While rare disease product approvals are held to the same evidentiary standards as common diseases, designing and conducting clinical trials in rare diseases face unique, complex and costly challenges in meeting these standards. Reducing costs and improving efficiencies is, therefore, paramount – are innovative, next-generation technologies the solution?
In this webinar, we explored:
• The use of data visualizations to interpret Big Data for biomarkers
• Applying artificial intelligence to guide decision making down the most optimal path, and collection of de-identified real-world evidence (RWE) for subsequent analysis
• Adoption of blockchain technologies to promote data integrity, transparency, collaboration amongst partners and availability of granular patient information in an anonymous fashion.
• Bluetooth-enabled wearables; remote data capture
• Social entrepreneurship in rare diseases clinical research
• Regulatory and non-technical aspects of rare disease clinical trials
This webinar is applicable to roles within sponsors, CROs and sites that:
• Are responsible for site identification, feasibility assessment, selection and activation
• Are responsible for collecting trial documentation
• Are responsible for project management of studies and operational excellence
Senior VP, Center for Rare Diseases
PRA Health Sciences
Scott is a clinical strategist with a 25-year background in clinical drug development focused on rare diseases, orphan drugs, and advanced therapeutics. Scott’s focus is on patient-centered approaches to drug development; reducing the burden of clinical trial participation; and leveraging novel and disruptive technologies to bring new therapies to patients faster. Committed to collaborative efforts to further drug development, Scott has developed active partnerships with rare disease patient-focused organizations, patient advocacy groups, drug development institutes, and academic research centers. Scott serves on leadership committees for a variety of rare disease societies and congresses, and also regularly speaks on novel strategies to accelerate rare disease drug development. Scott holds a master’s degree in Public Health (MPH) from the University of Utah School of Medicine and completed a Graduate Research Fellowship at The National Institutes of Health.
HARSHA K. RAJASIMHA, MS, PhD
Founder CEO, Jeeva Informatics Solutions Inc.
After losing a child to a rare congenital disease in 2012, Harsha was determined to apply his years of post-doctoral clinical genomics data research experience at National Institutes of Health and industry product development experience to develop products and solutions to help accelerate clinical research. His venture Jeeva is building a Uber-like model for clinical trials using digital health technologies to significantly improve efficiency while reducing patient burden and drop outs.
Harsha has authored 15 high impact journal publications, 2 book chapters, and inventor on 2 patents. He is a keynote speaker, convener of #DigitalOMICS meetup around the Capital Beltway and other part of several conference organizing committees. Harsha has received numerous Awards for his work in genomics, precision medicine and rare diseases including the Lead India 2020 Award for leadership and outstanding achievement for the cause of rare diseases, NTT DATA Healthcare Life Sciences Silver award for outstanding achievement 2017, Sanofi Genzyme patient advocacy leadership award 2016, Bioscience company of the year 2014, NEI director’s innovation award 2012 for building their genomics cyber infrastructure. Harsha earned his M.S. in Computer Science (2004) and Ph.D. in Genetics, Bioinformatics and Computational Biology (2007) from Virginia Tech.
VP of Marketing & Strategy
ClinX, parent company of ClinEdge
Joan is the Vice President of Marketing and Strategy at ClinX, a global service solutions provider for research sites, patients and healthcare organizations. Prior to joining ClinX, Joan's 20+ years in the health life sciences industry includes working in senior management roles at SCORR Marketing, CenterWatch, Cambridge Healthtech Institute, Tufts Center for the Study of Drug Development at Tufts University, and PAREXEL International. Joan holds a B.S. in Marketing.
Joan is on the Board of Directors for Greater Gift, The Center for Information and Study on Clinical Research Participation (CISCRP), the US PharmaTimes Steering Committee for CROY, Steering Committee for Pharma Intelligence/Informa Clinical & Research Excellence Awards, Steering Committee for PopUp Star and is an active member of the Association of Clinical Research Professionals (ACRP) and the Drug Information Association (DIA).
Director, Industry Strategy
Oracle Health Sciences
Elvin is a recognized industry expert in clinical trials, having over twenty years of experience working as a Clinical Business Analyst Consultant at major pharmaceutical companies including, Hoffmann-La Roche and Johnson & Johnson. Elvin works as a product strategist and system architect in goBalto's effort to optimize Study Startup. Elvin is the co-chair on the TMF Reference Model Exchange Mechanism and holds a Master of Science in Industrial Engineering from Aalborg Universitet.