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In this update, we discuss a position statement from the National Institute of Health and Care Excellence (NICE) on the use of artificial intelligence for evidence generation and publications reviewing the use of real-world data as external control arms. Finally, we discuss a number of recent studies investigating the real-world effectiveness of glucagon-like peptide-1 receptor agonists and whether these studies are informative for reimbursement decision making.

In this latest update we discuss real-world evidence (RWE) guidance from the leading oncology professional societies, the American Society of Clinical Oncology and the European Society for Medical Oncology, and the PRINCIPLED practical guide on the design and analysis of causal RWE studies.

Emerging health care research paradigms such as comparative effectiveness research (CER), patient-centered outcome research (PCOR), and precision medicine (PM) share one ultimate goal: constructing evidence to provide the right treatment to the right patient at the right time. We argue that to succeed at this goal, it is crucial to have both timely access to individual-level data and fine geographic granularity in the data. Existing data will continue to be an important resource for observational studies as new data sources are developed. We examined widely used publicly funded health databases and population-based survey systems and found four ways they could be improved to better support the new research paradigms: (1) finer and more consistent geographic granularity, (2) more complete geographic coverage of the US population, (3) shorter time from data collection to data release, and (4) improved environments for restricted data access. We believe that existing data sources, if utilized optimally, and newly developed data infrastructures will both play a key role in expanding our insight into what treatments, at what time, work for each patient.

Millions of dollars are spent on the development of new personalized medicine technologies. While these research costs are often supported by public research funds, many diagnostic tests and biomarkers are not adopted by the healthcare system due to lack of evidence on their cost-effectiveness. We describe a stepwise approach to conducting cost-effectiveness analyses that are performed early in the technology's development process and can help mitigate the potential risks of investment. Decision analytic modeling can identify the key drivers of cost effectiveness and provide minimum criteria that the technology needs to meet for adoption by public and private healthcare systems. A value of information analysis can quantify the added value of conducting more research to provide further evidence for policy decisions. These steps will allow public research funders to make better decisions on their investments to maximize the health benefits and to minimize the number of suboptimal technologies.

In order to monitor the net public health benefit of new drugs, especially in the light of recent stepwise approval approaches, there is a need to optimize real-time post-marketing evaluation of new drugs using data collected in routine care. Sweden, with its unique possibilities for observational research, can provide these data. We herein propose a framework for continuous monitoring of the effectiveness, safety, and cost-effectiveness of new drugs, using prospectively determined protocols designed in collaboration between all relevant stakeholders. We believe that this framework can be a useful tool for healthcare authorities and reimbursement agencies in the introduction of new drugs.

Background: Little is known about resource use in the care of neuroendocrine tumors (NETs). This study defined patterns of costs in NET management and compared them with those of a more common malignancy, colon cancer (CC)., Methods: Using a provincial cancer registry (2004-2012), NET patients were identified and matched at a ratio of 1-3 with CC patients. Four phases of care were examined: pre-diagnostic (PreDx: -2 years to -181 days), diagnostic (Dx: -180 days to +180 days), postdiagnostic (PostDx: +181 days to +3 years), and prolonged post-diagnostic (PPostDx: +181 days to +9 years). The mean costs per patient were compared, and cost predictors were analyzed with quintile regression., Results: Of 3827 NETs, 3355 were matched with 9320 CCs. The PreDx mean NET costs were higher than the CC costs ($5877 vs $5368; p = 0.06), driven by nondrug costs. They were lower in the Dx and PostDx phases (both p < 0.01). For PPostDx, the drug costs were higher for NETs ($26,788 vs $7827; p < 0.01), representing 41% of the costs versus 16% of the costs for CC. Older age and comorbidities predicted higher NET costs in all phases. Lower socioeconomic status (SES) predicted higher costs in the initial phases and higher SES costs in the PPost-Dx phase. Gastroenteric NETs were associated with lower costs in the Dx phase [parameter estimate (PE), -$13,644] and pancreatic NETs with higher costs in PostDx phase (PE, $3348)., Conclusion: Currently, NETs represent a potential important health care burden. The NET cost patterns differed from those for CC, with the highest costs during the PPostDx phase. The SES and primary NET site affected costs differently at different time points. These data can inform resource allocation tailored to the needs for NETs.

Aim: Health technology assessment (HTA) is a tool to help the decision-making process. The aim is to describe methods and processes used in the reimbursement decision making for drug-eluting stents (DES) in four different settings., Methods: DES as a technology under study was selected according to different criteria, all of them agreed by a working group. A survey of key informants was designed., Results: DES was evaluated following well-structured HTA processes. Nonetheless, scope for improvement was observed in relation to the data considered for the final decision, the transparency and inclusiveness of the process as well as in the methods employed., Conclusion: An attempt to describe the HTA processes of a well-known medical device.

Background: Payers are faced with making coverage and reimbursement decisions based on the best available evidence. Often these decisions apply to patient populations, provider networks, and care settings not typically studied in clinical trials. Treatment effectiveness evidence is increasingly available from electronic health records, registries, and administrative claims. However, little is known about when and what types of real-world evidence (RWE) studies inform pharmacy and therapeutic (P&T) committee decisions., Objective: To evaluate evidence sources cited in P&T committee monographs and therapeutic class reviews and assess the design features and quality of cited RWE studies., Methods: A convenience sample of representatives from pharmacy benefit management, health system, and health plan organizations provided recent P&T monographs and therapeutic class reviews (or references from such documents). Two investigators examined and grouped references into major categories (published studies, unpublished studies, and other/unknown) and multiple subcategories (e.g., product label, clinical trials, RWE, systematic reviews). Cited comparative RWE was reviewed to assess design features (e.g., population, data source, comparators) and quality using the Good ReseArch for Comparative Effectiveness (GRACE) Checklist., Results: Investigators evaluated 565 references cited in 27 monographs/therapeutic class reviews from 6 managed care organizations. Therapeutic class reviews mostly cited published clinical trials (35.3%, 155/439), while single-product monographs relied most on manufacturer-supplied information (42.1%, 53/126). Published RWE comprised 4.8% (21/439) of therapeutic class review references, and none (0/126) of the monograph references. Of the 21 RWE studies, 12 were comparative and assessed patient care settings and outcomes typically not included in clinical trials (community ambulatory settings [10], long-term safety [8]). RWE studies most frequently were based on registry data (6), conducted in the United States (6), and funded by the pharmaceutical industry (5). GRACE Checklist ratings suggested the data and methods of these comparative RWE studies were of high quality., Conclusions: RWE was infrequently cited in P&T materials, even among therapeutic class reviews where RWE is more readily available. Although few P&T materials cited RWE, the comparative RWE studies were generally high quality. More research is needed to understand when and what types of real-world studies can more routinely inform coverage and reimbursement decisions., Disclosures: This project was funded by the National Pharmaceutical Council. Hurwitz, Brown, Peters, and Malone have nothing to disclose. Graff is employed by the National Pharmaceutical Council Part of this study was presented as a poster presentation at the AMCP Managed Care & Specialty Pharmacy 2016 Annual Meeting; April 19-22, 2016; San Francisco, CA. Study concept and design were primarily contributed by Malone and Graff, along with Hurwitz and Brown. All authors participated in data collection, and data interpretation was performed by Malone, Hurwitz, and Graff, with assistance from Brown and Peters. The manuscript was written primarily by Hurwitz and Malone, along with Graff, Brown, and Peters, and revised by Malone, Brown, Peters, Hurwitz, and Graff.

Background: Randomized controlled trials provide robust data on the efficacy of interventions rather than on effectiveness. Health technology assessment (HTA) agencies worldwide are thus exploring whether real-world data (RWD) may provide alternative sources of data on effectiveness of interventions. Presently, an overview of HTA agencies' policies for RWD use in relative effectiveness assessments (REA) is lacking., Objectives: To review policies of six European HTA agencies on RWD use in REA of drugs. A literature review and stakeholder interviews were conducted to collect information on RWD policies for six agencies: the Dental and Pharmaceutical Benefits Agency (Sweden), the National Institute for Health and Care Excellence (United Kingdom), the Institute for Quality and Efficiency in Healthcare (Germany), the High Authority for Health (France), the Italian Medicines Agency (Italy), and the National Healthcare Institute (The Netherlands). The following contexts for RWD use in REA of drugs were reviewed: initial reimbursement discussions, pharmacoeconomic analyses, and conditional reimbursement schemes. We identified 13 policy documents and 9 academic publications, and conducted 6 interviews., Results: Policies for RWD use in REA of drugs notably differed across contexts. Moreover, policies differed between HTA agencies. Such variations might discourage the use of RWD for HTA., Conclusions: To facilitate the use of RWD for HTA across Europe, more alignment of policies seems necessary. Recent articles and project proposals of the European network of HTA may provide a starting point to achieve this., (Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Background: There is growing concern about appropriate disease management for peripheral artery disease (PAD) because of the rapidly expanding population at risk for PAD and the high burden of illness associated with symptomatic PAD. A better understanding of the potential economic impact of symptomatic PAD relative to a matched control population may help improve care management for these patients., Objective: To compare the medical resource utilization, costs, and medication use for patients with symptomatic PAD relative to a matched control population., Methods: In this retrospective longitudinal analysis, the index date was the earliest date of a symptomatic PAD record (symptomatic PAD cohort) or any medical record (control cohort), and a period of 1 year pre-index and 3 years post-index was the study time frame. Symptomatic PAD patients and control patients (aged ≥ 18 years) enrolled in the MarketScan Commercial and Encounters database from January 1, 2006, to June 30, 2010, were identified. Symptomatic PAD was defined as having evidence of intermittent claudication (IC) and/or acute critical limb ischemia requiring medical intervention. Symptomatic PAD patients were selected using an algorithm comprising a combination of PAD-related ICD-9-CM diagnostic and diagnosis-related group codes, peripheral revascularization CPT-4 procedure codes, and IC medication National Drug Code numbers. Patients with stroke/transient ischemic attack, bleeding complications, or contraindications to antiplatelet therapy were excluded from the symptomatic PAD group but not the control group. A final 1:1 symptomatic PAD to control population with an exact match based on age, sex, index year, and Charlson Comorbidity Index (CCI) was identified. Descriptive statistics comparing patient demographics, comorbidities, medical resource utilization, cost, and medication use outcomes were generated. Generalized linear models were developed to compare the outcomes while controlling for residual difference in demographics, comorbidities, pre-index resource use, and pre-index costs., Results: 3,965 symptomatic PAD and 3,965 control patients were matched. In both cohorts, 54.7% were male, with a mean age (SD) of 69.0 (12.9) years and a CCI score of 1.3 (0.9). Symptomatic PAD patients had more cardiovascular comorbidities than control patients (27.7% vs. 12.6% coronary artery disease, 27.1% vs. 15.9% hyperlipidemia, and 49.8% vs. 28.2% hypertension) in the pre-index period. Post-index rates of ischemic stroke, non-ST segment elevation myocardial infarction, unstable angina, and cardiovascular- or PAD-related procedures (limb amputations, endovascular procedures, open surgical procedures, percutaneous coronary intervention, and coronary artery bypass graft) were higher among symptomatic PAD patients versus control patients. All-cause annualized inpatient admissions (0.46 vs. 0.22 admissions), emergency department/urgent care days (0.27 vs. 0.22 days), and office visit days (12.5 vs. 10.2 days) were higher among symptomatic PAD versus control patients post-index. Annualized all-cause inpatient costs ($8,494 vs. $3,778); outpatient costs ($8,459 vs. $5,692); and total costs ($20,880 vs. $12,501) were higher among symptomatic PAD versus control patients post-index. Only 17.8% of symptomatic PAD patients versus 6.6% of control patients were on clopidogrel pre-index. In the post-index period, clopidogrel prescriptions in the symptomatic PAD population increased to 38.0%. Results were consistent in the regression models with the symptomatic PAD population having a higher number of all-cause post-index inpatient admissions, emergency department/urgent care days, office visit days, inpatient costs, outpatient costs, and total costs versus control patients (P ≤ 0.026)., Conclusions: Symptomatic PAD patients have significantly higher medical resource use and costs when compared with a matched control population. As the prevalence of symptomatic PAD increases, there will be a significant impact on the population and health care system. The rates of use of evidence-based secondary prevention therapies, such as antiplatelet medication, were low. Therefore, greater effort must be made to increase utilization rates of appropriate treatments to determine if the negative economic and clinical impacts of symptomatic PAD can be minimized., Disclosures: This study was funded by Merck & Co., Kenilworth, New Jersey. Chase and Heithoff are employees of Merck & Co., Kenilworth, New Jersey, and Upper Gwynedd, Pennsylvania. Friedman and Navaratnam are paid consultants for Merck & Co. Simpson is a paid consultant for Merck, Pfizer, and Amgen and has received speaker's fees from Merck and Pfizer. Study concept and design were contributed by Chase, Navaratnam, and Heilhoff, along with Simpson and Friedman. Friedman collected the data, which was interpreted by Simpson and Navaratnam, along with Friedman. The manuscript was written by Navaratnam and Friedman, along with Chase, Heilhoff and Simpson, and revised by all of the authors.

Although image interpretation is an essential part of radiologists' value, there are other ways in which we contribute to patient care. Part II of the value of imaging series reviews current initiatives that demonstrate value beyond the image interpretation. Standardizing processes, reducing the radiation dose of our examinations, clarifying written reports, improving communications with patients and providers, and promoting appropriate imaging through decision support are all ways we can provide safer, more consistent, and higher quality care. As payers and policy makers push to drive value, research that demonstrates the value of these endeavors, or lack thereof, will become increasingly sought after and supported., (Copyright © 2016 The Association of University Radiologists. Published by Elsevier Inc. All rights reserved.)

Background: Patients and healthcare stakeholders are increasingly becoming engaged in the planning and conduct of biomedical research. However, limited research characterizes this process or its impact., Objective: We aimed to characterize patient and stakeholder engagement in the 50 Pilot Projects funded by the Patient-Centered Outcomes Research Institute (PCORI), and identify early contributions and lessons learned., Design: A self-report instrument was completed by researchers between 6 and 12 months following project initiation., Participants: Forty-seven principal investigators or their designees (94 % response rate) participated in the study. MAIN MEASURES Self-report of types of stakeholders engaged, stages and levels of engagement, facilitators and barriers to engagement, lessons learned, and contributions from engagement were measured., Key Results: Most (83 %) reported engaging more than one stakeholder in their project. Among those, the most commonly reported groups were patients (90 %), clinicians (87 %), health system representatives (44 %), caregivers (41 %), and advocacy organizations (41 %). Stakeholders were commonly involved in topic solicitation, question development, study design, and data collection. Many projects engaged stakeholders in data analysis, results interpretation, and dissemination. Commonly reported contributions included changes to project methods, outcomes or goals; improvement of measurement tools; and interpretation of qualitative data. Investigators often identified communication and shared leadership strategies as "critically important" facilitators (53 and 44 % respectively); lack of stakeholder time was the most commonly reported challenge (46 %). Most challenges were only partially resolved. Early lessons learned included the importance of continuous and genuine partnerships, strategic selection of stakeholders, and accommodation of stakeholders' practical needs., Conclusions: PCORI Pilot Projects investigators report engaging a variety of stakeholders across many stages of research, with specific changes to their research attributed to engagement. This study identifies early lessons and barriers that should be addressed to facilitate engagement. While this research suggests potential impact of stakeholder engagement, systematic characterization and evaluation of engagement at multiple stages of research is needed to build the evidence base.

Background: This paper reports the process of establishing a transparent, accountable, evidence-based program for introduction of new technologies and clinical practices (TCPs) in a large Australian healthcare network. Many countries have robust evidence-based processes for assessment of new TCPs at national level. However many decisions are made by local health services where the resources and expertise to undertake health technology assessment (HTA) are limited and a lack of structure, process and transparency has been reported., Methods: An evidence-based model for process change was used to establish the program. Evidence from research and local data, experience of health service staff and consumer perspectives were incorporated at each of four steps: identifying the need for change, developing a proposal, implementation and evaluation. Checklists assessing characteristics of success, factors for sustainability and barriers and enablers were applied and implementation strategies were based on these findings. Quantitative and qualitative methods were used for process and outcome evaluation. An action research approach underpinned ongoing refinement to systems, processes and resources., Results: A Best Practice Guide developed from the literature and stakeholder consultation identified seven program components: Governance, Decision-Making, Application Process, Monitoring and Reporting, Resources, Administration, and Evaluation and Quality Improvement. The aims of transparency and accountability were achieved. The processes are explicit, decisions published, outcomes recorded and activities reported. The aim of ascertaining rigorous evidence-based information for decision-making was not achieved in all cases. Applicants proposing new TCPs provided the evidence from research literature and local data however the information was often incorrect or inadequate, overestimating benefits and underestimating costs. Due to these limitations the initial application process was replaced by an Expression of Interest from applicants followed by a rigorous HTA by independent in-house experts., Conclusion: The program is generalisable to most health care organisations. With one exception, the components would be achievable with minimal additional resources; the lack of skills and resources required for HTA will limit effective application in many settings. A toolkit containing details of the processes and sample materials is provided to facilitate replication or local adaptation by those wishing to establish a similar program.

One of the provisions of the health care reform legislation in 2010 was for funding pragmatic clinical trials or large observational studies for comparing the effectiveness of different approved medical treatments, involving broadly representative patient populations. After reviewing pragmatic clinical trials and the issues and challenges that have made them just a small fraction of comparative effectiveness research (CER), we focus on a recent development that uses point-of-care (POC) clinical trials to address the issue of "knowledge-action gap" in pragmatic CER trials. We give illustrative examples of POC-CER trials and describe a trial that we are currently planning to compare the effectiveness of newly approved oral anticoagulants. We also develop novel stage-wise designs of information-rich POC-CER trials under competitive budget constraints, by using recent advances in adaptive designs and other statistical methodologies., (Published by Elsevier Inc.)

Purpose: Meeting marketplace demands for proving the value of new products requires more data than the industry has routinely produced. These data include evidence from comparative effectiveness research (CER), including randomized, controlled trials; pragmatic clinical trials; observational studies; and meta-analyses., Methods: We designed and conducted a survey to examine the industry's perceptions on new data requirements regarding CER evidence, the acceptability of postapproval study types, payer-specific issues related to CER, communication of data being generated postapproval, and methods used for facilitating postapproval evidence generation., Findings: CER is being used by payers for most types of postapproval decisions. Randomized, controlled trials were indicated as the most acceptable form of evidence. At the same time, there was support for the utility of other types of studies, such as pragmatic clinical trials and observational studies. Respondents indicated the use of multiple formats for communicating postapproval data with many different stakeholders including regulators, payers, providers, and patients. Risk-sharing agreements with payers were unanimously supported by respondents with regard to certain products with unclear clinical and economic outcomes at launch. In these instances, conditional reimbursement through coverage with evidence development was considered a constructive option. The Food and Drug Administration's initiative called Regulatory Science was considered by the respondents as having the most impact on streamlining the generation of postapproval research-related evidence., Implications: The biopharmaceutical industry is faced with a broad and complex set of challenges related to evidence generation for postapproval decisions by a variety of health care system stakeholders. Uncertainty remains as to how the industry and payers use postapproval studies to guide decision making with regard to pricing and reimbursement status. Correspondingly, there is uncertainty regarding whether the industry's investment in CER will have a positive return on investment in terms of reimbursement and market access., (Copyright © 2015 Elsevier HS Journals, Inc. All rights reserved.)

Aim: Explore key factors influencing future expectations for the production of evidence from comparative effectiveness research for drugs in the USA in 2020 and construct three plausible future scenarios., Materials & Methods: Semistructured key informant interviews and three rounds of modified Delphi with systematic scenario-building methods., Results & Conclusion: Most influential key factors were: health delivery system integration; electronic health record development; exploitation of very large databases and mixed data sources; and proactive patient engagement in research. The scenario deemed most likely entailed uneven development of large integrated health systems with pockets of increased provider risk for patient care, enhanced data collection systems, changing incentives to do comparative effectiveness research and new opportunities for evidence generation partnerships.

Purpose: Comparative effectiveness research (CER) is designed to support informed decision making at both the individual, population, and policy levels. The American College of Sports Medicine and partners convened a conference with the focus of building an agenda for CER within the context of physical activity and nonpharmacological lifestyle approaches in the prevention and treatment of chronic disease. This report summarizes the conference content and consensus recommendations that culminated in a CER roadmap for physical activity and lifestyle approaches to reducing the risk of chronic disease., Methods: This conference focused on presentations and discussion around the following topic areas: 1) defining CER, 2) identifying the current funding climate to support CER, 3) summarizing methods for conducting CER, and 4) identifying CER opportunities for physical activity., Results: This conference resulted in consensus recommendations to adopt a CER roadmap for physical activity and lifestyle approaches to reducing the risk of chronic disease. In general, this roadmap provides a systematic framework by which CER for physical activity can move from a planning phase to a phase of engagement in CER related to lifestyle factors with particular emphasis on physical activity to a societal change phase that results in changes in policy, practice, and health., Conclusions: It is recommended that physical activity researchers and health care providers use the roadmap developed from this conference as a method to systematically engage in and apply CER to the promotion of physical activity as a key lifestyle behavior that can be effective at making an impact on a variety of health-related outcomes.

Drug development takes place in a global marketplace, albeit with the USA and EU markets currently dominating. In the USA, demands for comparative effectiveness research have gained traction against a backdrop of health delivery reform, while European stakeholders deliberate the role of relative effectiveness in health technology assessment, trying to reduce the duplication of effort by regulators and health technology assessment bodies. In both arenas, drug-makers are faced with mounting drug development costs, and uncertainty over the types of evidence acceptable for a growing list of stakeholders. This article reports and compares future scenarios for evidence expectations for drugs for the USA and EU in 2020. The similarities, differences, and joint implications of the scenarios are considered to create an view of future evidence generation for drugs developed for these markets.

The American Recovery and Reinvestment Act (ARRA) of 2009 directed US$1.1 billion to the US Department of Health and Human Services for support of comparative effectiveness research (CER). As part of this investment, US Department of Health and Human Services commissioned a midstream evaluation of the ARRA CER portfolio. One goal of the evaluation was to identify issues to consider for a future evaluation of the long-term impact of this portfolio and other CER investments. In planning the ARRA CER evaluation, we developed and revised a conceptual framework and related policy research questions that may be useful to future efforts to assess the impact of CER or patient-centered outcomes research investments. In addition, we explored methodological challenges related to designing an evaluation to assess investments in CER that may be informative to any future plans to evaluate the long-term impact of ARRA CER as well subsequent investments made from the Patient-Centered Outcomes Research Trust Fund.

Multidisciplinary, multi-institutional collaboration has become a key feature of comparative effectiveness research (CER), and CER funders have made promotion of these types of collaboration an implicit, and sometimes explicit, goal of funding. An important challenge in evaluating CER programs is understanding if and how different forms of collaboration are associated with successful CER projects. This article explores the potential use of social network analysis to address research questions about the associations between collaboration and the success of CER projects.

Background: The American Recovery and Reinvestment Act of 2009 (ARRA) directed nearly US$29.2 million to comparative effectiveness research (CER) methods development., Aim: To help inform future CER methods investments, we describe the ARRA CER methods projects, identify barriers to this research and discuss the alignment of topics with published methods development priorities., Methods: We used several existing resources and held discussions with ARRA CER methods investigators., Results & Conclusion: Although funded projects explored many identified priority topics, investigators noted that much work remains. For example, given the considerable investments in CER data infrastructure, the methods development field can benefit from additional efforts to educate researchers about the availability of new data sources and about how best to apply methods to match their research questions and data.

Aim: To describe the evaluation design of the American Recovery and Reinvestment Act of 2009 comparative effectiveness research (CER) investment, how funds were allocated and how CER priorities were addressed., Materials & Methods: Primary and secondary data included information from redacted project proposals, an investigator survey and federal project officers, investigators and expert panel discussions., Results: More than 420 projects (US$1.1 billion) were awarded. Those generating new or synthesizing existing CER made up the plurality (194, or US$524 million). Data infrastructure projects were the second-largest area (28%, US$302 million). More than three-fourths addressed at least one priority population, condition category or intervention category., Conclusion: These investments expanded the nation's CER activities and its future capacity to conduct CER.

The Recovery Act provided a substantial, one-time investment in data infrastructure for comparative effectiveness research (CER). A review of the publications, data, and tools developed as a result of this support has informed understanding of the level of effort undertaken by these projects. Structured search queries, as well as outreach efforts, were conducted to identify and review resources from American Recovery and Reinvestment Act of 2009 CER projects building electronic clinical data infrastructure. The findings from this study provide a spectrum of productivity across a range of topics and settings. A total of 451 manuscripts published in 192 journals, and 141 data resources and tools were identified and address gaps in evidence on priority populations, conditions, and the infrastructure needed to support CER.

Aim: This article describes American Reinvestment and Recovery Act comparative effectiveness research data infrastructure (DI) investments and identifies facilitators and barriers to implementation., Materials & Methods: We reviewed original project proposals, conducted an investigator survey and interviewed project officers and principal investigators., Results: DI projects assembled or enhanced existing clinical datasets, established linkages between public and private data sources and built infrastructure. Facilitators included building on existing relationships across organizations and making collection as seamless as possible for clinicians., Conclusion: To sustain DI, investigators should reduce the burden of comparative effectiveness research data collection on practices, adequately address data privacy and security issues, resolve or lessen the impact of data-linking issues and build research capacity for other investigators and clinicians.

There are large gaps in our knowledge on the potential impact of diagnostics and therapeutics on outcomes of patients treated in the real world. Comparative effectiveness research aims to fill these gaps to maximize effectiveness of these interventions. Health information technology has the potential to dramatically improve the practice of medicine and of research. This is an overview of about US$100 million of American Recovery and Reinvestment Act investment in 12 projects managed by the Agency for Healthcare Research and Quality to build an electronic clinical data infrastructure that connects research with healthcare delivery. The achievements and lessons learned from these projects provided a foundation for the National Patient-Centered Clinical Research Network (PCORnet)and will help to guide future infrastructure development needed to build an efficient, scalable and sustainable learning health system.

The 11 big health data networks participating in the AcademyHealth Electronic Data Methods Forum represent cutting-edge efforts to harness the power of big health data for research and quality improvement. This paper is a comparative case study based on site visits conducted with a subset of these large infrastructure grants funded through the Recovery Act, in which four key issues emerge that can inform the evolution of learning health systems, including the importance of acknowledging the challenges of scaling specialized expertise needed to manage and run CER networks; the delicate balance between privacy protections and the utility of distributed networks; emerging community engagement strategies; and the complexities of developing a robust business model for multi-use networks.

The Affordable Care Act is the largest and most comprehensive overhaul of the United States health care industry since the inception of the Medicare and Medicaid. Contained within the 10 titles are a multitude of provisions that will change how hand surgeons practice medicine and how they are reimbursed. It is imperative that surgeons are equipped with the knowledge of how this law will affect all physician practices and hospitals., (Copyright © 2014 Elsevier Inc. All rights reserved.)

Cost-effectiveness research is a component of clinical outcomes that addresses both cost and outcomes simultaneously, providing an understanding of what incremental costs, if any, are required for better clinical outcomes. In the current health care climate, these analyses are increasingly performed, and critical, as practitioners must optimize patient care at lower costs. This review discusses cost effectiveness research, its utilization in surgical oncology, and future opportunities provided by its methodologies., (© 2013 Wiley Periodicals, Inc.)

The Patient-Centered Outcomes Research Institute (PCORI) has established distinctive pathways for funding and conducting practical research and has awarded over $318 million for studies covering a wide range of conditions, locations, and socioeconomic characteristics.

With the Patient Protection and Affordable Care Act ("ACA") set to dramatically increase access to medical care, the problem of rising costs will move center stage in health law and policy discussions. "Consumer directed health care" proposals, which provide patients with financial incentives to equate marginal costs and benefits of care at the point of treatment, demand more decisionmaking ability from consumers than is plausible due to bounded rationality. Proposals that seek to change the incentives of health care providers threaten to create conflicts of interest between doctors and patients. New approaches are desperately needed. This Article proposes a government-facilitated but market-based approach to improving efficiency in the private market for medical care that I call "relative value health insurance." This approach focuses on the "choice architecture" necessary to enable even boundedly rational patients to contract for an efficient level of health care services through their health insurance purchase decisions. It uses comparative effectiveness research, which the ACA funds at a significant level for the first time, to rate medical treatments on a scale of one to ten based on their relative value, taking into account expected costs and benefits. These relative value ratings would enable consumers to contract with insurers for different levels of medical care at different prices, reflecting different cost-quality trade-offs. The Article describes both the benefits of relative value health insurance and the impediments to its implementation. It concludes with a brief discussion of how relative value ratings could also help to rationalize expenditures on public health insurance programs.

Comparative effectiveness research (CER) is a broad category of outcomes research encompassing many different methods employed by researchers and clinicians from numerous disciplines. The goal of cancer-focused CER is to generate new knowledge to assist cancer stakeholders in making informed decisions that will improve health care and outcomes of both individuals and populations. There are numerous CER methods that may be used to examine specific questions, including randomized controlled trials, observational studies, systematic literature reviews, and decision sciences modeling. Each has its strengths and weaknesses. To both inform and serve as a reference for readers of this issue of Seminars in Radiation Oncology as well as the broader oncology community, we describe CER and several of the more commonly used approaches and analytical methods., (© 2013 Published by Elsevier Inc.)

Technological advances are a major contributor to rising costs in health care, including radiation oncology. Despite the large amount spent on new technologies, technology assessment remains inadequate, leading to potentially costly and unnecessary use of new technologies. Comparative effectiveness studies have an important role to play in evaluating the benefits and harms of new technologies compared with older technologies and have been identified as a priority area for research by the Radiation Oncology Institute. This article outlines the elements of effective technology assessment, identifies key challenges to comparative effectiveness studies of new radiation oncology technologies, and reviews several examples of comparative effectiveness studies in radiation oncology, including studies on conformal radiation, IMRT, proton therapy, and other concurrent new technologies., (© 2014 Elsevier Inc. All rights reserved.)

Although the analysis of real-world data is the foundation of comparative effectiveness analysis, not all clinical questions are easily approached with patient-derived information. Decision analysis is a set of modeling and analytic tools that simulate treatment and disease processes, including the incorporation of patient preferences, thus generating optimal treatment strategies for varying patient, disease, and treatment conditions. Although decision analysis is informed by evidence-derived outcomes, its ability to test treatment strategies under different conditions that are realistic but not necessarily reported in the literature makes it a useful and complementary technique to more standard data analysis. Similarly, cost-effectiveness analysis is a discipline in which the relative costs and benefits of treatment alternatives are rigorously compared. With the well-recognized increase in highly technical, costly radiation therapy technologies, the cost-effectiveness of these different treatments would come under progressively more scrutiny. In this review, we discuss the theoretical and practical aspects of decision analysis and cost-effectiveness analysis, providing examples that highlight their methodology and utility., (Copyright © 2014 Elsevier Inc. All rights reserved.)

Comparative effectiveness research (CER) and community- based participatory research (CBPR) are two fields of research that do not have a history of strong collaboration. However, CER and CBPR researchers could benefit from interdisciplinary collaboration to design and implement relevant, timely, action-oriented research. This commentary explores field-specific definitions of stakeholders and then outlines various roles stakeholders might play within grant-funded research. Questions such as "What stakeholders should be involved?" and "How are stakeholders involved?" are addressed. The goal of this commentary is to highlight how the expertise and experiences of CBPR investigators can enhance the field of CER and to describe strategies for encouraging stakeholder involvement in CER research through the lens of CBPR. It is recommended that a team-based approach to conducting stakeholder-engaged CER encourages multiple stakeholders and "end users" to contribute their diverse expertise to the research process and contributes to the development of research with an increased likelihood of improving patient health and healthcare., (© 2013 Wiley Periodicals, Inc.)