On Tuesday, Tai Pasquini, CHI’s Chief Research Officer attended the Pediatric Inclusion Roundtable: Effective Inclusion of Children Early in Clinical Trials in Washington, DC. The event was hosted by FARA, a rare disease patient organization, and Leavitt Partners, a law firm, along with stakeholders across the rare disease community. Prominent leaders from the FDA, NIH, industry, and patient advocacy shared their visions for how clinical trials should evolve to appropriately include younger participants and innovative approaches to decrease barriers to rare disease drug development in the neonatal and adolescent period. For the hyperinsulinism community, this is critical. Current treatment options are limited and individuals with the most severe forms of HI often must try a complicated mix of medications to manage their HI or undergo a pancreatectomy, generally before their 4th birthday. Clinical trials are critical for ensuring the safety and effectiveness of a new drug, but access currently is not always available to the youngest patients who could benefit. The conversations were incredibly productive, outlining not only the regulatory but the ethical and practical considerations as well. There was a clear commitment from all participants to continue investing in this dialogue and CHI was thrilled to participate and represent the patient advocacy perspective.
If you’d like to learn more about the role of clinical trials for hyperinsulinism, as well as current clinical trial opportunities, visit our Clinical Research Trials page here.
