Clinical Comparative Effectiveness Research Research Articles

ADRD: A REVIEW OF THE PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE’S (PCORI) RESEARCH PORTFOLIO AND PRIORITIES

Abstract Given the aging of the U.S. population, the number of individuals living with Alzheimer’s Disease and Related Dementias (ADRD) is projected to reach 13 million by 2050. There is an urgent need for research efforts to advance prevention, diagnosis, and treatment strategies for ADRD. Furthermore, caregivers of individuals with ADRD often face distinct challenges and experience significant caregiving burden that has an impact on their well-being. To address unmet needs of individuals with ADRD and their caregivers, PCORI has invested nearly $85 million in patient-centered comparative clinical effectiveness research (CER) focused on ADRD and has initiated several stakeholder convenings to identify future research priorities. PCORI’s ADRD portfolio of 15 CER studies includes interventions focused on caregiving, polypharmacy, telehealth, care models, and palliative care. Additionally, PCORI has funded engagement awards, dissemination & implementation initiatives, and research infrastructure projects to help facilitate the conduct and awareness of research on persons living with ADRD and their caregivers. To support continued research in this area, PCORI plans to re-issue a $50 million funding initiative on Healthy Aging with a special area of emphasis on interventions for caregivers of older adults living with ADRD. By systematically synthesizing PCORI’s portfolio and themes from stakeholder convenings in ADRD, we can identify the strengths of current investments and understand evidence gaps to guide PCORI’s future investments. This will enable us to holistically address future real-world, decisional dilemmas most important to patients, caregivers, and other stakeholders.

Surgeon variation in glenoid bone reconstruction procedures for shoulder instability.

Advances in the understanding and management of glenoid bone loss in shoulder instability have led to the development of alternative bony reconstruction techniques to the Latarjet using free bone grafts, but little is known about surgeon adoption of these procedures. This study sought to characterize surgeon variation in the use of glenoid bone reconstruction procedures for shoulder instability and ascertain reasons underlying procedure choice. A 9-question survey was created and distributed to 160 shoulder surgeons members of the PacWest Shoulder and Elbow Society, of whom 65 (41%) responded. The survey asked questions regarding fellowship training, years in practice, surgical volume, preferred methods of glenoid bone reconstruction, and reasons underlying treatment choice. All surgeons completed a fellowship, with an equal number of sports medicine fellowship-trained (46%) and shoulder and elbow fellowship-trained (46%) physicians. The majority had been in practice for at least 6 years (6-10 years: 25%; >10 years: 59%). Most (78%) performed ≤10 glenoid bony reconstructions per year, and 66% indicated that bony procedures represented <10% of their total annual shoulder instability case volume. The open Latarjet was the preferred primary reconstruction method (69%), followed by open free bone block (FBB) (22%), arthroscopic FBB (8%), and arthroscopic Latarjet (1%). Distal tibia allograft (DTA) was the preferred graft (74%) when performing an FBB procedure, followed by iliac crest autograft (18%), and distal clavicle autograft (6%). The top 5 reasons for preferring Latarjet over FBB were the sling effect (57%), the autologous nature of the graft (37%), its robust clinical evidence (22%), low cost (17%), and availability (11%). The top 5 reasons for choosing an FBB procedure were less anatomic disruption (58%), lower complication rate (21%), restoration of articular cartilage interface (16%), graft versatility (11%), and technical ease (11%). Only 20% of surgeons indicated always performing a bony glenoid reconstruction procedure in the noncontact athlete with less than 20% glenoid bone loss. However, that percentage rose to 62% when considering a contact athlete with the same amount of bone loss. Although open Latarjet continues to be the most popular glenoid bony primary reconstruction procedure in shoulder instability, nearly 30% of shoulder surgeons in the western United States have adopted FBB techniques as their preferred treatment modality--with DTA being the most frequently used graft. High-quality comparative clinical effectiveness research is needed to reduce decisional conflict and refine current evidence-based treatment algorithms.

Emerging healthcare interventions: Patient-Centered Outcomes Research Institute's programmatic initiative.

The Patient-Centered Outcomes Research Institute (PCORI) is a nonprofit, nongovernmental organization established by the U.S. Congress to fund comparative clinical effectiveness research focusing on patient-centered outcomes through the engagement of stakeholders. Evaluation of emerging healthcare innovations is one of PCORI's five National Priorities for Health. One such initiative is PCORI's Emerging Technologies and Therapeutics Reports program, established to provide timely overviews of evidence on new drugs and other healthcare technologies. This article provides an overview of completed and ongoing Emerging Technologies and Therapeutics Reports including lessons learned to date. In addition to systematic searches, systematic selection of studies, and transparent reporting of the available evidence, informed by a select number of stakeholders (i.e., key informants), these reports focus on contextual factors shaping the diffusion of emerging technologies that are often not reported in the medical literature. This article also compares processes and methodologies of health technology assessments (HTAs) from a selected number of national and international publicly funded agencies with a goal toward potential future enhancement of PCORI's Emerging Technologies and Therapeutics Reports program. HTAs vary considerably in terms of funding, types of assessments, the role of manufacturers, stakeholder engagement, timeline to complete from the start to the finish of a draft report publication, and communication of uncertainty for informed decision making. Future Emerging Technologies and Therapeutics Reports may focus on rapid reports to support a more expedient development of evidence. Future research could explore the role of contextual factors identified in these reports on targeted evidence generation.

Celebrating 10 years of the Journal of Comparative Effectiveness Research.

Celebrating 10 years of the Journal of Comparative Effectiveness Research.

Patient-Centered Approaches to Transitional Care Research and Implementation: Overview and Insights From Patient-Centered Outcomes Research Institute's Transitional Care Portfolio.

Background:This Special Issue, Future Directions in Transitional Care Research, focuses on the approaches used and lessons learned by researchers conducting care transitions studies funded by the Patient-Centered Outcomes Research Institute (PCORI). PCORI’s approach to transitional care research augments prior research by encouraging researchers to focus on head-to-head comparisons of interventions, the use of patient-centered outcomes, and the engagement of stakeholders throughout the research process.Objectives:This paper introduces the themes and topics addressed by the articles that follow, which are focused on opportunities and challenges involved in conducting patient-centered clinical comparative effectiveness research in transitional care. It provides an overview of the state of the care transitions field, a description of PCORI’s programmatic objectives, highlights of the patient and stakeholder engagement activities that have taken place during the course of these studies, and a brief overview of PCORI’s Transitional Care Evidence to Action Network, a learning community designed to foster collaboration between investigators and their research teams and enhance the collective impact of this body of work.Conclusions:The papers in this Special Issue articulate challenges, lessons learned, and new directions for measurement, stakeholder engagement, implementation, and methodological and design approaches that reflect the complexity of transitional care comparative effectiveness research and seek to move the field toward a more holistic understanding of transitional care that integrates social needs and lifespan development into our approaches to improving care transitions.

CAREGIVERS OF OLDER ADULTS: AN ANALYSIS OF THE PCORI FUNDED RESEARCH PORTFOLIO

A family-centered approach to care is vital, and caregivers play an important role in patient-centered care for older adults. This analysis of the Patient-Centered Outcomes Research Institute’s (PCORI) portfolio of clinical comparative effectiveness research (CER) trials explores the extent to which caregiving for older adults is a focus within our funded studies and examines how these studies incorporate interventions and outcomes related to caregivers. Of 116 studies in the portfolio with a caregiving component, only 35 studies focus on caregivers of older adults. Approximately half of these studies (16) were not focused on a specific disease, but rather included older adults with a variety of diseases. Caregivers were the target of a delivered intervention in 18 studies. Among these studies, all but one included caregivers as part of a multicomponent intervention. The most common intervention components were caregiver training (14 studies) and inclusion of caregivers in the delivery of health services, notably coordination of care (17), home visits (9), integrated care (9), multidisciplinary care teams (9), and clinical decision tools (8). Caregiver-focused outcomes were assessed in 26 studies. The most frequently assessed domains include measures of health and well-being (most commonly psychosocial status; n=20), evaluation of care (most commonly satisfaction; n=8), and health behavior (most commonly attitudes; n=6). In general, given stakeholder interest in family-centered research on older adults, future CER research should include caregivers and/or compare interventions focused solely on the unique needs of caregivers of older adults. Inclusion of caregiver-related outcomes should also be promoted.

Lessons on Patient and Stakeholder Engagement Strategies for Pipeline to Proposal Awards.

The Patient Centered Outcomes Research Institute (PCORI) supports patient-centered clinical comparative effectiveness research (CER) including health disparities and engagement portfolios. In 2013, PCORI launched the Pipeline to Proposal (P2P) mechanism to support development of novel patient- and stakeholder-centered partnerships focused on designing clinical CER funding proposals. By providing a tiered structure of successive small contracts and technical assistance, the P2P mechanism encourages development of new research partnerships among diverse stakeholders. As a comparatively new field, patient-centered outcomes research (PCOR) has few well-delineated methods for engaging patients and other non-scientists in effective teams with academics or clinicians to develop and implement rigorous, scientific research proposals. Community partnered participatory research (CPPR) provides a useful framework for structuring new partnerships. In this article we highlight the origins, development, and prospects of three current examples of funded P2P initiatives based in New Orleans and Los Angeles. We outline how these projects - Prisoner to Patient, the NOLA Partnership, and Resilience Among African American Men - use CPPR principles. We also describe how they have collaborated with, and contributed to, a two-way learning and knowledge exchange among members of the PCORI-funded Community and Patient Partnered Research Network. Lessons learned may be applicable to other groups planning to create new partnerships focused on implementing PCOR.

A Portfolio Analysis of Culturally Tailored Trials to Address Health and Healthcare Disparities.

In 2010, Patient-Centered Outcomes Research Institute (PCORI) was authorized by Congress to improve the quality and relevance of evidence available to help patients, caregivers, employers, insurers, and policy makers make better-informed health decisions. We conducted a qualitative analysis of behavioral health trials in the PCORI Addressing Disparities portfolio to examine cultural tailoring strategies across the following priority populations: racial and ethnic minorities, rural populations, people with low-income or low socioeconomic status, individuals with disabilities, people with low health literacy, and lesbian, gay, bisexual, and transgender (LGBT) communities. The Common Strategies for Enhancing Cultural Appropriateness model was used to examine cultural tailoring strategies within trials. We hypothesized increased intersectionality within a patient population at risk for disparities would correlate with the dosage and type of cultural tailoring strategies applied. Thirty-three behavioral health trials applied cultural tailoring strategies and a majority of trials (n = 30) used three or more strategies. Trends in cultural tailoring were associated with certain racial and ethnic groups; however, increased use of tailoring was not associated with the number of priority populations included in a trial. The PCORI Addressing Disparities portfolio demonstrates how a range of cultural tailoring strategies are used, within comparative clinical effectiveness research trials, to address the needs and intersectionality of patients to reduce health and healthcare disparities.

Engaging Patients in Setting a Patient-Centered Outcomes Research Agenda in Hematopoietic Cell Transplantation

The goal of patient-centered outcomes research (PCOR) is to help patients and those who care for them make informed decisions about healthcare. However, the clinical research enterprise has not involved patients, caregivers, and other nonproviders routinely in the process of prioritizing, designing, and conducting research in hematopoietic cell transplantation (HCT). To address this need the National Marrow Donor Program/Be The Match engaged patients, caregivers, researchers, and other key stakeholders in a 2-year project with the goal of setting a PCOR agenda for the HCT community. Through a collaborative process we identified 6 major areas of interest: (1) patient, caregiver, and family education and support; (2) emotional, cognitive, and social health; (3) physical health and fatigue; (4) sexual health and relationships; (5) financial burden; and (6) models of survivorship care delivery. We then organized into multistakeholder working groups to identify gaps in knowledge and make priority recommendations for critical research to fill those gaps. Gaps varied by working group, but all noted that a historical lack of consistency in measures use and patient populations made it difficult to compare outcomes across studies and urged investigators to incorporate uniform measures and homogenous patient groups in future research. Some groups advised that additional pre-emptory work is needed before conducting prospective interventional trials, whereas others were ready to proceed with comparative clinical effectiveness research studies. This report presents the results of this major initiative and makes recommendations by working group on priority questions for PCOR in HCT.

Combining Propensity Score and Random Coefficient Modelling as an Approach to Analyse Complex Longitudinal Data

ABSTRACT&#x0D; ObjectivesWe looked for an approach to analyze/visualize a set of repeated measures of renal laboratory data (eGFR [estimated Glomerular Filtration Rate] from an observational population-based data set) as safety parameters in a longitudinal design and calculate annual changes in different sub-cohorts. Previous meta-analyses had struggled to address this problem (due to poor data quality and strong heterogeneity in underlying historical studies) and previous large population-based observational studies had only looked into binary outcomes.&#x0D; Particular challenges lay (1) in the complexity of the data set with irregularly spaced observation points, (2) in the observational character of the data with associated bias and confounding by indication and co-medication and (3) in the change of lab method during the observation period.&#x0D; ResultsOut of a population base of 400.000 we analysed linked longitudinal data of more than 1000 eligible patients with over 1000.000 prescription records of index drug or co-prescription drugs. Data were provided by the Dundee University Health Informatics Centre (HIC).&#x0D; We addressed the differences in covariates (which typically can lead to biased estimates of treatment effects in observational studies) via individual propensity scores (to reduce this bias by balancing the covariates in the two groups) and a hierarchical modelling approach.&#x0D; A Random Coefficient Model (via proc mixed in SAS 9.3) proved a much more powerful statistical tool than analysis of covariance of the summary measure during follow-up, particularly as the latter approach is less efficient when applied to longitudinal data with missing data points and irregularly spaced repeated measures.Visualization was achieved with the SAS 9.3 GPLOT procedure combined with a spline function. The historical change in lab method was addressed via a conversion of lab results to an internationally recognized standard (IDMS aligned method).&#x0D; We were able to achieve plausible and more precise estimates of the annual decline in eGFR in the patient group of interest than previous attempts from other research publications. This led to a publication in a high profile journal.&#x0D; ConclusionOur approach of combining a Propensity Score and Random Coefficient Modelling was successful to answer a question in drug safety using repeated measurement data from a longitudinal observational population based data set. This approach may be useful for other research questions in Drug Safety or in Comparative Clinical Effectiveness Research for continuous outcome measures.

The PCORI Engagement Rubric: Promising Practices for Partnering in Research.

Engaging patients, caregivers, and other health care stakeholders as partners in planning, conducting, and disseminating research is a promising way to improve clinical decision making and outcomes. Many researchers, patients, and other stakeholders, however, lack clarity about when and how to engage as partners within the clinical research process. To address the need for guidance on creating meaningful stakeholder partnerships in patient-centered clinical comparative effectiveness research, the Patient-Centered Outcomes Research Institute (PCORI) developed the PCORI Engagement Rubric (Rubric). PCORI developed the Rubric drawing from a synthesis of the literature, a qualitative study with patients, a targeted review of engagement plans from PCORI-funded project applications, and a moderated discussion and review with PCORI's Advisory Panel on Patient Engagement. The Rubric provides a framework for operationalizing engagement to incorporate patients and other stakeholders in all phases of research. It includes: principles of engagement; definitions of stakeholder types; key considerations for planning, conducting, and disseminating engaged research; potential engagement activities; and examples of promising practices from PCORI-funded projects. PCORI designed the Rubric to illustrate opportunities for engagement to researchers interested in applying for PCORI funding and to patients and other stakeholders interested in greater involvement in research. By encouraging PCORI applicants, awardees, and others to apply the rubric, PCORI hopes to shift the research paradigm from one of conducting research on patients as subjects to a pursuit carried out in collaboration with patients and other stakeholders to better reflect the values, preferences, and outcomes that matter to the patient community.

Facilitating the delivery of patient-centered cancer care: Insights from the Patient-Centered Outcomes Research Institute's (PCORI) cancer portfolio.

e18234Background: PCORI funds comparative clinical effectiveness research (CER) that aims to produce evidence-based information to help people make informed health care decisions and improve outcom...

Continuous patient engagement in cardiovascular disease clinical comparative effectiveness research

ABSTRACTResearchers have produced evidence that identifies interventions that reduce cardiovascular disease (CVD) risk; however, despite a significant investment in research CVD remains the leading cause of death. Engaging patients in the research process has the potential to ensure that evidence-based treatments are adopted in real-world practice to improve patient outcomes. The Patient-Centered Outcomes Research Institute has created an Engagement Rubric to guide meaningful engagement in the research process. A 10-step systematic framework to enhance patient engagement throughout the comparative effectiveness research process also has been proposed. This special report identifies the relationship between these two approaches to patient engagement and describes examples of how patients could be engaged in a hypothetical CVD study.

Perioperative outcomes and adverse events of minimally invasive versus open posterior lumbar fusion: meta-analysis and systematic review.

The objective of this study was to determine the clinical comparative effectiveness and adverse event rates of posterior minimally invasive surgery (MIS) compared with open transforaminal or posterior lumbar interbody fusion (TLIF/PLIF). A systematic review of the Medline, EMBASE, PubMed, Web of Science, and Cochrane databases was performed. A hand search of reference lists was conducted. Studies were reviewed by 2 independent assessors to identify randomized controlled trials (RCTs) or comparative cohort studies including at least 10 patients undergoing MIS or open TLIF/PLIF for degenerative lumbar spinal disorders and reporting at least 1 of the following: clinical outcome measure, perioperative clinical or process measure, radiographic outcome, or adverse events. Study quality was assessed using the Grades of Recommendation, Assessment, Development, and Evaluation (GRADE) protocol. When appropriate, a meta-analysis of outcomes data was conducted. The systematic review and reference list search identified 3301 articles, with 26 meeting study inclusion criteria. All studies, including 1 RCT, were of low or very low quality. No significant difference regarding age, sex, surgical levels, or diagnosis was identified between the 2 cohorts (856 patients in the MIS cohort, 806 patients in the open cohort). The meta-analysis revealed changes in the perioperative outcomes of mean estimated blood loss, time to ambulation, and length of stay favoring an MIS approach by 260 ml (p < 0.00001), 3.5 days (p = 0.0006), and 2.9 days (p < 0.00001), respectively. Operative time was not significantly different between the surgical techniques (p = 0.78). There was no significant difference in surgical adverse events (p = 0.97), but MIS cases were significantly less likely to experience medical adverse events (risk ratio [MIS vs open] = 0.39, 95% confidence interval 0.23-0.69, p = 0.001). No difference in nonunion (p = 0.97) or reoperation rates (p = 0.97) was observed. Mean Oswestry Disability Index scores were slightly better in the patients undergoing MIS (n = 346) versus open TLIF/PLIF (n = 346) at a median follow-up time of 24 months (mean difference [MIS - open] = 3.32, p = 0.001). The result of this quantitative systematic review of clinical comparative effectiveness research examining MIS versus open TLIF/PLIF for degenerative lumbar pathology suggests equipoise in patient-reported clinical outcomes. Furthermore, a meta-analysis of adverse event data suggests equivalent rates of surgical complications with lower rates of medical complications in patients undergoing minimally invasive TLIF/PLIF compared with open surgery. The quality of the current comparative evidence is low to very low, with significant inherent bias.

Perioperative Outcomes and Adverse Events of Minimally Invasive versus Open Posterior Lumbar Fusion: Meta-Analysis

Introduction The perioperative outcomes and adverse events of minimally invasive surgery (MIS) for lumbar fusion remain unclear. The purpose of this systematic review and meta-analysis is to summarize the perioperative outcomes and adverse event rates for MIS transforaminal or posterior lumbar interbody fusion (TLIF/PLIF) compared with traditional open midline techniques. Materials and Methods A systematic review of Medline, EMBASE, PubMed, Web of Science, and Cochrane databases were performed. A hand search of reference lists was performed. Studies were reviewed by two independent assessors to identify randomized controlled trials or comparative cohort studies including ≥ 10 patients undergoing MIS or open TLIF/PLIF for degenerative lumbar spinal disorders and reporting at least one of the following: clinical outcome measure; perioperative surgical performance or outcome measure; radiographic outcome; or adverse events. Study quality was assessed using the GRADE protocol. When appropriate, a meta-analysis of outcomes data was conducted. Results The systematic review and reference list search identified 3,306 articles, with 26 meeting study inclusion criteria. All studies, including one randomized controlled trial, were of low or very low quality. No significant difference regarding age, gender, surgical levels, or diagnosis was identified between the two cohorts ( n = 856 MIS/ n = 806 open). Meta-analysis revealed changes in the perioperative outcomes of mean estimated blood loss, time to ambulation, and length of stay favoring an MIS approach by 260 mL ( p &lt; 0.00001), 3.5 days ( p = 0.0006) and 2.9 days ( p &lt; 0.00001), respectively. Operative time was not significantly different between the surgical techniques ( p = 0.78). There was no significant difference in surgical adverse events ( p = 0.97); however, MIS cases were significantly less likely to experience medical adverse events (RR [95% CI] MIS vs. open = 0.39 [0.23–0.69], p = 0.001). No difference in nonunion ( p = 0.97) or reoperation rates ( p = 0.97) was observed. Mean Oswestry Disability Index scores were slightly better in the patients with MIS at a median follow-up time of 24 months (mean difference MIS [ n = 346] vs. open [ n = 346] = 3.32, p = 0.001). Conclusion The quality of the current comparative evidence is low to very low with significant inherent bias. The result of this quantitative systematic review of clinical comparative effectiveness research examining MIS versus open TLIF/PLIF for degenerative lumbar pathology suggests equipoise in patient reported clinical outcomes. Furthermore, meta-analysis of adverse event data suggests equivalent rates of surgical complications with lower rates of medical complications in patients undergoing MIS TLIF/PLIF compared with open surgery.

The patient-centered outcomes research institute: research done differently.

The Patient-Centered Outcomes Research Institute (PCORI) was authorized by the Patient Protection and Affordable Care Act of 2010 to fund comparative clinical effectiveness research to provide reliable evidence to help patients and their healthcare providers make informed decisions. The aim of this paper is to describe the synergy between nursing research and PCORI funding priorities, discuss unique aspects of PCORI funding, and identify the implications for nurse researchers. Goals of nursing research are well aligned with PCORI interests. Given this synergy, many phenomena that nursing scientists study could become topics of a competitive proposal for PCORI funding. Major aspects of PCORI funding with implications for nurse researchers include the nature of the questions asked, funding priorities, engagement of patients and stakeholders, compliance with the PCORI's methodology standards, and the proposal review criteria and process.

What does US$1.1 billion buy? An investment in the future.

Journal of Comparative Effectiveness ResearchVol. 3, No. 6 OpinionWhat does US$1.1 billion buy? An investment in the futureLisa A SimpsonLisa A SimpsonE-mail Address: Lisa.Simpson@academyhealth.orgAcademyHealth, 1150 17th Street, NW, Suite 600, Washington, DC 20036, USASearch for more papers by this authorPublished Online:12 Dec 2014https://doi.org/10.2217/cer.14.74AboutSectionsView ArticleView Full TextPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail View articleKeywords: academicCERfundingimpactincentivespolicyresearch infrastructureworkforceReferences1 National Academies of Science. Rising Above the Gathering Storm: Energizing and Employing America for a Brighter Economic Future. National Academy Press, Washington, DC, USA (2007).Google Scholar2 Institute of Medicine, National Academy of Sciences, and National Academy of Engineering. 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The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.No writing assistance was utilized in the production of this manuscript.PDF download

The PCORI Perspective on Patient-Centered Outcomes Research

The Patient-Centered Outcomes Research Institute (PCORI) was established as part of the US Patient Protection and Affordable Care Act of 2010 to fund patient-centered comparative clinical effectiveness research, extending the concept of patient-centeredness from health care delivery to health care research. In the United States, patient-centered outcomes research is new and not defined in the legislation, and the rationale is unclear to many. In this Viewpoint, we address 2 related questions: What does patientcenteredness in research mean? Why conduct patientcentered outcomes research? The essence of the PCORI definition of patientcentered outcomes research is the evaluation of questions and outcomes meaningful and important to patients and caregivers. The definition rests on the axiom that patients have unique perspectives that can change and improve the pursuit of clinical questions. Relevant to both the definition and rationale is the hypothesis that including the perspectives of end users of the research, which include patients, physicians, and other health care stakeholders, will enhance the relevance of research to actual health decisions these end users face. In turn, increased relevance is hypothesized to improve uptake of the evidence and improve the likelihood that patients will achieve the health outcomes they desire. Although this proposed causal chain is readily understood in relation to questions of direct interest to pa

Recommendations for Incorporating Patient-Reported Outcomes Into Clinical Comparative Effectiveness Research in Adult Oncology

Examining the patient's subjective experience in prospective clinical comparative effectiveness research (CER) of oncology treatments or process interventions is essential for informing decision making. Patient-reported outcome (PRO) measures are the standard tools for directly eliciting the patient experience. There are currently no widely accepted standards for developing or implementing PRO measures in CER. Recommendations for the design and implementation of PRO measures in CER were developed via a standardized process including multistakeholder interviews, a technical working group, and public comments. Key recommendations are to include assessment of patient-reported symptoms as well as health-related quality of life in all prospective clinical CER studies in adult oncology; to identify symptoms relevant to a particular study population and context based on literature review and/or qualitative and quantitative methods; to assure that PRO measures used are valid, reliable, and sensitive in a comparable population (measures particularly recommended include EORTC QLQ-C30, FACT, MDASI, PRO-CTCAE, and PROMIS); to collect PRO data electronically whenever possible; to employ methods that minimize missing patient reports and include a plan for analyzing and reporting missing PRO data; to report the proportion of responders and cumulative distribution of responses in addition to mean changes in scores; and to publish results of PRO analyses simultaneously with other clinical outcomes. Twelve core symptoms are recommended for consideration in studies in advanced or metastatic cancers. Adherence to methodologic standards for the selection, implementation, and analysis/reporting of PRO measures will lead to an understanding of the patient experience that informs better decisions by patients, providers, regulators, and payers.

In Tribute: Dennis L. Hart, PT, PhD, A Functional Outcome Measurement Visionary

Previous article Next article Full AccessEditorialIn Tribute: Dennis L. Hart, PT, PhD, A Functional Outcome Measurement VisionaryMark W. Werneke, PT, MSc, , PT, MScDaniel Deutscher, PT, PhD, , PT, PhDSteven Z. George, PT, PhD, PT, PhDAuthorsMark W. Werneke, PT, MScSearch for more papers by this author, Daniel Deutscher, PT, PhDSearch for more papers by this author, Steven Z. George, PT, PhDSearch for more papers by this authorJournal of Orthopaedic & Sports Physical TherapyPublished OnlineJune 1, 2012Volume42Issue6Pages489-490https://www.jospt.org/doi/10.2519/jospt.2012.0109SectionsPDFPDF PLUS ToolsAdd to FavoritesDownload CitationsTrack CitationsPermissions ShareShare onFacebookTwitterLinkedInRedditEmail AboutAbstractIn remembrance of Dennis L. Hart, PT, PhD.J Orthop Sports Phys Ther 2012;42(6):489–490. doi:10.2519/jospt.2012.0109On April 11, 2012, our physical therapy profession lost yet another esteemed colleague after an extended battle with cancer. Although Dennis is not with us physically, we are confident that his uncompromising love and contributions to our physical therapy profession will live on. Dennis was a clinician, national and international speaker, and renowned researcher, educator, and author. He started his education at Northeastern University as a math major before changing his major and graduating in 1971 to begin his career in physical therapy. He continued his formal education by completing his Master of Public Administration at New Haven University in 1976 and his PhD in Industrial Engineering at West Virginia University in 1985.Dennis's scholarly contributions were extensive. He published over 85 articles in scientifically refereed journals and authored chapters in several books on industrial rehabilitation. He also served on the editorial board for the Journal of Rehabilitation Outcomes Measurement; reviewed manuscripts and NIH grants; mentored students who became prominent authors in the physical therapy literature; and received multiple awards, including the 2010 John Medeiros, Distinguished Authorship Award for best clinical research paper published in the Journal of Manual & Manipulative Therapy, the 2004 APTA Chattanooga Research Award for best clinical research paper published in Physical Therapy, and the 2000 Rose Excellence in Research Award. Dennis will be best remembered for his leadership and expertise in the development and clinical application of rehabilitation functional outcome measures using modern psychometrics based on item response theory mathematics.4 Over the past 2 years, Dennis challenged a group of physical therapy researchers to publish a series of papers related to efficient measurement of key clinical predictors and outcomes. His last article, in print in this issue of the Journal of Orthopaedic & Sports Physical Therapy, compared the relative validity and important change between 2 disease-specific functional outcome measures for patients with lumbar impairments: the modified Oswestry Low Back Pain Disability Questionnaire and the functional status lumbar computerized adaptive test.5Download FigureDownload PowerPointDennis believed in continuous clinical data collection and performance assessment. Over the past 14 years, he was a founding member and Director of Consulting and Research at Focus On Therapeutic Outcomes, Inc. Dennis's professional vision for future research was the use of large clinical databases to promote clinical comparative effectiveness research with the ultimate goal to identify the right treatment for the right patient.1 Comparative effectiveness research is now consistently recognized by many prominent researchers and clinicians as an important research method complementing data and results from other research designs, such as the randomized control trial.2,6 Dennis was the first physical therapist to develop, implement, and publish a value-based purchasing method for quantifying the quality of orthopaedic outpatient rehabilitation, demonstrating treatment effectiveness and efficiency when managing patients.3 His favorite saying was, “In God we trust—everyone else better bring data.”With this, Dennis's high professional productivity left us a rich heritage of knowledge to be studied and implemented in routine care for the benefit of our patients. His work influenced numerous researchers and brought thousands of physical therapists both in the United States and Israel to better understand and integrate data collected during everyday clinical practice to help guide patient management and improve patient outcomes. Dennis will be greatly missed, but his contributions to the physical therapy profession will be long lasting.