BEGIN:VCALENDAR VERSION:2.0 PRODID:-//wp-events-plugin.com//7.2.3.1//EN TZID:America/Phoenix X-WR-TIMEZONE:America/Phoenix BEGIN:VEVENT UID:1510@azbio.org DTSTART;TZID=America/Phoenix:20200918T100000 DTEND;TZID=America/Phoenix:20200918T110000 DTSTAMP:20200820T134059Z URL:https://www.azbio.org/events/accelerating-drug-development-for-rare-di seases-through-data-sharing-and-analysis SUMMARY:Accelerating Drug Development for Rare Diseases through Data Sharin g and Analysis - - 18 Sep 20 10:00 DESCRIPTION:\nAccelerating Drug Development \; for Rare Diseases \; through Data Sharing and Analysis \nDATE: \; Friday\, September 18\, 2 020\n\nTIME: 10:00 - 11:00 AM AZ/MST/PDT\n\nThis is a free virtual event t hat is available to signup for via your AZBW app after registration.\n\nPr esented Virtually Via Webex\n\nRegister for this and other free Arizona Bi oscience Week Educational Events via the AZBW 2020 App\nRegister for AZBW 2020\nAccelerating Drug Development for Rare Diseases through Data Sharing and Analysis\n \;Despite increasing numbers of drug targets and techn ologies\, rare disease drug development is frequently slowed by the low nu mbers of patients and the lack of comprehensive quantitative characterizat ion of diseases. \nTucson's Critical Path Institute (C-Path)\, along with the National Organization of Rare Disorders and the FDA\, have developed a n integrated database and analytics hub called the Rare Disease Cures Acce lerator-Data and Analytics Platform (RDCA-DAP) to help \;accelerate cl inical development\, make it less expensive\, and encourage new companies to develop treatments for rare diseases. \;\n \;\n \;The Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP) is an int egrated database and analytics hub\, designed to help build tools to accel erate drug development across rare diseases. It is being developed by Tucs on's Critical Path Institute (C-Path) and the National Organization for Ra re Disorders (NORD) through a collaborative grant from the U.S. Food and D rug Administration (FDA). \nRDCA-DAP promotes sharing of patient-level dat a from diverse sources (clinical trials\, natural history studies\, patien t registries\, etc.) and encourages the standardization of prospective dat a collection\, as well as generating advanced data analysis solutions. Des pite increasing numbers of drug targets and technologies\, rare disease dr ug development is frequently slowed by the low numbers of patients and the lack of comprehensive quantitative characterization of diseases. This mea ns that design of clinical trials that can reliably evaluate the efficacy and safety of a potential therapy is challenging. Defining appropriate out come assessments and biomarkers and how these can best be applied is essen tial to running an informative clinical trial. Developing a clear understa nding of how each disease changes over time and what the relevant sources of variability are within the populations will allow development of clinic al trial protocols that efficiently determine if a new therapeutic is effe ctive or not. This would accelerate clinical development\, make it less ex pensive\, and encourage new companies to develop rare disease drugs. \nRDC A-DAP launched in September 2019\, and several pilots are underway to demo nstrate the utility of the platform.\n\n \;Presenters: \;\nJane La rkindale\, DPhil\,\, Executive Director Rare Disease Cures Accelerator-Dat a and Analytics Platform\; Duchenne Regulatory Science Consortium \;\n Jane Larkindale\, DPhil\, is the Executive Director for both the Duchenne Regulatory Sciences Consortium (D-RSC) and the Rare Disease Cures Accelera tor-Data and Analytics Platform (RDCA-DAP) at the Critical Path Institute in Tucson\, Arizona. Larkindale was a key leader in launching RDCA-DAP in September 2019. She launched D-RSC in 2005 and has been its leader since i nception. She is a molecular biologist by training\, having completed her D.Phil. (Ph.D.) in the department of plant sciences at Oxford University i n 2001\, which she attended on a Rhodes Scholarship. In the laboratory\, s he did research in areas as diverse as molecular biology\, biochemistry\, genomics\, plant science\, medical physics\, marine biology\, and industri al chemistry. In the course of this research\, she published numerous orig inal research papers and review articles in several disciplines. Her exper ience in drug development and neuromuscular diseases started at the Muscul ar Dystrophy Association\, an international non-profit covering over forty neuromuscular diseases\, where she ended as Vice President for Research. Dr. Larkindale was instrumental in the start-up of MDA Venture Philanthrop y (MDA’s drug development arm)\, which invested in 21 drug development p rojects\, of which 10 entered clinical trials\, and several of which have been licensed by large pharmaceutical companies. After leaving MDA\, Dr. L arkindale started a consulting company in the area of drug development for rare neuromuscular diseases and worked for the Friedreich’s Ataxia Rese arch Alliance\, developing biomarker and patient reported outcomes program s and working on a new patient registry.\n\n \;\nVanessa Boulanger\, M Sc\, National Organization for Rare Disorders\, Director of Research Progr ams\nAs Director of Research Programs at NORD\, Vanessa Boulanger\, MSc\, is responsible for leading the growth and development of NORD’s research department. Current initiatives include the IAMRARE Registry Program and NORD’s research grants program\, which has resulted in the development o f at least two FDA-approved orphan products. Previously\, Vanessa held res earch and leadership positions at the Dana-Farber Cancer Institute and the Harvard T.H. Chan School of Public Health\, where she earned her Master o f Science degree\, and a lecturer position at Regis College in the School of Health Sciences Public Health Program. She has more than 10 years of ex perience addressing health\, human rights and social inequalities from a r ange of health and development perspectives.\n\n \;\nMichelle Campbell \, PhD\, U.S. Food and Drug Administration\, \;Sr. Clinical Analyst fo r Stakeholder Engagement and Clinical Outcomes\, Division of Neurology Pro ducts\nDr. Michelle Campbell is a reviewer on the Clinical Outcome Assessm ents (COA) Staff and Scientific Coordinator of the COA Qualification Progr am in the Office of New Drugs (OND)\, Center for Drug Evaluation and Resea rch (CDER)\, U.S. Food and Drug Administration (FDA). COA Staff advises ON D review divisions and other FDA centers by providing consultation and adv ice on clinical outcome assessment development\, validation\, and interpre tation of clinical benefit endpoints in clinical trials to support drug de velopment\, labeling\, and promotion. Additionally\, the COA Staff leads a nd manages CDER’s Clinical Outcome Assessment qualification program and engages with internal and external stakeholders to advance good scientific clinical outcome measurement standards and policy development. Her prior research experience includes the use of both qualitative and quantitative methods to develop instruments\, program evaluation and the application of various study designs including clinical trials. Dr. Campbell earned her BA in Biology from the College of Notre Dame\, her MS in Health Science (c oncentration in Community Health Education) from Towson University and her PhD in Pharmaceutical Health Services Research from the University of Mar yland School of Pharmacy. END:VEVENT BEGIN:VTIMEZONE TZID:America/Phoenix X-LIC-LOCATION:America/Phoenix BEGIN:STANDARD DTSTART:20190919T100000 TZOFFSETFROM:-0700 TZOFFSETTO:-0700 TZNAME:MST END:STANDARD END:VTIMEZONE END:VCALENDAR