Pediatric PBPK models have broad application in the drug development process and are being used increasingly to optimize and replace clinical studies. However, the approach has yet to become fully integrated in regulatory submissions. Emerging data support an expanded integration of the PBPK model informed approach in regulatory guidances on pediatrics. Best practice standards are presented for further development through interaction between regulators, industry, and model providers.
- An increasing number of examples of the application of PBPK to replace studies in the pediatric population are beginning to emerge.
- A pediatric study (PSP) or investigation plan (PIP) has to be submitted to the regulators early in drug development. Both the FDA and the EMA guidelines for developing medicines for pediatrics support the use of PBPK models to determine the optimal dose for children.
- The recent EMA guidelines present a framework related to the intended use of PBPK separated into low, medium, and high impact applications which is linked to the required level of qualification.
- A set of best practice standards, based on the intended use of the PBPK model, is clearly needed.
- A summary of the pediatric regulatory guidance documents and case studies supporting best practice approaches will be presented.
- Karen Rowland Yeo, PhD. – SVP, Client and Regulatory Strategy, Certara
- Trevor Johnson, PhD- Principal Scientist, Certara
- Eva Gil Berglund, PhD, Senior Director, Certara