Clinical Evidence Supporting US Food and Drug Administration Clearance of Novel Therapeutic Devices via the De Novo Pathway Between 2011 and 2019.

This cross-sectional study characterizes the premarket clinical evidence supporting clearance of moderate-risk novel therapeutic medical devices by the US Food and Drug Administration.

Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study.

Objective: To characterize the therapeutic value of new drugs approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the association between these ratings and regulatory approval through expedited programs.

Design: Retrospective cohort study.

Setting: New drugs approved by the FDA and EMA between 2007 and 2017, with follow-up through 1 April 2020.

Comparative Effectiveness and Safety of Oral Anticoagulants Across Kidney Function in Patients With Atrial Fibrillation.

Background: Patients with atrial fibrillation and severely decreased kidney function were excluded from the pivotal non-vitamin K antagonist oral anticoagulants (NOAC) trials, thereby raising questions about comparative safety and effectiveness in patients with reduced kidney function. The study aimed to compare oral anticoagulants across the range of kidney function in patients with atrial fibrillation.

Methods And Results: Using a US administrative claims database with linked laboratory data, 34 569 new users of oral anticoagulants with atrial fibrillation and estimated glomerular filtration rate ≥15 mL/(min·1.

Comparative Effectiveness of Generic vs Brand-Name Levothyroxine in Achieving Normal Thyrotropin Levels.

Importance: Whether the use of generic vs brand levothyroxine affects thyrotropin levels remains unclear.

Objective: To compare the effectiveness of generic vs brand levothyroxine in achieving and maintaining normal thyrotropin levels among new users.

Design, Setting, And Participants: This retrospective, 1:1 propensity score-matched longitudinal cohort study used the OptumLabs Data Warehouse administrative claims database linked to laboratory results from commercially insured and Medicare Advantage enrollees throughout the United States.

Postmarket Clinical Evidence for High-Risk Therapeutic Medical Devices Receiving Food and Drug Administration Premarket Approval in 2010 and 2011.

This cross-sectional study provides a 5-year update on the status and availability of postmarket evidence for high-risk medical devices that received FDA premarket approval in 2010 and 2011.

Non-inferiority trials using a surrogate marker as the primary endpoint: An increasing phenotype in cardiovascular trials.

Background/aims: Non-inferiority trials are increasing in cardiovascular medicine, with approval of many drugs and devices on the basis of such studies. Surrogate markers as primary endpoints have been also more frequently used for efficient assessment of cardiovascular interventions. However, there is uncertainty about their concordance with clinical outcomes.

Disagreements Within the US Food and Drug Administration Regarding Approval of Novel Therapeutic Agents, 2011-2015.

This cross-sectional study examines the frequency of disagreements within the US Food and Drug Administration (FDA) regarding approval of novel therapeutic agents.

Consistency of trial reporting between ClinicalTrials.gov and corresponding publications: one decade after FDAAA.

The FDA Amendments Act (FDAAA) required that information for certain clinical trials, such as details about study design features and endpoints, as well as results, be publicly reported in ClinicalTrials.gov . We conducted a cross-sectional analysis of phase III trials with primary results published between January 1, 2016, and June 30, 2017, in high-impact journals and found 74% contained at least one discrepancy between results reported in ClinicalTrials.

Assessment of Preprint Policies of Top-Ranked Clinical Journals.

This cross-sectional study examines the preprint publication policies of 100 clinical journals with the highest impact factor.

Characteristics of Ongoing Clinical Trials for Alcohol Use Disorder Registered on ClinicalTrials.gov.

This cross-sectional study evaluates the characteristics of ongoing clinical trials for alcohol use disorder medications registered on ClinicalTrials.gov, including the medications, populations, and end points currently being studied.

Clinical Outcomes With Beta-Blocker Use in Patients With Recent History of Myocardial Infarction.

Background: It is uncertain whether beta-blockers (BBs) are beneficial in contemporary stable patients with prior myocardial infarction (MI). Therefore, we sought to examine the effectiveness of BB use in this population.

Methods: We conducted a cohort study with the use of administrative databases of patients ≥ 65 years of age, alive on April 1, 2012 (index date) with a hospital discharge diagnosis of MI within the previous 3 years.

Assessment of Health Status and Barriers to Employment Among Medicaid Beneficiaries Not Meeting Work Requirements After Accounting for State Medical Frailty Exemptions.

This cross-sectional study quantifies the proportion of nonexempt Medicaid beneficiaries not meeting work requirements, compares their health with nonexempt beneficiaries fulfilling requirements, and assesses their inability to work for health-related reasons.

Aggregating multiple real-world data sources using a patient-centered health-data-sharing platform.

Real-world data sources, including electronic health records (EHRs) and personal digital device data, are increasingly available, but are often siloed and cannot be easily integrated for clinical, research, or regulatory purposes. We conducted a prospective cohort study of 60 patients undergoing bariatric surgery or catheter-based atrial fibrillation ablation at two U.S.

Patient and provider-level factors associated with changes in utilization of treatments in response to evidence on ineffectiveness or harm.

High-quality health care not only includes timely access to effective new therapies but timely abandonment of therapies when they are found to be ineffective or unsafe. Little is known about changes in use of medications after they are shown to be ineffective or unsafe. In this study, we examine changes in use of two medications: fenofibrate, which was found to be ineffective when used with statins among patients with Type 2 diabetes (ACCORD lipid trial); and dronedarone, which was found to be unsafe in patients with permanent atrial fibrillation (PALLAS trial).

Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017.

Importance: Since the introduction of the Fast Track designation in 1988, the number of special regulatory programs available for the approval of new drugs and biologics by the US Food and Drug Administration (FDA) has increased, offering the agency flexibility with respect to evidentiary requirements.

Objective: To characterize pivotal efficacy trials supporting the approval of new drugs and biologics during the past 3 decades.

Design, Setting, And Participants: This cross-sectional study included 273 new drugs and biologics approved by the FDA for 339 indications from 1995 to 1997, from 2005 to 2007, and from 2015 to 2017.

Medicare Prescription Drug Plan Coverage of Hormone Therapies Used by Transgender Individuals.

Although pharmacologic hormone therapy represents one of the mainstays of gender-affirming therapy for transgender individuals, there are many access barriers for these therapies, including insurance coverage of these drugs. The purpose of this study was to examine Medicare coverage of hormone therapies used by transgender individuals. Using Centers for Medicare and Medicaid Services prescription drug plan formulary files, we determined plan coverage, coverage restrictions, and out-of-pocket (OOP) costs for all 10 drugs recommended in the 2009 and 2017 Endocrine Society treatment guidelines for transgender patients.

Prostaglandin Coverage and Costs to Medicare and Medicare Beneficiaries, 2009-2017.

Background: Prostaglandin analogs are the most effective treatment for glaucoma, a common condition among older adults. Despite the availability of generic drugs, the costs associated with these prescription drugs are rising.

Objective: To characterize Medicare prescription drug plan (PDP) formulary coverage and beneficiary out-of-pocket cost for prostaglandin analogs from 2009 to 2017 and Medicare spending on prostaglandin analogs from 2013 to 2017.

Impact of left ventricular assist devices and heart transplants on acute myocardial infarction and heart failure mortality and readmission measures.

Background: Concern has been raised about consequences of including patients with left ventricular assist device (LVAD) or heart transplantation in readmission and mortality measures.

Methods: We calculated unadjusted and hospital-specific 30-day risk-standardized mortality (RSMR) and readmission (RSRR) rates for all Medicare fee-for-service beneficiaries with a primary diagnosis of AMI or HF discharged between July 2010 and June 2013. Hospitals were compared before and after excluding LVAD and heart transplantation patients.