Showing papers in "Journal of Comparative Effectiveness Research in 2016"
TL;DR: This work evaluates evidence for their use and examine barriers to their uptake, and presents an evidence-based framework for the successful implementation of PROs in clinical practice.
Abstract: Patient-reported outcomes (PROs) are reports of the status of a patient's health condition that come directly from the patient. While PRO measures are a well-developed technology with robust standards in research, their use for informing healthcare decisions is still poorly understood. We review relevant examples of their application in the provision of healthcare and examine the challenges associated with implementing PROs in clinical settings. We evaluate evidence for their use and examine barriers to their uptake, and present an evidence-based framework for the successful implementation of PROs in clinical practice. We discuss current and future developments for the use of PROs in clinical practice, such as individualized measurement and computer-adaptive testing.
131 citations
TL;DR: It is believed that meaningful progress toward the goals behind the significant investments in patient-centered outcomes research and comparative effectiveness research will depend on a serious effort to address these issues.
Abstract: Policy makers have clearly indicated--through heavy investment in the Patient Centered Outcomes Research Institute--that reporting outcomes that are meaningful to patients is crucial for improvement in healthcare delivery and cost reduction. Better interpretation and generalizability of clinical research results that incorporate patient-centered outcomes research can be achieved by accelerating the development and uptake of core outcome sets (COS). COS provide a standardized minimum set of the outcomes that should be measured and reported in all clinical trials of a specific condition. The level of activity around COS has increased significantly over the past decade, with substantial progress in several clinical domains. However, there are many important clinical conditions for which high-quality COS have not been developed and there are limited resources and capacity with which to develop them. We believe that meaningful progress toward the goals behind the significant investments in patient-centered outcomes research and comparative effectiveness research will depend on a serious effort to address these issues.
72 citations
47 citations
TL;DR: This meta-epidemiological study provides indirect evidence that for knee osteoarthritis pain, the effects from exercise and from oral analgesics are comparable, and may support shared decision-making where a patient for some reason is unable to exercise or who consider exercise as unviable and analgesics as a more feasible choice.
Abstract: Aim: Evidence of comparative effectiveness of different treatment approaches is important for clinical decision-making, yet absent for most recommended treatments of knee osteoarthritis pain. The objective of this study was to estimate the comparative effectiveness of exercise versus orally administered analgesics for pain in patients with knee osteoarthritis. Methods: The Cochrane Database of systematic reviews was searched for meta-analyses of randomized controlled studies comparing exercise or analgesics with a control group (placebo or usual care) and with pain as an outcome. Individual study estimates were identified and effect sizes were calculated from group differences. We combined study-level effects on pain with a random effects meta-analysis and compared effect sizes between exercise trials and trials with analgesic interventions. Results: We included six Cochrane reviews (four pharmacology, two exercise). From these, 54 trials were eligible (20 pharmacology, 34 exercise), with 9806 participant...
38 citations
TL;DR: Engaging end-users in contextual inquiry resulted in CAPE (CHICAGO Action Plan after ED discharge), a new stakeholder-balanced discharge tool, which is being tested in a multicenter comparative effectiveness trial.
Abstract: Aim: To present the methods and outcomes of stakeholder engagement in the development of interventions for children presenting to the emergency department (ED) for uncontrolled asthma. Methods: We engaged stakeholders (caregivers, physicians, nurses, administrators) from six EDs in a three-phase process to: define design requirements; prototype and refine; and evaluate. Results: Interviews among 28 stakeholders yielded themes regarding in-home asthma management practices and ED discharge experiences. Quantitative and qualitative evaluation showed strong preference for the new discharge tool over current tools. Conclusion: Engaging end-users in contextual inquiry resulted in CAPE (CHICAGO Action Plan after ED discharge), a new stakeholder-balanced discharge tool, which is being tested in a multicenter comparative effectiveness trial.
24 citations
TL;DR: Direct adjustment by lasso regression and ensemble models involving multiple resamples have performance comparable to expert-based propensity scores and thus, may help provide real-time EHR-based evidence for timely clinical decisions.
Abstract: Aims: Electronic health records (EHR), containing rich clinical histories of large patient populations, can provide evidence for clinical decisions when evidence from trials and literature is absent. To enable such observational studies from EHR in real time, particularly in emergencies, rapid confounder control methods that can handle numerous variables and adjust for biases are imperative. This study compares the performance of 18 automatic confounder control methods. Methods: Methods include propensity scores, direct adjustment by machine learning, similarity matching and resampling in two simulated and one real-world EHR datasets. Results & conclusions: Direct adjustment by lasso regression and ensemble models involving multiple resamples have performance comparable to expert-based propensity scores and thus, may help provide real-time EHR-based evidence for timely clinical decisions. First draft submitted: 9 September 2015; Accepted for publication: 8 October 2015; Published online: 4 December 2015
23 citations
TL;DR: Findings provide actionable guidance across research and practice settings to promote and sustain widespread adoption of patient-reported outcomes across patient populations, healthcare settings and electronic health record systems.
Abstract: In recent years, patient-reported outcomes have become increasingly collected and integrated into electronic health records. However, there are few cross-cutting recommendations and limited guidance available in this rapidly developing research area. Our goal is to report key findings from a 2013 Patient-Centered Outcomes Research Institute workshop on this topic and a summary of actions that followed from the workshop, and present resulting recommendations that address patient, clinical and research/quality improvement barriers to regular use. These findings provide actionable guidance across research and practice settings to promote and sustain widespread adoption of patient-reported outcomes across patient populations, healthcare settings and electronic health record systems.
19 citations
TL;DR: A pragmatic framework of collaborative engagement and partnership between research investigators and patient andfamily advisors from existing patient and family advisory councils (PFACs) at an academic medical center is outlined.
Abstract: In response to the creation of the Patient-Centered Outcomes Research Institute in 2010, researchers have begun to incorporate patient and family stakeholders into the research process as equal partners, bringing their unique perspectives and experiences to the table. Nonetheless, there is a dearth of literature around how best to engage patients and families and many barriers to doing so effectively. This paper outlines a pragmatic framework of collaborative engagement and partnership between research investigators and patient and family advisors from existing patient and family advisory councils (PFACs) at an academic medical center. This framework includes the role for each party throughout the clinical research process (launch, hypothesis, specific aims, measures/methods, results, interpretations/recommendation and dissemination).
14 citations
TL;DR: This real-world study demonstrated that adalimumab and etanercept are equal alternatives for RA treatment and both were well tolerated.
Abstract: Aim: Biological disease-modifying antirheumatic drugs (bDMARDs) are used to treat rheumatoid arthritis (RA) with adalimumab and etanercept the most used bDMARDs in Brazil. This open prospective cohort study evaluated their effectiveness and safety among RA patients in the Brazilian Public Health System given their costs. Methods: The Clinical Disease Activity Index was primarily used to assess their effectiveness after 6 and 12 months of follow-up. The Health Assessment Questionnaire and EuroQol-5D were also used. Results: A total of 266 RA patients started treatment with adalimumab or etanercept. Adalimumab was the most widely used bDMARD (70%). In total, 46% achieved remission or low-disease activity at 12 months with no difference in effectiveness between them (p = 0.306). bDMARDs were more effective in patients who had better functionality at treatment onset and had spent longer in education. Conclusion: This real-world study demonstrated that adalimumab and etanercept are equal alternatives for RA tr...
14 citations
TL;DR: Findings suggest theTCM is more effective than ASC, however, potential effects of the RNC relative to the TCM warrant further study.
Abstract: Aim: Compare within site effects of three interventions designed to enhance outcomes of hospitalized cognitively impaired elders. Methods: Prospective, nonrandomized, confirmatory phased study. In Phase I, 183 patients received one of three interventions: augmented standard care (ASC), resource nurse care (RNC) or Transitional Care Model (TCM). In Phase II, 205 patients received the TCM. Results: Time to first rehospitalization or death was longer for the TCM versus ASC group (p = 0.017). Rates for total all-cause rehospitalizations and days were significantly reduced in the TCM versus ASC group (p < 0.001, both). No differences were observed between RNC versus TCM. Conclusion: Findings suggest the TCM is more effective than ASC. However, potential effects of the RNC relative to the TCM warrant further study.
13 citations
TL;DR: Future work is needed to systematically assess the impact of PCTs on clinicians in meeting their ethical obligations to patients and the burdens clinicians are willing to accept in exchange for potential benefits.
Abstract: Aim: Practicing physicians inevitably become involved in pragmatic clinical trials (PCTs), including comparative effectiveness research. We sought to identify physicians’ perspectives related to PCTs. Methods: In-depth semistructured interviews with 20 physicians in the USA. Results: Although physicians are generally willing to participate in PCTs, their support is predicated on several factors including expected benefits, minimization of time and workflow burdens and physician engagement. Physicians communicated a desire to respect patients’ rights and interests while maintaining a high level of care. Conclusion: Future work is needed to systematically assess the impact of PCTs on clinicians in meeting their ethical obligations to patients and the burdens clinicians are willing to accept in exchange for potential benefits.
TL;DR: Despite similar survival outcomes between SX versus TA, management of T1a renal cell carcinoma lesions may be influenced by a variety of nonclinical sociodemographic factors.
Abstract: Aim: To investigate national practice trends in the use of surgical resection (SX) versus thermal ablation (TA) for the management of T1aN0M0 renal cell carcinoma with regard to sociodemographic factors and associated outcomes. Materials & methods: Patients diagnosed in 2004–2011 were identified using the Surveillance, Epidemiology and End Results registry. Sociodemographic factors predicting the use of surgery versus TA were determined using logistic regression. Cancer-specific survival was estimated using Kaplan–Meier method. Results: Overall, 19,136 (92.9%) patients underwent SX versus 1468 (7.1%) TA. Patients who were unmarried, diagnosed between 2008 and 2011, Caucasian, aged ≥70 years, insured, residing in Pacific Coast and Northern Plains, and in metropolitan areas with higher median family income had higher likelihoods of undergoing TA. Age-adjusted cancer-specific survival was similar in the two groups. Conclusion: Despite similar survival outcomes between SX versus TA, management of T1a renal ce...
TL;DR: This work outlines the common investigations used in STEMI and their role in risk assessment of patients with an acute STEMI, and suggests that during emergency care increasing access to tools can provide additional information helping to further risk stratify patients.
Abstract: ST-elevation myocardial infarction (STEMI) is one of the leading causes of mortality and morbidity worldwide. While the survival after acute STEMI has considerably improved, mortality rate still remains high, especially in high-risk patients. Survival after acute STEMI is influenced by clinical characteristics such as age as well as the presence of comorbidities. However, during emergency care increasing access to tools such as the electrocardiogram, chest x-ray and echocardiography can provide additional information helping to further risk stratify patients. In the invasive setting, this can also include coronary angiography, invasive hemodynamic recordings and angiographic assessments of coronary flow and myocardial perfusion. We outline the common investigations used in STEMI and their role in risk assessment of patients with an acute STEMI.
TL;DR: Neither the augmented nor basic intervention was more effective than usual care in improving BP control, systolic BP, medication intensification or patient self-management, creating a high comparative effectiveness threshold.
Abstract: Aim: Assess the comparative effectiveness of two blood pressure (BP) control interventions for black patients with uncontrolled hypertension. Patients & methods: A total of 845 patients were enrolled in a three-arm cluster randomized trial. On admission of an eligible patient, field nurses were randomized to usual care, a basic or augmented intervention. Results: Across study arms there were no significant 12 months differences in BP control rates (primary outcome) (25% usual care, 26% basic intervention, 22% augmented intervention); systolic BP (143.8 millimeters of mercury [mmHg], 146.9 mmHG, 143.9 mmHG, respectively); medication intensification (47, 43, 54%, respectively); or self-management score (18.7, 18.7, 17.9, respectively). Adjusted systolic BP dropped more than 10 mmHg from baseline to 12 months (155.5–145.4 mmHg) among all study participants. Conclusion: Neither the augmented nor basic intervention was more effective than usual care in improving BP control, systolic BP, medication intensificat...
TL;DR: It is shown that incisional and other surgical procedures are effective treatments for patients with open-angle glaucoma and there were no differences in treatment response by race or ethnicity.
Abstract: Aims: The RiGOR study's primary outcome measure was a 15% reduction in intraocular pressure (IOP) for patients with open-angle glaucoma at 1 year. Methods: Patients received treatment according to the ophthalmologist's usual practice. Results: A higher proportion of patients in the incisional and other surgery group achieved a 15% reduction in IOP than in the laser surgery or additional medication groups (82, 57, and 57% respectively). In multivariate regression analyses, incisional surgery patients were 2.7-times as likely as patients treated with additional medication to achieve a 15% reduction in IOP (odds ratio: 2.67; 95% CI: 2.01–3.57). Conclusion: Incisional and other surgical procedures are effective treatments. There were no differences in treatment response by race or ethnicity
TL;DR: Five annual surveys of healthcare decision-makers potentially affected by comparative effectiveness research (CER) indicate sustained recognition of its importance and potential impact, but greater emphasis on translating and disseminating research findings is needed.
Abstract: Five annual surveys of healthcare decision-makers potentially affected by comparative effectiveness research (CER) indicate sustained recognition of its importance and potential impact. Initial expectations of immediate CER impact have over time turned to stakeholder assessments of little short-term impact. In successive surveys, they project a continuous horizon of 3-5 years for CER to have a moderate or substantial improvement in decision making. The prominence of Patient-Centered Outcomes Research Institute in CER is highlighted by stakeholders, but greater emphasis on translating and disseminating research findings is needed, a role in which Agency for Healthcare Research and Quality is expected to contribute. Stakeholders, including patients, must be engaged throughout to ensure that findings provide the flexibility for decision makers to apply them to their patient populations.
TL;DR: A shift toward increased prescribing generic statins is recommended to minimize costs and improve access in patients with hypertension in Nigeria.
Abstract: Background: Statins reduce cardiovascular risk, especially in patients with hypertension due to their concomitant blood pressure reducing effects. Prescribing generic statins minimizes cost and improves access. Aims: Ascertain current prescribing of statins in Nigeria and potential savings from the increased use of generic statins. Methods: Prospective study involving hypertensive patients attending University College Hospital (Ibadan, Nigeria). Results: In total, 228 hypertensive patients received statins. Atorvastatin was the most prescribed statin, followed by simvastatin, rosuvastatin and finally fluvastatin. Prescribed doses were less than one defined daily dose in the majority, with high use of originators. Average monthly potential savings from increased prescribing of generic statins was US$2635 for atorvastatin and US$10,578 for rosuvastatin. Conclusion: A shift toward increased prescribing generic statins is recommended to minimize costs.
TL;DR: In this article, the authors compared the effectiveness of primary percutaneous coronary intervention (pPCI) and fibrinolytic therapy (FL) for the acute management of ST elevation myocardial infarction (STEMI).
Abstract: Aim: To compare the effectiveness of primary percutaneous coronary intervention (pPCI) and fibrinolytic therapy (FL) for the acute management of ST elevation myocardial infarction (STEMI). Methods: A review of guidelines and PubMed literature comparing clinical outcomes of patients with STEMI treated with pPCI or FL. Results: Earlier trials reported reduced mortality and reinfarction with pPCI. Recent randomized data suggest similar outcomes for delayed pPCI compared with FL, especially in geographically remote areas. Guidelines recommend pPCI as the preferred reperfusion strategy for STEMI, if available within 120 mins of first medical contact. Conclusion: pPCI is the preferred treatment strategy for STEMI. However, FL with subsequent percutaneous coronary intervention remains a viable option for those in rural areas.
TL;DR: Compared with the other commonly used antibiotics, matched patients in the ceftaroline fosamil treatment group had an equivalent or lower in-hospital mortality rate, and significantly lower average unadjusted and regression-adjusted length of stay and inpatient costs.
Abstract: Aim: Compare clinical and cost outcomes associated with ceftaroline fosamil with other commonly used antibiotics in complicated skin and soft tissue infections. Methods: Retrospective analysis of hospital records from 2010 to 2013 in Premier’s Perspective comparative database for adults with complicated skin and soft tissue infection treated with intravenous ceftaroline fosamil, vancomycin, daptomycin, linezolid or tigecycline. Length of stay, inpatient costs and mortality were compared between propensity score-matched treatment groups. Results & conclusion: Compared with the other commonly used antibiotics, matched patients in the ceftaroline fosamil treatment group had an equivalent (1%) or lower (compared with linezolid, 2%) in-hospital mortality rate, and significantly lower (p < 0.001) average unadjusted and regression-adjusted length of stay and inpatient costs (savings of $3398.80 compared with daptomycin).
TL;DR: There was no differential risk of hip fractures between paroxetine and other SSRIs, and future studies are needed to evaluate other anticholinergic effects of parxetine.
Abstract: Aim: To evaluate comparative safety of paroxetine and other selective serotonin reuptake inhibitors (SSRIs) for the risk of hip fractures. Patients & methods: A propensity score-matched retrospective cohort study was conducted using 2007–2010 Minimum Data Set linked Medicare data. Robust Cox proportional hazards model was used to evaluate the risk of hip fractures in depressed elderly nursing home residents. Results: Cox analysis did not find any significant difference in the risk of hip fractures for the paroxetine users (hazard ratio: 1.09; 95% CI: 0.91–1.32) when compared with other SSRIs. Results from the sensitivity analysis supported the main findings. Conclusion: There was no differential risk of hip fractures between paroxetine and other SSRIs. Future studies are needed to evaluate other anticholinergic effects of paroxetine.
TL;DR: A Markov decision analysis model to represent two alternative treatment approaches for prostate cancer after prostatectomy over a 10-year time horizon was designed and results were sensitive to genomics-based estimates of cancer recurrence and to nonprostate cancer mortality.
Abstract: Aim: We developed a decision analysis framework to simulate the clinical choice of early adjuvant versus delayed salvage radiation therapy after radical prostatectomy. Materials & methods: We designed a Markov decision analysis model to represent two alternative treatment approaches for prostate cancer after prostatectomy over a 10-year time horizon. The model contained individualized inputs including genomic classifier score. Sensitivity analyses were performed to evaluate model results. Results: Observation with delayed salvage radiation is preferred according to the base case, with greater average length and quality of life. However, adjuvant therapy is preferred over observation with salvage when genomics-based estimates of recurrence are high. Conclusion: Model results were sensitive to genomics-based estimates of cancer recurrence and to nonprostate cancer mortality.
TL;DR: Perceived positive outcomes included diversity of perspectives provided, better understanding their own health and decision-making and improving CER.
Abstract: Aim: Explore perspectives of adolescent and young adult (AYA) and parent stakeholders regarding their engagement in comparative effectiveness research (CER) evaluating cholesterol screening and treatment strategies for 17–21 year olds. Methods: All nine AYAs and parent stakeholders participating in a 20-member panel of AYAs, parents and professionals (i.e., clinicians, researchers, policy makers, payers), completed a quantitative survey and a semistructured interview at the completion of the core CER study. Results & Conclusion: AYAs and parents stakeholders emphasized the role of power differentials regarding shared knowledge, relationships and trust, and logistics. To mitigate power differentials, stakeholders recommended more materials, clearer definition of roles and in-person meetings. Perceived positive outcomes included diversity of perspectives provided, better understanding their own health and decision-making and improving CER.
TL;DR: DEB-TACE in patients with unresectable AJCC stage I/II HCC was a significant independent prognostic factor for greater OS in a population-based study.
Abstract: Aim: To evaluate overall survival (OS) in unresectable American Joint Committee on Cancer (AJCC) stage I/II hepatocellular carcinoma (HCC) treated with drug-eluting-bead transarterial chemoembolization (DEB-TACE) versus best supportive care. Materials & methods: OS in consecutive patients with AJCC stage I/II unresectable HCC diagnosed in 2005–2010 who underwent DEB-TACE and similar patients from SEER with no surgery/radiation recommended/performed was evaluated. Results: Median OS from HCC diagnosis was 28.9 months (DEB-TACE) versus 10.0 months (SEER), p < 0.0001. Median OS was 36.3 months (DEB-TACE) versus 12.0 months (SEER) in AJCC I, and 27.9 months (DEB-TACE) versus 10.0 months (SEER) in AJCC II, p < 0.0001. Significant independent prognostic factors for OS were single primary tumor, no vascular invasion, normal α-fetoprotein and DEB-TACE. Conclusion: DEB-TACE in patients with unresectable AJCC stage I/II HCC was a significant independent prognostic factor for greater OS in a population-based study.
TL;DR: A vision where big data can be used not only to retrospectively assess the quality of oncologic care, but help physicians deliver high-quality care in real time is outlined.
Abstract: There is increasing attention in the US healthcare system on the delivery of high-quality care, an issue central to oncology. In the report ‘Crossing the Quality Chasm’, the Institute of Medicine identified six aims for improving healthcare quality: safe, effective, patient-centered, timely, efficient and equitable. This article describes how current big data resources can be used to assess these six dimensions, and provides examples of published studies in oncology. Strengths and limitations of current big data resources for the evaluation of quality of care are also discussed. Finally, this article outlines a vision where big data can be used not only to retrospectively assess the quality of oncologic care, but help physicians deliver high-quality care in real time.
TL;DR: Government should see public funding of pragmatic practice-oriented clinical trials as a good opportunity to improve the selection and quality of treatments and stimulate efficient use of limited resources.
Abstract: Aim: Many questions of relevance to patients/society are not answered by industry-sponsored clinical trials. We consider whether there are benefits to governments in funding practice-oriented clinical trials. Methodology: A literature search including publications on institutions’ websites was performed and supplemented with information gathered from (inter)national stakeholders. Results: Areas were identified where public funding of clinical trials is of importance for society, such as head-to-head comparisons or medical areas where companies have no motivation to invest. The available literature suggests publicly funded research programs could provide a positive return on investment. The main hurdles (e.g., sufficient funding and absence of equipoise) and success factors (e.g., selection of research questions and research infrastructure) for the successful conduct of publicly funded trials were identified. Conclusion: Governments should see public funding of pragmatic practice-oriented clinical trials a...
TL;DR: The incidence and hospital costs associated with hypoglycemic episodes (HEs) requiring hospital admission or emergency room (ER) visits in Denmark are estimated to be €3.0 million in 2008 and €2.3 million in 2011, respectively.
Abstract: Aims: The purpose of this study was to estimate the incidence and hospital costs associated with hypoglycemic episodes (HEs) requiring hospital admission or emergency room (ER) visits in Denmark. Materials & methods: This study analyzed data from the National Patient Registry. Data on HE-related hospital admissions or ER visits occurring between 2008 and 2011 were collected and analyzed. Results: There were 1906 hospital admissions and 803 ER visits in 2008 compared with 1646 hospital admissions and 547 ER visits in 2011, corresponding to a decrease in incidence from 10.6 to 7.1. The estimated annual total hospital costs ranged from €3.0 million in 2008 to €2.3 million in 2011. Conclusion: HEs represent a major burden for the Danish healthcare system.
TL;DR: With its high efficacy and improved tolerability, DCV + SOF is an important treatment for hepatitis C genotype 3.
Abstract: Aims: To compare the efficacy and tolerability of daclatasvir and sofosbuvir (DCV + SOF) versus SOF and ribavirin (SOF + R) and versus peginterferon-alfa plus ribavirin (A/R) in patients infected with hepatitis C genotype 3. Patients & methods: Clinical trials of SOF + R or A/R were identified in systematic literature reviews. The DCV+SOF population was adjusted via propensity score weighting to match average baseline characteristics to those reported for the comparator regimens. Results: The SVR12 rate was similar between DCV + SOF and SOF + R, and significantly higher with DCV + SOF than A/R. Rates of discontinuation due to AEs were similar or significantly lower in patients treated with DCV + SOF than SOF + R or A/R. Conclusion: With its high efficacy and improved tolerability, DCV + SOF is an important treatment for hepatitis C genotype 3.
TL;DR: For 36% of patients, a treatment cascade involves two or more therapies over a year, demonstrating the complex nature of open-angle glaucoma treatment.
Abstract: Aims: The RiGOR study provides a current picture of the types of glaucoma treatment over 12 months. Methods: Patients were identified and enrolled at the time of decision to proceed with laser surgery procedure or other procedure such as incisional surgery or drainage device implantation, or initiation of a new or additional course of therapy with medication for glaucoma treatment. Results: The most frequent type of treatments were prostaglandin analogues (60%) among patients with additional medication, selective laser trabeculoplasty (87%) among patients with laser surgery and trabeculectomy (57%) among patients with incisional surgery. Conclusion: For 36% of patients, a treatment cascade involves two or more therapies over a year. This demonstrates the complex nature of open-angle glaucoma treatment.