Webinar to tackle challenges in pediatric rare disease trials

Strategies to decentralize trials to aid patient recruitment will also be discussed

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by Mary Chapman |

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A Sept. 28 webinar, hosted by Xtalks, will explore the challenges of clinical research in pediatric rare disorders, along with strategies in decentralizing clinical trials to aid in recruiting and enrolling patients.

Registration is required for the free hour-long webinar, titled “The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life.” It begins at 11 a.m. EDT.

The webinar is mostly aimed at biotechnology, biopharmaceutical, and medical technology professionals involved in preclinical and clinical trials, clinical operations, treatment development, project management, and regulatory affairs.

Attendees will learn about the challenges of conducting clinical trials in pediatric rare disorders such as Batten disease, a group of rare inherited neurological conditions that can cause progressive cognitive and motor decline, vision loss, and seizures. While symptoms can manifest at any age, childhood Batten is the most common.

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Clinical research matters in studying rare pediatric diseases

Batten disease is thought to affect 2 to 4 of every 100,000 U.S. residents, and about 1 in every 100,000 people globally.

Because more than 90% of the approximately 7,000 known rare diseases have no approved treatment, clinical research is crucial to identify therapeutic options. Study design and execution are often hampered by recruitment and retention issues, since it can be difficult for patients and their families to get to where studies are conducted.

“Pediatric rare disease trials combine rarity of disease with a restricted and geographically dispersed population of vulnerable subjects for research, creating unique challenges for study design and execution,” Xtalks stated in a press release. “Recruitment is challenging, as patients, parents, and caregivers need to juggle school schedules, work responsibilities, and family obligations to accommodate study participation.”

Possible applications, risks, considerations, and the future of decentralized clinical trial (DCT) strategies in pediatric rare disease studies will also be discussed.

In DCTs, patients participate in clinical research at various sites scattered nationally, or even around the world. Such approaches may feature virtual consultations and data collection through wearable technology, mitigating the need for in-person site visits.

Incorporating DCT strategies can help attract patients and improve access, enrollment, and retention by reducing the need for site visits, facilitating participation among people for whom traveling long distances may not be feasible.

Webinar presenters represent Premier Research, a global clinical research organization designed to help biotech and pharma companies navigate product development and clinical trials to achieve product approval.

These will include Betsy Reid, Premier’s senior vice president of pediatrics, and Adam Bloomfield, MD, its executive director of medical affairs, rare diseases and pediatrics. Xtalks provides educational webinars to the global life science, food, and medical device community.