Real-world evidence in the reassessment of oncology therapies: payer perceptions from five countries.

Aim: This study explored the perceived value of real-world evidence (RWE) in the reassessment of oncology therapies by collecting the perspectives of health technology assessment/payer decision-makers. Materials & methods: A web-based survey was conducted using the Market Access Transformation Rapid Payer Response online portal. 30 participants from France, Germany, Spain, the UK and the USA were recruited based on their expertise. Results: Participants agreed that the most common uses of RWE are to confirm efficacy and safety results from randomized controlled trials and to reevaluate the projected utilization of an oncology therapy. We found variability in other reported uses of RWE. Conclusion: The organizations developing RWE should ensure that their plans recognize the heterogeneity in payer perceptions.

Extending the vision of adaptive point-of-care platform trials to improve targeted use of drug therapy regimens: An agile approach in the learning healthcare system toolkit.

OBJECTIVES: Improving the targeted use of drug regimens requires robust real-world evidence (RWE) to address the uncertainties that remain regarding their real-world performance following market entry. However, challenges in the current state of RWE production limit its impact on clinical decisions, as well as its operational scalability and sustainability. We propose an adaptive point-of-care (APoC) platform trial as an approach to RWE production that improves both clinical and operational efficiencies. METHODS AND FINDINGS: We explored design innovations, operational challenges, and infrastructure needs within a multi-stakeholder consortium to evaluate the potential of an APoC platform trial for studying chronic disease treatment regimens using rheumatoid arthritis as a case study. The concept integrates elements from adaptive clinical trials (dynamic treatment regimen strategies) and point-of-care trials (research embedded into routine clinical care) under a perpetual platform infrastructure. The necessary components to implement an APoC platform trial within outpatient settings exist, and present an opportunity for a cross-disciplinary, multi-stakeholder approach. Effective engagement of key stakeholders involved in and impacted by the platform is critical to success. Our collaborative design process identified three high-impact stakeholder-engagement areas: (1) focus on research question(s), (2) design and implementation planning such that it is feasible and fit-for-purpose, and (3) measurement, or meaningful metrics for both clinical (patient outcomes) and system (operational efficiencies) impact. CONCLUSIONS: An APoC platform trial for rheumatoid arthritis integrating innovative design elements in a scalable infrastructure has the potential to reduce important uncertainties about the real-world performance of biomedical innovations and improve clinical decisions.

Examining Endpoint Concordance in Clinical Trials and Real-World Clinical Practice to Advance Real-World Evidence Utilization.

Real-world evidence (RWE) is increasingly contributing to more informed decisions regarding the optimal access to and use of therapeutics to improve patient outcomes. However, in many cases, a disconnect between evidence derived from clinical trials and the RWE that follows market approval impedes the potential value and widespread adoption of RWE to optimize patient care. Collaborators with the Learning Ecosystems Accelerator for Patient-centered, Sustainable innovation (LEAPS), a major project of the Tufts Medical Center [formally Massachusetts Institute of Technology (MIT)] NEW Drug Development ParadIGmS (NEWDIGS) initiative, propose assessing the relationship between efficacy endpoints used in randomized controlled trials (RCTs) and effectiveness measures that inform treatment decisions within real-world clinical settings as one way to bridge this divide and further leverage RWE to improve care and patient outcomes. This commentary outlines elements of an endpoint concordance study using Rheumatoid Arthritis as a case study. The authors describe the ways in which better understanding of the relationship between effectiveness and RCT endpoints could improve the confidence in and adoption of RWE by both contextualizing existing RWE as well as identifying ways in which to improve the value of RWE in improving care and outcomes.

Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process.

OBJECTIVES: The Joint ISPOR-ISPE Special Task Force on Real-World Evidence included patient/stakeholder engagement as a recommended good procedural practice when designing, conducting, and disseminating real-world evidence (RWE). However, there are no guidelines describing how patient experience data (PED) can be applied when designing real-world data (RWD) studies. This article describes development of consensus recommendations to guide researchers in applying PED to develop patient-centered RWE. METHODS: A multidisciplinary advisory board, identified through recommendations of collaborators, was established to guide development of recommendations. Semistructured interviews were conducted to identify how experienced RWD researchers (n = 15) would apply PED when designing a hypothetical RWD study. Transcripts were analyzed and emerging themes developed into preliminary methods recommendations. An eDelphi survey (n = 26) was conducted to refine/develop consensus on the draft recommendations. RESULTS: We identified 13 recommendations for incorporating PED throughout the design, conduct, and translation of RWE. The recommendations encompass themes related to the development of a patient-centered research question, designing a study, disseminating RWE, and general considerations. For example, consider how patient input can inform population/subgroups, comparators, and study period. Researchers can leverage existing information describing PED and may be able to apply those insights to studies relying on traditional RWD sources and/or patient registries. CONCLUSIONS: Applying these emerging recommendations may improve the patient centricity of RWE through improved relevance of RWE to patient communities of interest and foster greater multidisciplinary participation and transparency in RWD research. As researchers gather experience by applying the methods recommendations, further refinement of these consensus recommendations may lead to "best practices."

Maintenance and Concomitant Therapy Use with Chlormethine Gel Among Patients with Stage IA/IB Mycosis Fungoides-Type Cutaneous T-Cell Lymphoma (MF-CTCL): A Real-World Evidence Study.

INTRODUCTION: Chlormethine (CL) gel is a skin-directed therapy approved for treatment of stage IA/IB mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) in the USA. MF-CTCL has a chronic clinical course, requiring long-term maintenance therapy with one or more therapies. This analysis describes real-world patterns of maintenance therapy and use of concomitant therapy with CL gel among patients with stage IA/IB MF-CTCL. METHODS: In a US-based registry, MF-CTCL patients treated with CL gel were enrolled between 3/2015 and 10/2018 across 46 centers and followed for up to 2 years. Patient demographics, clinical characteristics, CL gel treatment patterns, concomitant treatments, clinical response, and adverse events (AEs) were collected from medical records. Descriptive statistics are reported. RESULTS: Of the 206 patients with stage IA/IB MF-CTCL, 58.7% were male, and average age was 60.7 years with 4.6 years since diagnosis. Topical steroids, phototherapy, and topical retinoids were used concomitantly with CL gel in 62.6%, 26.2%, and 6.3% of patients, respectively. Most concomitant therapies (up to 85%) were started before CL gel initiation and, in about half of the cases (up to 57%), were used concurrently for ≥ 12 months. Overall, 158 (76.7%) patients experienced partial response (PR) and 144 continued with maintenance therapy. After achieving PR, most patients (74.3%) kept the same maintenance therapy schedule, most commonly once daily. Of patients who had any skin-related AE (31.6%) or skin-related AEs associated with CL gel (28.2%), nearly half experienced CL gel treatment interruption and ~40% had a dosing reduction. The observed real-world treatment patterns were concordant with National Comprehensive Cancer Network (NCCN) guidelines. CONCLUSION: The study results suggest that continuing CL gel maintenance therapy and combining treatments with CL gel are common practice in the real-world setting, with most maintained on a stable dosing schedule. Careful management of AEs may help patients maintain long-term optimal dosing with less treatment interruptions and dosing reductions.

The Current Landscape and Emerging Applications for Real-World Data in Diagnostics and Clinical Decision Support and its Impact on Regulatory Decision Making.

Real-world data (RWD) and real-world evidence (RWE) are becoming essential tools for informing regulatory decision making in health care and offer an opportunity for all stakeholders in the healthcare ecosystem to evaluate medical products throughout their lifecycle. Although considerable interest has been given to regulatory decisions supported by RWE for treatment authorization, especially in rare diseases, less attention has been given to RWD/RWE related to in vitro diagnostic (IVD) products and clinical decision support systems (CDSS). This review examines current regulatory practices in relation to IVD product development and discusses the use of CDSS in assisting clinicians to retrieve, filter, and analyze patient data in support of complex decisions regarding diagnosis and treatment. The review then explores how utilizing RWD could augment regulatory body understanding of test performance, clinical outcomes, and benefit-risk profiles, and how RWD could be leveraged to augment CDSS and improve safety, quality, and efficiency of healthcare practices. Whereas we present examples of RWD assisting in the regulation of IVDs and CDSS, we also highlight key challenges within the current healthcare system which are impeding the potential of RWE to be fully realized. These challenges include issues such as data availability, reliability, accessibility, harmonization, and interoperability, often for reasons specific to diagnostics. Finally, we review ways that these challenges are actively being addressed and discuss how private-public collaborations and the implementation of standardized language and protocols are working toward producing more robust RWD and RWE to support regulatory decision making.

Bridging the Gap Between RCTs and RWE Through Endpoint Selection.

This commentary is authored by several industry real-world evidence (RWE) experts, with support from IQVIA, as part of the 'RWE Leadership Forum': a group of Industry Leaders who have come together as non-competitive partners to understand and respond to RWD/E challenges and opportunities with a single expert voice. Here, the forum discusses the value in bridging the industry disconnect between RTCs and RWE, with a view to promoting the use of RWE in the RCT environment. RCT endpoints are explored along several axes including their clinical relevance and their measure of direct patient benefit, and then compared with their real-world counterparts to identify suitable paths, or gaps, for assimilating RWE endpoints into the RCT environment.

Strengthening pharma's contract with society: the value of trusted partnerships between pharma and healthcare facilitated by real-world data.

This White Paper is authored by 11 industry real-world evidence (RWE) experts, with support from IQVIA, as part of the 'RWE Leadership Forum': a group of industry leaders who come together as noncompetitive partners to understand and respond to internal or external RWD/E challenges and opportunities with a single expert voice. Herein we aim to clarify the rules of engagement between pharma and healthcare in order to establish trust-based partnerships, which will unlock unique value for society, including the medical community and the ultimate beneficiary, the patient.

Health-related quality of life outcomes and economic burden of inflammatory bowel disease in Japan.

PURPOSE: Previous Japanese studies have not compared health-related quality of life (HRQoL), work productivity and activity impairment, health care resource utilization (HRU), and costs in inflammatory bowel disease (IBD) patients with non-IBD controls, leading to insufficient evidence regarding IBD's true burden. The aim of this study was to examine the impact of IBD on patient-reported outcomes and costs among Japanese adults (≥18 years). PATIENTS AND METHODS: This retrospective cross-sectional study used data from the 2012-2014 Japan National Health and Wellness Survey (N=83,505). HRQoL (SF-36v2), work productivity and activity impairment (work productivity and activity impairment-General Health Questionnaire), HRU, and annual costs were compared between respondents with IBD (n=441) and non-IBD controls (n=82,944), and within IBD subtypes (Crohn's disease [CD] and ulcerative colitis [UC]) using chi-square and ANOVA tests. RESULTS: Mental Component Summary (MCS), Physical Component Summary (PCS), and health state utility (Short-Form-6 Dimensions [SF-6D]) scores were significantly lower in IBD respondents than in controls (differences of 2.2 points, 2.6 points, and 0.041 points, respectively; all P<0.001). However, only differences in SF-6D scores reached the minimally important difference threshold. Furthermore, IBD-diagnosed respondents reported greater absenteeism, presenteeism, overall work productivity loss and activity impairment, and HRU than controls (all P<0.001). Consequently, direct and indirect costs were 3-fold and 1.5-fold higher in IBD-diagnosed respondents than in controls (both, P<0.001). Additionally, CD-diagnosed respondents had lower MCS, PCS, and SF-6D scores (all P<0.01) and higher direct costs (P<0.001) than UC-diagnosed respondents. CONCLUSION: IBD and its subtype CD were associated with lower HRQoL, greater impairment to work and non-work activities, HRU, and costs among Japanese adults. This reinforces the general consensus that IBD patients, specifically those diagnosed with CD, require support from their family and society to combat the disease.

Health-related quality of life outcomes, economic burden, and associated costs among diagnosed and undiagnosed depression patients in Japan.

PURPOSE: Depression is associated with substantial health and economic burden. This study examined the impact of diagnosed and undiagnosed depression on health-related outcomes and costs among Japanese adults. METHODS: A retrospective, observational study was conducted using 2012-2014 Japan National Health and Wellness Survey (N=83,504) data. Differences between respondents diagnosed with depression (n=2,843) and undiagnosed with depression (weighted n=2,717) and controls without depression (weighted n=2,801) in health-related quality of life, impairment to work productivity and daily activities (Work Productivity and Activity Impairment Questionnaire), health care resource utilization, and annual costs were evaluated. Propensity score weighting and weighted generalized linear models were used to compare groups on the outcome variables, after adjusting for covariates. RESULTS: Overall, respondents with undiagnosed depression had significantly better outcomes than those diagnosed with depression, but significantly worse outcomes than controls (for all, P<0.001). The mean Mental Component Summary scores were lower in the diagnosed group when compared with undiagnosed respondents and controls (33.2 vs 34.5 vs 48.6). Similar findings were obtained for mean Physical Component Summary (49.2 vs 49.5 vs 52.8) and health state utility scores (0.61 vs 0.62 vs 0.76). Additionally, the diagnosed group reported greater absenteeism (13.1 vs 6.6 vs 2.5%), presenteeism (41.4 vs 38.1 vs 18.8%), overall work productivity impairment (47.2 vs 41.1 vs 20.2%), and activity impairment (48.4 vs 43.3 vs 21.1%) than the undiagnosed and control groups, respectively. Consistently, patients with diagnosed depression had higher annual per patient direct (1.6-fold) and indirect costs (1.1-fold) than those in the undiagnosed depression group. CONCLUSION: Diagnosed depression was associated with lower health-related quality of life and greater impairment in work productivity and daily activities, higher health care resource utilization, and higher costs, compared with undiagnosed respondents and controls. These study findings suggest a need for greater awareness of depression symptoms among Japanese adults, which is needed to facilitate proper diagnosis and treatment.

Health-related outcomes, health care resource utilization, and costs of multiple sclerosis in Japan compared with US and five EU countries.

PURPOSE: Multiple sclerosis (MS) imposes a huge burden on patients. This study examined the relationship between MS and health-related and economic burden in Japan; secondarily, health status was compared across patients with MS in Japan, US, and five European Union (5EU) countries (France, Germany, Italy, Spain, and UK). METHODS: A retrospective cross-sectional study was conducted using self-reported data from 2009 to 2014 Japan National Health and Wellness Survey (n=145,759). Health status, work productivity loss, activity impairment, health care resource utilization, and annual costs associated with MS (n=85) were compared with controls without MS (n=145,674). Propensity score matching and multivariable linear regressions determined the effect of MS after controlling for confounders. Health status in Japan was also compared with that of 5EU (n=62) and US (n=67) patients with MS. RESULTS: Patients with MS in Japan reported significantly worse health status via mental component summary score (MCS; 40.1 vs 45.8) and physical component summary score (PCS; 41.4 vs 51.2) and health state utility scores (0.63 vs 0.74; all P<0.001). They also reported more absenteeism (12.0% vs 3.7%), presenteeism (33.8% vs 19.8%), overall work impairment (40.9% vs 21.6%), and activity impairment (43.6% vs 24.0%), with higher indirect costs (¥2,040,672/US $20,102 vs ¥1,076,306/US$10,603) than controls (all P<0.001). Patients with MS reported higher resource use, including provider visits (8.0 vs 4.7), emergency room visits (0.03 vs 0.1), and hospitalizations (2.7 vs 0.69) in the past 6 months, with higher direct costs (¥3,670,906/US$36,162 vs ¥986,099/US$9,714) than controls (all P<0.001). Finally, Japanese patients with MS reported lower MCSs and higher PCSs than their US and 5EU counterparts. CONCLUSION: MS in Japan is associated with poor health status and high work productivity loss, resource use, and costs, underscoring the need for improved treatment, especially vis-à-vis mental health, when comparing Japanese patients with their 5EU and US counterparts.

Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group.

BACKGROUND: Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated. OBJECTIVE: To develop for the first time a catalog of primary impediments to RD research and HTA, and to describe the cause and effect of individual challenges. METHODS: Challenges were identified by an international 22-person expert working group and qualitative outreach to colleagues with relevant expertise. A broad range of stakeholder perspectives is represented. Draft results were presented at annual European and North American International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congresses, and written comments were received by the 385-strong ISPOR Rare Disease Review Group from two rounds of review. Findings were refined and confirmed via targeted literature search. RESULTS: Research-related challenges linked to the low prevalence of RDs were categorized into those pertaining to disease recognition and diagnosis, evaluation of treatment effect, and patient recruitment for clinical research. HTA-related challenges were classified into issues relating to the lack of a tailored HTA method for RD treatments and uncertainty for HTA agencies and health care payers. CONCLUSIONS: Identifying and highlighting diverse, but interrelated, key challenges in RD research and HTA is an essential first step toward developing implementable and sustainable solutions. A collaborative multistakeholder effort is required to enable faster and less costly development of safe, efficacious, and appropriate new RD therapies that offer value for money.

The Role of the Surgeon on Outcomes of Vaginal Prolapse Surgery With Mesh.

INTRODUCTION: Adverse outcomes after surgery for pelvic organ prolapse (POP) with mesh are often attributed to the mesh material with little attention paid to the influence of surgeon factors. We used a national data set to determine whether surgeon case volume and specialty influenced vaginal prolapse surgery outcomes with mesh. MATERIALS AND METHODS: Public Use File data on a 5% random national sample of female Medicare beneficiaries were obtained from the Centers for Medicare and Medicaid Services. Women with a diagnosis of POP who underwent surgery with mesh between 2007 and 2008 were identified by relevant International Classification of Diseases, 9th Revision, Clinical Modification and Current Procedural Terminology, 4th Edition procedure codes. Outcomes were compared by surgeon case volume and specialty. RESULTS: From 2007 to 2008, 1657 surgeries for POP were performed with mesh. Low-, intermediate-, and high-volume surgeons performed 881 (53%), 408 (25%), and 368 (22%) of the cases with mesh, respectively. The cumulative reoperation rates for low-, intermediate-, and high-volume providers were 6%, 2%, and 3%, respectively. The difference in reoperation rates between low and intermediate and low- and high-volume surgeons was statistically significant (P = 0.007 and 0.003, respectively). There was no significant difference in reoperation rates between gynecologists and urologists when vaginal mesh was implanted for POP surgery. CONCLUSIONS: Low-volume surgeons performed most of the vaginal prolapse repairs with mesh and had significantly higher reoperation rates. Surgeon experience must be a consideration when reporting mesh-related complications of POP surgery.

Caring for Children With Tuberous Sclerosis Complex: What Is the Physical and Mental Health Impact on Caregivers?

Disease burden associated with tuberous sclerosis complex, a genetic disorder characterized by benign tumor growth including lesions in multiple organs, puts tremendous demands on families. This analysis examines the physical and mental health burden of tuberous sclerosis complex caregivers in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex caregivers collected information; descriptive analyses were conducted on age-based subgroups. A total of 275 caregivers of tuberous sclerosis complex patients responded. Mean patient age ≤ 18 years was 6.9 (±4.4) and 42.3 (±18.2) for patients >18 years of age. Caregivers reported multiple tuberous sclerosis complex manifestations and high health care utilization for patients. Caregivers spending more time on doctor visits or researching tuberous sclerosis complex had lower physical and mental health-related quality of life scores and more depressive symptoms. Tuberous sclerosis complex caregivers had significantly lower physical and mental health-related quality of life scores and more depressive symptomatology compared to US healthy adult population norms.

Characteristics and temporal trends in patient registries: focus on the life sciences industry, 1981-2012.

PURPOSE: Patient registries are used to monitor safety, examine real-world effectiveness, and may potentially contribute to comparative effectiveness research. To our knowledge, life sciences industry (LSI)-sponsored registries have not been systematically categorized. This study represents a first step toward understanding such registries over time. METHODS: Studies described as registries were identified in the ClinicalTrials.gov database. Characteristics from these registry records were abstracted and analyzed. RESULTS: Of 1202 registries identified, approximately 47% reported LSI sponsorship. These 562 LSI registries varied in focus: medical devices (n = 193, 34%), specific drugs (n = 173, 31%), procedures (n = 29, 5%), or particular diseases (n = 139, 25%). Thirty-three registries (<6%) evaluated pregnancy outcomes. The most common therapeutic area was cardiovascular (n = 234, 42%); others included endocrinology, immunology, oncology, musculoskeletal disorders, and neurology. The two most often measured outcomes were clinical effectiveness and safety, each of which appeared in 363/562 (65%) of LSI registries. Other outcomes included real-world clinical practice patterns (n = 122, 22%), patient-reported outcomes (n = 106, 19%), disease epidemiology/natural history (n = 69, 12%), and economic outcomes (n = 30, 5%). The number of LSI registries and their geographic diversity has increased over time. CONCLUSIONS: The LSI registries represent a substantial proportion of all patient registries documented in ClinicalTrials.gov. These prospective studies are growing in number and encompass diverse therapeutic areas and geographic regions. Most registries measure multiple outcomes and capture real-world data that may be unavailable through other study designs. This classification of LSI registries documents their use for studying heterogeneity of diseases, examining treatment patterns, measuring patient-reported outcomes, examining economic outcomes, and performing comparative effectiveness research.

Trends in management of pelvic organ prolapse among female Medicare beneficiaries.

OBJECTIVE: In the last decade, many new surgical treatments have been developed to achieve less-invasive approaches to prolapse management. However, limited data exist on how the patterns of care for women with pelvic organ prolapse (POP) may have changed over the last decade, and whether mesh implantation techniques have influenced the type of specific compartment repair performed. We used a national data set to analyze the temporal trends in patterns of care for women with POP. STUDY DESIGN: Data were obtained from Public Use Files from the Centers for Medicare and Medicaid Services for a 5% random sample of national beneficiaries with an International Classification of Diseases, Ninth Revision, Clinical Modification diagnosis of POP from 1999 through 2009. Current Procedural Terminology, 4th Edition and International Classification of Diseases, Ninth Revision, Clinical Modification procedure codes were used to evaluate nonsurgical and surgical management trends for this cohort. Types of surgery were categorized by prolapse compartment and combinations of repairs. After 2005, when applicable codes became available, mesh or graft repairs were also analyzed. RESULTS: Over the study time period, the number of women with a diagnosis of POP in any 1 year in our 5% sample of Medicare beneficiaries remained relatively stable (range, 21,245-23,268 per year). Rates of pessary insertion were also consistent at 11-13% over the study period. Of the women with a prolapse diagnosis, 14-15% underwent surgical repair, and there was little change over time in surgical management patterns based on compartment. Most commonly, multiple compartments were repaired simultaneously. There was a rapid increase in mesh use such that in 2009, 41% of all women who underwent surgery (5.8% of the total cohort) had mesh or graft inserted in their repair. Hysterectomy rates for prolapse decreased over time. Rates of vault suspension at the time of hysterectomy for prolapse were low; however, they showed a relative increase over time (22% in 1999 to 26% in 2009). CONCLUSION: Patterns and rates of prolapse repairs remained relatively unchanged from 1999 through 2009, with an exception of a rapid rise in mesh use. These data suggest that the majority of mesh techniques were used for augmentation purposes only, but did not result in an increase in apical repairs performed in the United States. There remains a disappointingly low rate of vault suspension repairs concomitantly at time of hysterectomy for POP.

The humanistic burden of head and neck cancer: a systematic literature review.

BACKGROUND: Head and neck cancer (HNC) and its treatment can affect communication, nutrition, and physical appearance, and the global impact of this disease on patients' quality of life may be substantial. OBJECTIVE: The aim of this systematic literature review was to describe the impact of HNC and its treatment on the physical, emotional, and social well-being of patients over time, by examining longitudinal studies of patient-reported outcomes (PRO) evaluating these domains. METHODS: Databases (MEDLINE and Embase) were searched to identify studies published in English between January 2004 and January 2014 analyzing the humanistic aspects of HNC in adult patients. Additional relevant publications were identified through manual searches of abstracts from recent conference proceedings. RESULTS: Of 1,566 studies initially identified, 130 met the inclusion criteria and were evaluated in the assessment. Investigations using a variety of PRO instruments in heterogeneous patient populations consistently reported that PRO scores decrease significantly from diagnosis through the treatment period, but generally recover to baseline in the first year post-treatment. This trend was observed for many functional domains, although some side effects, such as xerostomia, persisted well beyond 1 year. In addition, considerable evidence exists that baseline PRO scores can predict clinical endpoints such as overall and progression-free survival. CONCLUSIONS: Many aspects of HNC, both disease and treatment specific, profoundly affect patients' quality of life. Improved knowledge of these effects on PRO may allow for more informed treatment decisions and can help physicians to better prepare patients for changes they may experience during therapy. Furthermore, the predictive value of baseline PRO data may enable healthcare providers to identify at-risk patients in need of more intensive intervention.

The economic burden of head and neck cancer: a systematic literature review.

BACKGROUND: This systematic literature review aimed to evaluate and summarize the existing evidence on resource use and costs associated with the diagnosis and treatment of head and neck cancer (HNC) in adult patients, to better understand the currently available data. The costs associated with HNC are complex, as the disease involves multiple sites, and treatment may require a multidisciplinary medical team and different treatment modalities. METHODS: Databases (MEDLINE and Embase) were searched to identify studies published in English between October 2003 and October 2013 analyzing the economics of HNC in adult patients. Additional relevant publications were identified through manual searches of abstracts from recent conference proceedings. RESULTS: Of 606 studies initially identified, 77 met the inclusion criteria and were evaluated in the assessment. Most included studies were conducted in the USA. The vast majority of studies assessed direct costs of HNC, such as those associated with diagnosis and screening, radiotherapy, chemotherapy, surgery, side effects of treatment, and follow-up care. The costs of treatment far exceeded those for other aspects of care. There was considerable heterogeneity in the reporting of economic outcomes in the included studies; truly comparable cost data were sparse in the literature. Based on these limited data, in the US costs associated with systemic therapy were greater than costs for surgery or radiotherapy. However, this trend was not seen in Europe, where surgery incurred a higher cost than radiotherapy with or without chemotherapy. CONCLUSIONS: Most studies investigating the direct healthcare costs of HNC have utilized US databases of claims to public and private payers. Data from these studies suggested that costs generally are higher for HNC patients with recurrent and/or metastatic disease, for patients undergoing surgery, and for those patients insured by private payers. Further work is needed, particularly in Europe and other regions outside the USA; prospective studies assessing the cost associated with HNC would allow for more systematic comparison of costs, and would provide valuable economic information to payers, providers, and patients.

Short-term outcomes of vaginal mesh placement among female Medicare beneficiaries.

OBJECTIVE: To compare short-term outcomes between prolapse repairs with and without mesh using a national data set. Mesh use in surgical treatment of pelvic organ prolapse has gained wide popularity. However, mesh complications have increased concomitantly with its use. METHODS: Public Use File data were obtained for a 5% random national sample of female Medicare beneficiaries aged 65 years and older. Women who underwent prolapse surgery were identified using Current Procedural Terminology Coding System, Fourth Edition (CPT-4) codes. Because the code for mesh placement was effected in 2005, we separated patients into 3 cohorts as follows: those who underwent prolapse repairs from 1999 to 2000 (presumably without mesh), those who underwent repairs from 2007 to 2008 (presumably without mesh), and those with mesh (based on CPT-4 code 57267) from 2007 to 2008. One-year outcomes were identified using International Classification of Diseases, Ninth Revision diagnosis and procedure codes and CPT-4 procedure codes. RESULTS: A total of 9180 prolapse repairs without mesh were performed from 1999 to 2000, 7729 without mesh from 2007 to 2008, and 1804 prolapse repairs with mesh from 2007 to 2008. Prolapse reoperation within 1 year of surgery was higher in nonmesh vs mesh cohorts (6%-7% vs 4%, P <.02). Mesh removal rates were higher in mesh vs nonmesh group (4% vs 0%-1%, P <.001). Mesh use was associated with more dyspareunia, mesh-related complications, and urinary retention, even when controlling for concomitant sling. CONCLUSION: Mesh to treat pelvic organ prolapse and stress urinary incontinence was associated with a small decrease in early reoperation for prolapse. This decrease came at the expense of increased rates of pelvic pain, retention, mesh-related complications, and mesh removal.