MIDD - An Investment for the Future

抄録

Over the last two decades, we have impacted decision making on numerous frontsthrough the application of model-informed drug discovery and development (MIDD)across the whole spectrum of drug development, from bridging animal data topredict human data when clinical studies are unethical, to avoiding Phase IIIstudies, to approving drugs in a population not studied in Phase III trials. Thesingle most important strength of MIDD is its ability to integrate knowledgeacross the development program thereby improving the efficiency of drugdevelopment and probability of success (POS) of new therapeutics. The goal ofdrug development teams should be to fully integrate modeling and simulation(M&S) end-to-end across programs agnostic of therapeutic areas of interesttogether with deep therapeutic area (TA) expertise, pharmacology and competitiveknowledge to enable MIDD.

Historically, the regulatory agencies have played a key role in advocating forthe modernization of drug development. With the recent inclusion of MIDD as oneof the goals in PDUFA VI authorization, it is all the more imperative that weunleash the full potential of M&S in decision making. In addition to thetraditional impacts of M&S i.e., informing dose, regimen, population, trialdesign, Go/No Go decisions etc., there is a huge opportunity to leverage thepredictive capabilities of novel and advancing technologies for e.g., MBMA, QSP,CTS for the evaluation and incorporation of novel endpoints in Phase 2/3 trials,or providing confirmatory evidence of efficacy/safety in lieu of clinical datafor regulatory submissions by conducting integrated analysis of data from acrosstrials and drug classes. There are also untapped benefits of applying AI/MLacross both the traditional and advanced technologies to gain efficiencies indecision making. The key feature driving successful application of MIDD isdependent on the strong collaboration and partnership between all stakeholdersinvolved in drug development, including regulatory colleagues and the agenciesto improve the POS of candidates that are progressed in the pipeline using arisk-based approach to portfolio investment decisions and thereby enable fasteraccess to new and safe treatments with reduced costs.