Accelerating Drug Development for Rare Diseases through Data Sharing and Analysis

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Date(s) - 18 Sep 2020 until 18 Sep 2020
10:00 AM - 11:00 AM

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Accelerating Drug Development  for Rare Diseases through Data Sharing and Analysis

DATE:  Friday, September 18, 2020

TIME: 10:00 – 11:00 AM AZ/MST/PDT

This is a free virtual event that is available to signup for via your AZBW app after registration.

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Accelerating Drug Development for Rare Diseases through Data Sharing and Analysis

 Despite increasing numbers of drug targets and technologies, rare disease drug development is frequently slowed by the low numbers of patients and the lack of comprehensive quantitative characterization of diseases.

Tucson’s Critical Path Institute (C-Path), along with the National Organization of Rare Disorders and the FDA, have developed an integrated database and analytics hub called the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) to help accelerate clinical development, make it less expensive, and encourage new companies to develop treatments for rare diseases. 

 

 The Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP) is an integrated database and analytics hub, designed to help build tools to accelerate drug development across rare diseases. It is being developed by Tucson’s Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD) through a collaborative grant from the U.S. Food and Drug Administration (FDA).

RDCA-DAP promotes sharing of patient-level data from diverse sources (clinical trials, natural history studies, patient registries, etc.) and encourages the standardization of prospective data collection, as well as generating advanced data analysis solutions. Despite increasing numbers of drug targets and technologies, rare disease drug development is frequently slowed by the low numbers of patients and the lack of comprehensive quantitative characterization of diseases. This means that design of clinical trials that can reliably evaluate the efficacy and safety of a potential therapy is challenging. Defining appropriate outcome assessments and biomarkers and how these can best be applied is essential to running an informative clinical trial. Developing a clear understanding of how each disease changes over time and what the relevant sources of variability are within the populations will allow development of clinical trial protocols that efficiently determine if a new therapeutic is effective or not. This would accelerate clinical development, make it less expensive, and encourage new companies to develop rare disease drugs.

RDCA-DAP launched in September 2019, and several pilots are underway to demonstrate the utility of the platform.

 Presenters

Jane Larkindale, DPhil,, Executive Director Rare Disease Cures Accelerator-Data and Analytics Platform; Duchenne Regulatory Science Consortium 

Jane Larkindale, DPhil, is the Executive Director for both the Duchenne Regulatory Sciences Consortium (D-RSC) and the Rare Disease Cures Accelerator-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 inception. She is a molecular biologist by training, having completed her D.Phil. (Ph.D.) in the department of plant sciences at Oxford University in 2001, which she attended on a Rhodes Scholarship. In the laboratory, she did research in areas as diverse as molecular biology, biochemistry, genomics, plant science, medical physics, marine biology, and industrial chemistry. In the course of this research, she published numerous original research papers and review articles in several disciplines. Her experience in drug development and neuromuscular diseases started at the Muscular 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 Philanthropy (MDA’s drug development arm), which invested in 21 drug development projects, of which 10 entered clinical trials, and several of which have been licensed by large pharmaceutical companies. After leaving MDA, Dr. Larkindale started a consulting company in the area of drug development for rare neuromuscular diseases and worked for the Friedreich’s Ataxia Research Alliance, developing biomarker and patient reported outcomes programs and working on a new patient registry.

 

Vanessa Boulanger, MSc, National Organization for Rare Disorders, Director of Research Programs

As 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 of at least two FDA-approved orphan products. Previously, Vanessa held research and leadership positions at the Dana-Farber Cancer Institute and the Harvard T.H. Chan School of Public Health, where she earned her Master of 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 experience addressing health, human rights and social inequalities from a range of health and development perspectives.

 

Michelle Campbell, PhD, U.S. Food and Drug Administration, Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, Division of Neurology Products

Dr. Michelle Campbell is a reviewer on the Clinical Outcome Assessments (COA) Staff and Scientific Coordinator of the COA Qualification Program in the Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). COA Staff advises OND review divisions and other FDA centers by providing consultation and advice on clinical outcome assessment development, validation, and interpretation of clinical benefit endpoints in clinical trials to support drug development, labeling, and promotion. Additionally, the COA Staff leads and 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 (concentration in Community Health Education) from Towson University and her PhD in Pharmaceutical Health Services Research from the University of Maryland School of Pharmacy.