The Role of Precompetitive Consortia, Data Sharing and Regulatory Science in Catalyzing Innovation for Autism Spectrum Disorders

Background:  The lack of success in development of effective therapies for Autism Spectrum Disorders (ASD) suggests that public private partnerships are catalysts to tackling the challenges and sharing costs and risks amongst diverse stakeholders. The Innovative Medicine’s Initiative’s European Autism Interventions - A Multicentre Study for Developing New Medications (EU-AIMS) and the nascent Autism Biomarkers Consortium for Clinical Trials (ABC-CT) initiatives serve as a striking examples of the positive impact that can be achieved in precompetitive alliances for ASD. Regulatory agencies in the U.S., Europe and Japan have identified drug development tool platforms to accelerate drug development.

Objectives:  This presentation aims to highlight existing initiatives in ASD and opportunities for future expansion and extension in the areas of regulatory science and innovation.

Methods:  

Critical Path Institute is a nonprofit organization that is dedicated to accelerating drug development by delivering on the mission outlined by the U.S. Food and Drug Administration’s (FDA’s) critical path initiative. Fundamental to the mission of C-Path consortia is the sharing of patient level data from longitudinal natural history studies and legacy clinical trials, and transformation of those data into generalizable and applicable knowledge to advance therapies for specific diseases. C-Path consortia are comprised of industry members, regulatory agencies, academic experts, government agencies and patient advocacy organizations that collaborate to achieve regulatory milestones not achievable by any one organization.  Data standardization, database development and integration are core to the success of all C-Path consortia.

Results:  

Diseases to date that have achieved positive qualification opinions from both European Medicines Agency and FDA enabled by C-Path’s consortia include Alzheimer’s disease, Polycystic Kidney Disease and Tuberculosis. For example, an Alzheimer’s disease (AD) clinical trial simulation tool has been made publically available and represents a milestone that serves to encourage the advancement of drug-disease-trial models and promises to increase the probability of success in future AD therapeutic trials. The applicability of a similar path for ASD holds tremendous promise. Recently launched C-Path consortia are focused on pediatric drug development including Duchenne Muscular Dystrophy (D-RSC), International Neonatal consortium (INC) and a pediatric clinical trial network (PTC) focused on trial recruitment.  Success of C-Path consortia is enabled by collaborations with other global initiatives. The prospects for the future in integrative collaborations and global data sharing initiatives include alliances with fNIH biomarkers consortium, NIMH, IMI EU-AIMS, SFARI/Simons Foundation, academic institutions and investigators, industry sponsors, Autism Speaks and other patient-driven initiatives.

Conclusions:  Regulatory science strategies enabled by active collaboration of diverse stakeholders promises to encourage and incentivize sponsors to discover and advance therapies for ASD.