Novel Endpoints and Analyses in Multisystemic Rare Disease Trials

Dr. Dunnmon is the acting Medical Team Leader in the Division of Cardiology and Nephrology as part of the Office of New Drugs (OND), Office of Drug Evaluation I (ODEI), Division of Cardiovascular and Renal Products (DCRP) within the Center for Drug Evaluation and Research (CDER), to serve as the CDER Public Private Partnership (PPP) Liaison to ARC.

Spencer Guthrie brings significant expertise with over 23 years in drug development and commercialization experience across large and small biotech. He has dedicated more than 12 years of his career to amyloid-related diseases, including AL and ATTR amyloidosis, Alzheimer’s and Parkinson’s disease, and has authored over 30 scientific publications in amyloidosis.

Dr. Finkelstein is the director of Biostatistics at the Massachusetts General Hospital Cancer Center. Dr. Finkelstein’s research is focused on the development of semi-parametric methodology for analyzing the effect of covariates on failure time distributions that arise in Cancer and Acquired Immune Deficiency Syndrome (AIDS) epidemiological studies and clinical trials. Dr. Finkelstein’s research has also focused on the development of methods for analyzing longitudinal and survival outcomes, and on animal carcinogenicity experiments. She has also developed along with David Schoenfeld a method of combining endpoints in clinical trials which is favored by the Food and Drug Administration.

Dr. Kakkis leads a team developing and commercializing multiple rare and ultra-rare disease treatments. Over the last 25 years, Dr. Kakkis is best known for his work developing novel treatments for rare diseases and for advocating on policy issues by founding and supporting the EveryLife Foundation for Rare Diseases. Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing more therapeutics for rare and ultra-rare diseases. Since its founding, Ultragenyx has worked on developing treatments for twenty different genetic diseases and has now received four approvals.

Dr. Maurer directs the Clinical Cardiovascular Research Laboratory for the Elderly (CCRLE) at the Allen Hospital of NewYork-Presbyterian Hospital. Dr. Maurer is a member of the Advanced Cardiac Care Center at NewYork-Presbyterian/Columbia. He has extensive expertise in amyloidosis and has served as a principle investigator in clinical trials for hereditary

Dr. Prasad is a senior medical assessor at MHRA with management responsibility for Cardiovascular- diabetes, anti-infective agents, and oncology therapy areas. His additional roles include Cardiology consultancy at St. Thomas’ hospital, London. He has worked for MHRA, the UK regulatory agency since 2002. Dr Prasad’s areas of special interests include heart failure, sudden death, cardiomyopathies and arrhythmias. He is a member of the Cardiovascular working party since 2008, assuming responsibility for coordinating European guidance on heart failure, lipid modifying agents as well as pediatric guidance in these areas.

Dr. Rothmann earned his Ph. D. in Statistics at the University of Iowa in 1990. He then spent the next nine years as a professor at various universities before coming to the FDA in 1999. At the FDA, he has since been involved in the review on Oncology, Hematology, and Metabolism and Endocrinology products and has been a Deputy Division Director or Acting Director since 2016. He has done research in several areas involving the design and analysis of clinical trials.

Dr. Tandon provides strategic direction and leadership in the clinical development of products and in the design, analysis, and reporting of late stage clinical trials.

He trained in hematology in the UK and Canada. His academic and clinical interests are focused on amyloidosis and multiple myeloma. In particular, he is interested in characterization and treatment of systemic AL amyloidosis as well as multiple myeloma, including clinical trials of new and novel agents in these disorders. He has published widely in these areas.

Jialu Zhang is a mathematical statistician in FDA with over 10 years of experience in reviewing clinical studies. She has worked in multiple divisions including cardiology/nephrology, psychiatry, and neurology. She is currently the statistical team leader supporting the Division of Cardiology and Nephrology in CDER.