Rare Diseases

Biography

Biography: Noopur Singh

Abstract

As study and trial facilitation in rare disease struggle with a limited patient population from which to pull, remain high-risk and under-funded, and often produce smaller data sets with less impact than large cohorts, it becomes apparent that non-profits can address the gaps by resourcing the space with infrastructure with multi-site entities through which to run these trials. By providing such elements as core funding, scientific, administrative, budgetary and operational management, clinical studies networks can both produce studies that otherwise would not occur and they can provide industry a lower risk platform through which to run studies. This resource bridges the gap between the science of drug treatment and its practice through lessening the cost and risk to run scientifically based interventions in real world settings. In addition, larger cohort populations can be recruited resulting in more impactful, rigorous and valid studies and trials.