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The next step in translational research: lessons learned from the first preclinical randomized controlled trial
Author(s) -
Llovera Gemma,
Liesz Arthur
Publication year - 2016
Publication title -
journal of neurochemistry
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.75
H-Index - 229
eISSN - 1471-4159
pISSN - 0022-3042
DOI - 10.1111/jnc.13516
Subject(s) - preclinical research , clinical trial , translational research , medicine , randomized controlled trial , medical physics , research design , clinical research , clinical study design , bridging (networking) , protocol (science) , computer science , alternative medicine , pathology , computer network , social science , sociology
For years, low reproducibility of preclinical trials and poor translation of promising preclinical therapies to the clinic have posed major challenges to translational research in most biomedical fields. To overcome the limitations that stand between experimental and clinical research, international consortia have attempted to establish standardized guidelines for study design and for reporting the resulting data. In addition, multicenter preclinical randomized controlled trials (p RCT s) have been proposed as a suitable tool for ‘bridging the gap’ between experimental research and clinical trials. We recently reported the design and results of the first such p RCT in which we confirmed the feasibility of using a coordinated approach with standardized protocols in collaboration with independent multinational research centers. However, despite its successes, this first p RCT also had several difficulties, particularly with respect to following the protocols established in the study design and analyzing the data. Here, we review our experiences performing the study, and we analyze and discuss the lessons learned from performing the first p RCT . Moreover, we provide suggestions regarding how obstacles can be overcome to improve the performance and outcome of future p RCT studies.Translational research is hampered by low reproducibility of preclinical studies and countless failed clinical trials. International consortia have proposed preclinical multicenter trials as an intermediate step to overcome this ‘translational roadblock’. We have recently performed the first such preclinical randomized controlled trial (pRCT) by adopting key elements of clinical study design to preclinical research. In this review, we discuss the lessons learned from this trial and provide suggestions how to optimize future pRCTs. This article is part of the 60th Anniversary special issue .