scholarly journals Use of routinely collected data in a UK cohort of publicly funded randomised clinical trials

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 323
Author(s):  
Andrew J. McKay ◽  
Ashley P. Jones ◽  
Carrol L. Gamble ◽  
Andrew J. Farmer ◽  
Paula R. Williamson
Keyword(s):  
Clinical Trials ◽  
Data Extraction ◽  
Sole Source ◽  
Outcome Data ◽  
Data Sources ◽  
Publicly Funded ◽  
Routinely Collected Data ◽  
Routinely Collected Health Data ◽  
Service Resource ◽  
Randomised Controlled

Routinely collected data about health in medical records, registries and hospital activity statistics is now routinely collected in an electronic form. The extent to which such sources of data are now being routinely accessed to deliver efficient clinical trials, is unclear. The aim of this study was to ascertain current practice amongst a United Kingdom (UK) cohort of recently funded and ongoing randomised controlled trials (RCTs) in relation to sources and use of routinely collected outcome data. Recently funded and ongoing RCTs were identified for inclusion by searching the National Institute for Health Research journals library. Trials that have a protocol available were assessed for inclusion and those that use or plan to use routinely collected health data for at least one outcome were included. Routinely collected data sources and outcome information were extracted. A total of 279 studies were identified with 102 eligible for data extraction. An Electronic Health Record (EHR) was the sole source of outcome data for at least one outcome in 46 trials. The most frequent sources are Hospital Episode Statistics (HES) and Office for National Statistics (ONS), with the most common outcome data to be extracted being on mortality, hospital admission, and health service resource use. Our study has found that around half of publicly funded trials in a UK cohort plan to collect outcome data from routinely collected data sources. This is much higher than the figure of 8% found in a cohort of 189 RCTs published since 2000, the majority of were carried out in North America (McCord et al., 2019).

scholarly journals Use of routinely collected data in a UK cohort of publicly funded randomised clinical trials

F1000Research ◽  
2021 ◽  
Vol 9 ◽  
pp. 323
Author(s):  
Andrew J. McKay ◽  
Ashley P. Jones ◽  
Carrol L. Gamble ◽  
Andrew J. Farmer ◽  
Paula R. Williamson
Keyword(s):  
Clinical Trials ◽  
Sole Source ◽  
Outcome Data ◽  
Publicly Funded ◽  
Routinely Collected Data ◽  
Routinely Collected Health Data ◽  
Outcome Information ◽  
Service Resource ◽  
Randomised Controlled ◽  
Health Service Resource

Routinely collected data about health in medical records, registries and hospital activity statistics is now routinely collected in an electronic form. The extent to which such sources of data are now being routinely accessed to deliver efficient clinical trials, is unclear. The aim of this study was to ascertain current practice amongst a United Kingdom (UK) cohort of recently funded and ongoing randomised controlled trials (RCTs) in relation to sources and use of routinely collected outcome data. Recently funded and ongoing RCTs were identified for inclusion by searching the National Institute for Health Research journals library. Trials that have a protocol available were assessed for inclusion and those that use or plan to use routinely collected health data (RCHD) for at least one outcome were included. RCHD sources and outcome information were extracted. Of 216 RCTs, 102 (47%) planned to use RCHD. A RCHD source was the sole source of outcome data for at least one outcome in 46 (45%) of those 102 trials. The most frequent sources are Hospital Episode Statistics (HES) and Office for National Statistics (ONS), with the most common outcome data to be extracted being on mortality, hospital admission, and health service resource use. Our study has found that around half of publicly funded trials in a UK cohort (NIHR HTA funded trials that had a protocol available) plan to collect outcome data from routinely collected data sources.

scholarly journals Use of routinely collected data in a UK cohort of publicly funded randomised clinical trials

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 323
Author(s):  
Andrew J. McKay ◽  
Ashley P. Jones ◽  
Carrol L. Gamble ◽  
Andrew J. Farmer ◽  
Paula R. Williamson
Keyword(s):  
Clinical Trials ◽  
Sole Source ◽  
Outcome Data ◽  
Publicly Funded ◽  
Routinely Collected Data ◽  
Routinely Collected Health Data ◽  
Outcome Information ◽  
Service Resource ◽  
Randomised Controlled ◽  
Health Service Resource

Routinely collected data about health in medical records, registries and hospital activity statistics is now routinely collected in an electronic form. The extent to which such sources of data are now being routinely accessed to deliver efficient clinical trials, is unclear. The aim of this study was to ascertain current practice amongst a United Kingdom (UK) cohort of recently funded and ongoing randomised controlled trials (RCTs) in relation to sources and use of routinely collected outcome data. Recently funded and ongoing RCTs were identified for inclusion by searching the National Institute for Health Research journals library. Trials that have a protocol available were assessed for inclusion and those that use or plan to use routinely collected health data (RCHD) for at least one outcome were included. RCHD sources and outcome information were extracted. Of 216 RCTs, 102 (47%) planned to use RCHD. A RCHD source was the sole source of outcome data for at least one outcome in 46 (45%) of those 102 trials. The most frequent sources are Hospital Episode Statistics (HES) and Office for National Statistics (ONS), with the most common outcome data to be extracted being on mortality, hospital admission, and health service resource use. Our study has found that around half of publicly funded trials in a UK cohort (NIHR HTA funded trials that had a protocol available) plan to collect outcome data from routinely collected data sources. This is much higher than the figure of 8% found in a cohort of 189 RCTs published since 2000, the majority of which were carried out in North America (McCord et al., 2019).

scholarly journals Access to routinely collected health data for clinical trials – review of successful data requests to UK registries

2020 ◽  
Author(s):  
Sarah Lensen ◽  
Archie Macnair ◽  
Sharon B Love ◽  
Victoria Yorke-Edwards ◽  
Nurulamin M Noor ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Data Extraction ◽  
Health Data ◽  
Lack Of Information ◽  
Routinely Collected Health Data ◽  
Long Term Follow Up ◽  
Randomised Controlled ◽  
The Uk

ABSTRACTBackgroundClinical trials generally each collect their own data despite routinely-collected health data (RCHD) increasing in quality and breadth. Our aim is to quantify UK-based randomised controlled trials (RCTs) accessing RCHD for participant data, characterise how these data are used and thereby recommend how more trials could use RCHD.MethodsWe conducted a systematic review of RCTs accessing RCHD from at least one registry in the UK between 2013-2018, for the purposes of informing or supplementing participant data. A list of all registries holding RCHD in the UK was compiled. In cases where registries published release registers, these were searched for RCTs accessing RCHD. Where no release register was available, registries were contacted to request a list of RCTs. For each identified RCT, information was collected from all publicly available sources (release registers, websites, protocol etc.). The search and data extraction was undertaken between Jan-2019 and May-2019.ResultsWe identified 160 RCTs accessing RCHD between 2013 and 2018 from a total of 22 registries; this corresponds to only a very small proportion of all UK RCTs (approximately 3%). RCTs accessing RCHD were generally large (median sample size 1590), commonly evaluating treatments for cancer or cardiovascular disease. Most of the included RCTs accessed RCHD from NHS Digital (68%), and the most frequently accessed datasets were mortality (76%) and hospital visits (55%). RCHD was used to inform the primary trial (82%) and long-term follow-up (57%). There was substantial variation in how RCTs used RCHD to inform participant outcome measures. A limitation was the lack of information and transparency from registries and RCTs with respect to which datasets have been accessed and for what purposes.ConclusionsIn the last five years, only a small minority of UK-based RCTs have accessed RCHD to inform participant data. We ask for improved accessibility, confirmed data quality and joined up thinking between the registries and the regulatory authorities.RegistrationPROSPERO CRD42019123088

5-Azacytidine and Decitabine Monotherapies of Myelodysplastic Disorders

2005 ◽  
Vol 39 (10) ◽  
pp. 1700-1709 ◽  
Author(s):  
Jim R Kuykendall
Keyword(s):  
Clinical Trials ◽  
Data Extraction ◽  
Supportive Therapy ◽  
Clinical Effectiveness ◽  
Data Sources ◽  
Hypomethylating Agents ◽  
Medline Search ◽  
Study Selection ◽  
Response To Chemotherapy ◽  
Leukemia Treatment

OBJECTIVE: To review and differentiate the pharmacology, toxicology, pharmacokinetics, and results of major clinical trials of 5-azacytidine (5-AzaC) and 5-aza-2'-deoxycytidine (decitabine) therapy of myelodysplastic disorders. DATA SOURCES: A PubMed/MEDLINE search was conducted (1966–October 2004) using the following terms: DNA methylation, myelodysplastic disorders, 5-azacytidine, and 5-aza-2'-deoxycytidine (decitabine). Additional data sources included bibliographies from identified articles and manufacturer information. STUDY SELECTION AND DATA EXTRACTION: Clinical trials for the treatment of various malignancies by hypomethylating agents were selected from data sources. All published, major clinical trials evaluating 5-AzaC or decitabine in myelodysplastic disorders and transformed myeloid leukemia treatment were included. DATA SYNTHESIS: Myelodysplastic disorders are a group of bone marrow stem cell hyperplasias and dysplasias that result in ineffective hematopoiesis. Myelodysplastic disorders and transformed leukemia have poor prognosis and minimal response to chemotherapy. DNA hypomethylating agents have been shown to improve overall response rates (increased neutrophil, leukocyte, and platelet counts), time to leukemic progression, and quality of life compared with supportive therapy. The incidence of the most common adverse effects (nausea, vomiting, myelosuppression) can be reduced by low-dose, continuous, or extended-interval infusion. CONCLUSIONS: Since appropriate dosing schedules of decitabine are being investigated, comparison of the clinical effectiveness of 5-AzaC and decitabine would be premature at this time. DNA hypomethylating agents show promise as monotherapies of myelodysplastic disorders and transformed leukemia and may be useful as a component of combination chemotherapy of various malignancies.

scholarly journals Benchmarks for detecting ‘breakthroughs’ in clinical trials: empirical assessment of the probability of large treatment effects using kernel density estimation

BMJ Open ◽  
2014 ◽  
Vol 4 (10) ◽  
pp. e005249 ◽  
Author(s):  
Branko Miladinovic ◽  
Ambuj Kumar ◽  
Rahul Mhaskar ◽  
Benjamin Djulbegovic
Keyword(s):  
Clinical Trials ◽  
Density Estimation ◽  
Kernel Density Estimation ◽  
Treatment Effects ◽  
Kernel Density ◽  
Data Sources ◽  
Cooperative Groups ◽  
Publicly Funded ◽  
Adaptive Kernel ◽  
Improve Health

ObjectiveTo understand how often ‘breakthroughs,’ that is, treatments that significantly improve health outcomes, can be developed.DesignWe applied weighted adaptive kernel density estimation to construct the probability density function for observed treatment effects from five publicly funded cohorts and one privately funded group.Data Sources820 trials involving 1064 comparisons and enrolling 331 004 patients were conducted by five publicly funded cooperative groups. 40 cancer trials involving 50 comparisons and enrolling a total of 19 889 patients were conducted by GlaxoSmithKline.ResultsWe calculated that the probability of detecting treatment with large effects is 10% (5–25%), and that the probability of detecting treatment with very large treatment effects is 2% (0.3–10%). Researchers themselves judged that they discovered a new, breakthrough intervention in 16% of trials.ConclusionsWe propose these figures as the benchmarks against which future development of ‘breakthrough’ treatments should be measured.

scholarly journals Identifying patient-important outcomes for treatment of bipolar disorder: a systematic review protocol

BMJ Open ◽  
2021 ◽  
Vol 11 (12) ◽  
pp. e050453
Author(s):  
Alessia D'Elia ◽  
Olivia Orsini ◽  
Stephanie Sanger ◽  
Alannah Hillmer ◽  
Nitika Sanger ◽  
...  
Keyword(s):  
Systematic Review ◽  
Bipolar Disorder ◽  
Clinical Trials ◽  
Systematic Reviews ◽  
Treatment Effectiveness ◽  
Data Extraction ◽  
Risk Of Bias ◽  
Observational Research ◽  
Cochrane Library ◽  
Randomised Controlled

IntroductionTreatment of bipolar disorder is the focus of several clinical trials, however the understanding of the outcomes for establishing treatment effectiveness within these trials is limited. Further, there is limited literature which reports on the outcomes considered to be important to patients, indicating that patient perspectives are often not considered when selecting outcomes of effectiveness within trials. This protocol describes a systematic review which aims to describe the outcomes being used within trials to measure treatment effectiveness, commenting on the inclusion of patient-important outcomes within previous trials.Methods and analysisThis protocol is reported using the Preferred Reporting Items for Systematic Reviews and Meta-analyses Protocols statement. OVID MEDLINE, OVID Embase, OVID APA PsycINFO, Web of Science, the Wiley Cochrane Library, ClinicalTrials.gov and the International Clinical Trials Registry Platform databases will be searched for eligible studies. Screening, full-text and data extraction stages will be completed in duplicate using the Covidence platform for systematic reviews. Eligible studies will include clinical trials of interventions in bipolar disorder, in order to identify outcomes used to assess treatment effectiveness, and qualitative studies, to determine which outcomes have been reported as important by patients. Risk of bias for included studies will be assessed using the Cochrane Risk of Bias Tool for randomised controlled trials, and the Newcastle-Ottawa Scale for observational research.Ethics and disseminationThis review will involve dissemination to key stakeholders, including primary end users such as patients, clinicians and trialists. Knowledge translation tools will be generated to share the relevant conclusions of this review. Results will be communicated to the scientific community through peer-reviewed publications, conferences and workshops. No ethics approval will be sought as this study is based on literature.PROSPERO registration numberCRD42021214435.

scholarly journals Analysis and reporting of adverse events in randomised controlled trials: a review

BMJ Open ◽  
2019 ◽  
Vol 9 (2) ◽  
pp. e024537 ◽  
Author(s):  
Rachel Phillips ◽  
Lorna Hazell ◽  
Odile Sauzet ◽  
Victoria Cornelius
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Hypothesis Testing ◽  
Randomised Controlled Trials ◽  
Data Extraction ◽  
Safety Data ◽  
Multiple Hypothesis Testing ◽  
Controlled Trials ◽  
Reporting Practices ◽  
Randomised Controlled

ObjectiveTo ascertain contemporary approaches to the collection, reporting and analysis of adverse events (AEs) in randomised controlled trials (RCTs) with a primary efficacy outcome.DesignA review of clinical trials of drug interventions from four high impact medical journals.Data sourcesElectronic contents table of the BMJ, the Journal of the American Medical Association (JAMA), the Lancet and the New England Journal of Medicine (NEJM) were searched for reports of original RCTs published between September 2015 and September 2016.MethodsA prepiloted checklist was used and single data extraction was performed by three reviewers with independent check of a randomly sampled subset to verify quality. We extracted data on collection methods, assessment of severity and causality, reporting criteria, analysis methods and presentation of AE data.ResultsWe identified 184 eligible reports (BMJ n=3; JAMA n=38, Lancet n=62 and NEJM n=81). Sixty-two per cent reported some form of spontaneous AE collection but only 29% included details of specific prompts used to ascertain AE data. Numbers that withdrew from the trial were well reported (80%), however only 35% of these reported whether withdrawals were due to AEs. Results presented and analysis performed was predominantly on ‘patients with at least one event’ with 84% of studies ignoring repeated events. Despite a lack of power to undertake formal hypothesis testing, 47% performed such tests for binary outcomes.ConclusionsThis review highlighted that the collection, reporting and analysis of AE data in clinical trials is inconsistent and RCTs as a source of safety data are underused. Areas to improve include reducing information loss when analysing at patient level and inappropriate practice of underpowered multiple hypothesis testing. Implementation of standard reporting practices could enable a more accurate synthesis of safety data and development of guidance for statistical methodology to assess causality of AEs could facilitate better statistical practice.

scholarly journals Protocol for a systematic scoping review of reasons given to justify the performance of randomised controlled trials

BMJ Open ◽  
2019 ◽  
Vol 9 (7) ◽  
pp. e027575 ◽  
Author(s):  
Brian Dewar ◽  
Mark Fedyk ◽  
Lucas Jurkovic ◽  
Stephanie Chevrier ◽  
Rosendo Rodriguez ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Randomised Controlled Trials ◽  
Data Extraction ◽  
Grey Literature ◽  
Medical Knowledge ◽  
Controlled Trials ◽  
Trial Methodology ◽  
Clinical Trial Methodology ◽  
Scoping Reviews ◽  
Randomised Controlled

IntroductionRandomised controlled trials (RCTs) are widely viewed to generate the most reliable medical knowledge. However, RCTs are not always scientifically necessary and therefore not always ethical. Unfortunately, it is not clear when an RCT is not necessary or how this should be established. This study seeks to systematically catalogue justifications offered throughout the medical and ethics literature for performing randomisation within clinical trials.Methods and analysisWe will systematically search electronic databases of the medical literature including MEDLINE, EMBASE, Cochrane Database of Systematic Reviews, Cochrane Clinical Trials Register, Web of Science Proceedings, ClinicalTrials.gov; databases of philosophical literature including Philosopher’s Index, Phil Papers, JSTOR, Periodicals Archive Online, Project MUSE, National Reference Centre for Bioethics; the library catalogue at the University of Ottawa; bibliographies of retrieved papers; and the grey literature. We will also pursue suggestions from experts in the fields of medical ethics, philosophy and clinical trial methodology. Article screening, selection and data extraction will be performed by two independent reviewers based on prespecified inclusion/exclusion criteria. A third reviewer will be consulted to resolve any discrepancies. We will then extract the reasons given to justify randomisation using methodology established to extract data in a defensible, systematic manner. We will track the reasons given, their frequency of use and changes over time. Finally, using grounded theory, we will combine the reasons into broader themes. These themes will form the foundation of our subsequent analysis from qualitative and quantitative perspectives. This review will map existing arguments that clinicians, ethicists and philosophers use to ethically justify randomisation in clinical trials.Ethics and disseminationNo research ethics board approval is necessary because we are not examining patient-level data. This protocol complies with the reported guidance for conducting systematic scoping reviews. The findings of this paper will be disseminated via presentations and academic publication. In a subsequent phase of this research, we hope to engage with stakeholders and translate any recommendations derived from our findings into operational guidelines.

scholarly journals Title: The Effectiveness of Participant Blinding of Non-Penetrating Sham/Placebo Acupuncture in Clinical Trials: A Systematic Review with Meta-Analysis

2021 ◽  
Author(s):  
Yee Hung Gan ◽  
Nway Aye Saint ◽  
Yen Suan Sin
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Control Method ◽  
Data Extraction ◽  
Meta Analysis ◽  
Sham Acupuncture ◽  
Placebo Control ◽  
Skin Contact ◽  
Randomised Controlled ◽  
Healthy Participants

Abstract Background: Acupuncture clinical trial is important to evaluate the efficacy of acupuncture. However, it is challenging to achieve effective blinding due to the nature of acupuncture. A standardised placebo control method of acupuncture has yet to be established. The study focuses on the non-penetrating sham acupuncture because it eliminates the placebo effect and generates lesser physiological responses. The study aims to evaluate and compare the participant blinding effectiveness of non-penetrating sham acupuncture devices, and analyse the factors which may influence the participant blinding.Methods: The study followed the PRISMA guidelines. An electronic search was conducted on PubMed, Ovid and CNKI up until 1st of October 2020 to include English and Chinese randomised controlled trials which evaluated the awareness on the type of acupuncture (real or sham) in any population who received acupuncture. Data screening, data extraction and quality assessment were done independently by two researchers and discrepancies were sorted out via discussion with a co-researcher. Data analysis was performed using RevMan 5.4.1.Results: 34 full-text articles had been included in the systematic review and meta-analysis. The quality of the studies ranged from moderate to good. Generally, non-penetrating sham acupuncture devices were effective in blinding participants in clinical trials. The foam device demonstrated a better blinding effect, followed by Streitberger, Park and Takakura devices. Sham needles with no skin contact could not blind the participants successfully. Naive, experienced, healthy and diseased participants all could be blinded using non-penetrating sham acupuncture devices but naive and healthy participants could be blinded comparatively easily. Acupoints from different regions could achieve blinding, however, the acupoints on the back could blind the participants more easily compared to the other areas.Conclusion: Non-penetrating sham acupuncture devices are valid placebo control for acupuncture clinical trials. The foam device has a better blinding effect, followed by Streitberger, Park and Takakura devices. Recruiting naive healthy participants and choosing acupoints from the back can achieve better blinding effects in the participants.

Efficacy, Acceptability, and Tolerability of Lisdexamfetamine, Mixed Amphetamine Salts, Methylphenidate, and Modafinil in the Treatment of Attention-Deficit Hyperactivity Disorder in Adults: A Systematic Review and Meta-analysis

2018 ◽  
Vol 53 (2) ◽  
pp. 121-133 ◽  
Author(s):  
Matej Stuhec ◽  
Petar Lukić ◽  
Igor Locatelli
Keyword(s):  
Attention Deficit Hyperactivity Disorder ◽  
Clinical Trials ◽  
Effect Size ◽  
Attention Deficit ◽  
Data Extraction ◽  
Meta Analysis ◽  
Sole Source ◽  
Adhd Symptoms ◽  
Double Blind ◽  
Hyperactivity Disorder

Objective: Psychostimulants are the first-line treatment in adults with attention-deficit hyperactivity disorder (ADHD). This meta-analysis aimed to evaluate the efficacy, acceptability, and tolerability of lisdexamfetamine (LDX), mixed amphetamine salts (MASs), modafinil (MDF), and methylphenidate (MPH) in comparison with placebo. Data Sources: We systematically searched PubMed/MEDLINE and Clinicaltrials.gov in May 2016, along with CENTRAL and EU Clinical Trials Register in February 2016, for the randomized, double-blind, placebo-controlled, parallel-group clinical trials conducted on adults diagnosed with ADHD. Study Selection and Data Extraction: Substantial comorbidity, substance abuse or dependence, and nonpharmacological interventions represented grounds for exclusion. Published reports were the sole source for data extraction. Improvement in ADHD symptoms was the primary outcome. Random-effects model meta-analysis was applied to calculate the standardized mean difference (SMD) with 95% CIs. Data Synthesis: The search retrieved 701 records, of which 20 studies were eligible for analysis. High effect size (expressed as SMD) in reducing ADHD symptoms was observed for LDX (−0.89; 95% CI = −1.09, −0.70), whereas MASs (−0.64; 95% CI = −0.83, −0.45) and MPH (−0.50; 95% CI = −0.58, −0.41) reduced symptoms moderately compared with placebo. No efficacy was shown for MDF (0.08; 95% CI; −0.18, 0.34). Relevance to Patient Care and Clinical Practice: In this meta-analysis, the efficacy, tolerability, and acceptability of psychostimulants were compared with that for placebo. Five of the included trials have not been evaluated in any of the previously published meta-analyses. Conclusions: The results suggest that LDX has the largest effect size and has a promising potential for treating adults with ADHD.

Sign in / Sign up

Export Citation Format

Share Document