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Pharmacoepidemiology and Drug Safety Oct 2020There is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to... (Review)
Review
PURPOSE
There is a need to develop hybrid trial methodology combining the best parts of traditional randomized controlled trials (RCTs) and observational study designs to produce real-world evidence (RWE) that provides adequate scientific evidence for regulatory decision-making.
METHODS
This review explores how hybrid study designs that include features of RCTs and studies with real-world data (RWD) can combine the advantages of both to generate RWE that is fit for regulatory purposes.
RESULTS
Some hybrid designs include randomization and use pragmatic outcomes; other designs use single-arm trial data supplemented with external comparators derived from RWD or leverage novel data collection approaches to capture long-term outcomes in a real-world setting. Some of these approaches have already been successfully used in regulatory decisions, raising the possibility that studies using RWD could increasingly be used to augment or replace traditional RCTs for the demonstration of drug effectiveness in certain contexts. These changes come against a background of long reliance on RCTs for regulatory decision-making, which are labor-intensive, costly, and produce data that can have limited applicability in real-world clinical practice.
CONCLUSIONS
While RWE from observational studies is well accepted for satisfying postapproval safety monitoring requirements, it has not commonly been used to demonstrate drug effectiveness for regulatory purposes. However, this position is changing as regulatory opinions, guidance frameworks, and RWD methodologies are evolving, with growing recognition of the value of using RWE that is acceptable for regulatory decision-making.
Topics: Decision Making; Drug Approval; Humans; Observational Studies as Topic; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Research Design
PubMed: 31823482
DOI: 10.1002/pds.4932 -
Atencion Primaria May 2022To evaluate the experiencie with a health education program in Primary Care in patients with chronic shoulder pain of musculoskeletal origin, on pain and disability and... (Observational Study)
Observational Study
OBJECTIVE
To evaluate the experiencie with a health education program in Primary Care in patients with chronic shoulder pain of musculoskeletal origin, on pain and disability and establish the protocol in primary care.
DESIGN
Quasi-experimental longitudinal descriptive observational study.
LOCATION
Arroyo de la Vega Health Center, Alcobendas, Madrid.
PARTICIPANTS
Patients referred by their Primary Care Physician to the Primary Care Physiotherapy Unit for shoulder pain of musculoskeletal origin.
INTERVENTION
7 group sessions of health education and therapeutic exercise.
MAIN MEASUREMENTS
Pain intensity was assessed through the Visual Analogue Scale (VAS), the disability of the upper limb with the Disabilities of the Arm, Shoulder and Hand (DASH) questionnaire and the level of disability and shoulder pain with the Shoulder Pain and Disability Index (SPADI) questionnaire.
RESULTS
Statistically significant differences were found in the reduction of pain and disability (P<.01), in addition, drug use and recurrences were reduced.
CONCLUSIONS
The shoulder physiotherapy protocol with health education was effective in reducing pain and disability in patients with chronic shoulder pain of musculoskeletal origin in Primary Care.
Topics: Chronic Pain; Health Education; Humans; Musculoskeletal Pain; Observational Studies as Topic; Physical Therapy Modalities; Primary Health Care; Shoulder; Shoulder Pain; Upper Extremity
PubMed: 35461039
DOI: 10.1016/j.aprim.2022.102284 -
International Heart Journal Apr 2017The FMDJ study, a multicenter prospective observational study conducted in Japan, demonstrated the acceptable reliability of measurement of flow-mediated vasodilatation... (Review)
Review
The FMDJ study, a multicenter prospective observational study conducted in Japan, demonstrated the acceptable reliability of measurement of flow-mediated vasodilatation (FMD) using a semi-automatic device in individual institutions. However, in about 10% of Japanese subjects, adequate scans to determine the brachial arterial diameter failed to be obtained. The prevalence of inadequate scans was higher in women than in men, while obesity had no influence on the inadequate scan rate. The FMDJ study also proposed that attending periodic refresher courses on the measurement of FMD is needed for maintaining competency. Finally, the FMDJ study proposed reference values for FMD. Thus, FMD measurement may be categorized as a clinically applicable tool on the basis of class IIb (exploratory cohort study with good reference standards) evidence.
Topics: Diagnostic Techniques, Cardiovascular; Humans; Multicenter Studies as Topic; Observational Studies as Topic; Reference Values; Vasodilation
PubMed: 28367852
DOI: 10.1536/ihj.17-013 -
Osteoarthritis and Cartilage Nov 2022
Topics: Causality; Observational Studies as Topic
PubMed: 36063988
DOI: 10.1016/j.joca.2022.08.010 -
Archives of Cardiovascular Diseases 2021Exactly two decades have elapsed since pacemakers first provided automatic remote monitoring. This innovation has been well received by patients. However, there is still... (Review)
Review
Exactly two decades have elapsed since pacemakers first provided automatic remote monitoring. This innovation has been well received by patients. However, there is still a widely held perception that remote monitoring of pacemakers is non-essential, despite the very similar gains that are achieved compared with remote monitoring of implantable cardioverter defibrillators. Reducing in-office evaluations and overall staff workload is important when these resources are stretched to their limits. The early detection ability provided by remote monitoring facilitates device management (extending battery longevity) and the ability to exercise vigilance over recalled components. Clinical complications, such as arrhythmic events, are also detected earlier. Remote monitoring has been shown to produce similar reductions in the risk of all-cause hospitalization and death for pacemakers and implantable cardioverter defibrillators in a mega-cohort observational study. This review is an evidence-based plea for the recognition and systematic implementation of remote monitoring for pacemakers.
Topics: Defibrillators, Implantable; Humans; Observational Studies as Topic; Pacemaker, Artificial
PubMed: 34561150
DOI: 10.1016/j.acvd.2021.06.007 -
Journal of Diabetes and Its... 2013Medical research continues to progress in its ability to identify treatments and characteristics associated with benefits and adverse outcomes. The principal engine for... (Review)
Review
Medical research continues to progress in its ability to identify treatments and characteristics associated with benefits and adverse outcomes. The principal engine for the evaluation of treatment efficacy is the randomized controlled trial (RCT). Due to the cost and other considerations, RCTs cannot address all clinically important decisions. Observational research often is used to address issues not addressed or not addressable by RCTs. This article provides an overview of the benefits and limitations of observational research to serve as a guide to the interpretation of this category of research designs in diabetes investigations. The potential for bias is higher in observational research but there are design and analysis features that can address these concerns although not completely eliminate them. Pharmacoepidemiologic research may provide important information regarding relative safety and effectiveness of diabetes pharmaceuticals. Such research must effectively address the important issue of confounding by indication in order to produce clinically meaningful results. Other methods such as instrumental variable analysis are being employed to enable stronger causal inference but these methods also require fulfillment of several key assumptions that may or may not be realistic. Nearly all clinical decisions involve probabilistic reasoning and confronting uncertainly, so a realistic goal for observational research may not be the high standard set by RCTs but instead the level of certainty needed to influence a diagnostic or treatment decision.
Topics: Bias; Case-Control Studies; Causality; Clinical Trials as Topic; Cohort Studies; Diabetes Mellitus; Humans; Observational Studies as Topic; Research Design; Treatment Outcome
PubMed: 24055326
DOI: 10.1016/j.jdiacomp.2013.07.007 -
European Journal of Epidemiology Jan 2023A detailed examination of the 1930 Lanarkshire Milk Experiment (LME) by the famous statistician William Sealy Gossett ("Student"), which appeared in Biometrika in 1931,... (Review)
Review
A detailed examination of the 1930 Lanarkshire Milk Experiment (LME) by the famous statistician William Sealy Gossett ("Student"), which appeared in Biometrika in 1931, is re-examined from a more modern perspective. The LME had a complicated design whereby 67 schools in Lanarkshire were allocated to receive either raw or pasteurised milk but pupils within the schools were allocated to either receive milk or to act as controls. Student's criticisms are considered in detail and examined in terms of subsequent developments on the design and analysis of experiments, in particular as regards appropriate estimation of standard errors of treatment estimates when an incomplete blocks structure has been used. An analogy with a more modern trial in osteoarthritis is made. Suggestions are made as to how analysis might proceed if the original data were available. Some lessons for observational studies in epidemiology are drawn and it is speculated that hidden clustering structures might be an explanation as to why results may vary from observational study to observational study by more than conventionally calculated standard errors might suggest.
Topics: Humans; Animals; Milk; Schools; Observational Studies as Topic
PubMed: 36477576
DOI: 10.1007/s10654-022-00941-x -
Contraception Aug 2022Systematically review the existing evidence about couples-based interventions and postpartum contraceptive uptake and generate recommendations for future research. (Review)
Review
OBJECTIVE
Systematically review the existing evidence about couples-based interventions and postpartum contraceptive uptake and generate recommendations for future research.
DATA SOURCES
PubMed, Web of Science, PsycINFO, Embase, and CINAHL through June 7, 2021.
STUDY SELECTION AND DATA EXTRACTION
Studies with a couples-based intervention assessing postpartum contraceptive uptake. Two independent reviewers screened studies, extracted data, and assessed risk of bias with RoB-2 (Cochrane Risk of Bias 2) for randomized and ROBINS-I (Risk of Bias in Non-Randomized Studies - Interventions) for observational studies. Data were synthesized in tables, figures, and a narrative review.
RESULTS
A total of 925 papers were identified, 66 underwent full text review, and 17 articles, which included 18 studies - 16 randomized, 2 observational - were included. The lack of intervention and outcome homogeneity precluded meta-analysis and isolating the effect of partner involvement. Four studies were partner-required, where partner involvement was a required component of the intervention, and 14 were partner-optional. Unadjusted risk differences ranged from 0.01 to 0.51 in favor of couples-based interventions increasing postpartum contraceptive uptake versus standard of care. Bias assessment of the 16 randomized studies classified 8, 3, and 5 studies as at a high, some concern, and low risk of bias. Common sources of bias included intervention non-adherence and missing outcome data. One observational study was at a high and the other at a low risk of bias.
CONCLUSIONS
Future studies that assess couples-based interventions must clearly define and measure how partners are involved in the intervention and assess how intervention adherence impacts postpartum contraceptive uptake.
Topics: Contraceptive Agents; Contraceptive Devices; Female; Humans; Observational Studies as Topic; Postpartum Period; Text Messaging
PubMed: 35577147
DOI: 10.1016/j.contraception.2022.05.001 -
International Journal of Clinical... Jun 2016Observational studies have been recognised to be essential for investigating the safety profile of medications. Numerous observational studies have been conducted on the... (Review)
Review
Observational studies have been recognised to be essential for investigating the safety profile of medications. Numerous observational studies have been conducted on the platform of large population databases, which provide adequate sample size and follow-up length to detect infrequent and/or delayed clinical outcomes. Cohort and case-control are well-accepted traditional methodologies for hypothesis testing, while within-individual study designs are developing and evolving, addressing previous known methodological limitations to reduce confounding and bias. Respective examples of observational studies of different study designs using medical databases are shown. Methodology characteristics, study assumptions, strengths and weaknesses of each method are discussed in this review.
Topics: Databases, Pharmaceutical; Humans; Observational Studies as Topic; Pharmacoepidemiology; Research Design
PubMed: 27003827
DOI: 10.1007/s11096-016-0285-6 -
European Journal of Vascular and... Jan 2016
Topics: Humans; Observational Studies as Topic; Prospective Studies; Research Design; Time Factors
PubMed: 26538072
DOI: 10.1016/j.ejvs.2015.09.021