Recommendations for achieving interoperable and shareable medical data in the USA.

Easy access to large quantities of accurate health data is required to understand medical and scientific information in real-time; evaluate public health measures before, during, and after times of crisis; and prevent medical errors. Introducing a system in the USA that allows for efficient access to such health data and ensures auditability of data facts, while avoiding data silos, will require fundamental changes in current practices. Here, we recommend the implementation of standardized data collection and transmission systems, universal identifiers for individual patients and end users, a reference standard infrastructure to support calibration and integration of laboratory results from equivalent tests, and modernized working practices.

How the Critical Path Initiative Addresses CDER's Regulatory Science Needs: Some Illustrative Examples.

Since 2008, the Critical Path Initiative has supported FDA's program of intramural research projects in regulatory science, with the goal of improving translation of advances in emerging sciences to the development of safe and effective medical products. Since 2011, the research of FDA's Center for Drug Evaluation and Research (CDER), including the work supported by the Critical Path Initiative, has been guided by the regulatory science needs identified in the CDER science and research needs report. In this review, the authors highlight a few of CDER'S Critical Path Initiative research projects, each addressing a different regulatory science need, to illustrate the diversity of regulatory science at CDER.

Catalyzing the Critical Path Initiative: FDA's progress in drug development activities.

The US Food and Drug Administration (FDA) has directed considerable effort towards modernizing its regulatory processes over the past decade to address the challenges in the drug development sector. Through partnerships and input from stakeholders, multiple initiatives are under way, many projects have been launched, several have resulted in tangible results, and many are ongoing and under discussion. We are learning that collaborative efforts can better inform and leverage existing knowledge, that the challenges of data sharing and intellectual property can be overcome, and that there is wide interest in partnering to address key public health regulatory science issues.

An introduction to RNA-mediated gene silencing.

Careful analysis of cases where introduction of additional copies of endogenous genes caused coordinate silencing of both the transgene and the endogenous gene laid the ground work for the discovery of RNA-mediated silencing. Silencing begins with the expression and recognition of double-stranded RNA, which is cleaved into short RNAs that recognize, by complementarity, sequences that are targets for down regulation. An RNA target can be regarded (post-transcriptional gene silencing), but the small RNAs can also direct the sequence-specific modification of DNA and chromatin.

Glomerular disease workshop.

Recent observations regarding intrinsic glomerular cell biology, particularly in the podocyte, have provided exciting new insights into potential pathogenic mechanisms of human glomerular disease. Although both immune and nonimmune mechanisms of glomerular injury have been studied previously, experimental models of disease and recent techniques that provide tools for molecular profiling show great promise for identifying glomerular disease biomarkers. Despite these recent advances, additional work in both basic and clinical studies of glomerular disease is needed to advance the field.