Model-informed drug development (MIDD) is an approach that involves developing and applying exposure-based, biological and statistical models derived from preclinical and clinical data sources to inform drug development and decision-making. MIDD can be seen as foundational to efficient and effective drug development and regulatory evaluation of small molecule drugs and biological products. There have been many regulatory applications of MIDD to address a variety of drug development and regulatory questions. These applications can be broadly classified into four categories: dose optimization, supportive evidence for efficacy, clinical trial design, and informing policy.
With nearly two decades of experience in demonstrating the relevance and value, MIDD has been recognized as a potential enabler of efficient drug development and decision making. With rapid evolution in new methodologies and the enthusiasm for newer applications, here is arguably a greater appreciation of the benefits and promise of MIDD now more than ever. This is reflected by the inclusion of MIDD‐related performance goals in the latest congressionally reauthorized Prescription Drug User Fee Act (PDUFA) VI. Against this backdrop, the key question is how we capitalize on this unique opportunity to mainstream MIDD.
The presentation will provide a brief history of the evolution of MIDD in the Office of Clinical Pharmacology and outline the commitments included in the PDUFA VI. This will be followed by a summary of the various activities under the PDUFA VII MIDD imitative including the early experience and impact. Lastly the presentation will close out with a call for action aimed at mainstreaming MIDD to achieve consistent and relevant application for patient and societal benefit.