How Agile Clinical Trials Unlock Universal Access to Rare Disease Research

Life Sciences, Clinical Trials, Patient Recruitment & Retention,
  • Tuesday, October 04, 2022

Arguably, the greatest challenge in rare disease clinical research is finding enough diagnosed patients who can travel frequently to trial sites. The greatest benefit, however, of a decentralized clinical trial (DCT) is the ability to break down geographical barriers, enabling universal access to all patients and providers. Does this mean, therefore, that DCTs and rare diseases are a match made in heaven?

The agile clinical trial of the future, comprising a bespoke mix of traditional and decentralized components, has the potential to transform and accelerate the development of treatments for rare diseases. Advanced enrollment techniques bring clinical trials to the diagnosed patients who need them most, while also increasing the diversity of study populations. Meanwhile, the replacement of numerous clinic-based activities with home visits and remote digital tools greatly reduces the burden on participants and creates a highly patient-focused environment.

Science 37’s research shows that rare diseases will be among the most prevalent categories for incorporating decentralized approaches into future clinical trial designs.

This webinar will explore some of the key issues, opportunities and best practices around the following topics:

  • Why the agile clinical trial model is potentially a great fit for many rare disease categories
  • How agile clinical trials are intrinsically patient-focused, and how to maximize engagement
  • What successful enrollment looks like for an agile clinical trial in a rare disease category
  • Navigating the regulatory landscape for agile clinical trials in rare diseases
  • Designing studies that are safe, appropriate and that generate reliable data
  • Case studies and appropriate use cases for agile clinical trials in rare disease categories

Join this webinar to learn about accelerating rare disease research with agile clinical trials.


Debra Weinstein, MD, CMI, Vice President, Internal Medicine, Science 37

Dr. Weinstein graduated with honors from the University of Pennsylvania in 1983 and received her medical degree from the New York University School of Medicine in 1987. In 1990, she became a diplomat of the American Board of Internal Medicine.Dr. Weinstein had a private practice in internal medicine in Palm Beach County for 16 years — Town & Country magazine named her a top internist in 2000 — before spending 15 years as CMO of her clinical research company.

She is an active member of the Academy of Clinical Research Professionals, The American Medical Women’s Association and The American College of Physicians. The National Association of Professional Women named Dr Weinstein “Woman of the Year” for her contribution to medical research.

Message Presenter
Suzanne Pendl, Science 37

Suzanne Pendl, PhD, Senior Director, Medical Affairs, Science 37

Suzanne Pendl provides medical, scientific and technical insight and consultation in support of unlocking universal access to clinical research.

Suzanne has an interdisciplinary background in biomedical science, neuroscience and biophysics and received her PhD in biomedical science from the Department of Biophysics at the Medical College of Wisconsin.

Prior to joining Science 37, Suzanne performed original research at Cedars-Sinai Medical Center’s Biomedical Imaging Research Institute, where she used functional MRI to probe the development of infant brain networks and the dynamics of adult neurological disease.

Message Presenter
James Chase, Science 37

James Chase (Moderator), Senior Director, Thought Leadership, Science 37

James is responsible for telling the story of the evolution of agile clinical trials, and for sharing the unique knowledge and expertise of Science 37’s thought leaders.

A seasoned Journalist, Editor and Content Strategist, James spent more than 20 years in the business media, having moved to the US from the UK in 1998 as part of the launch team of PR Week. After helping to roll out digital marketing publication Revolution two years later, James took the reins at Medical Marketing & Media, where he spent nine years as Editor In Chief, overseeing the launch of many successful initiatives, including the MM&M Awards.

Prior to joining Science 37, James was Digital Director at Ad Age, where he oversaw editorial content on the media outlet’s high-traffic website — winning the coveted Jesse Neal Award for “Website of the Year” in both 2020 and 2021, under his stewardship.

Message Presenter

Britanny Groner, Associate Director, BridgeBio

Brittany has worked in clinical research for over 11 years, across all phases of clinical trials in multiple indications with a focus on rare disease and gene therapy.

As a CRA, Brittany traveled to over 200 research facilities across almost all 50 states.  As a Project Manager and Associate Director of Clinical Operations, Brittany manages clinical trials, overseeing activities of multiple cross-functional groups, sites and vendors to ensure successful execution of these trials in an effort to bring new therapies to patients.

Message Presenter

Who Should Attend?

This insightful session is unmissable for any biopharmaceutical sponsor in the rare diseases space, or for any clinical research professional interested in learning about the positive impact of agile (hybrid) clinical trials and decentralized approaches.

Titles can include:

  • C-suite in Clinical Trials Management
  • Therapeutic Lead/Head
  • Clinical Development Director
  • Clinical Development Manager
  • Clinical Operations
  • Clinical Risk Manager
  • Information System Management
  • Information Technology
  • Innovation Lead/Head
  • Medical Director
  • Operational Director
  • Patient Advocacy
  • Project Management
  • Research and Development
  • Scientific Affairs

What You Will Learn

Attendees will learn:

  • Why the agile clinical trial model is a great fit for many rare disease categories
  • How agile clinical trials are intrinsically patient-focused, and how to maximize patient involvement
  • What successful enrollment looks like for an agile clinical trial in a rare disease category
  • How to navigate the regulatory landscape for agile clinical trials in rare diseases
  • How to design studies that are safe, appropriate and reliable so they can generate data to meet the endpoints
  • About case studies and appropriate use cases for agile clinical trials in rare disease categories

Xtalks Partner

Science 37

Science 37, Inc.’s (Nasdaq: SNCE) mission is to enable universal access to clinical research, making it easier for patients and providers to participate from anywhere. Since 2014, we’ve pioneered decentralized and agile clinical trial approaches and having conducted more than 125 agile clinical trials, we’re helping forge the future of research. The Science 37 Operating System (OS) supports today’s more agile clinical research design, enabling up to 15x faster enrollment, 28% better retention, and 3x more diverse patient population.

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