Will 2008 mark the start of a new clinical trial era in gastroenterology?


he randomized trial is the “gold standard” design for most clinical esearch questions related to efficacy of linical interventions. However, there re numerous barriers to the conduct f such studies. Clinical trials typically equire a complex infrastructure to enure data quality and patient safety. dditionally, there are many regulaory hurdles. Clinical trials also conume huge amounts of resources, both n terms of absolute dollar costs and ime commitments. Recruitment of a ufficient number of patients is often mong the greatest challenges faced by nvestigators. To help achieve suffiient sample sizes, many clinical trials re conducted across multiple centers. owever, this serves to further increase he intrinsic complexity and expense of hese trials. Despite the large number of disases encompassed by Gastroenterolgy, the field lags behind others in erms of conduct of clinical trials. A ecent search of www.clinicaltrials. ov (December 22, 2007) identified 5 open clinical trials targeting paients with Crohn’s diseases, 44 with lcerative colitis, 130 with hepatitis , 89 with hepatitis B, 16 with nonlcoholic fatty liver disease or nonaloholic steatohepatitis, 69 with cirhosis, 23 with peptic ulcer disease, 4 with colon polyps, and 2 with eliac disease. By contrast, there were 35 open studies targeting patients ith rheumatoid arthritis, 155 with eart attacks, 239 with congestive eart failure, 515 with AIDS, and ,075 with lymphoma. Recently, several important strateies have been implemented at the Naional Institutes of Health (NIH) that ill impact the potential for future linical trials in Gastroenterology. The linical and Translational Science wards (CTSA) are designed to faciliate the translation of research findc ngs from the bench to the bedside and rom the bedside to populations. Curently, there are 24 funded centers ithin 18 states. By 2012, it is anticiated that 60 centers will be funded. he funded centers are expected to ulimately function as a consortium that ill redefine how collaborative translaional research is conducted. The goals re to “encourage the development of ew methods and approaches to clincal and translational research, imrove training and mentoring to enure that new investigators can avigate the increasingly complex reearch system, design new and imroved clinical research informatics ools, assemble interdisciplinary teams hat cover the complete spectrum of edical research, and forge new parterships with private and public ealth care organizations” (http:// ww.ctsaweb.org/index.cfm). An obvious byproduct of the CTSA rogram will be the identification and esting of new therapeutics. However, he CTSA alone are not sufficient to nsure the execution of high-quality linical trials that define new effective herapies and optimal treatment stratgies. Rather, such studies require arge-scale grants from the NIH that re specific to the study. Competition or such grants is highly challenging. n investigator is expected to convince he review committee that a proposed tudy is novel, well designed, and able o be accomplished in the timeline upported by the grant. Even with a ovel idea, emerging investigators can truggle to convince reviewers that the tudy is well designed and able to be ccomplished. Supporting evidence ay include completion of approprite preliminary studies and developent of a complete research protocol, anual of procedures, data and safety onitoring plan, data management ystem, case report forms, training maerials, and other materials. In my peronal experience, developing such maerials for a complicated, multicenter linical trial requires a year or more of art-time work by the principal invesigator and full-time work by support taff. This substantial burden of proof emanded by review committees may erve as a disincentive for potential rantees to pursue such funding. Understanding this predicament, he NIH recently announced the ew NIDDK Multi-Center Clinical tudy Implementation Planning rants (U34) (PAR-08-057). The 34 grant mechanism will provide p to 2 years of funding of up to 250,000 per year in direct costs to und such activities in preparation or a clinical trial. As stated in the nnouncement (http://grants.nih. ov/grants/guide/pa-files/PAR-0857.html), this funding mechanism ill:

DOI: 10.1053/j.gastro.2008.03.030

Cite this paper

@article{Lewis2008Will2M, title={Will 2008 mark the start of a new clinical trial era in gastroenterology?}, author={James D. Lewis}, journal={Gastroenterology}, year={2008}, volume={134 5}, pages={1289} }