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FDA: Modeling, simulation offer opportunity to advance public health
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Modeling and simulation are valuable research and product development tools that are helping to advance regulatory science, according to the FDA.
The FDA is using modeling and simulation to help achieve its objectives of improving health care, broadening access and advancing public health goals, Tina Morrison, PhD, chair of FDA’s agency-wide modeling and simulation working group and regulatory advisor of computational modeling for FDA’s Office of Device Evaluation, said at an FDA Grand Rounds meeting.
‘Investment to the future’
Modeling and simulation can help predict clinical outcomes and product safety, inform clinical trial designs and support evidence of effectiveness, according to the FDA.
“Ideally, we’d like to be able to promote consistent review and decision-making with modeling and simulation,” Morrison said.
“In the area of drug product quality, it is possible that in silico, or computerized, clinical trials can be performed to identify clinically relevant specifications, key components of drug product quality and how clinical performance may be affected,” she added.
The FDA recognizes that there is an aspect of uncertainty that comes with modeling and simulation, she said. Therefore, it is important for scientists to demonstrate credibility of models and determine the appropriate level of information needed to support modeling in making a regulatory decision, she said.
Modeling and simulations are an investment to the future, according to Morrison.
“There is an important opportunity here for simulation to really support personalized medicine, clinical decision support, software, medical device and digital health,” Morrison said.
Strengths, limitations
“The current cost of new drug discovery, development and approval is unsustainable,” Robert A. Beckman, MD, professor of oncology and biostatistics, bioinformatics and biomathematics at the Lombardi Comprehensive Cancer Center and Innovation Center for Biomedical Informatics at Georgetown University Medical Center, told Healio Internal Medicine.
“This is in part due to the stratification of patients into smaller and smaller subgroups by molecular and other biomarkers, and the very large number of experimental therapies currently available for testing,” he added. “This, in turn, creates a combinatorial explosion of possible clinical trials involving small patient groups that are difficult to enroll in the numbers required to demonstrate efficacy according to conventional clinical trial designs.”
The cost-effectiveness of drug development may dramatically increase with the use of complex innovative clinical trial designs, Beckman said. Increased cost-effectiveness may lead to more life-altering medicines for people with serious illnesses, but at a lower cost, he said.
“Simulations are essential for two purposes: optimizing these complex designs for best performance and judging the performance of the final innovative design,” he said. “To the latter point, health authorities need to be able to quantify the risk of a false positive that might lead to marketing approval of an ineffective agent, whereas pharmaceutical companies want to understand the risk of a false negative that limits their opportunities.”
Researchers must quantify the risks of the designs, their cost-effectiveness in delivering answers with fewer patients and their ability to accurately and precisely estimate drug effects in order to benefit public health, he said.
“Simulations can deliver these answers, but the choice of scenarios to simulate must be a fair and complete representation of likely scenarios to avoid misleading conclusions,” Beckman said. “Simulation methodology and reporting must be transparent to allow critical evaluation by health care decision makers.” – by Alaina Tedesco
Disclosure: Beckman is a consultant for Astra Zeneca and EMD Serono, a stockholder in Johnson & Johnson and the Chief Scientific Officer of Onco-mind LLC. Morrison is an employee of the FDA.