艾昆纬（IQVIA）：2026早期临床试验优化白皮书：降低风险并提升决策效能（英文版）（16页）.pdf

1、White PaperOptimizing Early-Phase Clinical Trials to Minimize Risk and Improve Decision-Making ELIZABETH ALLEN,PhD,Vice President,Early Phase Development Centre of Excellence,Scientific Strategy,IQVIA RAYMOND COOK,MD,Vice President,Medical Strategy for Early Phase Development,IQVIA DAPHNE C.TSITOURA

2、,MD,PhD,Vice President,Therapeutic Strategy,IQVIATable of contentsIntroduction:The increasing importance of early-phase trials in a changing environment 3Pillars of success 4Pillar one:Innovative trial design frameworks 4 Adaptive and hybrid trial designs 4 Basket trials 5 Master protocols 6 Bayesia

3、n analytics 7Pillar two:Selecting CRO partners that align scientific expertise with operational capabilities 9 Global strategies 10Pillar three:Integrated delivery 11Case Study:Early-phase study in sickle cell disease 12Future horizons:Early stage and beyond 13About the authors increasing importance

4、 of early-phase trials in a changing environmentFor more than half a century,clinical trials have adopted a model wherein phase I and II trials examine the safety,pharmacokinetics,dose range,optimal dosing,and early efficacy of a new molecule in small groups of healthy volunteers and patients.These

5、phases have traditionally operated consecutively,with phase II studies beginning after the results of phase I have been reported and statistically analyzed.Today,this paradigm is changing as the result of science evolution,intensified economic pressures,and constrained capital that have raised the c

6、osts inherent in drug development while simultaneously heightening scrutiny over investment decisions.From the earliest stages of drug development,sponsors are expected to provide strong scientific justification for investment decisions while delivering credible investment-related evidence that supp