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1.
BMJ ; 368: l7078, 2020 Feb 05.
Artigo em Inglês | MEDLINE | ID: mdl-32024657

RESUMO

OBJECTIVES: To conduct a systematic review and meta-analysis of the effects of rosiglitazone treatment on cardiovascular risk and mortality using multiple data sources and varying analytical approaches with three aims in mind: to clarify uncertainties about the cardiovascular risk of rosiglitazone; to determine whether different analytical approaches are likely to alter the conclusions of adverse event meta-analyses; and to inform efforts to promote clinical trial transparency and data sharing. DESIGN: Systematic review and meta-analysis of randomized controlled trials. DATA SOURCES: GlaxoSmithKline's (GSK's) ClinicalStudyDataRequest.com for individual patient level data (IPD) and GSK's Study Register platforms, MEDLINE, PubMed, Embase, Web of Science, Cochrane Central Registry of Controlled Trials, Scopus, and ClinicalTrials.gov from inception to January 2019 for summary level data. ELIGIBILITY CRITERIA FOR SELECTING STUDIES: Randomized, controlled, phase II-IV clinical trials that compared rosiglitazone with any control for at least 24 weeks in adults. DATA EXTRACTION AND SYNTHESIS: For analyses of trials for which IPD were available, a composite outcome of acute myocardial infarction, heart failure, cardiovascular related death, and non-cardiovascular related death was examined. These four events were examined independently as secondary analyses. For analyses including trials for which IPD were not available, myocardial infarction and cardiovascular related death were examined, which were determined from summary level data. Multiple meta-analyses were conducted that accounted for trials with zero events in one or both arms with two different continuity corrections (0.5 constant and treatment arm) to calculate odds ratios and risk ratios with 95% confidence intervals. RESULTS: 33 eligible trials were identified from ClinicalStudyDataRequest.com for which IPD were available (21 156 patients). Additionally, 103 trials for which IPD were not available were included in the meta-analyses for myocardial infarction (23 683 patients), and 103 trials for which IPD were not available contributed to the meta-analyses for cardiovascular related death (22 772 patients). Among 29 trials for which IPD were available and that were included in previous meta-analyses using GSK's summary level data, more myocardial infarction events were identified by using IPD instead of summary level data for 26 trials, and fewer cardiovascular related deaths for five trials. When analyses were limited to trials for which IPD were available, and a constant continuity correction of 0.5 and a random effects model were used to account for trials with zero events in only one arm, patients treated with rosiglitazone had a 33% increased risk of a composite event compared with controls (odds ratio 1.33, 95% confidence interval 1.09 to 1.61; rosiglitazone population: 274 events among 11 837 patients; control population: 219 events among 9319 patients). The odds ratios for myocardial infarction, heart failure, cardiovascular related death, and non-cardiovascular related death were 1.17 (0.92 to 1.51), 1.54 (1.14 to 2.09), 1.15 (0.55 to 2.41), and 1.18 (0.60 to 2.30), respectively. For analyses including trials for which IPD were not available, odds ratios for myocardial infarction and cardiovascular related death were attenuated (1.09, 0.88 to 1.35, and 1.12, 0.72 to 1.74, respectively). Results were broadly consistent when analyses were repeated using trials with zero events across both arms and either of the two continuity corrections was used. CONCLUSIONS: The results suggest that rosiglitazone is associated with an increased cardiovascular risk, especially for heart failure events. Although increased risk of myocardial infarction was observed across analyses, the strength of the evidence varied and effect estimates were attenuated when summary level data were used in addition to IPD. Because more myocardial infarctions and fewer cardiovascular related deaths were reported in the IPD than in the summary level data, sharing IPD might be necessary when performing meta-analyses focused on safety. SYSTEMATIC REVIEW REGISTRATION: OSF Home https://osf.io/4yvp2/.

2.
J Am Heart Assoc ; 9(4): e013606, 2020 Feb 18.
Artigo em Inglês | MEDLINE | ID: mdl-32063087

RESUMO

Background More than 600 000 coronary stents are implanted during percutaneous coronary interventions (PCIs) annually in the United States. Because no real-world surveillance system exists to monitor their long-term safety, claims data are often used for this purpose. The extent to which adverse events identified with claims data can be reasonably attributed to a specific medical device is uncertain. Methods and Results We used deterministic matching to link the NCDR (National Cardiovascular Data Registry) CathPCI Registry to Medicare fee-for-service claims for patients aged ≥65 years who underwent PCI with drug-eluting stents (DESs) between July 1, 2009 and December 31, 2013. We identified subsequent PCIs within 1 year of the index procedure in Medicare claims as potential safety events. We linked these subsequent PCIs back to the NCDR CathPCI Registry to ascertain how often the revascularization could be reasonably attributed to the same coronary artery as the index PCI (ie, target vessel revascularization). Of 415 306 DES placements in 368 194 patients, 33 174 repeat PCIs were identified in Medicare claims within 1 year. Of these, 28 632 (86.3%) could be linked back to the NCDR CathPCI Registry; 16 942 (51.1% of repeat PCIs) were target vessel revascularizations. Of these, 8544 (50.4%) were within a previously placed DES: 7652 for in-stent restenosis and 1341 for stent thrombosis. Of 16 176 patients with a claim for acute myocardial infarction in the follow-up period, 4446 (27.5%) were attributed to the same coronary artery in which the DES was implanted during the index PCI (ie, target vessel myocardial infarction). Of 24 288 patients whose death was identified in claims data, 278 (1.1%) were attributed to the same coronary artery in which the DES was implanted during the index PCI. Conclusions Most repeat PCIs following DES stent implantation identified in longitudinal claims data could be linked to real-world registry data, but only half could be reasonably attributed to the same coronary artery as the index procedure. Attribution among those with acute myocardial infarction or who died was even less frequent. Safety signals identified using claims data alone will require more in-depth examination to accurately assess stent safety.

3.
JAMA ; 2020 Feb 10.
Artigo em Inglês | MEDLINE | ID: mdl-32040163

RESUMO

Importance: Acute myocardial infarction (AMI) complicated by cardiogenic shock is associated with substantial morbidity and mortality. Although intravascular microaxial left ventricular assist devices (LVADs) provide greater hemodynamic support as compared with intra-aortic balloon pumps (IABPs), little is known about clinical outcomes associated with intravascular microaxial LVAD use in clinical practice. Objective: To examine outcomes among patients undergoing percutaneous coronary intervention (PCI) for AMI complicated by cardiogenic shock treated with mechanical circulatory support (MCS) devices. Design, Setting, and Participants: A propensity-matched registry-based retrospective cohort study of patients with AMI complicated by cardiogenic shock undergoing PCI between October 1, 2015, and December 31, 2017, who were included in data from hospitals participating in the CathPCI and the Chest Pain-MI registries, both part of the American College of Cardiology's National Cardiovascular Data Registry. Patients receiving an intravascular microaxial LVAD were matched with those receiving IABP on demographics, clinical history, presentation, infarct location, coronary anatomy, and clinical laboratory data, with final follow-up through December 31, 2017. Exposures: Hemodynamic support, categorized as intravascular microaxial LVAD use only, IABP only, other (such as use of a percutaneous extracorporeal ventricular assist system, extracorporeal membrane oxygenation, or a combination of MCS device use), or medical therapy only. Main Outcomes and Measures: The primary outcomes were in-hospital mortality and in-hospital major bleeding. Results: Among 28 304 patients undergoing PCI for AMI complicated by cardiogenic shock, the mean (SD) age was 65.0 (12.6) years, 67.0% were men, 81.3% had an ST-elevation myocardial infarction, and 43.3% had cardiac arrest. Over the study period among patients with AMI, an intravascular microaxial LVAD was used in 6.2% of patients, and IABP was used in 29.9%. Among 1680 propensity-matched pairs, there was a significantly higher risk of in-hospital death associated with use of an intravascular microaxial LVAD (45.0%) vs with an IABP (34.1% [absolute risk difference, 10.9 percentage points {95% CI, 7.6-14.2}; P < .001) and also higher risk of in-hospital major bleeding (intravascular microaxial LVAD [31.3%] vs IABP [16.0%]; absolute risk difference, 15.4 percentage points [95% CI, 12.5-18.2]; P < .001). These associations were consistent regardless of whether patients received a device before or after initiation of PCI. Conclusions and Relevance: Among patients undergoing PCI for AMI complicated by cardiogenic shock from 2015 to 2017, use of an intravascular microaxial LVAD compared with IABP was associated with higher adjusted risk of in-hospital death and major bleeding complications, although study interpretation is limited by the observational design. Further research may be needed to understand optimal device choice for these patients.

5.
Int J Epidemiol ; 2020 Jan 22.
Artigo em Inglês | MEDLINE | ID: mdl-31967637

RESUMO

BACKGROUND: Different analytical approaches can influence the associations estimated in observational studies. We assessed the variability of effect estimates reported within and across observational studies evaluating the impact of alcohol on breast cancer. METHODS: We abstracted largest harmful, largest protective and smallest (closest to the null value of 1.0) relative risk estimates in studies included in a recent alcohol-breast cancer meta-analysis, and recorded how they differed based on five model specification characteristics, including exposure definition, exposure contrast levels, study populations, adjustment covariates and/or model approaches. For each study, we approximated vibration of effects by dividing the largest by the smallest effect estimate [i.e. ratio of odds ratio (ROR)]. RESULTS: Among 97 eligible studies, 85 (87.6%) reported both harmful and protective relative effect estimates for an alcohol-breast cancer relationship, which ranged from 1.1 to 17.9 and 0.0 to 1.0, respectively. The RORs comparing the largest and smallest estimates in value ranged from 1.0 to 106.2, with a median of 3.0 [interquartile range (IQR) 2.0-5.2]. One-third (35, 36.1%) of the RORs were based on extreme effect estimates with at least three different model specification characteristics; the vast majority (87, 89.7%) had different exposure definitions or contrast levels. Similar vibrations of effect were observed when only extreme estimates with differences based on study populations and/or adjustment covariates were compared. CONCLUSIONS: Most observational studies evaluating the impact of alcohol on breast cancer report relative effect estimates for the same associations that diverge by >2-fold. Therefore, observational studies should estimate the vibration of effects to provide insight regarding the stability of findings.

6.
J Am Geriatr Soc ; 68(1): 96-102, 2020 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-31603248

RESUMO

BACKGROUND/OBJECTIVE: Heart failure (HF) readmission rates have plateaued despite scrutiny of hospital discharge practices. Many HF patients are discharged to skilled nursing facility (SNF) after hospitalization before returning home. Home healthcare (HHC) services received during the additional transition from SNF to home may affect readmission risk. Here, we examined whether receipt of HHC affects readmission risk during the transition from SNF to home following HF hospitalization. DESIGN: Retrospective cohort study. SETTING: Fee-for-service Medicare data, 2012 to 2015. PARTICIPANTS: Beneficiaries, aged 65 years and older, hospitalized with HF who were subsequently discharged to SNF and then discharged home. MEASUREMENTS: The primary outcome was unplanned readmission within 30 days of discharge to home from SNF. We compared time to readmission between those with and without HHC services using a Cox model. RESULTS: Of 67 585 HF hospitalizations discharged to SNFs and subsequently discharged home, 13 257 (19.6%) were discharged with HHC, and 54 328 (80.4%) were discharged without HHC. Patients discharged home from SNFs with HHC had lower 30-day readmission rates than patients discharged without HHC (22.8% vs 24.5%; P < .0001) and a longer time to readmission. In an adjusted model, the hazard for readmission was 0.91 (0.86-0.95) with receipt of HHC. CONCLUSIONS: Recipients of HHC were less likely to be readmitted within 30 days vs those discharged home without HHC. This is unexpected, as patients discharged with HHC likely have more functional impairments. Since patients requiring a SNF stay after hospital discharge may have additional needs, they may particularly benefit from restorative therapy through HHC; however, only approximately 20% received such services. J Am Geriatr Soc 68:96-102, 2019.

8.
Drug Alcohol Depend ; 205: 107671, 2019 Dec 01.
Artigo em Inglês | MEDLINE | ID: mdl-31706248

RESUMO

OBJECTIVES: To examine trends in polysubstance detection associated with drug-related overdose deaths in Connecticut. METHODS: We used 2012-2018 data provided by Connecticut's Office of the Chief Medical Examiner (OCME) on accidental overdose deaths. We estimated annual trends, standardizing the number of deaths per 100,000 Connecticut residents each year. We then conducted stratified analyses by polysubstance use status. We also examined the numbers of deaths involving fentanyl in a separate analysis. We obtained data in April 2019, and statistical analyses were performed from April to September 2019. RESULTS: The rate of overdose deaths in Connecticut increased from 9.9 per 100,000 residents in 2012 to 28.5 per 100,000 residents in 2018-a 221 % increase-with the majority occurring among persons aged 35-64 (65.3 %), men (73.9 %), and non-Hispanic whites (78.5 %). Among deaths involving fentanyl, the overall deaths escalated from 5.2 deaths per 100,000 residents in 2015 to 21.3 deaths per 100,000 residents in 2018, and more than 50% of these fentanyl-related deaths involved polysubstance use. CONCLUSIONS: Connecticut experienced a more-than doubling of opioid-involved overdose deaths, largely driven by fentanyl and polysubstance use. The role of polysubstance use should be considered in efforts toward reducing opioid-related overdose incidents.

9.
Ann Intern Med ; 2019 Nov 12.
Artigo em Inglês | MEDLINE | ID: mdl-31711094

RESUMO

The PATH (Predictive Approaches to Treatment effect Heterogeneity) Statement was developed to promote the conduct of, and provide guidance for, predictive analyses of heterogeneity of treatment effects (HTE) in clinical trials. The goal of predictive HTE analysis is to provide patient-centered estimates of outcome risk with versus without the intervention, taking into account all relevant patient attributes simultaneously, to support more personalized clinical decision making than can be made on the basis of only an overall average treatment effect. The authors distinguished 2 categories of predictive HTE approaches (a "risk-modeling" and an "effect-modeling" approach) and developed 4 sets of guidance statements: criteria to determine when risk-modeling approaches are likely to identify clinically meaningful HTE, methodological aspects of risk-modeling methods, considerations for translation to clinical practice, and considerations and caveats in the use of effect-modeling approaches. They discuss limitations of these methods and enumerate research priorities for advancing methods designed to generate more personalized evidence. This explanation and elaboration document describes the intent and rationale of each recommendation and discusses related analytic considerations, caveats, and reservations.

10.
Ann Intern Med ; 2019 Nov 12.
Artigo em Inglês | MEDLINE | ID: mdl-31711134

RESUMO

Heterogeneity of treatment effect (HTE) refers to the nonrandom variation in the magnitude or direction of a treatment effect across levels of a covariate, as measured on a selected scale, against a clinical outcome. In randomized controlled trials (RCTs), HTE is typically examined through a subgroup analysis that contrasts effects in groups of patients defined "1 variable at a time" (for example, male vs. female or old vs. young). The authors of this statement present guidance on an alternative approach to HTE analysis, "predictive HTE analysis." The goal of predictive HTE analysis is to provide patient-centered estimates of outcome risks with versus without the intervention, taking into account all relevant patient attributes simultaneously. The PATH (Predictive Approaches to Treatment effect Heterogeneity) Statement was developed using a multidisciplinary technical expert panel, targeted literature reviews, simulations to characterize potential problems with predictive approaches, and a deliberative process engaging the expert panel. The authors distinguish 2 categories of predictive HTE approaches: a "risk-modeling" approach, wherein a multivariable model predicts the risk for an outcome and is applied to disaggregate patients within RCTs to define risk-based variation in benefit, and an "effect-modeling" approach, wherein a model is developed on RCT data by incorporating a term for treatment assignment and interactions between treatment and baseline covariates. Both approaches can be used to predict differential absolute treatment effects, the most relevant scale for clinical decision making. The authors developed 4 sets of guidance: criteria to determine when risk-modeling approaches are likely to identify clinically important HTE, methodological aspects of risk-modeling methods, considerations for translation to clinical practice, and considerations and caveats in the use of effect-modeling approaches. The PATH Statement, together with its explanation and elaboration document, may guide future analyses and reporting of RCTs.

11.
Am Heart J ; 218: 110-122, 2019 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-31726314

RESUMO

BACKGROUND: Medicare insurance claims may provide an efficient means to ascertain follow-up of older participants in clinical research. We sought to determine the accuracy and completeness of claims- versus site-based follow-up with clinical event committee (+CEC) adjudication of cardiovascular outcomes. METHODS: We performed a retrospective study using linked Medicare and Duke Database of Clinical Trials data. Medicare claims were linked to clinical data from 7 randomized cardiovascular clinical trials. Of 52,476 trial participants, linking resulted in 5,839 (of 10,497 linkage-eligible) Medicare-linked trial participants with fee-for-service A and B coverage. Death, myocardial infarction (MI), stroke, and revascularization incidences were compared using Medicare inpatient claims only, site-reported events (+CEC) only, or a combination of the 2. Randomized treatment effects were compared as a function of whether claims-based, site-based (+CEC), or a combined system was used for event detection. RESULTS: Among the 5,839 study participants, the annual event rates were similar between claims- and site-based (+CEC) follow-up: death (overall rate 5.2% vs 5.2%; adjusted κ 0.99), MI (2.2% vs 2.3%; adjusted κ 0.96), stroke (0.7% vs 0.7%; adjusted κ 0.99), and any revascularization (7.4% vs 7.9%; adjusted κ 0.95). Of events detected by claims yet not reported by CEC, a minority were reported by sites but negatively adjudicated by CEC (39% of MIs and 18% of strokes). Differences in individual case concordance led to higher event rates when claims- and site-based (+CEC) systems were combined. Randomized treatment effects were similar among the 3 approaches for each outcome of interest. CONCLUSIONS: Claims- versus site-based (+CEC) follow-up identified similar overall cardiovascular event rates despite meaningful differences in the events detected. Randomized treatment effects were similar using the 2 methods, suggesting claims data could be used to support clinical research leveraging routinely collected data. This approach may lead to more effective evidence generation, synthesis, and appraisal of medical products and inform the strategic approaches toward the National Evaluation System for Health Technology.

14.
JAMA Netw Open ; 2(10): e1913298, 2019 Oct 02.
Artigo em Inglês | MEDLINE | ID: mdl-31617923

RESUMO

Importance: Out-of-hospital cardiac arrest is a common scenario facing prehospital emergency medical services (EMS) professionals and nearly always involves either manual or mechanical cardiopulmonary resuscitation (CPR). Mechanical CPR devices are expensive and prior clinical trials have not provided evidence of benefit for patients when compared with manual CPR. Objectives: To investigate the use of mechanical CPR in the prehospital setting and determine whether patient demographic characteristics or geographical location is associated with its use. Design, Setting, and Participants: A retrospective cross-sectional study was performed using the 2010 through 2016 National Emergency Medical Services Information System data. Participants included all patients identified by EMS professionals as having out-of-hospital cardiac arrest. Main Outcomes and Measures: Use of CPR, categorized as manual or mechanical. Results: From 2010 to 2016, 892 022 patients (38.6% female, 60.4% male, missing for 1%; mean [SD] age, 61.1 [20.5] years) with out-of-hospital cardiac arrest were identified by EMS professionals. Overall, manual CPR was used for 618 171 patients (69.3%) and mechanical CPR was used for 45 493 patients (5.1%). The risk-standardized rate of mechanical CPR use, accounting for patient demographic and geographical characteristics, rose from 1.9% in 2010 to 8.0% in 2016 (P < .001). In multivariable analyses, use of mechanical CPR devices was increasingly likely over time among patients identified with out-of-hospital cardiac arrest treated by EMS professionals, increasing from an adjusted odds ratio of 1.58 (95% CI, 1.42-1.77; P < .001) when comparing 2011 with 2010, to an adjusted odds ratio of 11.32 (95% CI, 10.22-12.54; P < .001) when comparing 2016 with 2010. In addition, several other patient demographic and geographical characteristics were associated with a higher likelihood of receiving mechanical CPR, including being 65 years or older, being male, being Hispanic, as well as receiving treatment in the Northeast Census Region, in a suburban location, or in a zip code with a median annual income greater than $20 000. Conclusions and Relevance: Mechanical CPR device use increased more than 4-fold among patients with out-of-hospital cardiac arrest treated by EMS professionals. Given the high costs of mechanical CPR devices, better evidence is needed to determine whether these devices improve clinically meaningful outcomes for patients treated for out-of-hospital cardiac arrest by prehospital EMS professionals to justify the significant increase in their use.

16.
JAMA Intern Med ; : 1-8, 2019 Oct 28.
Artigo em Inglês | MEDLINE | ID: mdl-31657836

RESUMO

Litigation involving drug and medical device manufacturers has the potential to reveal important information about product efficacy and safety as well as company marketing. Prevailing legal standards recognize the public's interest in having access to certain types of information in lawsuits. However, in practice, courts and litigants commonly use overly broad or unwarranted confidentiality orders, which can prevent the public from accessing important public health information that emerges during litigation. This Special Communication reviews the rules governing confidentiality orders and discusses the tension between these rules and prevailing legal practices relating to court secrecy in medical product litigation, including competing interests among manufacturers, plaintiffs, and courts. Using examples of successful efforts to challenge confidentiality orders, we describe how these prevailing legal practices can undermine access to information by patients, clinicians, and the US Food and Drug Administration and also obscure patterns of injury and disease associated with the drugs and medical devices at issue. We then discuss several ways to advance access to information important to public health that emerges during litigation, focusing particularly on the role of medical experts engaged in cases.

17.
JAMA Intern Med ; 2019 Oct 28.
Artigo em Inglês | MEDLINE | ID: mdl-31657845
18.
J Manag Care Spec Pharm ; 25(11): 1201-1217, 2019 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-31663461

RESUMO

BACKGROUND: The boxed warning (also known as "black box warning") is one of the FDA's strongest safety actions for pharmaceuticals. After the FDA issues black box warnings for drugs, prescribing changes have been inconsistent. Formulary management may provide an opportunity to restrict access to drugs with serious safety concerns. OBJECTIVE: To examine Medicare prescription drug plan formulary changes after new FDA postmarket black box warnings and major updates to preexisting black box warnings. METHODS: In this cohort study, we identified each drug that received a new FDA postmarket black box warning or a major update to a preexisting black box warning from January 2008 through June 2015 and examined its formulary coverage. The main outcome measure was the proportion of Medicare prescription drug plan formularies providing unrestrictive coverage immediately before the black box warning, at least 1 year after the warning and at least 2 years after the warning. Unrestrictive formulary coverage was defined as coverage of a drug without prior authorization or step-therapy requirements. RESULTS: Of 101 new black box warnings and major updates to preexisting warnings affecting 68 unique drug formulations, the mean percentage of formularies providing unrestrictive coverage changed from 65.4% (95% CI = 59.6%-71.2%) prewarning; 62.6% (95% CI = 56.3%-68.9%, P = 0.04) at least 1 year postwarning; and 61.9% (95% CI = 55.4%-68.5%, P = 0.10) at least 2 years postwarning. CONCLUSIONS: The mean percentage of Medicare prescription drug plan formularies providing unrestrictive coverage decreased modestly by approximately 3 percentage points after drugs received postmarket FDA black box warnings. Formulary restrictions may present an underused mechanism to reduce use of potentially unsafe medications. DISCLOSURES: This study was supported by a student research grant received by Solotke and provided by the Yale School of Medicine Office of Student Research under National Institutes of Health training grant award T35DK104689. Karaca-Mandic, Shah, and Ross acknowledge support from Agency for Healthcare Research and Quality (AHRQ) grant R01 HS025164, which studies factors associated with de-adoption of drug therapies shown to be ineffective or unsafe. The content of this study is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. The authors assume full responsibility for the accuracy and completeness of the ideas presented. Ross has received support from the following: the U.S. Food and Drug Administration (FDA) as part of the Centers for Excellence in Regulatory Science and Innovation (CERSI) program; Johnson and Johnson through Yale University to develop methods of clinical trial data sharing; Medtronic and the FDA to develop methods for postmarket surveillance of medical devices; the Blue Cross Blue Shield Association to better understand medical technology evaluation; the Centers for Medicare & Medicaid Services (CMS) to develop and maintain performance measures that are used for public reporting; the AHRQ to examine community predictors of health care quality; and the Laura and John Arnold Foundation, which established the Collaboration for Research Integrity and Transparency at Yale University. Shah has received support from the FDA as part of the CERSI program. In addition, he has received support through the Mayo Clinic from CMS, AHRQ, National Science Foundation, and Patient-centered Outcomes Research Institute. Karaca-Mandic has provided consulting services to Precision Health Economics and Tactile Medical for work unrelated to this manuscript. Dhruva and Solotke have no conflicts of interest to report.

19.
JAMA Netw Open ; 2(10): e1912869, 2019 Oct 02.
Artigo em Inglês | MEDLINE | ID: mdl-31596493

RESUMO

Importance: Although randomized clinical trials are considered to be the criterion standard for generating clinical evidence, the use of real-world evidence to evaluate the efficacy and safety of medical interventions is gaining interest. Whether observational data can be used to address the same clinical questions being answered by traditional clinical trials is still unclear. Objective: To identify the number of clinical trials published in high-impact journals in 2017 that could be feasibly replicated using observational data from insurance claims and/or electronic health records (EHRs). Design, Setting, and Participants: In this cross-sectional analysis, PubMed was searched to identify all US-based clinical trials, regardless of randomization, published between January 1, 2017, and December 31, 2017, in the top 7 highest-impact general medical journals of 2017. Trials were excluded if they did not involve human participants, did not use end points that represented clinical outcomes among patients, were not characterized as clinical trials, and had no recruitment sites in the United States. Main Outcomes and Measures: The primary outcomes were the number and percentage of trials for which the intervention, indication, trial inclusion and exclusion criteria, and primary end points could be ascertained from insurance claims and/or EHR data. Results: Of the 220 US-based trials analyzed, 33 (15.0%) could be replicated using observational data because their intervention, indication, inclusion and exclusion criteria, and primary end points could be routinely ascertained from insurance claims and/or EHR data. Of the 220 trials, 86 (39.1%) had an intervention that could be ascertained from insurance claims and/or EHR data. Among the 86 trials, 62 (72.1%) had an indication that could be ascertained. Forty-five (72.6%) of 62 trials had at least 80% of inclusion and exclusion criteria data that could be ascertained. Of these 45 studies, 33 (73.3%) had at least 1 primary end point that could be ascertained. Conclusions and Relevance: This study found that only 15% of the US-based clinical trials published in high-impact journals in 2017 could be feasibly replicated through analysis of administrative claims or EHR data. This finding suggests the potential for real-world evidence to complement clinical trials, both by examining the concordance between randomized experiments and observational studies and by comparing the generalizability of the trial population with the real-world population of interest.

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