Changing the System to Meet the New Era of Individualized Medicine

This explored how we can build the systems needed to support individualized medicines safely, effectively, and at scale including: -Rethinking development pathways: Why models built for large patient populations don't work for individualized medicines at scale. -Global regulatory models: How different approaches around the world are reshaping the development and evaluation of individualized medicines. -Building the evidence base: How systematic learning across every individualized therapy can strengthen safety, improve evidence generation, and accelerate scalable development. Speaker Biographies: In 2016, Julia Vitarello's daughter, Mila, was diagnosed with a rare fatal genetic condition called Batten disease. In a race against time, Julia drove an unprecedented collaboration between scientists, clinicians and regulators which led to the development of her daughter's treatment, milasen—the first ever drug tailored to a single person. Since then, Julia has led an international movement to get from ‘Mila to Millions,’ turning her daughter’s groundbreaking story into an entirely new field of individualized medicines. Julia speaks regularly around the world, engaging academics, biotechs, governments, regulators, payers and families alike. Janet Woodcock recently completed a long career at FDA. She served as Director of the Center for Drug Evaluation and Research for over twenty years in several stretches. Most recently she served as Principal Deputy Commissioner and prior to that as Acting FDA Commissioner. She held multiple other senior positions at FDA including at the Center for Biologics Evaluation and Research. She was the therapeutics lead for “Operation Warp Speed” during the COVID pandemic. Her most recent effort was spearheading a major reorganization of FDA’s foods program and the Office of Regulatory Affairs. Dr. Woodcock completed many major regulatory initiatives during her FDA tenure. She was instrumental in getting the biosimilars legislation enacted and worked to ensure adoption in the clinical community. Additionally, she worked with industry and Congress to bring about the first GDUFA. After passage of the legislation, she oversaw an extensive reorganization of the generic drug review program at CDER, that successfully met the aggressive targets of the legislation and led to eventual reauthorizing, with elimination of backlogs and approval of thousands of generic drugs. Dr. Klaus Romero is a prominent clinician scientist and scholar, who serves as both the Chief Executive Officer and Chief Science Officer at Critical Path Institute. As a recognized thought-leader, Dr. Romero established C-Path’s Quantitative Medicine Program and has been an instrumental leader in the growth of the organization’s portfolio of transformative consortia and public-private-partnerships across more than 16 therapeutic development areas. As both a scientist and an executive, Dr. Romero led the generation of actionable drug development tools in Alzheimer’s disease, which introduced a transformation in the drug development process for this indication. In tuberculosis, Romero’s leadership was instrumental in generating a drug development infrastructure that allowed the approval of the first new individual drug and the first new regimen for this disease, in more than 50 years. Dr. Romero’s leadership has also resulted in the transformation of therapeutic development paradigms for many other diverse areas, like polycystic kidney, Parkinson’s and Huntington’s diseases, as well as type 1 diabetes prevention, kidney transplantation, Duchenne muscular dystrophy, and several other rare and orphan indications. As a trained clinical pharmacologist and epidemiologist, Dr. Romero is a fellow of the American College of Clinical Pharmacology, a founding member of the International Society of Pharmacometrics, as well as a member of the American Society for Clinical Pharmacology and Therapeutics, and the International Society of Pharmacoepidemiology. He is also an Associate Research Professor at the University of Arizona, as well as an Adjunct Professor at the University of Southern California and Arizona State University.