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Raghu G.,University of Washington | Chen S.-Y.,Biogen Idec | Yeh W.-S.,Biogen Idec | Maroni B.,Biogen Idec | And 3 more authors.
The Lancet Respiratory Medicine | Year: 2014

Background: Published data for the epidemiology of idiopathic pulmonary fibrosis in the USA are scarce. We sought to estimate the incidence, prevalence, and mortality risk of idiopathic pulmonary fibrosis among US Medicare beneficiaries. Methods: We used administrative claims from a 5% random sample of Medicare beneficiaries (aged 65 years and older) from the years 2000-11 as a data source. Idiopathic pulmonary fibrosis was defined by International Classification of Diseases, Ninth Revision, Clinical Modification diagnosis codes. We estimated annual incidence and cumulative prevalence of idiopathic pulmonary fibrosis, median survival time of patients, and potential risk factors for diagnosis of idiopathic pulmonary fibrosis and death between 2001 and 2011. We also estimated incidence and prevalence using more restrictive algorithms for diagnosis. Findings: The annual incidence of idiopathic pulmonary fibrosis in the Medicare population remained stable between 2001 and 2011, with an overall estimate of 93·7 cases per 100 000 person-years (95% CI 91·9-95·4) across the study period. The annual cumulative prevalence increased steadily from 202·2 cases per 100 000 people in 2001 to 494·5 cases per 100 000 people in 2011. Among newly diagnosed patients with Medicare (mean age 79·4 years [SD 7·2], 54% female, 91% white), the median survival time was 3·8 years (95% CI 3·5-3·8). Older age and male sex were associated with a higher incidence of disease and shorter survival time after diagnosis. Mortality risk was lower in patients diagnosed in more recent years (median survival time 3·3 years [95% CI 3·0-3·8] in 2001 vs 4·0 years [3·8-4·5] in 2007). Interpretation: The incidence and prevalence of idiopathic pulmonary fibrosis in people aged 65 years and older in the USA are substantially higher than previously reported, and prevalence is increasing annually, even in the subgroups based on more restrictive algorithms for diagnosis. Patients with idiopathic pulmonary fibrosis aged 65 years and older were living longer in 2011 than they were 10 years before, which could partly account for the steady increase in prevalence. Funding: Biogen Idec. © 2014 Elsevier Ltd.

Kistler K.D.,Evidera | Nalysnyk L.,Genzyme | Rotella P.,Evidera | Esser D.,Boehringer Ingelheim GmbH
BMC Pulmonary Medicine | Year: 2014

Background: Idiopathic pulmonary fibrosis (IPF) is a distinct form of interstitial pneumonia with unknown origin and poor prognosis. Current pharmacologic treatments are limited and lung transplantation is a viable option for appropriate patients. The aim of this review was to summarize lung transplantation survival in IPF patients overall, between single (SLT) vs. bilateral lung transplantation (BLT), pre- and post Lung Allocation Score (LAS), and summarize wait-list survival. Methods: A systematic review of English-language studies published in Medline or Embase between 1990 and 2013 was performed. Eligible studies were those of observational design reporting survival post-lung transplantation or while on the wait list among IPF patients. Results: Median survival post-transplantation among IPF patients is estimated at 4.5 years. From ISHLT and OPTN data, one year survival ranged from 75% - 81%; 3-year: 59% - 64%; and 5-year: 47% - 53%. Post-transplant survival is lower for IPF vs. other underlying pre-transplant diagnoses. The proportion of IPF patients receiving BLT has steadily increased over the last decade and a half. Unadjusted analyses suggest improved long-term survival for BLT vs. SLT; after adjustment for patient characteristics, the differences tend to disappear. IPF patients account for the largest proportion of patients on the wait list and while wait list time has decreased, the number of transplants for IPF patients has increased over time. OPTN data show that wait list mortality is higher for IPF patients vs. other diagnoses. The proportion of IPF patients who died while awaiting transplantation ranged from 14% to 67%. While later transplant year was associated with increased survival, no significant differences were noted pre vs. post LAS implementation; however a high LAS vs low LAS was associated with decreased one-year survival. Conclusions: IPF accounts for the largest proportion of patients awaiting lung transplants, and IPF is associated with higher wait-list and post-transplant mortality vs. other diagnoses. Improved BLT vs. SLT survival may be the result of selection bias. Survival pre- vs. post LAS appears to be similar except for IPF patients with high LAS, who have lower survival compared to pre-LAS. Data on post-transplant morbidity outcomes are sparse. © 2014 Kistler et al.

Leidy N.K.,Evidera | Murray L.T.,Evidera | Jones P.,St George's, University of London | Sethi S.,State University of New York at Buffalo
Annals of the American Thoracic Society | Year: 2014

Rationale: The EXAcerbations of Chronic Pulmonary Disease Tool (EXACT) is a patient-reported outcome measure to standardize the symptomatic assessment of chronic obstructive pulmonary disease exacerbations, including reported and unreported events. The instrument has been validated in a short-term study of patients with acute exacerbation and stable disease; its performance in longer-term studies has not been assessed. Objectives: To test the EXACT's performance in three randomized controlled trials and describe the relationship between resource-defined medically treated exacerbations (MTEs) and symptom (EXACT)-defined events. Methods: Prespecified secondary analyses of data from phase II randomized controlled trials testing new drugs for the management of chronic obstructive pulmonary disease: one 6-month trial (United States) (n = 235) and two 3-month, multinational trials (AZ 1 [n = 749], AZ 2 [n = 597]). In each case, the experimental drugs were found to be ineffective, permitting assessment of the EXACT's performance in three independent studies of moderate to severe high-risk patients on maintenance therapies. Measurements and Main Results: The mean age of subjects was 62 to 64 years; 48 to 76% were male. Mean FEV1 % predicted was 42 to 59%. EXACT scores exhibited internal consistency (Cronbach's α ≥ 0.90), reproducibility (intraclass correlation > 0.70), correlation with St. George's Respiratory Questionnaire (Spearman rho [rs] = 0.62, 0.46, 0.46 in the three trials; P < 0.001), and Breathlessness Cough and Sputum Scale (AZ 1, rs = 0.83; AZ 2, rs = 0.83; P < 0.001). EXACT-defined events had a high correspondence with alternative indicators of worsening (94, 88, and 93%). In each trial, unreported events were similar in severity (mean EXACT score, 56, 57, 61 vs. 53, 54 [P < 0.05], 57 [P < 0.05], respectively; 100-point scale) and longer (median, 9, 8, 7 vs. 8, 7 [P < 0.01], 6 days, respectively) than moderate MTEs. Conclusions: Data generated through the EXACT offers insight into the symptomatic nature of MTEs and the frequency, severity, and duration of unreported symptom-defined events. Clinical trials registered with www.clinicaltrials.gov (MPEX: NCT00739648; AZ 1: NCT00949975; AZ 2: NCT01023516). Copyright © 2014 by the American Thoracic Society.

Background: The aim was to assess the cost impact of daptomycin compared to vancomycin treatment in patients hospitalised for complicated skin and soft-tissue infection (cSSTI) with suspected methicillin-resistant Staphylococcus aureus infection in the UK. Methods: A decision model was developed to estimate the costs associated with cSSTI treatment. Data on efficacy, treatment duration and early discharge from published clinical trials were used, with data gaps on standard clinical practice being filled by means of clinician interviews. Results: Total health-care costs per patient were GBP 6,214 and GBP 6,491 for daptomycin and vancomycin, respectively. A sensitivity analysis suggested that modifying the parameters within a reasonable range does not impact on the conclusion that the higher cost of daptomycin is likely to be offset by lower costs of monitoring and hospitalisation. Conclusions: This study demonstrates that daptomycin not only provides an alternative treatment for multiple resistant infections, but may also reduce National Health Service costs. © 2014 S. Karger AG, Basel.

Coyne K.S.,Evidera | Thompson C.L.,Evidera | Lai J.-S.,Northwestern University | Sexton C.C.,Evidera
Neurourology and Urodynamics | Year: 2015

Aims The Overactive Bladder Questionnaire (OAB-q) has demonstrated robust psychometric properties in continent and incontinent OAB patients. However, there is a need for a short-form of this instrument for settings where completing the full OAB-q may be too burdensome. The purpose of this manuscript is to describe the validation of the OAB-q short-form. Methods Three studies were used to derive and validate the OAB-q SF: a 12-week, multicenter, open-label clinical trial of tolterodine ER (N-=-865 incontinent OAB [I-OAB]; the "Noble Nested Case-Control" [NCC] study; N-=-523 healthy controls; N-=-396 OAB); and a test-retest validation study (N-=-47). Rasch analysis and confirmatory factor analysis (CFA) were performed to assess the subscale structure, and the psychometric performance of the resulting scales was evaluated. Results Based on the Rasch analysis, 6-items were retained in the OAB-q SF Symptom Bother Scale and 13-items were retained in the HRQL scale. CFAs showed excellent model fit and internal consistency in the study populations. Both scales demonstrated good convergent validity, discriminant validity, internal reliability, reproducibility, and responsiveness to change. The OAB-q SF scales clearly differentiated among I-OAB, C-OAB, and healthy controls. Conclusion The OAB-q SF captures the full spectrum of OAB Symptom Bother and HRQL impact with good reliability, validity, and responsiveness, while being less time-consuming for patients to complete. © 2014 Wiley Periodicals, Inc.

Revicki D.A.,Evidera | Kleinman L.,Evidera | Cella D.,Northwestern University
Dialogues in Clinical Neuroscience | Year: 2014

Health-related quality of life (HRQoL) is a multidimensional concept that includes subjective reports of symptoms, side effects, functioning in multiple life domains, and general perceptions of life satisfaction and quality. Rather than estimating it from external observations, interview, or clinical assessment, it is best measured by direct query. Due to a perception that respondents may not be reliable or credible, there has been some reluctance to use self-report outcomes in psychiatry. More recently, and increasingly, HRQoL assessment through direct patient query has become common when evaluating a range of psychiatric, psychological, and social therapies. With few exceptions, psychiatric patients are credible and reliable reporters of this information. This article summarizes studies that highlight the development, validation, and application of HRQoL measures in psychiatry. Thoughtful application of these tools in psychiatric research can provide a much-needed patient perspective in the future of comparative effectiveness research, patient-centered outcomes research, and clinical care. © 2014, AICH.

Kansal A.R.,Evidera | Zheng Y.,Evidera | Pokora T.,Evidera | Sorensen S.V.,Evidera
Best Practice and Research: Clinical Haematology | Year: 2013

Atrial fibrillation (AF) is a common arrhythmia and the leading cause of stroke, an event with high human and economic burden. Novel oral anticoagulants have been approved in many markets as alternatives to warfarin for stroke prevention in patients with AF - dabigatran etexilate, apixaban and rivaroxaban. Given the high burden of AF, and given that new treatments can more effectively prevent stroke than warfarin, but at higher drug cost, there has been a need for systematic evaluation of the costs and benefits of these new treatments. In this study, we summarize the findings of a systematic literature review on the cost-effectiveness of the new oral anticoagulants. We find that there is substantial heterogeneity between the studies and their numerical findings, despite using a common set of four trials for their clinical inputs. However, there is broad consensus among them that each of the novel oral anticoagulants is cost-effective versus warfarin or aspirin. © 2013 Elsevier Ltd. All rights reserved.

Blieden M.,Evidera | Paramore L.C.,Evidera | Shah D.,Purdue Pharma | Ben-Joseph R.,Purdue Pharma
Expert Review of Clinical Pharmacology | Year: 2014

Acetaminophen is a commonly-used analgesic in the US and, at doses of more than 4 g/day, can lead to serious hepatotoxicity. Recent FDA and CMS decisions serve to limit and monitor exposure to high-dose acetaminophen. This literature review aims to describe the exposure to and consequences of high-dose acetaminophen among chronic pain patients in the US. Each year in the US, approximately 6% of adults are prescribed acetaminophen doses of more than 4 g/day and 30,000 patients are hospitalized for acetaminophen toxicity. Up to half of acetaminophen overdoses are unintentional, largely related to opioid-acetaminophen combinations and attempts to achieve better symptom relief. Liver injury occurs in 17% of adults with unintentional acetaminophen overdose. © 2014 Informa UK, Ltd.

Ward A.,Evidera | Alvarez P.,Evidera | Vo L.,Janssen Scientific Affairs LLC | Martin S.,Janssen Scientific Affairs LLC
Journal of Medical Economics | Year: 2014

Objective: To estimate the direct medical costs associated with managing complications, hypoglycemia episodes, and infections associated with type 2 diabetes expressed in 2012 United States dollars (USD). Methods: Direct data analysis and microcosting were used to estimate the costs for an event leading to either a hospital admission or outpatient care, and the post-acute care associated with managing macrovascular and microvascular complications, hypoglycemia episodes, and infections. Data were obtained from many sources, including inpatient and emergency department databases, national physician and laboratory fee schedules, government reports, and literature. Event-year costs reflect the resource use during an acute care episode (initial management in an inpatient or outpatient setting) and any subsequent care provided in the first year. State costs reflect annual resource use required beyond the first year for the ongoing management of complications and other conditions. Costs were assessed from the perspective of a comprehensive US healthcare payer and expressed in 2012 USD. Results: Event-year costs (and state costs) for macrovascular complications were as follows: myocardial infarction $56,445 ($1904); ischemic stroke $42,119 ($15,541); congestive heart failure $23,758 ($1904); ischemic heart disease $21,406 ($1904); and transient ischemic attack $7388 ($179). For two microvascular complications the event-year and state costs were assumed the same: $71,714 for end stage renal disease, and $2862 blindness. The event-year cost was $9041 for lower extremity amputations, and $2147 for diabetic foot ulcers. Costs were also determined for managing hypoglycemic episodes: $176-$16,478 (depending on treatment required), and infections: vulvovaginal candidiasis $111, lower urinary tract infection $105. Conclusions: This study, which provides up-to-date cost estimates per patient, found that managing macrovascular and microvascular complications results in substantial costs to the healthcare system. This study facilitates conduct of other research studies such as modeling the management of diabetes and estimating the economic burden associated with complications. © 2014 All rights reserved.

Yeung C.,Evidera | Dinh T.,Evidera | Lee J.,Evidera
PharmacoEconomics | Year: 2014

The purpose of this paper is to provide a current view of the economic burden of bladder cancer, with a focus on the cost effectiveness of available interventions. This review updates a previous systematic review and includes 72 new papers published between 2000 and 2013. Bladder cancer continues to be one of the most common and expensive malignancies. The annual cost of bladder cancer in the USA during 2010 was $US4 billion and is expected to rise to $US5 billion by 2020. Ten years ago, urinary markers held the potential to lower treatment costs of bladder cancer. However, subsequent real-world experiments have demonstrated that further work is necessary to identify situations in which these technologies can be applied in a cost-effective manner. Adjunct cytology remains a part of diagnostic standard of care, but recent research suggests that it is not cost effective due to its low diagnostic yield. Analysis of intravesical chemotherapy after transurethral resection of bladder tumor (TURBT), neo-adjuvant therapy for cystectomy, and robot-assisted laparoscopic cystectomy suggests that these technologies are cost effective and should be implemented more widely for appropriate patients. The existing literature on the cost effectiveness of bladder cancer treatments has improved substantially since 2000. The body of work now includes many new models, registry analyses, and real-world studies. However, there is still a need for new implementation guidelines, new risk modeling tools, and a better understanding of the empirical burden of bladder cancer. © 2014, Springer International Publishing Switzerland.

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