Patient and Caregiver Experiences With and Perceptions of Risk Evaluation and Mitigation Strategy Programs With Elements to Assure Safe Use.

Importance: The US Food and Drug Administration (FDA) Amendments Act of 2007 authorized the FDA to impose safety requirements on drugs with important risks, such as prescriber certification or routine laboratory testing, to ensure that the benefits of use outweighed the risks. However, little is known about patient and caregiver experiences with these Risk Evaluation and Mitigation Strategy (REMS) programs with Elements to Assure Safe Use (ETASU).

Objective: To understand patient and caregiver experiences with and perceptions of REMS programs with ETASU.

Regulatory authority and clinical acceptability: Physicians' responses to regulatory drug safety warnings.

Aims: Medicines regulators issue post-market safety warnings to advise of newly uncovered risks, but with mixed impacts. We aimed to identify factors influencing the use of regulatory warnings by primary care and specialist physicians in the US and Australia.

Methods: Semi-structured qualitative interviews were carried out with 40 primary care physicians, endocrinologists and other generalist specialists in Boston (USA) and Australia.

The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care.

Background: The EMPA-REG OUTCOME trial showed that empagliflozin reduced the risk of cardiovascular death and hospitalization for heart failure (HHF) in diabetic patients with cardiovascular disease. EMPRISE is a study programme on the effectiveness, safety and healthcare utilization of empagliflozin in routine care, leveraging real-world data from two commercial and one federal US data sources from 2014 to 2019.

Objectives: To describe rationale and design of EMPRISE, assess ability to minimize confounding and evaluate the time to reach sufficient statistical power for a key study outcome, HHF, using baseline information from the first year of EMPRISE.

Mapping from the International Classification of Diseases (ICD) 9th to 10th Revision for Research in Biologics and Biosimilars Using Administrative Healthcare Data.

Purpose: The Centers for Medicare and Medicaid Services (CMS) mandated the transition from ICD-9 to ICD-10 codes on October 1, 2015. Postmarketing surveillance of newly marketed drugs, including novel biologics and biosimilars, requires a robust approach to convert ICD-9 to ICD-10 codes for study variables. We examined three mapping methods for health conditions (HCs) of interest to the Biologics and Biosimilars Collective Intelligence Consortium (BBCIC) and compared their prevalence.

Validation of claims-based algorithms for psoriatic arthritis.

Purpose: An increasing number of new medications are being developed and approved for psoriatic arthritis (PsA). To generate real-world evidence on comparative safety and effectiveness of these drugs, a claims-based algorithm that can accurately identify PsA is greatly needed.

Methods: To identify patients with PsA, we developed seven claims-based algorithms based on a combination of diagnosis codes and medication dispensing using the claims data from Medicare parts A/B/D linked to electronic medical records (2012-2014).

Evaluating utilization patterns of oral anticoagulants in routine care.

Unlabelled: Essentials Unlike warfarin, treatment with DOACs do not require regular plasma level monitoring. We compared persistence of patients treated with DOACs compared to warfarin. Persistence at 12 months was higher for all DOACs compared to warfarin.

Empagliflozin and the Risk of Heart Failure Hospitalization in Routine Clinical Care.

Background: The EMPA-REG OUTCOME trial (Empagliflozin Cardiovascular Outcome Event Trial in Type 2 diabetes Mellitus Patients) showed that empagliflozin, a sodium-glucose cotransporter-2 inhibitor, reduces the risk of hospitalization for heart failure (HHF) by 35%, on top of standard of care in patients with type 2 diabetes mellitus (T2D) and established cardiovascular disease. The EMPRISE (Empagliflozin Comparative Effectiveness and Safety) study aims to assess empagliflozin's effectiveness, safety, and healthcare utilization in routine care from August 2014 through September 2019. In this first interim analysis, we investigated the risk of HHF among T2D patients initiating empagliflozin versus sitagliptin, a dipeptidyl peptidase-4 inhibitor.

Risk of serious infections in tocilizumab versus other biologic drugs in patients with rheumatoid arthritis: a multidatabase cohort study.

Objective: To investigate the rate of serious bacterial, viral or opportunistic infection in patients with rheumatoid arthritis (RA) starting tocilizumab (TCZ) versus tumour necrosis factor inhibitors (TNFi) or abatacept.

Methods: Using claims data from US Medicare from 2010 to 2015, and IMS and MarketScan from 2011 to 2015, we identified adults with RA who initiated TCZ or TNFi (primary comparator)/abatacept (secondary comparator) with prior use of ≥1 different biologic drug or tofacitinib. The primary outcome was hospitalised serious infection (SI), including bacterial, viral or opportunistic infection.

Study protocol for the dabigatran, apixaban, rivaroxaban, edoxaban, warfarin comparative effectiveness research study.

Unlabelled: Since 2010, four oral anticoagulants have been approved for marketing in addition to warfarin for treatment of thromboembolic disease. Limited head-to-head data exist comparing these treatments, leaving patients and clinicians with little guidance for selecting a strategy that balances recurrence reduction with bleeding risk. In the dabigatran, apixaban, rivaroxban, edoxaban and warfarin comparative effectiveness research study, we compare all five currently available oral anticoagulant agents for the extended treatment of deep venous thrombosis and pulmonary embolism, as well as no extended treatment, and evaluate whether results differ in specific sub-populations.