ORPHAN PRODUCT ACCELERATOR – INNOVATIONS INCUBATOR
The Orphan Product Accelerator - Innovations Incubator (OPA-II) module will provide infrastructure, assistance and training for CTR investigators in the development of orphan products, specifically those aimed at the diagnosis and treatment of rare diseases, many of which are particularly relevant to children. Innovation in orphan product development has become a top priority for governments, regulators, patient advocacy groups, industry, and stakeholder foundations worldwide. However, most child-health CTR investigators and team members are not knowledgeable in this area and do not have the supports that they need to compete in these new funding initiatives.
Aim 1: Provide a platform of experienced regulatory, business planning, and drug and device development services to foster the advance of orphan product development for pediatric patients.
Aim 2: Bring together innovators from CTSA hubs to collaborate with the CTSI-CN v2.0 to support milestonedriven, orphan product development involving a mix of projects at all stages of development.
Aim 3: Build sustainable support for the program by leveraging academic-private partnerships to pursue commercialization of orphan products (through licensing, partnership and company spin-offs).
CHILD HEALTH RESEARCH ACCELERATION THROUGH MULTISITE PLANNING
The Child Health research Acceleration through Multisite Planning (CHAMP) module seeks to provide the infrastructure, assistance and training for CTR investigators in the performance of multi-site clinical studies. For rare genetic diseases and other disorders of childhood, natural history studies, in combination with clinical trials, are essential for advancing child health. Both often require multiple-site involvement. The CHAMP program, co-directed by Children's National Hospital and University of California, Irvnie seeks to develop the infrastructure and training program for multi-center trials initially involving 16 CTSA hubs that have a strong pediatric focus. To achieve these goals we have developed the following specific aims:
Aim 1: Use an Immersion/“Boot-camp” approach at early stages of multi-site study incubation to support, organize, and lead one-day team-building conferences (TBCs) for nascent groups of child health researchers
focused on a specific project.
Aim 2: Impart best practices in team science at the early planning phases of study development.
Please visit the Faculty and Staff Directory to find the faculty/staff member(s) responsible for these resources.