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Randomized trials labelled as "pragmatic" are attractive to funders, patients, and clinicians as the label implies that the results are directly applicable to clinical care. We examined how authors justify use of the label (e.g., by referring to one or more PRECIS [PRagmatic Explanatory Continuum Indicator Summary]-2 domains).We reviewed primary trial reports published 2014-2019, registered in ClinicalTrials.gov and using the pragmatic label anywhere in the report.Among 415 trials, the label was justified by reference to at least one design element in 282 (68.0%); of these, 240 (85.1%) referenced trial characteristics that can be mapped to one or more of the PRECIS-2 domains, most commonly eligibility (91, 32.3%), setting (90, 31.9%), flexibility delivery (89, 31.6%), and organization (75, 26.6%); 42 (14.9%) referenced characteristics that are not PRECIS-2 domains, most commonly type of intervention/comparator (48, 17%), recruitment without consent (22, 7.8%), routinely collected data (22, 7.8%), and cluster randomization (20, 7.1%). Most reports referenced only one or two design elements. Overall, 9/415 (2.2%) provided PRECIS wheels.Current use of pragmatic labels is uninformative. Authors should clarify the decision the trial is intended to support and include a PRECIS-2 table to make the design transparent.

Background Addressing persistent and pervasive health inequities is a global moral imperative, which has been highlighted and magnified by the societal and health impacts of the COVID-19 pandemic. Observational studies can aid our understanding of the impact of health and structural oppression based on the intersection of gender, race, ethnicity, age and other factors, as they frequently collect this data. However, the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guideline, does not provide guidance related to reporting of health equity. The goal of this project is to develop a STROBE-Equity reporting guideline extension. Methods We assembled a diverse team across multiple domains, including gender, age, ethnicity, Indigenous background, disciplines, geographies, lived experience of health inequity and decision-making organizations. Using an inclusive, integrated knowledge translation approach, we will implement a five-phase plan which will include: (1) assessing the reporting of health equity in published observational studies, (2) seeking wide international feedback on items to improve reporting of health equity, (3) establishing consensus amongst knowledge users and researchers, (4) evaluating in partnership with Indigenous contributors the relevance to Indigenous peoples who have globally experienced the oppressive legacy of colonization, and (5) widely disseminating and seeking endorsement from relevant knowledge users. We will seek input from external collaborators using social media, mailing lists and other communication channels. Discussion Achieving global imperatives such as the Sustainable Development Goals (e.g., SDG 10 Reduced inequalities, SDG 3 Good health and wellbeing) requires advancing health equity in research. The implementation of the STROBE-Equity guidelines will enable a better awareness and understanding of health inequities through better reporting. We will broadly disseminate the reporting guideline with tools to enable adoption and use by journal editors, authors, and funding agencies, using diverse strategies tailored to specific audiences.

ObjectivesTo describe reporting of informed consent in pragmatic trials, justifications for waivers of consent and reporting of alternative approaches to standard written consent. To identify factors associated with (1) not reporting and (2) not obtaining consent.MethodsSurvey of primary trial reports, published 2014–2019, identified using an electronic search filter for pragmatic trials implemented in MEDLINE, and registered in ClinicalTrials.gov.ResultsAmong 1988 trials, 132 (6.6%) did not include a statement about participant consent, 1691 (85.0%) reported consent had been obtained, 139 (7.0%) reported a waiver and 26 (1.3%) reported consent for one aspect (eg, data collection) but a waiver for another (eg, intervention). Of the 165 trials reporting a waiver, 76 (46.1%) provided a justification. Few (53, 2.9%) explicitly reported use of alternative approaches to consent. In multivariable logistic regression analyses, lower journal impact factor (p=0.001) and cluster randomisation (pDiscussionNot obtaining consent seems to be increasing and is associated with the use of cluster randomisation and pragmatic aims, but neither cluster randomisation nor pragmatism are currently accepted justifications for waivers of consent. Rather than considering either standard written informed consent or waivers of consent, researchers and research ethics committees could consider alternative consent approaches that may facilitate the conduct of pragmatic trials while preserving patient autonomy and the public’s trust in research.

Background The transfer of information is a key aspect of the transition of adolescent patients with inflammatory bowel disease (IBD) from pediatric to adult care. This is typically accomplished through the use of a consultation letter with a medical summary of the patient being transferred. To improve the quality and completeness of information included in a transfer letter, we developed a standardized medical summary template by integrating the feedback of adult and pediatric health care providers. Methods To develop the letter template, we purposively sampled gastroenterologists or nurse practitioners caring for patients with IBD in four Canadian cities and invited them to take part in focus group discussions. Using a semi-structured approach, we explored the items deemed essential for inclusion in a transfer summary. Using the conventional content analysis framework, the focus group discussions were inductively coded to identify areas of priority for inclusion in the template. Results Four focus groups were conducted, comprising 17 health care providers of 30 invited (56.7% participation). The resulting medical summary template included the following major headings: patient/disease characteristics, therapeutics history (including medications and surgeries), clinical history and current status, noteworthy investigations, history of complications (including hospitalizations), family history, immunization history and psychosocial history. The template also addressed health system process factors (i.e., urgency of transfer, mode of delivery and confidentiality) to ensure a seamless transfer to adult care. Conclusions The standardized medical summary template should be used by pediatric providers to ensure that essential patient information and disease characteristics are sent to an adult provider.

The overall aim of this program of research is to assess when/how patient partners are compensated financially for their contributions to health research. The research program consists of three studies to address the following questions: (1) What is the prevalence of reporting patient partner financial compensation? (2) What are researcher and institutional attitudes around patient partner financial compensation? (3) What are the current practices of patient partner financial compensation and what guidance exists to inform these practices?In our first project, we will conduct a systematic review to assess the prevalence of reporting patient partner financial compensation and identify current financial compensation practices on an international scale. We will identify a cohort of published studies that have engaged patients as partners through a forward citation search of the Guidance for Reporting the Involvement of Patients and the Public (GRIPP I and II) checklists. We will extract details of financial compensation (type of financial compensation, amount, payment frequency etc.) and reported benefits, challenges, barriers and enablers to financially compensating patient partners. Quantitative data will be analyzed descriptively, and qualitative data will undergo thematic analysis. In our second project, we will conduct a cross-sectional survey of researchers who have engaged patient partners. We will also survey members of their affiliated institutions to gain further understanding of stakeholder experiences and attitudes with patient partner financial compensation. Survey responses will be analyzed by calculating prevalence. In our third project, we will conduct a scoping review to identify all published guidance and policy documents that guide patient partner financial compensation. Overton, the largest available online database of international policy documents, and the grey literature will be systematically searched. Data items will be extracted and presented descriptively. A comprehensive overview of guidance documents will be presented, which will represent a repository of resources that stakeholders can refer to when developing a financial compensation strategy.Our three studies will not only inform and assist patient partners and researchers by informing compensation strategies, but also support the inclusion of diverse perspectives. We will disseminate findings through traditional mediums (publications, conferences) as well as social media, non-technical summaries, and visual abstracts.Partnering with patients, caregivers and members of the public to co-develop and co-conduct research is becoming more accepted and prevalent. However, it is unclear how researchers recognize patient partners for their contributions to research projects. In this publication we outline a series of three studies that will help us better understand when and how patient partners are being financially compensated for their involvement.First, we will conduct a review of the literature to identify an international group of researchers that engage patients as research partners. Evaluating these studies will give us an idea about how often patient partners are compensated, and the methods of compensation being used (e.g. offered gifts, cash, etc.). Second, we will survey researchers and institutions who have experience with patient engagement to ask them for more details about their experiences with patient partner compensation (i.e. challenges that they have encountered and their attitudes). Third, we will conduct another review to identify any guidance documents or policies that can help guide researchers through the processes of financially compensating patient partners. We will provide a summary of all of these guidance documents so that researchers, patient partners, and institutions have a one-stop-shop of resources.We hope that our three studies will inform and help researchers and patient partners navigate the processes for compensating patient engagement in research.

IntroductionFor close to a century opioid administration has been a standard of care to complement anaesthesia during surgery. Considering the worldwide opioid epidemic, this practice is now being challenged and there is a growing use of systemic pharmacological opioid minimising strategies. Our aim is to conduct a scoping review that will examine clinical trials that have evaluated the impact of intraoperative opioid minimisation strategies on patient-centred outcomes and identify promising strategies.Methods and analysisOur scoping review will follow the framework developed by Arksey and O’Malley. We will search MEDLINE, Embase, CENTRAL, Web of Science and CINAHL from their inception approximately in March 2023. We will include randomised controlled trials, assessing the impact of systemic intraoperative pharmacologic opioid minimisation strategies on patient-centred outcomes. We define an opioid minimisation strategy as any non-opioid drug with antinociceptive properties administered during the intraoperative period. Patient-centred outcomes will be defined and classified based on the consensus definitions established by the Standardised Endpoints in Perioperative Medicine initiative (StEP-COMPAC group) and informed by knowledge users and patient partners. We will use a coproduction approach involving interested parties. Our multidisciplinary team includes knowledge users, patient partners, methodologists and knowledge user organisations. Knowledge users will provide input on methods, outcomes, clinical significance of findings, implementation and feasibility. Patient partners will participate in assessing the relevance of our design, methods and outcomes and help to facilitate evidence translation. We will provide a thorough description of available clinical trials, compare their reported patient-centred outcome measures with established recommendations and identify promising strategies.Ethics and disseminationEthics approval is not required for the review. Our scoping review will inform future research including clinical trials and systematic reviews through identification of important intraoperative interventions. Results will be disseminated through a peer-reviewed publication, presentation at conferences and through our network of knowledge user collaborators.RegistrationOpen Science Foundation (currently embargoed)

Background Major depressive disorder is common, debilitating, and affects feelings, thoughts, mood, and behaviors. Childhood and adolescence are critical periods for the development of depression and adolescence is marked by an increased incidence of mental health disorders. This protocol outlines the planned scope and methods for a systematic review update that will evaluate the benefits and harms of screening for depression in children and adolescents. Methods This review will update a previously published systematic review by Roseman and colleagues. Eligible studies are randomized controlled trials (RCTs) assessing formal screening in primary care to identify children or adolescents not already self-reporting symptoms of, diagnosed with, or treated for depression. If no or only a single RCT is available, we will consider controlled studies without random assignment. Studies of participants with characteristics associated with an elevated risk of depression will be analyzed separately. Outcomes of interest are symptoms of depression, classification of major depressive disorder based on a validated diagnostic interview, suicidality, health-related quality of life, social function, impact on lifestyle behavior (e.g., substance use, school performance, lost time at work, or school), false-positive results, overdiagnosis, overtreatment, labeling, and other harms such as those arising from treatment. We will search MEDLINE, Embase, PsycINFO, CINAHL, the Cochrane Library, and grey literature sources. Two reviewers will independently screen the titles and abstracts using the liberal accelerated method. Full-text screening will be performed independently by two reviewers using pre-specified eligibility criteria. Data extraction and risk of bias assessments will be performed independently by two reviewers. Pre-planned analyses, including subgroup and sensitivity analyses, are detailed within this protocol. Two independent reviewers will assess and finalize through consensus the certainty of evidence using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, and prepare GRADE evidence profiles and summary of findings tables for each outcome of interest. Discussion The systematic review will provide a current state of the evidence of benefits and harms of depression screening in children and adolescents. These findings will be used by the Canadian Task Force on Preventive Health Care to inform the development of recommendations on depression screening. Systematic review registration PROSPERO CRD42020150373

IntroductionChildren with inherited metabolic diseases (IMDs) often have complex and intensive healthcare needs and their families face challenges in receiving high-quality, family centred health services. Improvement in care requires complex interventions involving multiple components and stakeholders, customised to specific care contexts. This study aims to comprehensively understand the healthcare experiences of children with IMDs and their families across Canada.Methods and analysisA two-stage explanatory sequential mixed methods design will be used. Stage 1: quantitative data on healthcare networks and encounter experiences will be collected from 100 parent/guardians through a care map, 2 baseline questionnaires and 17 weekly diaries over 5–7 months. Care networks will be analysed using social network analysis. Relationships between demographic or clinical variables and ratings of healthcare experiences across a range of family centred care dimensions will be analysed using generalised linear regression. Other quantitative data related to family experiences and healthcare experiences will be summarised descriptively. Ongoing analysis of quantitative data and purposive, maximum variation sampling will inform sample selection for stage 2: a subset of stage 1 participants will participate in one-on-one videoconference interviews to elaborate on the quantitative data regarding care networks and healthcare experiences. Interview data will be analysed thematically. Qualitative and quantitative data will be merged during analysis to arrive at an enhanced understanding of care experiences. Quantitative and qualitative data will be combined and presented narratively using a weaving approach (jointly on a theme-by-theme basis) and visually in a side-by-side joint display.Ethics and disseminationThe study protocol and procedures were approved by the Children’s Hospital of Eastern Ontario’s Research Ethics Board, the University of Ottawa Research Ethics Board and the research ethics boards of each participating study centre. Findings will be published in peer-reviewed journals and presented at scientific conferences.

Acromio-clavicular (AC) joint pathology is a disabling condition that can restrict quality of life through chronic pain and functional limitation. The objective of this study was to determine symptoms and patient-centred outcomes identified by patients with AC pathology and from experienced shoulder surgeons.Qualitative research methods were undertaken with patient and surgeon focus groups. Patients with AC-joint pathology were invited to participate and were stratified into two cohorts: AC instability and AC osteoarthritis (OA).Sixteen patients participated in five patient focus group discussions including 10 patients with AC instability and six patients with AC OA. Four surgeons participated in two focus groups. Although some themes were common to both AC Instability and AC OA, a number of themes were unique to each. A number of themes were identified that do not appear in existing AC joint scores.Although many factors affecting the AC joint were common to instability and OA pathology, several factors appear to be unique to each and do not appear in existing AC joint metrics. These symptoms and patient-centered outcomes may be used by clinicians in the assessment of patients with AC pathology and in the development of rehabilitation programs.Implications for rehabilitationA thorough understanding of symptoms and patient-centred outcomes associated with acromio-clavicular arthritis and instability is necessary in order to conduct an accurate clinical assessment and design an effective rehabilitation program that meets the clinical needs of patients.Patients in this study identified several themes relevant to assessment and rehabilitation program development including pain location, type of pain (eg. burning pain), and specific activities that induced pain that do not exist in current existing tools.Our finding that certain themes were only raised in either acromio-clavicular instability or osteoarthritis suggests that there may be differences in important outcomes for patients depending on the underlying cause of the acromio-clavicular joint pathology. By incorporating these themes into clinical assessments, outcomes important to patients may be elicited to monitor response to rehabilitation following injury or surgery.

Background The hemodialysis setting is suitable for trials that use cluster randomization, where intact groups of individuals are randomized. However, cluster randomized trials (CRTs) are complicated in their design, analysis, and reporting and can pose ethical challenges. We reviewed CRTs in the hemodialysis setting with respect to reporting of key methodological and ethical issues. Methods We conducted a systematic review of CRTs in the hemodialysis setting, published in English, between 2000 and 2019, and indexed in MEDLINE or Embase. Two reviewers extracted data, and study results were summarized using descriptive statistics. Results We identified 26 completed CRTs and five study protocols of CRTs. These studies randomized hemodialysis centers (n = 17, 55%), hemodialysis shifts (n = 12, 39%), healthcare providers (n = 1, 3%), and nephrology units (n = 1, 3%). Trials included a median of 28 clusters with a median cluster size of 20 patients. Justification for using a clustered design was provided by 15 trials (48%). Methods that accounted for clustering were used during sample size calculation in 14 (45%), during analyses in 22 (71%), and during both sample size calculation and analyses in 13 trials (42%). Among all CRTs, 26 (84%) reported receiving research ethics committee approval; patient consent was reported in 22 trials: 10 (32%) reported the method of consent for trial participation and 12 (39%) reported no details about how consent was obtained or its purpose. Four trials (13%) reported receiving waivers of consent, and the remaining 5 (16%) provided no or unclear information about the consent process. Conclusion There is an opportunity to improve the conduct and reporting of essential methodological and ethical issues in future CRTs in hemodialysis. Review Registration We conducted this systematic review using a pre-specified protocol that was not registered.

Background: Novel rationales for randomizing clusters rather than individuals appear to be emerging from the push for more pragmatic trials, for example, to facilitate trial recruitment, reduce the costs of research, and improve external validity. Such rationales may be driven by a mistaken perception that choosing cluster randomization lessens the need for informed consent. We reviewed a random sample of published cluster randomized trials involving only individual-level health care interventions to determine (a) the prevalence of reporting a rationale for the choice of cluster randomization; (b) the types of explicit, or if absent, apparent rationales for the use of cluster randomization; (c) the prevalence of reporting patient informed consent for study interventions; and (d) the types of justifications provided for waivers of consent. We considered cluster randomized trials for evaluating exclusively the individual-level health care interventions to focus on clinical trials where individual randomization is only theoretically possible and where there is a general expectation of informed consent. Methods: A random sample of 40 cluster randomized trials were identified by implementing a validated electronic search filter in two electronic databases (Ovid MEDLINE and Embase), with two reviewers independently extracting information from each trial. Inclusion criteria were the following: primary report of a cluster randomized trial, evaluating exclusively an individual-level health care intervention, published between 2007 and 2016, and conducted in Canada, the United States, European Union, Australia, or low- and middle-income country settings. Results: Twenty-five trials (62.5%, 95% confidence interval = 47.5%–77.5%) reported an explicit rationale for the use of cluster randomization. The most commonly reported rationales were those with logistical or administrative convenience (15 trials, 60%) and those that need to avoid contamination (13 trials, 52%); five trials (20%) were cited rationales related to the push for more pragmatic trials. Twenty-one trials (52.5%, 95% confidence interval = 37%–68%) reported written informed consent for the intervention, two (5%) reported verbal consent, and eight (20%) reported waivers of consent, while in nine trials (22.5%) consent was unclear or not mentioned. Reported justifications for waivers of consent included that study interventions were already used in clinical practice, patients were not randomized individually, and the need to facilitate the pragmatic nature of the trial. Only one trial reported an explicit and appropriate justification for waiver of consent based on minimum criteria in international research ethics guidelines, namely, infeasibility and minimal risk. Conclusion: Rationales for adopting cluster over individual randomization and for adopting consent waivers are emerging, related to the need to facilitate pragmatic trials. Greater attention to clear reporting of study design rationales, informed consent procedures, as well as justification for waivers is needed to ensure that such trials meet appropriate ethical standards.

BackgroundThere is a concern that the apparent effectiveness of interventions tested in clinical trials may not be an accurate reflection of their actual effectiveness in usual practice. Pragmatic randomized controlled trials (RCTs) are designed with the intent of addressing this discrepancy. While pragmatic RCTs may increase the relevance of research findings to practice they may also raise new ethical concerns (even while reducing others). To explore this question, we interviewed key stakeholders with the aim of identifying potential ethical challenges in the design and conduct of pragmatic RCTs with a view to developing future guidance on these issues.MethodsInterviews were conducted with clinical investigators, methodologists, patient partners, ethicists, and other knowledge users (e.g., regulators). Interviews covered experiences with pragmatic RCTs, ethical issues relevant to pragmatic RCTs, and perspectives on the appropriate oversight of pragmatic RCTs. Interviews were coded inductively by two coders. Interim and final analyses were presented to the broader team for comment and discussion before the analytic framework was finalized.ResultsWe conducted 45 interviews between April and September 2018. Interviewees represented a range of disciplines and jurisdictions as well as varying content expertise. Issues of importance in pragmatic RCTs were (1) identification of relevant risks from trial participation and determination of what constitutes minimal risk; (2) determining when alterations to traditional informed consent approaches are appropriate; (3) the distinction between research, quality improvement, and practice; (4) the potential for broader populations to be affected by the trial and what protections they might be owed; (5) the broader range of trial stakeholders in pragmatic RCTs, and determining their roles and responsibilities; and (6) determining what constitutes “usual care” and implications for trial reporting.ConclusionsOur findings suggest both the need to discuss familiar ethical topics in new ways and that there are new ethical issues in pragmatic RCTs that need greater attention. Addressing the highlighted issues and developing guidance will require multidisciplinary input, including patient and community members, within a broader and more comprehensive analysis that extends beyond consent and attends to the identified considerations relating to risk and stakeholder roles and responsibilities.

Purpose To inform recommendations by the Canadian Task Force on Preventive Health Care on potentially inappropriate prescribing and over-the-counter (OTC) medication use among adults aged 65 years and older in primary care settings. This protocol outlines the planned scope and methods for a systematic review of the benefits and harms and acceptability of interventions to reduce potentially inappropriate prescriptions and OTC medication use. Methods De novo systematic reviews will be conducted to synthesize the available evidence on (a) the benefits and harms of interventions to reduce potentially inappropriate prescriptions and OTC medications compared to no intervention, usual care, or non- or minimally active intervention among adults aged 65 years and older and (b) the acceptability of these interventions or attributes among patients. Outcomes of interest for the benefits and harms review are all-cause mortality, hospitalization, non-serious adverse drug reactions, quality of life, emergency department visits, injurious falls, medical visits, and the number of medications (and number of pills). Outcomes for the acceptability review are the preference for and relative importance of different interventions or their attributes. For the benefits and harms review, we will search MEDLINE, Embase, and Cochrane Central Register of Controlled Trials for randomized controlled trials. For the acceptability review, we will search MEDLINE, Embase, PsycInfo, Cochrane Central Register of Controlled Trials, and the NHS Economic Evaluation Database for experimental and observational studies with a comparator. Websites of relevant organizations, other grey literature sources, and reference lists of included studies and reviews will be searched. Title and abstract screening will be completed by two independent reviewers using the liberal accelerated approach. Full-text review, data extraction, risk of bias assessments, and GRADE (Grading of Recommendations Assessment, Development and Evaluation) will be completed independently by two reviewers, with any disagreements resolved by consensus or by consulting with a third reviewer. The GRADE approach will be used to assess the certainty of the evidence for outcomes. Discussion The results of this systematic review will be used by the Canadian Task Force on Preventive Health Care to inform their recommendation on potentially inappropriate prescribing and OTC medication use among adults aged 65 years and older. Systematic review registration PROSPERO (KQ1: CRD42022302313; KQ2: CRD42022302324); Open Science Framework (https://osf.io/urj4b/).

This review aims to describe the landscape of pragmatic randomized controlled trials (RCTs) in the context of Alzheimer's disease (AD) and related dementias with respect to ethical considerations.Searches of MEDLINE were performed from January 2014 until April 2019. Extracted information included: trial setting, interventions, data collection, study population, and ethical protections (including ethics approvals, capacity assessment, and informed consent).We identified 62 eligible reports. More than two-thirds (69%) included caregivers or health-care professionals as research participants. Fifty-eight (94%) explicitly identified at least one vulnerable group. Two studies did not report ethics approval. Of 57 studies in which patients were participants, 55 (96%) reported that consent was obtained but in 37 studies (67%) no mention was made regarding assessment of the patients' capacity to consent to research participation.Few studies reported protections implemented when vulnerable participants were included. Shortcomings remain when reporting consent approaches and capacity assessment.

Background and Aims We need more pragmatic trials of interventions to improve care and outcomes for people living with Alzheimer’s disease and related dementias. However, these trials present unique methodological challenges in their design, analysis, and reporting—often, due to the presence of one or more sources of clustering. Failure to account for clustering in the design and analysis can lead to increased risks of Type I and Type II errors. We conducted a review to describe key methodological characteristics and obtain a “baseline assessment” of methodological quality of pragmatic trials in dementia research, with a view to developing new methods and practical guidance to support investigators and methodologists conducting pragmatic trials in this field. Methods We used a published search filter in MEDLINE to identify trials more likely to be pragmatic and identified a subset that focused on people living with Alzheimer’s disease or other dementias or included them as a defined subgroup. Pairs of reviewers extracted descriptive information and key methodological quality indicators from each trial. Results We identified N = 62 eligible primary trial reports published across 36 different journals. There were 15 (24%) individually randomized, 38 (61%) cluster randomized, and 9 (15%) individually randomized group treatment designs; 54 (87%) trials used repeated measures on the same individual and/or cluster over time and 17 (27%) had a multivariate primary outcome (e.g. due to measuring an outcome on both the patient and their caregiver). Of the 38 cluster randomized trials, 16 (42%) did not report sample size calculations accounting for the intracluster correlation and 13 (34%) did not account for intracluster correlation in the analysis. Of the 9 individually randomized group treatment trials, 6 (67%) did not report sample size calculations accounting for intracluster correlation and 8 (89%) did not account for it in the analysis. Of the 54 trials with repeated measurements, 45 (83%) did not report sample size calculations accounting for repeated measurements and 19 (35%) did not utilize at least some of the repeated measures in the analysis. No trials accounted for the multivariate nature of their primary outcomes in sample size calculation; only one did so in the analysis. Conclusion There is a need and opportunity to improve the design, analysis, and reporting of pragmatic trials in dementia research. Investigators should pay attention to the potential presence of one or more sources of clustering. While methods for longitudinal and cluster randomized trials are well developed, accessible resources and new methods for dealing with multiple sources of clustering are required. Involvement of a statistician with expertise in longitudinal and clustered designs is recommended.

ObjectivesTo describe the extent to which pragmatic trials underachieved or overachieved their target sample sizes, examine explanations and identify characteristics associated with under-recruitment and over-recruitment.Study design and settingSecondary analysis of an existing database of primary trial reports published during 2014–2019, registered in ClinicalTrials.gov, self-labelled as pragmatic and with target and achieved sample sizes available.ResultsOf 372 eligible trials, the prevalence of under-recruitment (achieving 110% of target) was 87 (23.4%). Under-recruiting trials commonly acknowledged that they did not achieve their targets (51, 71.8%), with the majority providing an explanation, but only 11 (12.6%) over-recruiting trials acknowledged recruitment excess. The prevalence of under-recruitment in individually randomised versus cluster randomised trials was 41 (17.0%) and 30 (22.9%), respectively; prevalence of over-recruitment was 39 (16.2%) vs 48 (36.7%), respectively. Overall, 101 025 participants were recruited to trials that did not achieve at least 90% of their target sample size. When considering trials with over-recruitment, the total number of participants recruited in excess of the target was a median (Q1–Q3) 319 (75–1478) per trial for an overall total of 555 309 more participants than targeted. In multinomial logistic regression, cluster randomisation and lower journal impact factor were significantly associated with both under-recruitment and over-recruitment, while using exclusively routinely collected data and educational/behavioural interventions were significantly associated with over-recruitment; we were unable to detect significant associations with obtaining consent, publication year, country of recruitment or public engagement.ConclusionsA clear explanation for under-recruitment or over-recruitment in pragmatic trials should be provided to encourage transparency in research, and to inform recruitment to future trials with comparable designs. The issues and ethical implications of over-recruitment should be more widely recognised by trialists, particularly when designing cluster randomised trials.

Changing circumstances may alter the benefit and risk profile of screening programmes. Fabienne G Ropers and colleagues propose a framework for re-evaluation to ensure continued public benefit

Background Core outcome sets (COS) are lists of consensus-determined outcomes to be measured and reported in all clinical research studies within a disease area. While including patients and families in COS development to improve their relevance and applicability to patient values is key, there is limited literature documenting practical barriers and facilitators to successful patient engagement in COS development. In this paper, as researchers and patient partners, we provide a resource for COS developers to meaningfully and effectively engage patients and families. Main body To establish a consensus-based COS for children with two inherited metabolic diseases (medium-chain acyl-CoA dehydrogenase deficiency and phenylketonuria), we conducted an evidence review, Delphi survey, and workshop. Two adult patient partner co-investigators co-developed the study protocol, co-designed strategies to address challenges with incorporating patient perspectives, and led all patient engagement activities, including communication with a group of family advisors. Seven adult family advisors received training about COS development and subsequently contributed to Delphi survey development, outcome definitions, the consensus workshop, and selection of outcome measurement instruments. Patient partner co-investigators and family advisors were essential to the successful design, conduct, and completion of the two COS. Patient partner co-investigators supported the understanding, inclusion and engagement of family advisors, and helped develop accessible tools to determine patient-oriented outcome measurement instruments. Patient partner co-investigators and family advisors collaborated with the study team to co-develop surveys, modify technical language, and recruit participants to the study. Together, we addressed challenges to patient engagement in COS development such as unfamiliarity with study methods, comprehensibility of materials and ongoing engagement, and power imbalances between team members. Conclusion Our approach to patient and family engagement in COS development for two rare conditions for children was feasible and considered valuable by all study team members, including patients and family members, in improving the relevance of the deliverable to patients. This approach to patient engagement in developing COS can be applied to other paediatric disease contexts, allowing patient and family perspectives to influence the direction of future studies to develop COS. Supplementary Information The online version contains supplementary material available at 10.1186/s40900-021-00304-y., Plain English summary Core outcome sets (COS) are lists of outcomes agreed upon by a group of people to be measured and reported in studies about certain diseases and populations. Core outcomes are meant to represent what is useful to study from the perspectives of health care providers, researchers and patients. For researchers who seek to include patients in the development of a COS, there is little guidance about how to do this well. We recently developed COS for two rare diseases in children, medium-chain acyl-CoA dehydrogenase deficiency and phenylketonuria. We did this by reviewing available information from published research, surveying health care providers, researchers, and patients, and eventually coming to agreement during a workshop. We included two adult patient partner co-researchers who helped design the COS study and co-developed the patient engagement strategy. These partners formed relationships with seven adult family advisors, who helped ensure that materials were accessible, participated in outcome selection, and helped select tools to measure core outcomes. Strategies we used to engage patient partners included a) training about both the scientific research process and how to help other researchers in the future, and b) frequent communication about study progress and how family advisor feedback was used. Also, we made sure that the impacts of power imbalances between health care providers, researchers and patients were low. Our approach to patient engagement in COS development for two rare conditions in children proved to be both feasible and considered valuable by all study team members, including patient partners and family advisors. To include patient perspectives and values, future COS developers may take a similar approach. Supplementary Information The online version contains supplementary material available at 10.1186/s40900-021-00304-y.

OBJECTIVES: To describe prevalence of multiple primary outcomes, changes in primary outcomes and target sample sizes between protocols and final reports, and how issues of multiplicity are addressed in pragmatic trials. STUDY DESIGN AND SETTING: Individually randomized trials labeled as pragmatic, published 2014–2019 in MEDLINE and registered with ClinicalTrials.gov. RESULTS: We identified 262 final reports and located protocols for 159 (61%); primary outcomes were clearly reported in 145 (91%) protocols and 256 (98%) final reports. Thirty (19%) protocols and 38 (15%) final reports had multiple primary outcomes. Primary outcomes were present and identical in 128 (81%) matched protocol-final reports. Among 140 pairs with target sample sizes reported, 28 (20.0%) reduced their target sample size (mean 543 fewer participants per trial) and 16 (11.4%) increased it (mean 192 more participants per trial). Thirteen (29.5%) provided an explanation. Only 2 of 30 (7%) protocols and 4 of 38 (11%) final reports with co-primary outcomes explained how results would be interpreted in light of multiplicity; 21 of 30 (70%) protocols and 20 of 38 (53%) final reports accounted for co-primary outcomes in power calculations. CONCLUSION: Co-primary outcomes are common in pragmatic trials; improved transparency around design and analysis decisions involving co-primary outcomes is required.

In their article “Misrepresenting ‘Usual Care’ in Research: An Ethical and Scientific Error” Macklin and Natanson (2020) use three exemplar randomized controlled trials (RCTs) to argue that—far fro...

Objective: This study describes the first use of a robotic walker in youth and young adults with cerebral palsy (CP) Gross Motor Function Classification (GMFCS) IV.Methods: Semi-structured intervie...

Most newborns undergo newborn screening blood tests. Breastfeeding, skin-to-skin care, and sweet solutions effectively reduce pain; however, these strategies are inconsistently used. We conducted a 2-armed pilot randomized controlled trial in a mother-baby unit to examine the feasibility and acceptability of a parent-targeted and -mediated video demonstrating use of these pain-reducing strategies and to obtain preliminary effectiveness data on uptake of pain management. One hundred parent-newborn dyads were randomized to view the video or receive usual care (51 intervention and 49 control arm). Consent and attrition rates were 70% and 1%, respectively. All participants in the intervention arm received the intervention as planned and reported an intention to recommend the video and to use at least 1 pain treatment with breastfeeding or skin-to-skin care preferred over sucrose. In the intervention arm, 60% of newborns received at least 1 pain treatment compared with 67% in the control arm (absolute difference, -7%; 95% confidence interval, -26 to 12). The video was well accepted and feasible to show to parents. As there was no evidence of effect on the use of pain management, major modifications are required before launching a full-scale trial. Effective means to translate evidence-based pain knowledge is warranted.

There are several reasons to believe that Institutional Review Boards (IRBs) and Human Research Protection Programs (HRPPs) contribute to ethical research and the protection of research participants, but there are also important reasons to interrogate this belief. Determining whether IRBs and HRPPs "work" requires empirical evaluation of whether and how well they actually achieve what they were designed to do. In other words, it is critical to examine their outcomes and not only their procedures and structures. In this response to Tsan, we argue that the concept of IRB and HRPP quality entails three dimensions: (1) effectiveness, (2) procedures and structures likely to promote effectiveness, and (3) features unrelated to effectiveness but nonetheless essential, such as efficiency, fairness, and proportionality. Because not all types of quality necessarily guarantee or entail effectiveness, we suggest that broad quality assessments, including such features as regulatory compliance and other procedural measures suggested by Tsan, are unhelpful as the first step in evaluating IRBs and HRPPs. Instead, we must start with outcomes relevant to effectiveness. To do this, we launched the Consortium to Advance Effective Research Ethics Oversight (AEREO), with a mission to define and specify ways to measure relevant outcomes for research ethics oversight, empirically evaluate whether those outcomes are achieved, test new approaches to achieving them, and ultimately, develop and implement empirically-based policy and practice to advance IRB and HRPP effectiveness. We describe several anticipated AEREO projects and call for collaboration between various stakeholders to more meaningfully evaluate IRB and HRPPs.

Objective We established a large database of trials to serve as a resource for future methodological and ethical analyses. Here, we use meta-data to describe the broad landscape of pragmatic trials including research areas, identification as pragmatic, quality of trial registry data and enrolment. Study Design and Setting Trials were identified by a validated search filter and included if a primary report of a health-related randomized trial published January 2014-April 2019. Data were collated from MEDLINE, Web of Science, ClinicalTrials.gov, and full text. Results 4337 eligible trials were identified from 13,065 records, of which 1988 were registered in ClinicalTrials.gov. Research areas were diverse, with the most common being general and internal medicine; public, environmental and occupational health; and health care sciences and services. The term “pragmatic” was seldom used in titles or abstracts. Several domains in ClinicalTrials.gov had questionable data quality. We estimated that one-fifth of trials under-accrued by at least 15%. Conclusion There is a need to improve reporting of pragmatic trials and quality of trial registry data. Under accrual remains a challenge in pragmatic RCTs despite calls for more streamlined recruitment approaches. The diversity of pragmatic trials should be reflected in future ethical analyses.

BACKGROUND Evidence to guide treatment of pediatric medium-chain acyl-coenzyme A dehydrogenase (MCAD) deficiency and phenylketonuria (PKU) is fragmented because of large variability in outcome selection and measurement. Our goal was to develop core outcome sets (COSs) for these diseases to facilitate meaningful future evidence generation and enhance the capacity to compare and synthesize findings across studies. METHODS Parents and/or caregivers, health professionals, and health policy advisors completed a Delphi survey and participated in a consensus workshop to select core outcomes from candidate lists of outcomes for MCAD deficiency and PKU. Delphi participants rated the importance of outcomes on a nine-point scale (1–3: not important, 4–6: important but not critical, 7–9: critical). Candidate outcomes were progressively narrowed down over 3 survey rounds. At the workshop, participants evaluated the remaining candidate outcomes using an adapted nominal technique, open discussion, and voting. After the workshop, we finalized the COSs and recommended measurement instruments for each outcome. RESULTS There were 85, 61, and 53 participants across 3 Delphi rounds, respectively. The candidate core outcome lists were narrowed down to 20 outcomes per disease to be discussed at the consensus workshop. Voting by 18 workshop participants led to COSs composed of 8 and 9 outcomes for MCAD deficiency and PKU, respectively, with measurement recommendations. CONCLUSIONS These are the first known pediatric COSs for MCAD deficiency and PKU. Adoption in future studies will help to ensure best use of limited research resources to ultimately improve care for children with these rare diseases.

Background Mental health (MH) problems are among the most important causes of morbidity and mortality for children and youth. Problems of lack of equity in child and youth MH services (CYMHS)—including, but not limited to, problems in inaccessibility and quality of services—are widespread. Characterizing the nature of equity in CYMHS is an ongoing challenge because the field lacks a consistent approach to conceptualizing equity. We will conduct a scoping review of how equity in MH services for children and youth has been defined, operationalized, and measured. Our objectives are to discover: (1) What conceptual definitions of equity are used by observational studies of CYMHS?; (2) What service characteristics of CYMHS care do indices of equity cover?; (3) What population dimensions have been used to operationalize equity?; (4) What statistical constructs have been used in indices that measure CYMHS equity?; and (5) What were the numerical values of those indices? Methods The following databases will be searched: Medline, Embase, PsycINFO, Cochrane Controlled Register of Trials, CINAHL, EconLit, and Sociological Abstracts. Searches will be conducted from the date of inception to the end of the last full calendar year (December 2019). Studies will be included if they include an evaluation of a mental health service for children or youth (defined as those under 19 years of age) and which quantify variation in some aspect of child or youth mental health services (e.g., accessibility, volume, duration, or quality) as a function of socio-demographic and/or geographic variables. Study selection will occur over two stages. Stage one will select articles based on title and abstract using the liberal-accelerated method. Stage two will review the full texts of selected titles. Two reviewers will work independently on full-text reviewing, with each study screened twice using pre-specified eligibility criteria. One reviewer will chart study characteristics and indices to be verified by a second reviewer. Reviewers will resolve full-text screening and data extraction disagreements through discussion. Synthesis of the collected data will focus on compiling and mapping the types and characteristics of the indices used to evaluate MH services equity. Discussion The planned, systematic scoping review will survey the literature regarding how equity in MH services for children and youth has been operationalized and help inform future studies of equity in CYMHS. Systematic review registration Open Science Foundation ID SYSR-D-19-00371, https://osf.io/58srv/.

Pragmatic cluster randomized trials (CRTs) offer an opportunity to improve health care by answering important questions about the comparative effectiveness of treatments using a trial design that can be embedded in routine care. There is a lack of empirical research that addresses ethical issues generated by pragmatic CRTs in hemodialysis.To identify stakeholder perceptions of ethical issues in pragmatic CRTs conducted in hemodialysis.Qualitative study using semi-structured interviews.In-person or telephone interviews with an international group of stakeholders.Stakeholders (clinical investigators, methodologists, ethicists and research ethics committee members, and other knowledge users) who had been involved in the design or conduct of a pragmatic individual patient or cluster randomized trial in hemodialysis, or their role would require them to review and evaluate pragmatic CRTs in hemodialysis.Interviews were conducted in-person or over the telephone and were audio-recorded with consent. Recorded interviews were transcribed verbatim prior to analysis. Transcripts and field notes were analyzed using a thematic analysis approach.Sixteen interviews were conducted with 19 individuals. Interviewees were largely drawn from North America (84%) and were predominantly clinical investigators (42%). Six themes were identified in which pragmatic CRTs in hemodialysis raise ethical issues: (1) patients treated with hemodialysis as a vulnerable population, (2) appropriate approaches to informed consent, (3) research burdens, (4) roles and responsibilities of gatekeepers, (5) inequities in access to research, and (6) advocacy for patient-centered research and outcomes.Participants were largely from North America and did not include research staff, who may have differing perspectives.The six themes reflect concerns relating to individual rights, but also the need to consider population-level issues. To date, concerns regarding inequity of access to research and the need for patient-centered research have received less coverage than other, well-known, issues such as consent. Pragmatic CRTs offer a potential approach to address equity concerns and we suggest future ethical analyses and guidance for pragmatic CRTs in hemodialysis embed equity considerations within them. We further note the potential for the co-creation of health data infrastructure with patients which would aid care but also facilitate patient-centered research. These present results will inform planned future guidance in relation to the ethical design and conduct of pragmatic CRTs in hemodialysis.Registration is not applicable as this is a qualitative study.Les essais pragmatiques randomisés par grappes fournissent une occasion d’améliorer les soins parce qu’ils répondent à des questions importantes sur l’efficacité comparative des traitements en utilisant des modèles pouvant être intégrés aux soins courants. On constate toutefois un manque de recherche empirique abordant les questions éthiques générées par ces essais en contexte d’hémodialyse.Connaître le point de vue d’intervenants sur les questions éthiques liées aux essais pragmatiques randomisés par grappes en contexte d’hémodialyse.Étude qualitative sous forme d’interviews semi-structurées.Interviews téléphoniques ou en personne avec des intervenants internationaux.Des intervenants (chercheurs cliniciens, spécialistes de la méthodologie, éthiciens, membres de comités d’éthique de la recherche et autres utilisateurs de connaissances) impliqués dans la conception ou la conduite d’essais pragmatiques randomisés menés sur un patient individuel, ou un groupe de patients, en contexte d’hémodialyse; ou des individus dont le rôle pourrait les amener à réviser et à évaluer ce type d’essais cliniques.Les interviews ont été menées en personne ou au téléphone, et ont été enregistrées avec le consentement des intervenants. Les enregistrements ont été transcrits verbatim pour l’analyse. Les transcriptions et les notes ont été analysées par une approche d’analyse thématique.Seize interviews ont été menées auprès de 19 intervenants, principalement des chercheurs cliniciens (42%) provenant en grande majorité d’Amérique du Nord (84 %). Ces discussions ont dégagé six thèmes pour lesquels les essais pragmatiques randomisés par grappes soulèvent des questions éthiques en contexte d’hémodialyse: 1) les patients hémodialysés en tant que population vulnérable; 2) les approches appropriées en matière de consentement éclairé; 3) la charge de la recherche; 4) les rôles et responsabilités des personnes responsables; 5) les inégalités dans l’accès à la recherche, et; 6) la promotion de la recherche et des résultats axés sur les patients.Les participants provenaient très majoritairement d’Amérique du Nord et aucun membre du personnel de recherche n’a été questionné, ceux-ci auraient pu fournir un point de vue différent.Les six thèmes rendent compte de préoccupations relatives aux droits individuels, mais indiquent également la nécessité de se pencher sur les enjeux relatifs à la population. À ce jour, les questions concernant l’inégalité dans l’accès à la recherche et la nécessité de faire de la recherche axée sur les patients ont reçu moins d’attention que d’autres enjeux notoires comme le consentement. Les essais pragmatiques randomisés par grappes constituent une approche susceptible d’aborder les questions d’équité; nous suggérons que les futures analyses et orientations éthiques intègrent des considérations d’équité à ce type d’essais en contexte d’hémodialyse. Nous notons également un potentiel pour la co-création d’une infrastructure de données sur la santé avec les patients, ce qui améliorerait les soins tout en facilitant la recherche axée sur les patients. Ces résultats éclaireront les orientations futures pour la conception et la conduite éthique d’essais pragmatiques randomisés par grappes menés en contexte d’hémodialyse.L’enregistrement n’est pas nécessaire puisqu’il s’agit d’une étude qualitative.

Background Inherited metabolic diseases (IMDs) are a group of individually rare single-gene diseases. For many IMDs, there is a paucity of high-quality evidence that evaluates the effectiveness of clinical interventions. Clinical effectiveness trials of IMD interventions could be supported through the development of core outcome sets (COSs), a recommended minimum set of standardized, high-quality outcomes and associated outcome measurement instruments to be incorporated by all trials in an area of study. We began the process of establishing pediatric COSs for two IMDs, medium-chain acyl-CoA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU), by reviewing published literature to describe outcomes reported by authors, identify heterogeneity in outcomes across studies, and assemble a candidate list of outcomes. Methods We used a comprehensive search strategy to identify primary studies and guidelines relevant to children with MCAD deficiency and PKU, extracting study characteristics and outcome information from eligible studies including outcome measurement instruments for select outcomes. Informed by an established framework and a previously published pediatric COS, outcomes were grouped into five, mutually-exclusive, a priori core areas: growth and development, life impact, pathophysiological manifestations, resource use, and death. Results For MCAD deficiency, we identified 83 outcomes from 52 articles. The most frequently represented core area was pathophysiological manifestations, with 33 outcomes reported in 29/52 articles (56%). Death was the most frequently reported outcome. One-third of outcomes were reported by a single study. The most diversely measured outcome was cognition and intelligence/IQ for which eight unique measurement instruments were reported among 14 articles. For PKU, we identified 97 outcomes from 343 articles. The most frequently represented core area was pathophysiological manifestations with 31 outcomes reported in 281/343 articles (82%). Phenylalanine concentration was the most frequently reported outcome. Sixteen percent of outcomes were reported by a single study. Similar to MCAD deficiency, the most diversely measured PKU outcome was cognition and intelligence/IQ with 39 different instruments reported among 82 articles. Conclusions Heterogeneity of reported outcomes and outcome measurement instruments across published studies for both MCAD deficiency and PKU highlights the need for COSs for these diseases, to promote the use of meaningful outcomes and facilitate comparisons across studies.

Objective It is now more than 50 years since the concepts of explanatory and pragmatic attitudes toward trials were first discussed by Schwartz and Lellouch in their influential 1967 paper. Since then, there has been increasing focus on design aspects that may be consistent with more pragmatic attitudes within clinical trials, and a number of tools developed to assist investigators prospectively think about their trial design. Researchers have subsequently expressed interest in using these tools retrospectively to characterize trials as pragmatic or explanatory. Results We suggest that recent attempts to retrospectively dichotomize trials solely on the basis of quantitative scoring of trial design features are flawed. Instead, we argue that there is a need to consider both the intent and design when assessing the degree of pragmatism within a trial. Conclusion The practical implication of our suggestion for trial reporting is that investigators should explicitly state the intent of the trial through a clear articulation of the decision that they hope will be informed by the trial results. This should be coupled with a completed PRagmatic-Explanatory Continuum Indicator Summary 2 assessment (or similar) with an explanation of study design choices to appropriately assess whether the study design is consistent with the study intent. We believe this will assist reviewers and knowledge users in making assessments of trials.

International audience; BACKGROUND: There is a widely recognized need for more pragmatic trials that evaluate interventions in real-world settings to inform decision-making by patients, providers, and health system leaders. Increasing availability of electronic health records, centralized research ethics review, and novel trial designs, combined with support and resources from governments worldwide for patient-centered research, have created an unprecedented opportunity to advance the conduct of pragmatic trials, which can ultimately improve patient health and health system outcomes. Such trials raise ethical issues that have not yet been fully addressed, with existing literature concentrating on regulations in specific jurisdictions rather than arguments grounded in ethical principles. Proposed solutions (e.g. using different regulations in "learning healthcare systems") are speculative with no guarantee of improvement over existing oversight procedures. Most importantly, the literature does not reflect a broad vision of protecting the core liberty and welfare interests of research participants. Novel ethical guidance is required. We have assembled a team of ethicists, trialists, methodologists, social scientists, knowledge users, and community members with the goal of developing guidance for the ethical design and conduct of pragmatic trials. METHODS: Our project will combine empirical and conceptual work and a consensus development process. Empirical work will: (1) identify a comprehensive list of ethical issues through interviews with a small group of key informants (e.g. trialists, ethicists, chairs of research ethics committees); (2) document current practices by reviewing a random sample of pragmatic trials and surveying authors; (3) elicit views of chairs of research ethics committees through surveys in Canada, UK, USA, France, and Australia; and (4) elicit views and experiences of community members and health system leaders through focus groups and surveys. Conceptual work will consist of an ethical analysis of identified issues and the development of new ethical solutions, outlining principles, policy options, and rationales. The consensus development process will involve an independent expert panel to develop a final guidance document. DISCUSSION: Planned output includes manuscripts, educational materials, and tailored guidance documents to inform and support researchers, research ethics committees, journal editors, regulators, and funders in the ethical design and conduct of pragmatic trials.

Background The transfer of information in a medical summary letter is a key aspect of the transition of adolescent patients with IBD from pediatric to adult care. The existing template from the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) is nearly 20 years old, and the many intervening changes to the care of children with IBD necessitate a new template to reflect modern care. Aims We developed a standardized medical summary template based on the feedback of adult and pediatric IBD health care providers. Methods We purposively sampled gastroenterologists (GIs) or nurse practitioners (NPs) caring for patients with IBD in Montreal, Ottawa, Toronto or Vancouver and invited them to take part in focus group discussions. Gastroenterologists caring for Results Content areas of importance (see Table) were: patient/disease characteristics, therapeutics history, clinical history and current status, noteworthy investigations, history of complications, family history, immunization history, and psychosocial history. In addition, the template addressed health system process factors (i.e., urgency of transfer, mode of delivery, confidentiality) to ensure a seamless transfer to adult care. Conclusions The standardized medical summary template should be used by pediatric providers to ensure that essential information is sent to the receiving adult provider. Funding Agencies Transition des patients avec Crohn et colite (TRACC) program

To assess the reach, acceptability, and effect of the BSweet2Babies video showing breast-feeding, skin-to-skin care, and sucrose during blood sampling on intention to recommend the video or advocate for use of the interventions. In July 2014, the video and an electronic survey were produced and posted. After 1 year, the online viewer survey responses and YouTube analytics were analyzed. One year after posting, the BSweet2Babies video had 10 879 views from 125 countries and 187 (1.7%) viewers completed the survey. Most respondents were aware of the analgesic effects of breast-feeding, skin-to-skin care, and sucrose. Nearly all respondents (n = 158, 92%) found the BSweet2Babies video to be a helpful resource and 146 (84%) answered that they would recommend the video to others. After viewing the video, 183 (98%) respondents answered that they would advocate for 1 or more of the interventions. The BSweet2Babies video showing effective pain treatment during blood sampling had a large reach but a very small response rate for the survey. Therefore, analysis of acceptability and effect on intention to recommend the video and advocate for the interventions depicted are limited. Further research is warranted to explore how to best evaluate videos delivered through social media and to determine the effect of the video to promote knowledge translation into clinical practice.

Proposed changes to the Common Rule are proffered to save almost 7,000 reviews annually and consequently vast amounts of investigator and IRB-member time. However, the proposed changes have been subject to criticism. While some have lauded the changes as being imperfect, but nevertheless as improvements, others have contended that ‘neither the scientific community nor the public can be confident that improved practices will emerge from the regulatory changes mandated by the NPRM.’ In the present article, I discuss an important aspect that has been overlooked: the question of whether benefits will emerge is demonstrably empirical, yet data upon which to draw conclusions are conspicuous by their absence. This is thrown into sharp relief when we consider the current environment in which health research is increasingly focused on providing evidence of need or benefit, where there is greater emphasis on evidence-based practice, and when we have the nascent field of implementation science.

KEY POINTS Obtaining research ethics approval is an important component of research conducted in humans. Increasingly, researchers undertake multicentre studies, which can require them to seek approvals from a number of research ethics boards (REBs). Many researchers have complained about

Objectives Identifying pragmatic trials from among all randomized trials is challenging because of inconsistent reporting. Our objective was to develop and validate a search filter to identify reports of pragmatic trials from Ovid MEDLINE. Study Design and Setting Two sets of known and probable pragmatic trial records were analyzed using text mining to generate candidate terms. Two large population sets comprising clinical trials and explanatory trials were used to select discriminating terms. Various combinations of terms were tested iteratively to achieve optimal search performance. Two externally derived sets were used to validate sensitivity and specificity of the derived filters. Results Our validated sensitivity-maximizing filter (combines trial design terms with terms relating to attributes of pragmatic trials) retrieves over 42,000 records in MEDLINE and has sensitivity of 46.4% (95% confidence interval (CI) 37.2 to 55.7%) and estimated specificity of 98.1% (95% CI 93.4 to 99.8%). Search performance is superior to other ad hoc filters for pragmatic trials. The Cochrane search for randomized trials has much better sensitivity (98.2%), but poorer specificity (1.9%) and retrieves 4.5 million records. Conclusion A highly specific filter (low false positive rate) with moderate sensitivity is available for identifying reports of trials more likely to be pragmatic.