Randomized controlled trials (RCTs) are designed to measure the effectiveness of a new intervention or treatment by minimizing bias through the random allocation of participants to one or more comparison groups. While RCTs are the gold standard for evaluating clinical interventions, there are limitations to their use in settings where strong patient preferences exist. Such limitations are pronounced in cardiovascular research trials comparing treatments that differ substantially in invasiveness, burden, or delivery.
In a commentary in the Journal of Clinical Epidemiology, Dr. Bjorn Redfors and colleagues describe patient-preference controlled designs, which offer a non-randomized alternative to RCTs or a complement to randomization. Researchers suggest that patient preference should be considered in settings where randomization might limit participation or introduce systematic bias through unequal treatment adherence or study discontinuation across trial arms. This format enables patients to select their preferred treatment, thereby improving trial representativeness and increasing patient compliance.
Dr. Redfors and colleagues outline a hybrid trial design that incorporates both randomized and preference-based cohorts. As mentioned, the patient-preference controlled approach can complement randomization within the same trial to increase efficiency and external validity. This trial design allows patients who are willing to be randomized to be enrolled in the randomized cohort, while patients with stronger preferences can be included in the patient-preference controlled cohort. This integrated approach could provide a more comprehensive framework for evaluating health care interventions and producing results that are practically meaningful across patient populations. Researchers advocate for the development of formal design and reporting guidelines for these methods and encourage their use in trials where patient empowerment and generalizability are paramount.
- Highlights