Registry randomised trials: a methodological perspective

Doherty, Dorota; Tong, Steven Y C; Reilly, Jennifer; Shrapnel, Jane; McDonald, S.; Ahern, Susannah; Harris, Ian; Tam, Charmaine S.; Brennan, A.; Hodgson, Carol; Wilcox, Leonie; Balagurunathan, Anitha; Butcher, Belinda; Reid, C.

doi:10.1136/bmjopen-2022-068057

Cited by 17 publications

(9 citation statements)

References 33 publications

(60 reference statements)

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“…One important and pragmatic opportunity arises from the development of large regional, national and international networks and registries for lipodystrophy (e.g. the Europe-based ECLip registry 62 ), allied to emergence of randomised registry-based trial (RRT) methodology 63,64 . RRTs have attracted increasing interest in several disease areas and are particularly suitable for evaluation of agents with well-established safety profiles.…”

Section: Discussionmentioning

confidence: 99%

“…In monogenic IR this would permit questions to be addressed about optimal usage of different "common" medications in different genetic subgroups, including the order of introduction of therapies, and their optimal combinations. The quality of such studies will critically rely on good registry design and quality and completeness of data capture 63,64 .…”

Section: Discussionmentioning

confidence: 99%

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Systematic review of genotype-stratified treatment for monogenic insulin resistance

Semple

Patel

Auh

et al. 2023

Preprint

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ObjectiveTo assess the effects of pharmacologic and/or surgical interventions in monogenic insulin resistance (IR), stratified by genetic aetiology.DesignSystematic review.Data sourcesPubMed, MEDLINE and Embase, from 1 January 1987 to 23 June 2021.Review methodsStudies reporting individual-level effects of pharmacologic and/or surgical interventions in monogenic IR were eligible. Individual subject data were extracted and duplicate data removed. Outcomes were analyzed for each affected gene and intervention, and in aggregate for partial, generalised and all lipodystrophy.Results10 non-randomised experimental studies, 8 case series, and 21 single case reports met inclusion criteria, all rated as having moderate or serious risk of bias. Metreleptin was associated with lower triglycerides and hemoglobin A1c in aggregated lipodystrophy (n=111), in partial lipodystrophy (n=71) and generalised lipodystrophy (n=41)), and inLMNA,PPARG,AGPAT2orBSCL2subgroups (n=72,13,21 and 21 respectively). Body Mass Index (BMI) was lower after treatment in partial and generalised lipodystrophy overall, and inLMNA or BSCL2, but notPPARGorAGPAT2subgroups. Thiazolidinedione use was associated with improved hemoglobin A1c and triglycerides in aggregated lipodystrophy (n=13), improved hemoglobin A1c only in thePPARGsubgroup (n=5), and improved triglycerides only in theLMNAsubgroup (n=7). InINSR-related IR, use of rhIGF-1, alone or with IGFBP3, was associated with improved hemoglobin A1c (n=15). The small size or absence of all other genotype-treatment combinations precluded firm conclusions.ConclusionsThe evidence guiding genotype-specific treatment of monogenic IR is of low to very low quality. Metreleptin and Thiazolidinediones appear to have beneficial metabolic effects in lipodystrophy, and rhIGF-1 appears to lower hemoglobin A1c in INSR-related IR. For other interventions there is insufficient evidence to assess efficacy and risks either in aggregated lipodystrophy or in genetic subgroups. There is a pressing need to improve the evidence base for management of monogenic IR.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Systematic review of genotype-stratified treatment for monogenic insulin resistance

Semple

Patel

Auh

et al. 2023

Preprint

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“…Real-world study (RWS) design and methodology have also been advanced, which facilitates the utilization of RWD. Based on advances in data quality and linkage, registry-based randomized control trials (RCTs), which are defined as pragmatic trials that use registry data for case records, data collection, randomization, and follow-up, are playing an important role in answering clinical questions pragmatically and cost effectively [ 6 , 7 ]. One example is the national-level SWEDEHEART (Swedish Web-system for Enhancement and Development of Evidence-based care in Heart disease Evaluated According to Recommended Therapies) registry database [ 8 ].…”

Section: Introductionmentioning

confidence: 99%

Maximizing the Value of Real-World Data and Real-World Evidence to Accelerate Healthcare Transformation in China: Summary of External Advisory Committee Meetings

Sun,

Bedenkov,

Liu

et al. 2024

Pharm Med

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Use of real-world data (RWD) is gaining wide attention. To bridge the gap between diverse healthcare stakeholders and to leverage the impact of Chinese real-world evidence (RWE) globally, a multi-stakeholder External Advisory Committee (EAC) and EAC meetings were initiated, aiming to elucidate the current and evolving RWD landscape in China, articulate the values of RWE in ensuring Chinese patients’ equitable access to affordable medicines and solutions, and identify strategic opportunities and partnerships for expansion of RWE generation in China. Chinese and international experts who are clinicians and academic researchers were selected as EAC members based on their professional background and familiarity with RWD/RWE. Three EAC meetings were held quarterly in 2023. Various topics were presented and discussed for insights and suggestions. Nine experts from China, one from South Korea, and two from Europe were selected as EAC members and attended these meetings. Experts’ presentations were summarized by theme, including the RWD landscape and RWE enablement in China, as well as global development of a patient-centric ecosystem. Experts’ insights and suggestions on maximizing the RWD/RWE value to accelerate healthcare transformation in China were collected. We concluded that though data access, sharing, and quality are still challenging, RWD is developing to support evidence generation in the medicinal product lifecycle, inform clinical practice, and empower patient management in China. RWD/RWE creates value, accelerates healthcare transformation, and improves patient outcomes. Fostering a patient-centric ecosystem across healthcare stakeholders and maintaining global partnerships and collaboration are essential for unlocking the power of RWD/RWE. Supplementary Information The online version contains supplementary material available at 10.1007/s40290-024-00520-3.

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“…4 The embedding of registry randomised clinical trials (RRCTs) into established registries may be a useful approach in the field of clinical research. 5 In particular, this approach has been suggested as a means to overcome issues with external generalisability associated with the strict inclusion criteria in traditionally-designed randomised clinical trials.…”

mentioning

confidence: 99%

“…4 Intelligent use of electronic medical records to leverage possibilities for efficient collection of data should be considered. 5 The proactive engagement of individual clinicians with registry participation is integral to registry maintenance. Finally, patient education, either on an individual or public level, may improve awareness of registries and improve understanding of their utility and acceptability.…”

mentioning

confidence: 99%