RARE Drug Development Symposium: Disruptive Innovations in Clinical Trials Workshop

June 3, 2022

The 2022 RARE Drug Development Symposium kicks off June 8, and Global Genes is ecstatic about the speaker lineup for all of the sessions this year. Going with this year’s theme of disrupting innovation, Global Genes is going for a different approach with sessions this year. Formats will be more laid back and inclusive, with fireside chats to start the day, and workshop-style sessions with experts and peers throughout the day that will be offered at multiple times so you don’t miss any content. 

Disruptive Innovations in Clinical Trials Workshop

Clinical trials for orphan diseases are often smaller than those of non-orphans due to low disease prevalence and patient heterogeneity. In this session, we’ll discuss the potential for using efficient trial design, data aggregation and sharing, to create inclusive data sets and reduce clinical trial delays and terminations. We’ll also touch on uses of real world data (RWD) to replace or boost clinical placebo arms, impact recruitment, or enhance impact by linking to surveys. Different platforms that manage, handle and connect data will be highlighted and advantages and limitations of specific features will be discussed.


Craig Lipset
Co-Chair, Decentralized Trials & Research Alliance
Adjunct Assistant Professor, Health Informatics, Rutgers University
Founder, Clinical Information Partners

Craig is an advisor to technology and biopharmaceutical companies, leading universities, and the venture community, bringing vision and driving action at the intersection of research, digital solutions, and patient engagement. He is the founder of Clinical Innovation Partners, where he provides growth advisory services for organizations developing and implementing innovative approaches for clinical research and medicine development.  

 Among the many roles he plays, he is co-chair for the Decentralized Trials & Research Alliance (DTRA). DTRA enables collaboration to accelerate the adoption of patient-focused, decentralized clinical trials and research within life sciences and healthcare. Craig also serves on the Board of Directors for the Foundation for Sarcoidosis Research and the MedStar Health Research Institute, as well as on the Editorial Board for Therapeutic Innovation & Regulatory Science. Craig is Adjunct Assistant Professor in Health Informatics at Rutgers University, and Adjunct Instructor in the Center for Health + Technology at University of Rochester.

Previously, Craig was the Head of Clinical Innovation and Venture Partner at Pfizer, on the founding Operations Committee for TransCelerate Biopharma, and on the founding management teams for two successful startup ventures (Perceptive Informatics and Adnexus Therapeutics). During that time, Craig designed and launched multiple industry firsts — from the first fully remote/virtual clinical trial for a new medicine to the first returning of results and data to research participants.

Learn More:
Decentralized Clinical Trials: Collaboration is Needed to Expand their Adoption and Impact
The New Normal in Clinical Trials: Decentralized Studies

Caitlin Nichols, PhD
Research Director, AllStripes Research 

In her role as Research Director at AllStripes, Dr. Nichols oversees scientific communications and the design and execution of real-world data research partnerships with industry, academic, government, and patient advocacy group stakeholders. She received a PhD in Biological and Biomedical Sciences from Harvard University. Dr. Nichols was formerly a scientific curator on the Product Science team at 23andMe, where she helped develop and improve consumer genetic test reports. 

Learn More: 
Patient Engagement and Real-World Data Drive Innovation in Orphan Disease Drug Development
The Importance of a Natural History Study, a webinar with CLOVES Syndrome Community, LGDAlliance, and Denise Adams (starts at 17:32)
The JUMP (Journey to Understand MMA and PA) Study: A Natural History Study, 43rd Annual Society for Inherited Metabolic Disorders (SIMD), April 2022
ONCE UPON A GENE – Episode 076 – AllStripes: Jump Start New Research for Your Rare Disease with Caitlin Nichols

Jennifer Tjernagel, MS
Senior Project Manager, Simons Foundation

Jennifer Tjernagel is the senior project manager for Simons Searchlight, an international research program funded by the Simons Foundation, with the goal of accelerating science and improving lives for people with rare genetic neurodevelopmental disorders.

This innovative data collection platform, designed to collect standardized data and biospecimens across over 170 genetic conditions associated with neurodevelopmental disorders, bridges the needs of researchers, families and industries.  Researchers can request access to phenotypic, genetic, or imaging data; order biospecimens and/or submit a research match application to recruit individuals or families for future research studies. Caregivers and patients learn how they compare to others in their genetic community through summary reports and presentations. They can also connect with others with the same diagnosis through various Facebook communities and by attending conferences.

Simons Searchlight collects family, medical, developmental and behavioral information through online surveys and phone interviews with families and individuals. Biospecimen collection is performed either remotely or at in-person family meetings. More than 6000 families have registered with Simons Searchlight to date. 

Jennifer Tjernagel’s personal connection to the world of rare disease began when her teenage son was diagnosed with Vascular Ehlers Danlos Syndrome (VEDS) in early 2017. She has served on both the Board of Directors and Medical and Scientific Board of the Ehlers Danlos Society since 2018.  Jennifer is also a member of the VEDS Collaborative, powered by the VEDS Movement as part of the Marfan Foundation.  Before her work with the Simons Foundation, Tjernagel spent ten years in the pharmaceutical industry as an associate director of statistical analysis and reporting at Pfizer.

Learn More: 
Simons Searchlight on YouTube – researchers share how they have used Simons searchlight data and collaborations to advance neurodevelopmental and rare disease research
Simons Searchlight FAQs
Simons Foundation Autism Research Initiative (SFARI) programs
SFARI resources for the research community learn more about funding opportunities, available resources (data, biospecimens, animal models), how to access resources and how to recontact cohort participants. 
SFARI funding opportunities 

Vanessa Vogel-Farley, MS
Research and Data Governance Lead, RARE-X

Vanessa Vogel-Farley serves as the Data Collection Platform Lead for RARE-X. She currently serves as the Chair of the Rare Epilepsy Network and is formerly the Executive Director of the Dup15q Alliance.  

Her work is informed by over 18 years of experience in data collection methods, including a position at the University of Minnesota, Center for Neurobehavioral Development and as the Clinical Research Coordinator for the Division of Developmental Medicine Laboratory of Cognitive Neuroscience, Boston Children’s Hospital, working on collaborations with scientists from MIT and Harvard examining several clinical populations, including autism.

She also brings expertise in non-profit and research operations, patient advocacy and support, non-profit management, and broad knowledge of child development neuroscience/psychology research and administration.

Learn More: 
A Patient-Centric Approach to Consent (White Paper)
Insights and Recommendations for Organizational DEI Strategies (Report and Study)
Enabling the Future of Cell & Gene Therapies through Non-Proprietary Patient-Owned Data Collection
Patient-Owned Health Data Registries – India Feasibility Study Report


See other workshops and speaker lineups:
AI-Driven Screening Platforms and New Approaches to Therapeutics
Emerging Models & Partnerships
Fostering Successful Connections Between Stakeholders to Accelerate Progress

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