Value of clinical research: Usefulness tool development and systematic review of 350 randomised controlled trials in preterm birth

Abstract

Objective: We developed a research usefulness tool collating published\ncriteria and examined if randomised controlled trials (RCTs) addressing\npreterm birth were useful. Search Strategy: Cochrane library. Selection\nCriteria: Published RCTs within 56 preterm birth Cochrane reviews. Data\nCollection and Analysis: A usefulness tool was developed with eight\ncriteria combining 13 items identified through literature searches and\nconsensus. RCTs were evaluated for compliance with each item by multiple\nassessors (reviewer agreement 95-98%). Proportions with 95% confidence\ninterval (CI) were calculated and compared for change over time using ≧\n2010 as a cut-off, with relative risks (RR). Main Results: Among 350\nselected RCTs, only 38 (11%, 95% CI 8-15%) met half of the usefulness\ncriteria. Compared to trials before 2010, recent trials used composite\nor surrogate (less informative) outcomes more often (13% vs 25%, RR\n1.87, 95% CI 1.19-2.93). Only 17 trials reflected real life\n(pragmatism) in design (5%, 95% CI 3-8%), with no improvements over\ntime. No trials reported involvement of mothers to reflect patients’ top\npriorities in question definition or outcomes selection. Recent trials\nwere more transparent with prospective registration (0.5% vs 28%, RR\n58, 95% CI 8-420%), availability of protocol (0.5% vs 15%, RR 32,\n95% CI 4-237%) and data sharing statements (2% vs 8%, RR 3, 95% CI\n1-10%). Conclusion: Clinical trials in preterm birth lacked many\nusefulness features, with one tenth of trials meeting half of the items\nevaluated. Use of informative outcomes, patient centeredness, pragmatism\nand transparency should be key targets for future research planning.