Background: Clinical trials mature from Phase I to Phase IV. The delay in licensure of new drugs for children and pregnant women is as long as 10 years post licensure for adult populations. Hypothetical use of EMRs for data retrieval along with use of AI to both streamline development of clinical protocols and data sets for submission to FDA and EMA are needed

Methods: Pediatric Investigation Plans (PIP) to EMA and Pediatric Study Plan (PSP) to FDA timing to development of clinical trials suggest long delays. The IMPAACT network protocols and planning will be used to demonstrate gaps in clinical trial and licensure submission

Results: Data from the past 10 years of IMPAACT protocol development to licensure will be presented. Gaps and delays across TB and HIV treatment studies will be discussed focusing on study oversight, interim analyses, trial master files and regulatory submissions.

Conclusions: Lack of creative planning for changes in drug availably and new guidelines suggest that more timely and better alternatives that include AI and EMR tools for clinical trial development are needed.

No abstract available.

The speaker is on the autism spectrum and lives between the worlds of science and self-advocacy. In interviewing experts and advocates, several themes emerge. The first is that the public disagreements in the autism world belie a growing convergence of the views that autism is a biological condition subject to intervention and a social difference that requires only acceptance. Scientific evidence, interviews with experts and advocates, and aspects of the speaker’s experience will be shared.

Our group identified the first genes associated with autism pointing at a key role of the synapse in this complex condition. These findings underscore the genetic diversity of autism while revealing shared biological mechanisms. The genetic architecture of autism involves a complex combination of de novo, rare and common genetic variants shared with other traits such as attention deficit hyperactivity disorders (ADHD), cognitive skills, and epilepsy. In this presentation, I will introduce recent results that shed new light on different subgroups of people with autism and how they differ at the clinical, brain imaging and genetic levels. I will also illustrate how we can explore synaptic genes such as SHANK2 and SHANK3 and their roles in cognition and social motivation. Finally, I will present how we are currently using participatory research to study Risk, Resilience and Developmental Diversity in Mental Health (The R2D2-MH project) to understand why some carriers of genetic variants seem to be protected from adverse symptoms while others have more difficulties to thrive in the society.