PPT-FDA Regulatory Requirements for Successful Biomarker Applications

Early Detection Research Network September 9 2019 Anand Pathak MD PhD MPH Division of Molecular Genetics and Pathology Molecular Genetics Branch OHT7OIRCDRHFDA No financial relationship to disclose. Bruce Ross. , . M.A. M.P.H.. Director, India Office. Agenda. Priorities. Challenges of globalization. cGMP. . deficiencies. Comparison. Post inspection regulatory . actions. 2. Advance Regulatory Science: the science of developing new tools, standards and approaches to assess the safety and effectiveness, quality and performance of FDA-regulated products. Director. Policy and Regulations Staff. Center for Veterinary Medicine. Foundations of Regulation. The Federal Government is composed of three distinct branches: legislative, executive, and judicial, which powers are vested by the U.S. Constitution. MBBS (Hons), . MMedSci. (. Clin. . Epid. ), . MBiostat. , PhD, FRACP. Biomarker-Based Clinical Trials: Practical and Design Considerations. Biomarker. “Any characteristic that can be objectively measured as an indicator of normal or pathological biological processes or the response to a therapy”. Worldwide Trends . 2014. What is a Biomarker?. Worldwide Trends. 2014. What is a Biomarker?. A biological marker, better known as a “biomarker”, is a characteristic . that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes or pharmacological responses to a therapeutic . Joan Wilmarth Blair. Sr. Advisor, International Affairs. Center for Biologics Evaluation and Research, FDA. Goal of Presentation. Provide an appreciation of the complex history of the Identification of Medical Product (IDMP) standards while noting the ongoing engagement of FDA, EMA and several EU MS’s. Not Quite Ready . for Prime Time. Scott Kopetz, MD, PhD. . Department of GI Medical Oncology. MD Anderson Cancer Center. NOT YET. YES. Individual Biomarkers versus . Molecular Subtypes. Individual biomarkers:. David W. Bates MD, . MSc. , Chair. 1. Committee Membership. David W. Bates, Chair. , Brigham and Women’s Hospital. Patricia Brennan. , University of Wisconsin-Madison. Geoff Clapp. , Better. Todd Cooper. (i.e. no one has seen or approved this). Draft 07/01/13. Coordination. This is the point at which we need to truly start integrating the work products of the subgroups. .. We need to look at the safety issues and assess whether they are addressed. Devices Applied to HIT. Bradley Merrill Thompson. Epstein Becker & . Green. June . 7, . 2013. 1. Roadmap. Exercise Overview . Health IT Use Case – Mechanical Ventilation Weaning. FDA Device Regulatory Framework. Investigational New Drug Application (IND); Summary of regulations and guidelines . Introduction of . cGMP's. /principles of validation . Introduction to QA/QC principles . Good Laboratory Practice (GLP) compliance . FDA Modeling and Simulation Working Group. Sponsored by the Office of the Chief . Scientist Fall 2016. Main . Objectives. :. Raise awareness of the successes, challenges and opportunities for modeling and simulation to advance regulatory science at the FDA;. a measurable indicator . that . can . tell . us something about a person’s health or disease . state, for example, . disease (pathological) processes in the body, for example, disease stage. , . biological processes in the body (heart rate, blood pressure, temperature), . Limitations of . Circulating miRNA. Performed by the . Data Management . a. nd Resource Repository (DMRR). ERCC . D. ata Analysis. 1. Williams Z., et al. . (. 2013. ) Comprehensive profiling of circulating microRNA via small RNA sequencing of cDNA libraries reveals biomarker potential and limitations. John Scott, Ph.D.. Director, Division of Biostatistics. Office of Biostatistics and Epidemiology. Center for Biologics Evaluation and Research. FDA. Disclaimer. This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.