Abstract
BackgroundPatients and their families often have preferences for medical care that relate to wider considerations beyond the clinical effectiveness of the proposed interventions. Traditionally, these preferences have not been adequately considered in research. Research questions where patients and families have strong preferences may not be appropriate for traditional randomized controlled trials (RCTs) due to threats to internal and external validity, as there may be high levels of drop-out and non-adherence or recruitment of a sample that is not representative of the treatment population. Several preference-informed designs have been developed to address problems with traditional RCTs, but these designs have their own limitations and may not be suitable for many research questions where strong preferences and opinions are present.MethodsIn this paper, we propose a novel and innovative preference-informed complementary trial (PICT) design which addresses key weaknesses with both traditional RCTs and available preference-informed designs. In the PICT design, complementary trials would be operated within a single study, and patients and/or families would be given the opportunity to choose between a trial with all treatment options available and a trial with treatment options that exclude the option which is subject to strong preferences. This approach would allow those with strong preferences to take part in research and would improve external validity through recruiting more representative populations and internal validity. Here we discuss the strengths and limitations of the PICT design and considerations for analysis and present a motivating example for the design based on the use of opioids for pain management for children with musculoskeletal injuries.ConclusionsPICTs provide a novel and innovative design for clinical trials with more than two arms, which can address problems with existing preference-informed trial designs and enhance the ability of researchers to reflect shared decision-making in research as well as improving the validity of trials of topics with strong preferences.
Highlights
Patients and their families often have preferences for medical care that relate to wider considerations beyond the clinical effectiveness of the proposed interventions
Patients and families are often presented with treatment options that require them to consider the efficacy of treatments, their personal preferences, and the values and weight they place on health outcomes [1]
In healthcare, patients and families may have strong preferences about the treatments they receive that go beyond considerations of efficacy
Summary
Patients and their families often have preferences for medical care that relate to wider considerations beyond the clinical effectiveness of the proposed interventions These preferences have not been adequately considered in research. In research, patients and families must choose whether to give consent and receive an allocated treatment or be excluded This can pose problems for research both ethically (i.e., shared decision-making) and practically (i.e., study feasibility, validity). Preferences in healthcare arise for a number of reasons and reflect patients’ and families’ personal values regarding treatment options When they are strongly held, preferences may drive treatment decisions regardless of the effectiveness of treatments and consideration of preferences is crucial to effective shared decision-making [1]. There is still limited focus on patient preferences and novel approaches are needed to ensure that preferences are considered when planning studies
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