guidance by the FDA, a sponsor must submit the iPSP no later than 60 calendar days after the date of the end-of-phase 2 meeting.

What are the important things you need to know about the RACE Act?  How can you set up your pediatric program for success? 

Learn more about the RACE for the Children Act
Set your pediatric program up for success

Certara scientists and experts meet the needs of the regulators, enhance feasibility of the studies, and increase safety for pediatrics.

Certara’s quantitative methods leverage sparse data and prior information from pre-clinical studies, adult trials, literature data, and pediatric studies of related indications or drug actions. We build that knowledge into models of patient physiology, drug actions and trial characteristics, which enable us to develop and iterate clinical trial design and explore alternative dosing scenarios, in silico patient responses, drug-drug interactions, and whole trial outcomes

Our consultants also use the Simcyp™ Pediatric PBPK Simulator, the industry’s most sophisticated physiologically-based pharmacokinetic (PBPK) technology for modeling drug performance and assessing drug-drug interactions in neonates, infants, and children. The Simcyp Pediatric PBPK Simulator, routinely used by the Simcyp Consortium’s 35 leading pharmaceutical companies, contains extensive libraries on demographics, developmental physiology, and the ontogeny of drug elimination pathways. It is used by all of the industry’s leading pharma companies.

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Regulatory writing, publishing and submission support for your PIP and PSP

Our regulatory science expertise in pediatrics spans more than 15 therapeutic areas, including oncology, infectious diseases, and cardiovascular disorders. Core competencies include consulting, writing, preparing and managing documents, including pediatric investigational plans (PIPs) and pediatric study plans (PSPs) through global regulatory processes, and authoring scientific and commercial documents.

Oftentimes, Certara will support the PIP/PSP development plan by creating a population PK (popPK) model using adult data scaled to pediatrics. That model will integrate a range of maturation and disease factors with allometric scaling to set the best dose for the first pediatric trial cohort.

Furthermore, our GlobalSubmit™ clinical trial operations technology facilitates the eCTD submittal process, providing scalability and consistency for sponsors.

Learn more about our regulatory science solutions
Lynne Georgopoulos, RN, MSHS, RAC Vice President, Regulatory Strategy

Lynne has over 30 years of biopharmaceutical and CRO experience providing strategic, clinical development and regulatory advice at all stages of product development. She has spent the last several years promoting innovative methods to accelerate pediatric drug development as VP of Regulatory Affairs and Pediatric Strategic Development at Synteract, and Sr. VP Regulatory Affairs and Clinical Development at KinderPharm.  She has lead numerous successful regulatory filings and agency interactions (e.g., Pre-IND, INDs, NDA/BLAs, EMA scientific advice, EOP2, and Pre-NDA).

Patrick Smith, PharmD Senior Vice President, Integrated Drug Development, Strategy

With over 20 years of global drug development experience, Patrick works across all phases of drug development life cycle with particular expertise in infectious diseases, oncology, and inflammation as well as novel early development program design and applying modeling and simulation to solve critical development problems. Patrick has over 125 peer reviewed publications, in journals including NEJM and Lancet.

Trevor Johnson, PhD Principal Scientist

Trevor leads the development of Simcyp pediatric software designed to predict dose, drug-concentration time profiles and likely drug response from birth onwards. His current areas of research focus on pediatric oral drug absorption & biologics, ontogeny of transporters and also special population’s particularly hepatic impairment.  He has over 50 research publications and 7 book contributions in the areas of pharmacokinetics, drug metabolism, pediatric drug therapy and hospital Pharmacy

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