Investigational Use of Real-World Data as a Hybrid Control in Pancreatic Ductal Adenocarcinoma from the Randomized Phase Ib/II MORPHEUS Trial.

Enrolling adequate numbers of patients into the control arm of randomized controlled trials (RCTs) often presents barriers. There is interest in leveraging real-world data (RWD) from electronic health records (EHRs) to construct external control (EC) arms to supplement RCT control arms and form hybrid control (HC) arms. This investigation showed the use of an HC arm in second-line metastatic pancreatic ductal adenocarcinoma (PDAC).

Real-World Progression-Free Survival as an Endpoint in Lung Cancer: Replicating Atezolizumab and Docetaxel Arms of the OAK Trial Using Real-World Data.

Evaluating cancer treatments in real-world data (RWD) requires informative endpoints. This study replicated the atezolizumab and docetaxel arms of the OAK trial using RWD and compared progression-free survival (PFS) outcomes derived from abstracted physician's notes in RWD (rwPFS) against PFS outcomes derived from the clinical trial PFS (ctPFS). Atezolizumab and docetaxel arms of the phase III OAK randomized controlled trial (RCT; NCT02008227) were replicated in a US nationwide real-world database using selected OAK inclusion/exclusion criteria and propensity score-based adjustment for baseline prognostic variables.

Hybrid-control arm construction using historical trial data for an early-phase, randomized controlled trial in metastatic colorectal cancer.

Background: Treatment for metastatic colorectal cancer patients beyond the second line remains challenging, highlighting the need for early phase trials of combination therapies for patients who had disease progression during or following two prior lines of therapy. Leveraging hybrid control design in these trials may preserve the benefits of randomization while strengthening evidence by integrating historical trial data. Few examples have been established to assess the applicability of such design in supporting early phase metastatic colorectal cancer trials.

Alpha-Fetoprotein as a Potential Surrogate Biomarker for Atezolizumab + Bevacizumab Treatment of Hepatocellular Carcinoma.

Purpose: Atezolizumab + bevacizumab is the new standard of care for systemic treatment-naïve, unresectable hepatocellular carcinoma (HCC). This exploratory study investigated on-treatment alpha-fetoprotein (AFP) response as a potential surrogate biomarker of prognosis for the combination therapy.

Experimental Design: Data from Group A of the phase Ib GO30140 study were used to identify the optimal time for AFP measurement and AFP cutoffs to differentiate patients by their best confirmed response per independent review facility-assessed RECIST (IRF-RECIST) version 1.

Natural History of Human Epidermal Growth Factor Receptor 2-Amplified and Human Epidermal Growth Factor Receptor 2 Wild-Type Refractory Metastatic Colorectal Cancer in US Clinical Practice.

Purpose: The molecular heterogeneity of metastatic colorectal cancer (mCRC) presents a therapeutic challenge, with few trials focused on patients with human epidermal growth factor receptor 2 amplification (HER2-Amp). Our limited understanding of real-world patterns and outcomes by HER2 status of treatment-refractory patients leaves treatment decisions with little contextual information. We conducted a retrospective cohort study to describe the natural disease history of patients with refractory mCRC using an electronic health record-derived database with oncogenomic information.

Building External Control Arms From Patient-Level Electronic Health Record Data to Replicate the Randomized IMblaze370 Control Arm in Metastatic Colorectal Cancer.

Purpose: External control (EC) arms derived from electronic health records (EHRs) can provide appropriate comparison groups when randomized control arms are not feasible, but have not been explored for metastatic colorectal cancer (mCRC) trials. We constructed EC arms from two patient-level EHR-derived databases and evaluated them against the control arm from a phase III, randomized controlled mCRC trial.

Methods: IMblaze370 evaluated atezolizumab with or without cobimetinib versus regorafenib in patients with mCRC.

"Real-world" outcomes and prognostic indicators among patients with high-risk muscle-invasive urothelial carcinoma.

Introduction: There is no current standard of care for patients with high-risk muscle-invasive urothelial carcinoma (MIUC) after neoadjuvant chemotherapy and surgical resection or for those who cannot receive or decline cisplatin-based perioperative chemotherapy. Understanding current, real-world treatment patterns may help inform decisions from clinical, research, and population health management perspectives. We examined real-world treatment patterns, survival outcomes, and prognostic factors among Medicare beneficiaries with high-risk MIUC who did not receive adjuvant treatment after surgical resection.

Real-world treatment patterns and adverse events in metastatic renal cell carcinoma from a large US claims database.

Background: Vascular endothelial growth factor (VEGF), tyrosine kinase (TK) and mechanistic target of rapamycin kinase (mTOR) inhibitors are common first-line (1 L) treatments for metastatic renal cell carcinoma (mRCC). Despite treatment availability, the 5-year survival rate in patients diagnosed at the metastatic stage is only ≈ 10%. To gain contemporary insights into RCC treatment trends that may inform clinical, scientific and payer considerations, treatment patterns and adverse events (AEs) associated with 1 L therapy were examined in a retrospective, longitudinal, population-based, observational study of patients with mRCC.

Vismodegib Use in Clinical Practice: Analysis of a United States Medical Claims Database.

Background: Information is limited on the use of vismodegib for treatment of advanced basal cell carcinoma beyond the setting of clinical trials.

Objective: To investigate the treatment patterns and characteristics of patients treated with vismodegib in clinical practice.

Methods: A longitudinal, retrospective cohort study was undertaken using data from a US commercial insurance claims (Truven Health Analytics MarketScan) database.

Effect of warning placement on the information processing of college students reading an OTC drug facts panel.

Objective: To compare three over-the-counter (OTC) Drug Facts panel versions for information processing optimization among college students.

Participants: University of Houston students (N = 210) participated in a cross-sectional survey from January to May 2010.

Methods: A current FDA label was compared to two experimental labels developed using the theory of CHREST to test information processing by re-positioning the warning information within the Drug Facts panel.

Risk of Nondherence to Diabetes Medications Among Medicare Advantage Enrollees: Development of a Validated Risk Prediction Tool.

Background: Low adherence to oral antidiabetic drugs (OADs) in the Medicare population can greatly reduce Centers for Medicare & Medicaid Services (CMS) star ratings for managed care organizations (MCOs).

Objective: To develop and validate a risk assessment tool (Prescription Medication Adherence Prediction Tool for Diabetes Medications [RxAPT-D]) to predict nonadherence to OADs using Medicare claims data.

Methods: In this retrospective observational study, claims data for members enrolled in a Medicare Advantage Prescription Drug (MA-PD) program in Houston, Texas, were used.

Importance of Risk Factors for Febrile Neutropenia Among Patients Receiving Chemotherapy Regimens Not Classified as High-Risk in Guidelines for Myeloid Growth Factor Use.

Background: Clinical practice guidelines recommend prophylaxis in patients with cancer receiving a colony-stimulating factor (CSF) when the risk of febrile neutropenia (FN) is high (>20%). For patients receiving chemotherapy regimens not documented as high-risk, the decision regarding CSF prophylaxis use can be challenging, because some patients may be at high risk based on a combination of the regimen and individual risk factors.

Methods: A retrospective cohort design and US private health care claims data were used.

Development of a conceptual model of health-related quality of life among hepatitis C patients: A systematic review of qualitative studies.

Aim: The Food and Drug Administration guidelines emphasize that patient-reported outcome (PRO) instruments used in clinical trials must be developed based on a conceptual model, yet existing PRO instruments currently used in clinical trials of hepatitis C virus (HCV) patients are not based on a predetermined model. The purpose of this study was to identify a comprehensive list of health-related quality of life (HRQoL) themes that may be unique to HCV by reviewing qualitative research articles of HCV patients. The information collected from the review was used to develop a preliminary model of HRQoL in HCV patients.

Assessing a conceptual model of over-the-counter medication misuse, adverse drug events and health-related quality of life in an elderly population.

Aim: The objectives of the present study were to test Spilker's quality of life model in the elderly population consuming over-the-counter (OTC) medications. It was hypothesized that OTC medication misuse increases adverse drug events (ADEs), ADEs as a result of OTC medication misuse decrease health-related quality of life (HRQoL) and the impact of OTC medication misuse on patients' HRQoL is fully medicated by ADEs associated with OTC medications.

Methods: Data were used from a previously carried out cross-sectional study using survey instruments with elderly patients consuming OTC medications in Houston, Texas, USA.

Impact of computerized provider order entry on pharmacist productivity.

Purpose: To examine the impact of computerized provider order entry (CPOE) implementation on average time spent on medication order entry and the number of order actions processed.

Methods: An observational time and motion study was conducted from March 1 to March 17, 2011. Two similar community hospital pharmacies were compared: one without CPOE implementation and the other with CPOE implementation.

Impact of over-the-counter medication use on patients' health-related quality of life: development and psychometric validation of Over-the-Counter Medication Impact Scale.

Background And Objective: Over-the-counter (OTC) medication use can impact an individual's health-related quality of life (HRQoL); however, there is no instrument specifically developed to measure the impact of OTC medication use on patients' HRQoL. The aim of the study was to develop an OTC Medication Impact Scale (OTC-MIS), a generic patient-reported outcome measure to assess the impact of OTC medication use on patients' HRQoL.

Methods: Items were generated based on the principles of the HRQoL theory, and were comparable to the domains of the Short-Form version 12 scale (SF-12).

Use of and attitude toward complementary and alternative medicine: understanding the role of generational influence.

Context: The prevalence of the use of complementary and alternative medicine (CAM) is increasing in developed countries, including the United States. Some patients use CAM without informing their physicians, subjecting themselves to potentially dangerous contraindications. Identifying patient populations that have an inclination to use CAM can help physicians provide patient-centered care, ensuring better health outcomes.