A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences

Trials

Lawrence Richer¹  Samina Ali²  Naveen Poonai³  Chris McCabe⁴  Gareth Hopkin⁴  Terry Paul Klassen⁵  Anna Heath⁶  Maryna Yaskina⁷ 

[1] Department of Pediatrics, University of Alberta, Edmonton, AB, Canada;Department of Pediatrics, University of Alberta, Edmonton, AB, Canada;Women and Children’s Health Research Institute, University of Alberta, Edmonton, Alberta, Canada;Departments of Pediatrics and Internal Medicine, Schulich School of Medicine & Dentistry, Childrens’ Health Research Institute, London, Ontario, Canada;Institute of Health Economics, Edmonton, Alberta, Canada;Max Rady College of Medicine, Pediatrics and Child Health, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, Manitoba, Canada;Children’s Hospital Research Institute of Manitoba, Winnipeg, Manitoba, Canada;The Hospital for Sick Children, Toronto, Ontario, Canada;University of Toronto, Toronto, Ontario, Canada;University College London, London, UK;Women and Children’s Health Research Institute, University of Alberta, Edmonton, Alberta, Canada;

关键词: Clinical trial;    Patient preference;    Methodology;    Caregiver preference;   

DOI  :  10.1186/s13063-021-05164-1

来源: Springer

【 摘 要 】

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.

【 授权许可】

CC BY