According to the U.S. Food & Drug Administration (FDA), “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.” In 2018, the FDA created a pilot program to explore MIDD further.
The MIDD pilot program is described by the FDA as follows:
…to facilitate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources, referred to as MIDD approaches. MIDD approaches use a variety of quantitative methods to help balance the risks and benefits of drug products in development. When successfully applied, MIDD approaches can improve clinical trial efficiency, increase the probability of regulatory success, and optimize drug dosing/therapeutic individualization in the absence of dedicated trials.
As model-informed drug development gains in importance to the pharmaceutical industry, we sit down with Pumas-AI Co-Founder and CEO Dr. Joga Gobburu to answer questions about MIDD, the future of pharma, and the field of pharmacometrics.
What steps is the FDA taking to explore model-informed drug development?
Answer: There is no “explore” anymore in this sense. These MIDD meetings with the FDA are here to stay.
What is your background in regards to model-informed drug development?
Answer: At a higher level, MIDD meetings with the FDA are a metamorphosis of a previous path called EOP2A meetings, which occurred between the FDA and sponsors for the purpose of more efficient development through clinical trial simulation and modeling.
During my tenure at the FDA, I was one of the authors of the EOP2A Guidance for Industry. Now that this has evolved, my team of scientists at Pumas-AI and I have been involved with several sponsors in successfully preparing and assisting them for the MIDD meeting process.
With the FDA only granting 2-4 meetings requests per quarter as part of the MIDD pilot program, what must sponsors expect when trying to participate?
Answer: Our scientists have first-hand experience working with sponsors and the FDA for direct support in preparation for and during these MIDD meetings, and we see how important the proper planning is. One critical aspect is to perform adequate modeling and simulations tailored to support the exact development questions that the Sponsor is seeking FDA responses to.
Since you have been so active in these MIDD meetings, can you share any expectations that Sponsors should have before attending?
Answer: MIDD meetings are perhaps the best vehicle in the pharmaceutical industry for discussing precedent-setting matters driven by modeling and simulation. All required disciplines from the FDA are in attendance for the meeting. In fact, due to the complex nature of model-informed drug development, the Sponsor receives two meetings to ensure that any matters from the first meeting are addressed.
Where do you see model-informed drug development in the future of pharma?
Answer: Treatment modalities have been changing in the pharmaceutical industry throughout recent years. More and more, novel treatments are emerging. These novel treatments require innovative drug development approaches, as conventional approaches apply less and less. For such topics, Sponsors have the unique opportunity to leverage model-informed drug development paths to make more appropriate decisions.
What advice do you have for a sponsor looking to explore model-informed drug development?
Answer: To be successful, Sponsors should first identify the key questions needed for discussion. In our experience, this is the most challenging step. To discover this more quickly, we developed a unique Decision Information Analysis (D-I-A) approach. This is a systematic approach to discovering the core topics for discussion, allowing the Sponsor to make the best use of this unique opportunity.
Is there anything else that you would like to share about model-informed drug development?
Answer: MIDD meetings are a reflection of the success that the field of pharmacometrics has seen over the years, collectively with regulatory bodies, the pharmaceutical industry, and academia together. The dream of clinical pharmacologists and pharmacometricians taking the helm of drug development strategy is being realized effectively through such opportunities as MIDD meetings. It puts our field on the power-map, and I am proud to be playing a key role in the growth of model-informed drug development.
Details on the FDA’s MIDD pilot program can be found here: https://www.fda.gov/drugs/development-resources/model-informed-drug-development-pilot-program