A Connected, Collaborative Approach to Streamlining Clinical Trials in Children

Melissa Stevens, Director, Strategic Initiatives

According to the Centers for Disease Control and Prevention, at least 14 million children in this country have a brain disorder for which there is no treatment or cure. This figure represents 17 percent of children between birth and 19 years of age.

The Children’s Neurobiological Solutions Foundation (CNS) recently convened a meeting that brought together policy makers, researchers, nonprofit organizations, and industry to address the barriers to clinical trials and treatments for children affected by neurological conditions. We participated at that meeting.

According to CNS, when research into a potential new treatment advances to the clinical stage, obstacles arise. Among the reasons are that few clinicians are experienced in devising pediatric clinical trials for brain disorders, the potential risks of pediatric trials discourage institutional review boards and scare drug companies, and biomarkers or imaging technologies readily used for adult trials have not been adapted to pediatric populations.

CNS is hoping to create the Centers for Excellence for Pediatric Neurological Disorders, a network of locations across the U.S. focused on conducting pediatric brain clinical trials. Because of its specialty focus, testing of treatments for pediatric neurological disorders would be accelerated, significantly enhancing the possibility that laboratory discoveries are translated into safe and effective treatments. The proposed network would be organized and managed under several Centers of Excellence – a hub and spokes system that would be populated by academic child neurologists across the United States. The network would also be charged with:

1. Providing training for child neurologists and their physician and nurse colleagues in clinical trial design management, and interpretation;
2. Exploiting existing infrastructure for clinical trials design, execution, and, evaluation, as well as creating infrastructure where needed;
3. Evaluating emerging basic science discoveries as potential treatments;
4. With colleagues at the FDA and industry, assessing the feasibility of developing discoveries into drugs or other therapeutic modalities;
5. Enlisting international partnerships whenever necessary to advance the mission; and
6. Communicating the results of studies to colleagues and to patients and their advocates, including assembling a searchable accessible database.

At FasterCures, we are firm believers that expediting cures requires collaboration. It was affirming to hear the enthusiasm of participants at this meeting all eager to see this network come to fruition and deliver on the promise of a more connected clinical trial system for children with neurological conditions.

At the upcoming Partnering for Cures meeting, we are specifically spotlighting innovative, cross-sector collaborations that are advancing medical progress. A call for applications for innovator presentations is now underway. Our goal is to feature up to 30 of the most forward-thinking, transformative efforts. To learn more, visit http://www.partneringforcures.org/.