Understanding the High Failure Rate in Rare Disease Trials and How to Change It

Upcoming Webinar: Rare Disease Trials, Rarely Ready



The clinical research landscape for rare diseases is fraught with challenges, leading to the unsettling reality that more than half of the trials fail before achieving publication. Understanding the underlying causes of these high failure rates is crucial for researchers and stakeholders alike. In an upcoming free webinar hosted by Xtalks, attendees will gain valuable insights into why these trials often end in discontinuation or remain unpublished, and explore strategies to avoid these pitfalls.

Why Do Trials Fail?


Many professionals in the healthcare and clinical research industries recognize that the reasons behind these failures are often not linked to the drugs themselves but rather to issues like poor-quality data, inadequately designed study frameworks, and outdated recruitment tactics. This results in not only significant resource wastage—often amounting to millions of dollars—but also delays in potentially life-saving treatments for patients awaiting innovative therapies.

Expert Insights


The webinar will feature Ediz Calay, PhD, Senior Manager of Commercial Strategy at IMO Health. Dr. Calay will explain how leveraging normalized clinical data can drastically reduce the time required for site activation and participant cohorting. Attendees will learn about the precarious state of rare disease trials and discover effective methodologies aimed at refining these processes.

Transforming Trial Outcomes


In this session, participants will be introduced to a next-gen trial model that emphasizes data normalization, intelligent cohorting, and streamlined workflow activation. This innovative approach is designed to diminish trial durations and cut costs significantly, making it an invaluable learning opportunity for sponsors and clinical research organizations (CROs). The goal is to replace traditional 'spray-and-pray' methodologies with precision-focused studies that complete faster and ensure inclusivity of all patients who stand to benefit from new treatments.

What to Expect


Webinar attendees can anticipate learning about:
  • - Strategies to overcome common hurdles in rare disease trials.
  • - How to implement a data-driven, precision-oriented workflow.
  • - Tips on increasing enrollment rates and minimizing screen failures within trials.
  • - Techniques that could save organizations substantial funds—over $50,000 per underperforming site.

Who Should Attend?


This session is ideal for clinical researchers, trial sponsors, and CROs looking to improve their trial outcomes substantially. By engaging with this content, participants will gain knowledge about cutting-edge strategies that help adapt to the evolving landscape of rare disease trials.

Register Now


The live webinar is scheduled for Thursday, October 2, 2025, at 12 PM EDT (5 PM BST).
Secure your place today by visiting the registration page: Rare Disease Trials, Rarely Ready: Why Most Fail Before They Begin.

About Xtalks


Xtalks is widely recognized as a leading platform for educational webinars and digital content serving the life sciences, healthcare, and food industries. Each year, thousands of professionals rely on Xtalks for access to high-quality information on the latest industry trends, regulations, and critical issues faced in their fields.
To learn more about Xtalks and its offerings, visit www.xtalks.com.

Join us as we bridge the gap in rare disease research and create opportunities for better outcomes in clinical trials!

Topics Health)

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