The Significance of Model-Based Strategies in Drug Development throughout the Lifecycle and Regulatory...

Model-informed drug development (MIDD) is a regulatory-endorsed approach that streamlines drug discovery, development, and approval. Actively promoted by the U.S. Food and Drug Administration (FDA), it integrates quantitative modelling to support decision-making across drugs, generics, and biologics.

This study aims to highlight the applications, benefits, and future perspectives of MIDD in optimizing clinical trial design, supporting regulatory review, advancing biopharmaceutics, and enabling innovation in emerging therapeutic areas.

Current applications of MIDD were evaluated, focusing on its role in new drug development, generic drug approval, biopharmaceutics, and early exploration in cell and gene therapy. The study emphasizes computational modelling, dose optimization, clinical trial refinement, and postapproval lifecycle management strategies.

MIDD has demonstrated considerable impact in optimizing dose selection, refining trial design, and addressing regulatory concerns regarding efficacy and safety. In the field of biopharmaceutics, computational modelling has guided formulation development and facilitated subsequent in vivo studies. In genetics, mathematical modelling has enabled efficient development and approval of complex formulations, reducing both time and cost. MIDD shows strong potential for quantitatively analysing biological activity, pharmacodynamics, transgene expression, immune responses, safety, and therapeutic effectiveness.

MIDD is a transformative approach in drug development, offering robust tools for decision- making and regulatory assessment. Its broader implications across therapeutic domains are expected to enhance innovation, improve patient outcomes, and reduce development costs. Future advancements, particularly in cell and gene therapy, will further expand its role as a cornerstone of drug development.