A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences
Abstract Background Patients 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 pati...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2021-03-01
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| Series: | Trials |
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| Online Access: | https://doi.org/10.1186/s13063-021-05164-1 |
| _version_ | 1818676871516127232 |
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| author | Samina Ali Gareth Hopkin Naveen Poonai Lawrence Richer Maryna Yaskina Anna Heath Terry Paul Klassen Chris McCabe on behalf of the KidsCAN PERC Innovative Pediatric Clinical Trials No OUCH Study Group the KidsCAN PERC Innovative Pediatric Clinical Trials Methods Core |
| author_facet | Samina Ali Gareth Hopkin Naveen Poonai Lawrence Richer Maryna Yaskina Anna Heath Terry Paul Klassen Chris McCabe on behalf of the KidsCAN PERC Innovative Pediatric Clinical Trials No OUCH Study Group the KidsCAN PERC Innovative Pediatric Clinical Trials Methods Core |
| author_sort | Samina Ali |
| collection | DOAJ |
| description | Abstract Background Patients 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. Methods In 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. Conclusions PICTs 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. |
| first_indexed | 2024-12-17T08:50:22Z |
| format | Article |
| id | doaj.art-1fbf35c71f7c415fa684f4cd1975fc22 |
| institution | Directory Open Access Journal |
| issn | 1745-6215 |
| language | English |
| last_indexed | 2024-12-17T08:50:22Z |
| publishDate | 2021-03-01 |
| publisher | BMC |
| record_format | Article |
| series | Trials |
| spelling | doaj.art-1fbf35c71f7c415fa684f4cd1975fc222022-12-21T21:56:05ZengBMCTrials1745-62152021-03-012211910.1186/s13063-021-05164-1A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferencesSamina Ali0Gareth Hopkin1Naveen Poonai2Lawrence Richer3Maryna Yaskina4Anna Heath5Terry Paul Klassen6Chris McCabe7on behalf of the KidsCAN PERC Innovative Pediatric Clinical Trials No OUCH Study Groupthe KidsCAN PERC Innovative Pediatric Clinical Trials Methods CoreDepartment of Pediatrics, University of AlbertaInstitute of Health EconomicsDepartments of Pediatrics and Internal Medicine, Schulich School of Medicine & Dentistry, Childrens’ Health Research InstituteDepartment of Pediatrics, University of AlbertaWomen and Children’s Health Research Institute, University of AlbertaThe Hospital for Sick ChildrenMax Rady College of Medicine, Pediatrics and Child Health, Rady Faculty of Health Sciences, University of ManitobaInstitute of Health EconomicsAbstract Background Patients 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. Methods In 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. Conclusions PICTs 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.https://doi.org/10.1186/s13063-021-05164-1Clinical trialPatient preferenceMethodologyCaregiver preference |
| spellingShingle | Samina Ali Gareth Hopkin Naveen Poonai Lawrence Richer Maryna Yaskina Anna Heath Terry Paul Klassen Chris McCabe on behalf of the KidsCAN PERC Innovative Pediatric Clinical Trials No OUCH Study Group the KidsCAN PERC Innovative Pediatric Clinical Trials Methods Core A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences Trials Clinical trial Patient preference Methodology Caregiver preference |
| title | A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences |
| title_full | A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences |
| title_fullStr | A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences |
| title_full_unstemmed | A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences |
| title_short | A novel preference-informed complementary trial (PICT) design for clinical trial research influenced by strong patient preferences |
| title_sort | novel preference informed complementary trial pict design for clinical trial research influenced by strong patient preferences |
| topic | Clinical trial Patient preference Methodology Caregiver preference |
| url | https://doi.org/10.1186/s13063-021-05164-1 |
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