The US Food and Drugs Administration (FDA) has outlined recommendations for submitting physiologically based pharmacokinetic (PBPK) analysis data to support regulatory applications. The new draft guidance describes the preferred format and content for a sponsor to submit PBPK analyses to support INDs, NDAs, BLAs and ANDAs. A previous lack of regulatory guidance has meant that the content and presentation of submitted PBPK analyses varied greatly. It is hoped that the new guidance will deliver a standard approach, in turn helping FDA be more efficient and timely with their reviews.
The new draft guidance recommends presenting the information in five sections: an executive summary including a rationale for the analyses and summarise the key conclusions; an introduction describing a synopsis of the drug’s properties and efficacy and safety data; methods and materials including the modelling strategy and parameters, stimulation design and software details; results demonstrating the relevancy of the model and presenting the results; discussion which should include how the analysis addresses the proposed scientific, regulatory, or clinical questions, the basis for any requests to waive clinical studies, proposed dosage recommendations if applicable and any limitations of the modelling approach; plus appendices.
To access the draft guidance, please click here.