FDA Lecture: Patient Experience in Real World Settings: The CBER Science of Patient Input Program at the FDA
Hussein Ezzeldin, PhD - Senior Staff Fellow, Office of Biostatistics and Epidemiology/CBER/FDA
Martin Ho, MS - Associate Director of Science for Patient Input and Real-World Patient Evidence, Office of Biostatistics and Epidemiology/CBER/FDA
Ting-Hsuan Lee, MHS - Research Fellow, Office of Biostatistics and Epidemiology/CBER/FDA
Xinyi Ng, PhD - Visiting Scientist, Office of Biostatistics and Epidemiology/CBER
Sarah Stothers, RN, MSN, MPH - Research Fellow, Office of Biostatistics and Epidemiology/CBER/FDA
Novel therapeutics such as gene therapies often have distinct benefit-risk profiles from those of traditional treatments. To fulfill its mission of protecting and promoting public health, the U.S. FDA Center for Biologics Evaluation and Research (CBER) not only adapts innovative ways to measure the performance of these breakthrough technologies, but also incorporates patient perspective into regulatory decision-making. Collecting patient experience data in real-world setting such as patient preference information (PPI) is a useful quantitative strategy to understand how patients make benefit-risk tradeoffs. The Science of Patient Input (SPI) team in CBER is conducting three patient preference studies in the disease areas of sickle cell disease (SCD), osteoarthritis of the knee (KOA), and hard-to-control type 1 diabetes mellitus.
To further inform therapeutic regulatory processes, the CBER SPI team, in collaboration with the National Organization for Rare Disorders (NORD), is piloting a natural history study in the rare disease Metachromatic leukodystrophy (MLD) population. Frequently rare disease patients choose not to enroll in clinical trials due to the possibility of being randomized to the control arm rather than the treatment arm. The purpose of this study is to capture MLD patient data for future use in augmenting control groups in clinical trials of potential lifesaving therapies. In tandem, the SPI team has partnered with IBM to develop a mobile app that will allow MLD patients to share their disease experience without needing to travel to a clinical site. This mobile app, named Survey of Health And Patient Experience (SHAPE), serves to eliminate missing or incomplete data needed for clinical research in the development of therapeutic products. This research effort highlights another way real world data can be used to inform regulatory decision-making.
Hussein Ezzeldin, PhD has been a member of the Analytics Benefit-Risk Assessment (ABRA) team since 2016. He received his PhD in Mechanical Engineering from the University of Maryland, College Park in 2012, and completed a postdoctoral fellowship at George Washington University in 2013. He then joined FDA as an Oak Ridge Institute for Science and Education Research Fellow from 2013-2016. Dr. Ezzeldin, currently co-leads BEST (Biologics Effectiveness and Safety) C2, which focuses on Development of New and Innovative Methods for Automated Reporting for CBER-Regulated Biological Products. Dr. Ezzeldin has worked on multiple risk assessments policy and research projects. Also, Dr. Ezzeldin works with OBE Leadership to develop training workshops and seminars that aim to increase the transparency of the modeling done in OBE and raise the awareness about the importance of conducting reproducible research projects. In addition, Dr. Ezzeldin is part of the science of patient input (SPI) working group, which aims to advance the Patient experience as part of the FDA regulatory-science strategic goals by developing the needed expertise in the SPI field.
Martin Ho, MS is the Associate Director of Science for Patient Input and Real-World Patient Evidence in Office of Biostatistics and Epidemiology at CBER, FDA. He practices the principle of “make one, do one, teach one” when it comes to innovations. At CBER, Martin develops new tools to inform regulatory decisions, applies these tools to submissions, builds review capacity by using the tools. He leads multiple pilot studies of using new tools such as data sources beyond traditional RCTs to inform regulatory decisions, including clinical outcome assessments (COAs), patient preference information (PPI), real-world evidence (RWE), digital health technologies (DHTs), and site-less clinical trials. He gains real-life experience by serving as methodological lead embedded in CBER and CDER review teams to evaluate first-of-a-kind applications. Martin shares what he learned to build review capacities by provide trainings to evaluate quantitative patient inputs such as PRO and PPI. Martin represents CBER in cross-Center Working Groups include Patient-Focused Drug Development (PFDD), COA, DHTs, and Site-less Clinical Trials Working Group to work with other Centers to develop guidance documents and organize FDA public workshops with other FDA Centers. He was elected the President of the FDA Statistical Association in 2017 and the Chair of the American Statistical Association Medical Devices and Diagnostic Section in 2020. Martin has strived to advance quantitative methodologies in medical product developments in pre-competitive space, where industry, academia and regulators collaborate and innovate. In 2017, he co-found the American Statistical Association Biopharmaceutical Section RWE Working Group, conducting statistical research on RWE with a group of 30 SMEs. Recently, he co-found ISPOR Emerging Good practices of Quantitative Benefit-Risk Assessments Task Force in October, 2020. Prior to CBER, Martin worked at CDRH for 10 years and was the founding Director of the Quantitative Innovation Program at CDRH, responsible for evaluating novel sources of data to inform regulatory decisions, elevating the appropriate ones to evidence grade through rigorous and early discussions with the sponsors at the study design stage, and interpreting the study analyses and conclusions from a regulatory perspective.
Ting-Hsuan “Joyce” Lee, MHS is a research fellow working on patient engagement projects (patient preferences) and a pilot natural history study for a rare disease. Before joining the Science of Patient Input (SPI) group in CBER, she had been with CDRH for 3 years working on patient preferences, patient-reported outcomes, real-world evidence on the topics of abdominal hernias, prostate cancer, T1D devices, and healthcare-associated infections (HAIs). She has a master’s degree in health economics and international health from Johns Hopkins Bloomberg School of Public Health.
Xinyi Ng, PhD is a health economics and outcomes research scientist at Center for Biologics Evaluation and Research, U.S. FDA. She has expertise in the elicitation of patients’ and physicians’ preferences for medical products and using stated preference data to inform benefit-risk assessments of health interventions. She has participated in several studies that elicited preferences using discrete choice experiment and best-worst scaling across therapeutic areas including oncology, mental health, rheumatology, and immunology. In addition, Dr. Ng has experience with using real-world data such as SEER-Medicare, MarketScan and CancerLinQ to address research questions.
Sarah Stothers, RN, MPH, MSN is a research fellow working to identify innovative strategies for the inclusion of patient preferences and experiences into the regulatory decision-making framework. Prior to CBER, she worked on public and patient engagement initiatives with the Patient-Centered Outcomes Research Institute (PCORI) to advance patient-centered research. For four years, Sarah served as the National Nurse Health Educator of the Lupus Foundation of America (www.lupus.org ), where she also led the development effort of the organization’s first patient registry. Sarah completed her nurse residency training in Geriatric Medical/Surgical acute care at MedStar Washington Hospital Center in Washington, DC. She has intimate knowledge of the healthcare system and has experience working in preventative health and infectious diseases. Sarah completed her Master of Public Health and Master of Science in Nursing at Johns Hopkins University.