The National Center for Advancing Translational Sciences (NCATS) at NIH is keenly interested in seeing more research studying rare diseases in which efforts and expertise are pooled across research groups and diseases to identify commonalities and shared targetable mechanisms. The intention is that such collaboration will increase efficiency and capacity in the development of new therapies compared to traditional approaches that focus within a single disease.
It is becoming increasingly clear that problems with some basic cellular processes can contribute to diseases affecting different organs. One cellular signaling pathway that appears to be involved in multiple diseases is the mTOR pathway.
Robert Desnick, PhD, MD, and Cynthia Tifft, PhD, MD, have been chosen by the National Organization for Rare Disorders (NORD) to receive 2017 Rare Impact Awards.
Projects supported under this program must involve collaboration with investigators from at least three different CTSA hubs and are intended to support the development of new technologies, methods or approaches to overcome roadblocks in translational science.
NIH has announced a mandate requiring use of a single Institutional Review Board (sIRB) review for multi-site federally-funded studies. The intent of the mandate is to increase efficiency, uniformity, and reduce the time to obtain IRB approval for multiple sites on the premise that individual sites submitting research to their local IRBs is costly, results in duplication of effort, results in inconsistencies among sites, and delays implementation of research.
Investigators from the 'MILES' (Multicenter International LAM Efficacy of Sirolimus) trial recount the unique path it took seeking FDA review of their drug.