Model-Informed Drug Development
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.
Greater understanding of the drug, a disease, and how a drug affects the human body
Streamline drug development by replacing some experimental data collection with simulated data
Generate information that cannot or would not be obtained experimentally
Move Smarter & Faster
MIDD can streamline and accelerate the development of new medical products by helping to design better future trials. In some instances, MIDD can reduce the need for additional clinical trials, require fewer patients for studies, or increase the probability of success by aiding in the selection of the right dosing, duration, and patient population. These advantages enable more informed decision-making, and reduce uncertainty when moving between phases of development, giving sponsors confidence to move more quickly to the next phase.
GAIN INSIGHTS
MIDD is unique in that it can provide insights about the benefits and risks of new therapies beyond the primary intent of clinical trials.
Clinical trials are carried out in a sample of patients with a disease, and are aimed mainly at answering one or several questions, such as whether the primary outcome measure differs significantly between or among treatment groups. But other questions may arise during regulatory review and once a new treatment is approved. For example:
- Can the dosing be optimized for the general population or high-risk subpopulations?
- Can we provide dosing for some unstudied subpopulations of interest?
- Are there risks when other medications are co-administered, and if so, how can these be mitigated?
MIDD can answer these and other questions by providing information to bridge efficacy and safety for certain unstudied patient subpopulations or use scenarios. This rational approach makes the most efficient use of available information to inform next steps or, in some cases, reduce or eliminate the need for additional trials.
"In silico clinical trials use computer models and simulations to develop and evaluate devices and drugs. Modeling and simulation play a critical role in organizing diverse data sets and exploring alternate study designs. This enables safe and effective new therapeutics to advance more efficiently through the different stages of clinical trials. FDA’s efforts in modeling and simulation are enabled through multiple collaborations with external parties that provide additional expertise and infrastructure to advance the development of these state-of-the-art technologies."
- Scott Gottlieb, M.D., Commissioner of the U.S. Food and Drug Administration
Companies and regulators have used MIDD to understand target concentrations and therapeutic windows, probe drug-drug interactions, select doses, optimize dosing in difficult-to-test populations such as children, and correlate trial endpoints with clinical outcomes. The approach has allowed some companies to run shorter or smaller trials, and conduct fewer postmarketing studies.