Sept. 20 Webinar to Offer Insight Into Rare Disease Clinical Trials

AADC deficiency patients, caregivers can register now for free live seminar

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by Mary Chapman |

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An illustration of a clinical trial word puzzle.

A free live webinar on Sept. 20 will explore various aspects of rare disease clinical trials — and offer insight into the challenges researchers face in finding and enrolling participants, particularly those of diverse backgrounds.

Those interested in attending can register now for the hour-long webinar, titled “Rare Disease Clinical Trials: Site, Advocate, and Technology Insights.” It will broadcast live at 12 p.m. EDT, according to a press release announcing the event.

Attendees will learn about the hope that clinical trials can represent to rare-disease families and caregivers, as well as what study participants can expect and how trials can be improved. In addition, speakers will discuss trial difficulties, including costs — estimated at more than $300,000 per patient — and making studies convenient for caregivers.

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The Road to Enrolling in a Clinical Trial

Further, attendees will hear about new technologies that can affect clinical study enrollment, retention, and compliance. Speakers will address the extent to which “adaptive experiences” improve studies for all participants. Trials with adaptive designs permit modifications without undermining their validity or integrity.

Clinical studies are at the heart of all medical progress, according to the webinar organizers. The goal of such trials is to determine whether a procedure, device, or pharmaceutical therapy is effective and safe. Trials also may evaluate novel ways of using cure treatments, gauge other aspects of care, or just record a condition’s progression over time to provide further data to researchers.

Some 300 million people worldwide are thought to have a rare disorder — such as AADC deficiency, which affects the nervous system and hinders communication between nerve cells.

Only approximately 5% of rare diseases have even one federally approved treatment. There is no cure for AADC, but treatments are available that can ease some symptoms and enhance patients’ life quality. Other therapies are being developed.

The webinar will feature speakers who are experts in rare diseases, including:

  • Daniel DeFabio, director of community engagement for Global Genes. His son, now deceased, had the rare disease Menkes, an inherited X-linked recessive disorder. DeFabio will discuss the expectations and hopes of trial participation.
  • Fabian Sandoval, MD, president and CEO of the Emerson Clinical Research Institute. His presentation will focus on driving diversity in clinical trials.
  • Andrea Valente, CEO of ClinOne, an advocate of adaptive experiences for clinical trial participants. She will speak about using technology to create adaptive experiences for all study participants.

Xtalks, which provides educational webinars to the global life science, food, and medical devices communities, is presenting the event. The webinar is hosted by ClinOne.