Rare Disease Clinical Trials: Site, Advocate and Technology Insights

Life Sciences, Clinical Trials, Patient Recruitment & Retention,
  • Tuesday, September 20, 2022

Rare disease clinical trials are challenging on many levels, from identifying eligible patients, to making it convenient for caregivers, to the outsized expense (over $300k per patient) compared to other therapeutic areas. Not to mention that, in many cases, families are looking to clinical trials as their last, best, or only hope for treatment or a cure — when in reality, clinical trials are more likely to benefit families down the line.

Join this webinar to hear key perspectives from a distinguished panel of experts in rare disease, rare disease clinical trials and diversity:

  • Featured speaker Daniel DeFabio will share the realities of rare disease families — the expectations and hope, frustrations and challenges and importance of maintaining a positive outlook — of participating in rare disease trials
  • Featured speaker Dr. Fabian Sandoval will focus on driving diversity in clinical trials — something that is already a patient recruitment challenge without the intersection of rare disease. But diverse patient populations get rare diseases too — so what can be done to serve this community
  • Featured speaker Andrea Valente will share trends and solutions that consider the participant experience just as important as the data captured during a trial, and how study sponsors and sites can get the most out of them in 2022 and beyond


Daniel Defabio, DISORDER

Daniel DeFabio, Co-Founder, The Disorder Channel

Daniel DeFabio didn’t aspire to work in the rare disease world. He was a successful video producer and director, with credits for American Cinematographer, PBS and TNT’s “The Closer”, among others.

But when his son Lucas was diagnosed with Menkes disease, he knew he could use his talents for more. Being in the larger rare disease community helped Daniel realize that they had hope in the form of clinical trials, whereas many rare diseases have no hope for treatment or a cure.

Daniel produced a 12-minute documentary about Menkes disease, a film that was well-received, and shared at many outlets including, DISORDER: The Rare Disease Film Festival.

After his son’s passing in June 2020, Daniel has continued to remain in the rare disease community, now as the Director of Community Engagement for Global Genes. As for his experience as a parent of a child with a rare disease, Daniel feels that he still has a lot that he can contribute to the community. Being an advocate for rare diseases provided Daniel with an opportunity to turn his experiences into a positive outcome.

Message Presenter
Fabian Sandoval, Emerson Clinical Research Institute

Fabian Sandoval, MD, President & CEO, Emerson Clinical Research Institute

Dr. Fabian Sandoval has over 25 years of bench to bedside research experience. His diversified research career has been in academia, healthcare systems and the public sector. After opening the Emerson Clinical Research Institute (ECRI), he started the Emerson Community Clinic in order to support uninsured patients across the DC metro area, as well as the Emerson Diversity Health Foundation, whose mission is to educate patients and providers in the importance of participating in clinical research and access to care.

In addition, Dr. Sandoval holds the following esteemed positions in the following community areas:

  • Emmy Winner; Host of a weekly medical TV show “Tu Salud Tu Familia” (Your Family Your Health”)
  • Medical Contributor for Telemundo WZDC Washington DC News and national spots
  • Leadership Council member, Society for Clinical Research Sites (SCRS)
  • Renowned invited speaker/presenter to multiple pharmaceutical and clinical trial organizations, on the topic of increasing minorities’ engagement, diversity education and participation, in clinical trials
Message Presenter
Andrea Valente, ClinOne

Andrea Valente, CEO, ClinOne

Andrea Valente is a transformational leader and a champion of adaptive experiences for participants in clinical trials. With more than 20 years of consistent over-achievement and demonstrated success in life sciences technology product development, she has proven expertise in delivery operations, customer loyalty and M&A strategy.

Andrea joined ClinOne in March of this year from Medable where she served as Chief Operating Officer. Prior to Medable, she was Vice President of Pandemic Response Systems at Oracle, where she led business development, product development and service delivery of the cloud-based Oracle Health Management System (OHMS). She also led the partnership with the Tony Blair Institute for Global Change to deploy OHMS in Africa. And before Oracle, she served as Executive Vice President, Chief Development Officer, and member of the Senior Leadership Team with a distinguished career at ERT.

Andrea has immediately made an impact at ClinOne, putting her strategic vision into place by focusing our technology roadmap to create adaptive experiences for all trial participants, building strategic partnerships to provide more value with a unified user experience, and most importantly bringing in a team of proven leaders to grow the company responsibly with an absolute commitment to customer success.

Message Presenter

Who Should Attend?

  • Clinical Operations
  • Rare Disease Programs
  • Diversity and Inclusion
  • Patient Recruitment and Retention
  • Decentralized Clinical Trials (DCT)
  • Caregivers and Advocacy
  • Clinical Research Technology
  • Innovation and Technology
  • Site Selection and Engagement
  • Clinical Trials as a Care Option

What You Will Learn

Attendees will gain insights into:

  • The realities, considerations and hope of clinical trials for rare disease families and caregivers
  • Challenges sites face finding and enrolling patients, especially when trying to ensure diversity
  • Best practices and lessons learned for running (and participating in) rare disease studies
  • What Adaptive Experiences are, and how they improve clinical trials for all participants
  • Existing and new technologies that can make an impact on enrollment, retention and compliance

Xtalks Partner


This webinar is hosted by ClinOne, whose technology creates Adaptive Experiences for all clinical participants. By offering choice to patients, caregivers, and site staff, our technology makes it easier to stay active, compliant, and comfortable in a trial – thereby expanding enrollment access, reducing drop-out rate, and shortening conduct timelines. Our true single platform features solutions for local HCP trial awareness, remote and on-site eConsent, Uber Health, medication adherence, and a DCT portal. ClinOne stands alone with the industry’s fastest implementation (average 2-4 weeks), cost-efficient scale, and seamless API integrations for complex therapeutic areas such as Oncology, CNS, and Rare Disease in over 60 languages. To learn why more than 55 sponsors and CROs trust ClinOne in 100+ currently active studies to connect, inform, and empower participants to take control of their clinical trial experience, begin a conversation with our experts at www.clinone.com.

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