Strategic Deployment of Technology Drives Rare Disease Research



ClinX, in Partnership with Oracle and goBalto: 

Strategic Deployment of Technology Drives Rare Disease Research

This webinar was recorded on Tuesday, March 12, 2019. 


Roughly 10% of the U.S. population suffers from a rare disease, with an estimated 350 million suffers worldwide. Since the passage of the Orphan Drug Act, the number of orphan indications approved in the United States has risen dramatically with 40%+ of NMEs in 2017 being orphan drugs.

Designing and conducting clinical trials in rare diseases comes with many challenges, as some diseases are not well-characterized, patients are scarce and geographically dispersed, and standards of care vary significantly across geographies. Technology that is available to us today can help us engage rare diseases patients better, ensure clinical trials fit better into patient's daily lives, and accelerate the development of new orphan drugs.

In this webinar, we explore:

  • The impact of patient registries
  • Use of real-world evidence (RWE) for better benchmarking, site selection, and trial design
  • Use of mobile technology and connected devices to engage, enroll, and retain patients in clinical trials
  • The impact social media is having in rare disease awareness
  • Social entrepreneurship in rare disease 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 study design and operationalizing clinical protocols
  • Are involved in site identification, feasibility assessment, selection and activation
  • Are responsible for collecting trial documentation
  • Are responsible for project management of studies and operational excellence


Webinar- Strategic Deployment of Technology Drives Rare Disease Research



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.


Founder CEO, Jeeva Digital Health

Co-Founder Board Director, Organization for Rare Diseases India, USA

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.

ClinEdge Staff

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