Steven B. Clauser

Standardizing patient-reported outcomes assessment in cancer clinical trials: a patient-reported outcomes measurement information system initiative.

Patient-reported outcomes (PROs), such as symptom scales or more broad-based health-related quality-of-life measures, play an important role in oncology clinical trials. They frequently are used to help evaluate cancer treatments, as well as for supportive and palliative oncology care. To be most beneficial, these PROs must be relevant to patients and clinicians, valid, and easily understood and interpreted. The Patient-Reported Outcomes Measurement Information System (PROMIS) Network, part of the National Institutes of Health Roadmap Initiative, aims to improve appreciably how PROs are selected and assessed in clinical research, including clinical trials. PROMIS is establishing a publicly available resource of standardized, accurate, and efficient PRO measures of major self-reported health domains (eg, pain, fatigue, emotional distress, physical function, social function) that are relevant across chronic illnesses including cancer. PROMIS is also developing measures of self-reported health domains specifically targeted to cancer, such as sleep/wake function, sexual function, cognitive function, and the psychosocial impacts of the illness experience (ie, stress response and coping; shifts in self-concept, social interactions, and spirituality). We outline the qualitative and quantitative methods by which PROMIS measures are being developed and adapted for use in clinical oncology research. At the core of this activity is the formation and application of item banks using item response theory modeling. We also present our work in the fatigue domain, including a short-form measure, as a sample of PROMIS methodology and work to date. Plans for future validation and application of PROMIS measures are discussed.

Development of the National Cancer Institute’s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

The standard approach for documenting symptomatic adverse events (AEs) in cancer clinical trials involves investigator reporting using the National Cancer Institutes (NCIs) Common Terminology Criteria for Adverse Events (CTCAE). Because this approach underdetects symptomatic AEs, the NCI issued two contracts to create a patient-reported outcome (PRO) measurement system as a companion to the CTCAE, called the PRO-CTCAE. This Commentary describes development of the PRO-CTCAE by a group of multidisciplinary investigators and patient representatives and provides an overview of qualitative and quantitative studies of its measurement properties. A systematic evaluation of all 790 AEs listed in the CTCAE identified 78 appropriate for patient self-reporting. For each of these, a PRO-CTCAE plain language term in English and one to three items characterizing the frequency, severity, and/or activity interference of the AE were created, rendering a library of 124 PRO-CTCAE items. These items were refined in a cognitive interviewing study among patients on active cancer treatment with diverse educational, racial, and geographic backgrounds. Favorable measurement properties of the items, including construct validity, reliability, responsiveness, and between-mode equivalence, were determined prospectively in a demographically diverse population of patients receiving treatments for many different tumor types. A software platform was built to administer PRO-CTCAE items to clinical trial participants via the internet or telephone interactive voice response and was refined through usability testing. Work is ongoing to translate the PRO-CTCAE into multiple languages and to determine the optimal approach for integrating the PRO-CTCAE into clinical trial workflow and AE analyses. It is envisioned that the PRO-CTCAE will enhance the precision and patient-centeredness of adverse event reporting in cancer clinical research.

The Organization of Multidisciplinary Care Teams: Modeling Internal and External Influences on Cancer Care Quality

Quality cancer treatment depends upon careful coordination between multiple treatments and treatment providers, the exchange of technical information, and regular communication between all providers and physician disciplines involved in treatment. This article will examine a particular type of organizational structure purported to regularize and streamline the communication between multiple specialists and support services involved in cancer treatment: the multidisciplinary treatment care (MDC) team. We present a targeted review of what is known about various types of MDC team structures and their impact on the quality of treatment care, and we outline a conceptual model of the connections between team context, structure, process, and performance and their subsequent effects on cancer treatment care processes and patient outcomes. Finally, we will discuss future research directions to understand how MDC teams improve patient outcomes and how characteristics of team structure, culture, leadership, and context (organizational setting and local environment) contribute to optimal multidisciplinary cancer care.

Impact of cancer on health-related quality of life of older Americans.

BACKGROUND The impact of cancer on health-related quality of life (HRQOL) is poorly understood because of the lack of baseline HRQOL status before cancer diagnosis. To our knowledge, this is the first population-based study to quantify the nature and extent of HRQOL changes from before to after cancer diagnosis for nine types of cancer patients and to compare their health with individuals without cancer. METHODS The Surveillance, Epidemiology, and End Results cancer registry data were linked with the Medicare Health Outcomes Survey (MHOS) data; data were collected from Medicare beneficiaries who were aged 65 years and older from 1998 through 2003. Cancer patients (n = 1432; with prostate, breast, colorectal, lung, bladder, endometrial, or kidney cancers; melanoma; or non-Hodgkin lymphoma [NHL]) were selected whose first cancer diagnosis occurred between their baseline and follow-up MHOS assessments. Control subjects without cancer (n = 7160) were matched to cancer patients by use of propensity scores that were estimated from demographics and comorbid medical conditions. Analysis of covariance models were used to estimate changes in HRQOL as assessed with the Medical Outcomes Study Short Form-36 survey (mean score = 50, SD = 10). All statistical tests were two-sided. RESULTS Patients with all cancer types (except melanoma and endometrial cancer) reported statistically significant declines in physical health (mean scores: prostate cancer = -3.4, 95% confidence interval [CI] = -2.5 to -4.2; breast cancer = -3.5, 95% CI = -2.5 to -4.5; bladder cancer = -4.3, 95% CI = -2.5 to -6.1; colorectal cancer = -4.4, 95% CI = -3.3 to -5.5; kidney cancer = -5.7, 95% CI = -3.2 to -8.2; NHL = -6.7, 95% CI = -4.4 to -9.1; and lung cancer = -7.5, 95% CI = -5.9 to -9.2) compared with the control subjects (mean score = -1.8, 95% CI = -1.6 to -2.0) (all P < .05). However, only lung (mean score = -5.4, 95% CI = -3.5 to -7.2), colorectal (mean score = -3.5, 95% CI = -2.2 to -4.7), and prostate (mean score = -2.8, 95% CI = -1.8 to -3.7) cancer patients showed statistically significant decreases in mental health relative to the mean change of the control subjects (mean score = -1.2, 95% CI = -0.9 to -1.4) (all P < .05). CONCLUSION These findings provide validation of the specific deleterious effects of cancer on HRQOL and an evidence base for future research and clinical interventions aimed at understanding and remediating these effects.

Introduction: Understanding and Influencing Multilevel Factors Across the Cancer Care Continuum

Health care in the United States is notoriously expensive while often failing to deliver the care recommended in published guidelines. There is, therefore, a need to consider our approach to health-care delivery. Cancer care is a good example for consideration because it spans the continuum of health-care issues from primary prevention through long-term survival and end-of-life care. In this monograph, we emphasize that health-care delivery occurs in a multilevel system that includes organizations, teams, and individuals. To achieve health-care delivery consistent with the Institute of Medicines six quality aims (safety, effectiveness, timeliness, efficiency, patient-centeredness, and equity), we must influence multiple levels of that multilevel system. The notion that multiple levels of contextual influence affect behaviors through interdependent interactions is a well-established ecological view. This view has been used to analyze health-care delivery and health disparities. However, experience considering multilevel interventions in health care is much less robust. This monograph includes 13 chapters relevant to expanding the foundation of research for multilevel interventions in health-care delivery. Subjects include clinical cases of multilevel thinking in health-care delivery, the state of knowledge regarding multilevel interventions, study design and measurement considerations, methods for combining interventions, time as a consideration in the evaluation of effects, measurement of effects, simulations, application of multilevel thinking to health-care systems and disparities, and implementation of the Affordable Care Act of 2010. Our goal is to outline an agenda to proceed with multilevel intervention research, not because it guarantees improvement in our current approach to health care, but because ignoring the complexity of the multilevel environment in which care occurs has not achieved the desired improvements in care quality outlined by the Institute of Medicine at the turn of the millennium.

Patient-Reported Outcomes Assessment in Cancer Trials: Taking Stock, Moving Forward

To evaluate and improve the use of cancer trial end points that reflect the patients own perspective, the National Cancer Institute organized an international conference, Patient-Reported Outcomes Assessment in Cancer Trials (PROACT), in 2006. The 13 preceding articles in this special issue of the Journal were commissioned in preparation for or in response to the PROACT conference, which was cosponsored by the American Cancer Society. Drawing from these articles and also commentary from the conference itself, this concluding report takes stock of what has been learned to date about the successes and challenges in patient-reported outcome (PRO) assessment in phase III, phase II, and symptom management trials in cancer and identifies ways to improve the scientific soundness, feasibility, and policy relevance of PROs in trials. Building on this synthesis of lessons learned, this article discusses specific administrative policies and management procedures to improve PRO data collection, analysis, and dissemination of findings; opportunities afforded by recent methodologic and technologic advances in PRO data collection and analysis to enhance the scientific soundness and cost efficiency of PRO use in trials; and the importance of better understanding the usefulness of PRO data to the full spectrum of cancer decision makers, including patients and families, health providers, public and private payers, regulatory agencies, and standards-setting organizations.

The role of primary care physicians in cancer care.

ABSTRACTBACKGROUNDThe demand for oncology services in the United States (US) is increasing, whereas a shortage of oncologists looms. There is the need for a better understanding of the involvement of primary care physicians (PCPs) in cancer care.OBJECTIVETo characterize the role of PCPs in cancer care, compare it with that of oncologists, and identify factors explaining greater PCP involvement in cancer care.DESIGNNational survey of physicians caring for cancer patients conducted by the Cancer Care Outcomes Research and Surveillance Consortium.PARTICIPANTS1694 PCPs; 1621 oncologists.MEASUREMENTSQuestionnaires mailed during 2005 and 2006 examined the participation of physicians in 12 aspects of care for cancer patients.MAIN RESULTSOver 90% of PCPs fulfilled general medical care roles for patients with cancer such as managing comorbid conditions, chronic pain, or depression; establishing do-not-resuscitate status; and referring patients to hospice. Oncologists were less involved in these roles. Determining the treatment preferences of individual patients and deciding on the use of surgery were the only cancer care roles in which ≥50% of PCPs participated. Twenty-two percent of PCPs reported no direct involvement in cancer care roles while 19% reported heavy involvement. PCPs who were aged ≥50 years, were internists or geriatricians, taught medical students, saw more cancer patients, or experienced referral barriers fulfilled more roles. Rural practice location was not associated with greater PCP involvement in cancer care.CONCLUSIONSPCPs across the US have an active role in cancer patient management. Determining the optimal interface between PCPs and oncologists in delivering and coordinating cancer care is an important area for future research.

Understanding high-quality cancer care: a summary of expert perspectives.

The Institute of Medicine (IOM) report Crossing the Quality Chasm proposed 6 aims for high‐quality healthcare: effective, safe, timely, efficient, equitable, and patient‐centered, and emphasized care coordination. Through interviews with nationally recognized experts in healthcare quality, perspectives on barriers and facilitators to achieving these aims for cancer patients were elicited.

Impact of diagnosis and treatment of clinically-localized prostate cancer on health-related quality of life for older Americans: a population-based study

Few studies have measured longitudinal changes in health‐related quality of life (HRQOL) among patients with prostate cancer starting before their cancer diagnosis or have provided simultaneous comparisons with a matched noncancer cohort. In the current study, the authors addressed these gaps by providing unique estimates of the effects of a cancer diagnosis on HRQOL accounting for the confounding effects of ageing and comorbidity.

In Search of Synergy: Strategies for Combining Interventions at Multiple Levels

The social ecological perspective provides a compelling justification for multilevel intervention. Yet, it offers little guidance for selecting interventions that work together in complementary or synergistic ways. Using a causal modeling framework, we describe five strategies for increasing potential complementarity or synergy among interventions that operate at different levels of influence: accumulation, amplification, facilitation, cascade, and convergence. We illustrate these strategies with examples of multilevel interventions to improve the quality of cancer treatment.