From the Guidelines
This study is a randomized controlled trial (RCT) comparing Drug A to a standard drug, which is considered the gold standard for evaluating the efficacy and safety of new medications. In this type of study, participants are randomly assigned to either receive the experimental drug (Drug A) or the standard drug, which serves as the control. RCTs are considered the highest level of evidence for therapeutic interventions, allowing for direct causal inferences about treatment effects, as noted in the 2017 ELN recommendations from an international expert panel 1. The control group receiving the standard drug (rather than a placebo) makes this an active-controlled trial, which is ethically appropriate when an effective standard treatment already exists. Key characteristics of RCTs include:
- Random assignment of participants to treatment groups
- Comparison of the experimental drug to a standard drug
- Evaluation of efficacy and safety outcomes
- Minimization of selection bias through randomization
- Creation of comparable groups at baseline, as described in the context of trial design for new therapies 1. Understanding RCT design is crucial for evaluating medical literature and making evidence-based clinical decisions.
From the Research
Study Design
- A randomized controlled trial comparing Drug A to a standard drug is an example of a randomized controlled trial (RCT), which is considered the gold standard for evaluating the causal effects of medications 2.
- RCTs are prospective, comparative, quantitative studies/experiments performed under controlled conditions with random allocation of interventions to comparison groups 3.
- The primary purpose of an RCT is to determine whether a cause-effect relation exists between an intervention and an outcome 3.
Key Characteristics
- RCTs involve random allocation of participants to either an intervention group (receiving Drug A) or a control group (receiving the standard drug) 3, 4.
- The use of randomization helps to minimize bias and ensures that the groups are comparable in terms of known and unknown factors that may affect the outcome 3, 4.
- RCTs can be used to evaluate the effectiveness and safety of an intervention, and can provide high-quality evidence for informing clinical decisions 3, 4.
Examples
- An example of an RCT is the STRIDENT trial, which compared intensive drug therapy (adalimumab plus thiopurine) to standard drug therapy (adalimumab monotherapy) for symptomatic intestinal Crohn's disease strictures 5.
- This trial demonstrated that Crohn's disease strictures are responsive to drug treatment, and that intensive drug therapy may be more effective than standard drug therapy in improving symptoms and stricture morphology 5.