Use of electronic healthcare records in large‐scale simple randomized trials at the point of care for the documentation of value‐based medicine

Staa, Tjeerd van; Klungel, Olaf H.; Smeeth, Liam

doi:10.1111/joim.12211

Cited by 20 publications

(18 citation statements)

References 25 publications

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“…There is a vital need for trial designs that promote increased external validity and cost efficiency so we can continue to deliver medical benefits to patients [ 2 ]. To this end, there have been frequent calls for more pragmatic generalizable trials [ 3 , 4 ]. A simplification of trial eligibility criteria and integration of routine electronic health records to monitor trial outcomes should play a key role in the development of pragmatic trials [ 1 , 3 ].…”

Section: Introductionmentioning

confidence: 99%

Evaluation of biases present in the cohort multiple randomised controlled trial design: a simulation study

Candlish

Pate

Sperrin

et al. 2017

BMC Med Res Methodol

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BackgroundThe cohort multiple randomised controlled trial (cmRCT) design provides an opportunity to incorporate the benefits of randomisation within clinical practice; thus reducing costs, integrating electronic healthcare records, and improving external validity. This study aims to address a key concern of the cmRCT design: refusal to treatment is only present in the intervention arm, and this may lead to bias and reduce statistical power.MethodsWe used simulation studies to assess the effect of this refusal, both random and related to event risk, on bias of the effect estimator and statistical power. A series of simulations were undertaken that represent a cmRCT trial with time-to-event endpoint. Intention-to-treat (ITT), per protocol (PP), and instrumental variable (IV) analysis methods, two stage predictor substitution and two stage residual inclusion, were compared for various refusal scenarios.ResultsWe found the IV methods provide a less biased estimator for the causal effect when refusal is present in the intervention arm, with the two stage residual inclusion method performing best with regards to minimum bias and sufficient power. We demonstrate that sample sizes should be adapted based on expected and actual refusal rates in order to be sufficiently powered for IV analysis.ConclusionWe recommend running both an IV and ITT analyses in an individually randomised cmRCT as it is expected that the effect size of interest, or the effect we would observe in clinical practice, would lie somewhere between that estimated with ITT and IV analyses. The optimum (in terms of bias and power) instrumental variable method was the two stage residual inclusion method. We recommend using adaptive power calculations, updating them as refusal rates are collected in the trial recruitment phase in order to be sufficiently powered for IV analysis.

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Section: Introductionmentioning

confidence: 99%

Evaluation of biases present in the cohort multiple randomised controlled trial design: a simulation study

Candlish

Pate

Sperrin

et al. 2017

BMC Med Res Methodol

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“…As these datasets develop, their strengths in size and in reflecting actual clinical practice can be maintained by conducting a randomised trial prospectively through the CPRD general practices [ 56 ]. For instance, it is now possible to alert CPRD-linked general practitioners through their electronic records system when they consult patients potentially eligible for a trial [ 56 , 57 ].…”

Section: Discussionmentioning

confidence: 99%

Calculating Total Health Service Utilisation and Costs from Routinely Collected Electronic Health Records Using the Example of Patients with Irritable Bowel Syndrome Before and After Their First Gastroenterology Appointment

2015

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IntroductionHealth economic models are increasingly important in funding decisions but most are based on data, which may therefore not represent the general population. We sought to establish the potential of real-world data available within the Clinical Practice Research Datalink (CPRD) and linked Hospital Episode Statistics (HES) to determine comprehensive healthcare utilisation and costs as input variables for economic modelling.MethodsA cohort of patients with irritable bowel syndrome (IBS) who first saw a gastroenterologist in 2008 or 2009, and with 3 years of data before and after their appointment, was created in the CPRD. Primary care, outpatient, inpatient, prescription and colonoscopy data were extracted from the linked CPRD and HES. The appropriate cost to the NHS was attached to each event. Total and stratified annual healthcare utilisation rates and costs were calculated before and after the gastroenterology appointment with distribution parameters. Absolute differences were calculated with 95 % confidence intervals.ResultsTotal annual healthcare costs over 3 years increase by £935 (95 % CI £928–941) following a gastroenterology appointment for IBS. We derived utilisation and cost data with parameter distributions stratified by demographics and time. Women, older patients, smokers and patients with greater comorbidity utilised more healthcare resources, which generated higher costs.ConclusionsThese linked datasets provide comprehensive primary and secondary care data for large numbers of patients, which allows stratification of outcomes. It is possible to derive input parameters appropriate for economic models and their distributions directly from the population of interest.

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“…16 Until then, PCTs have their main role when there is insufficient knowledge on comparative effectiveness and effectiveness in the real world clinical setting with heterogeneous populations. 17 Our interviews highlighted 3 major considerations that must be addressed for embedded PCTs to become more widely used:…”

Section: Conclusion and Recommendationsmentioning

confidence: 99%

Opportunities and barriers for pragmatic embedded trials: Triumphs and tribulations

Ramsberg

Platt

2017

Learning Health Systems

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Results: Embedded pragmatic clinical trials (PCTs) are set in routine health care, have broad eligibility criteria, and use routinely collected electronic data. Many consider them a breakthrough innovation in clinical research and a necessary step in clinical trial development. To identify barriers and success factors, we reviewed published embedded PCTs and interviewed 30 researchers and clinical leaders in 7 US delivery systems.Literature: We searched PubMed, the Cochrane library, and clinicaltrials.gov for studies reporting embedded PCTs. We identified 108 embedded PCTs published in the last 10 years.The included studies had a median of 5540 randomized patients, addressed a variety of diseases, and practice settings covering a broad range of interventions. Eighty-one used cluster randomization. The median cost per patient was $97 in the 64 trials for which it was possible to obtain cost data.Interviews: Delivery systems required research studies to align with operational priorities, existing information technology capabilities, and standard quality improvement procedures.Barriers that were identified included research governance, requirements for processes that were incompatible with clinical operations, and unrecoverable costs.Conclusions: Embedding PCTs in delivery systems can provide generalizable knowledge that is directly applicable to practice settings at much lower cost than conventional trials. Successful embedding trials require accommodating delivery systems' needs and priorities. | INTRODUCTIONIn a learning health system, evidence is both generated and applied as a natural product of the care process.1 An essential element of a learning health system is to collect and analyze data to generate knowledge. 2 Many study designs exist to achieve this. While there is no single best approach, the randomized controlled trial (RCT) is the gold standard in knowledge generation. -------------------------This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

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Use of electronic healthcare records in large‐scale simple randomized trials at the point of care for the documentation of value‐based medicine

Cited by 20 publications

References 25 publications

Evaluation of biases present in the cohort multiple randomised controlled trial design: a simulation study

Evaluation of biases present in the cohort multiple randomised controlled trial design: a simulation study

Calculating Total Health Service Utilisation and Costs from Routinely Collected Electronic Health Records Using the Example of Patients with Irritable Bowel Syndrome Before and After Their First Gastroenterology Appointment

Opportunities and barriers for pragmatic embedded trials: Triumphs and tribulations

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