Improving Comparative Effectiveness Research of Complex Health Interventions: Standards from the Patient-Centered Outcomes Research Institute (PCORI).

INTRODUCTION: Complex health interventions (CHIs) are increasingly studied in comparative effectiveness research (CER), and there is a need for improvements in CHI research practices. The Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee (MC) launched an effort in 2016 to develop formal guidance on this topic. OBJECTIVE: To develop a set of minimal standards for scientifically valid, transparent, and reproducible CER studies of CHIs. The standards are intended to apply to research examining a broad range of healthcare interventions including delivery system, behavior change, and other non-pharmacological interventions. METHODS: We conducted a literature review, reviewed existing methods guidance, and developed standards through an iterative process involving the MC, two panels of external research methods experts, and a 60-day public comment period. The final standards were approved by the PCORI MC and adopted by the PCORI Board of Governors on April 30, 2018. RESULTS: The final standards include the following: (1) fully describe the intervention and comparator and define their core functions, (2) specify the hypothesized causal pathways and their theoretical basis, (3) specify how adaptations to the form of the intervention and comparator will be allowed and recorded, (4) plan and describe a process evaluation, and (5) select patient outcomes informed by the causal pathway. DISCUSSION: The new standards offer three major contributions to research: (1) they provide a simple framework to help investigators address the major methodological features of a CHI study, (2) they emphasize the importance of the causal model and the need to understand how a CHI achieves its effects rather than simply measuring these effects, and (3) they require description of a CHI using the concepts of core functions and forms. While these standards apply formally to PCORI-funded CER studies, they have broad applicability.

Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute.

Charged with ensuring that research produces useful evidence to inform health decisions, the Patient-Centered Outcomes Research Institute (PCORI) requires investigators to engage patients and other health care stakeholders, such as clinicians and payers, in the research process. Many PCORI studies result in articles published in peer-reviewed journals that detail research findings and engagement's role in research. To inform practices for engaging patients and others as research partners, we analyzed 126 articles that described engagement approaches and contributions to research. PCORI projects engaged patients and others as consultants and collaborators in determining the study design, selecting study outcomes, tailoring interventions to meet patients' needs and preferences, and enrolling participants. Many articles reported that engagement provided valuable contributions to research feasibility, acceptability, rigor, and relevance, while a few noted trade-offs of engagement. The findings suggest that engagement can support more relevant research through better alignment with patients' and clinicians' real-world needs and concerns.

Patient-centered Outcomes Research in Pulmonary, Critical Care, and Sleep Medicine. An Official American Thoracic Society Workshop Report.

Patient-centered outcomes research (PCOR) represents a paradigm shift in research methods aimed to create the body of evidence that supports clinical practice and informs health care decisions. PCOR integrates patients and other key stakeholders including family members, policy makers, clinicians, and patient advocates and advocacy groups as research partners throughout all stages of the research process. The importance of PCOR has received increased recognition, yet there is little evidence available to help guide researchers interested in the design and conduct of PCOR. In May 2014, we convened a workshop to identify key issues related to designing, conducting, and disseminating findings from PCOR studies. Workshop participants included a diverse group of patients, patient advocates, clinicians (physicians, nurses, psychologists, and advanced practice providers), researchers, administrators, and funders within and beyond the pulmonary, critical care, and sleep medicine communities. Participants identified important issues and considerations to address when undertaking PCOR. In this report, we summarize the results of this workshop to inform members of the pulmonary, sleep, and critical care community interested in participating in PCOR. Key findings include the following: 1) requirements for research to be considered PCOR; 2) the potential significant impact of PCOR on patients, clinicians, and researchers; 3) guiding principles and practical strategies to form successful patient-centered research partnerships, conduct PCOR, and disseminate study results to a broad audience of stakeholders; 4) benefits and challenges of PCOR for researchers; and 5) resources available within the American Thoracic Society to help with the conduct of PCOR.

Providers' Note-Writing Practices for Post-traumatic Stress Disorder at Five United States Veterans Affairs Facilities.

The capacity of electronic health records (EHRs) to capture desired information depends on the practices of health care providers. These practices have not been well studied in relation to post-traumatic stress disorder (PTSD). This qualitative study investigated how providers write EHR notes on PTSD through 38 interviews with providers working at five Veterans Affairs (VA) hospitals across the United States of America. Two overarching themes were prominent in the results. Providers used progress notes primarily to remember and access details for direct patient care, but only rarely for care coordination. Providers infrequently recorded information not judged to directly contribute to improved care, sometimes deliberately omitting information perceived to jeopardize patients' access to, or quality of, care. Omitted information frequently included sexual or non-military trauma. Understanding providers' thought processes can help clinicians be aware of the limitations of EHR notes as a tool for learning the histories of new patients. Similarly, researchers relying on EHR data for PTSD research should be aware of likely areas of missing data.

Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases.

Clinical research in rare diseases, including alpha-1 antitrypsin deficiency (AATD), faces challenges not shared by common disease research. These challenges may include the limited number of patient volunteers available for research, lack of natural history studies on which to base many clinical trial interventions, an urgency for the development of drug therapies given the often poor prognosis of rare diseases and uncertainties about appropriate biomarkers and clinical outcomes critical to clinical trial design. To address these challenges and initiate formal discussions among key stakeholders-patients, researchers, industry, federal regulators-the Alpha-1 Foundation hosted the Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases conference February 3-4, 2014 in Bethesda, Maryland. Discussions at the conference led to the conclusions that 1) adaptive designs should be considered for rare disease clinical trials yet more dialogue and study is needed to make these designs feasible for smaller trials and to address current limitations; 2) natural history studies, including the identification of appropriate biomarkers are critically needed and precompetitive collaborations may offer a means of creating these costly studies; and 3) patient registries and databases within the rare disease community need to be more publicly available and integrated, particularly for AATD. This report summarizes the discussions leading to these conclusions.

A systematic review of approaches for engaging patients for research on rare diseases.

BACKGROUND: Patients with rare diseases have limited access to useful information to guide treatment decisions. Engagement of patients and other stakeholders in clinical research may help to ensure that research efforts in rare diseases address relevant clinical questions and patient-centered health outcomes. Rare disease organizations may provide an effective means to facilitate patient engagement in research. However, the effectiveness of patient-engagement approaches, particularly for the study of rare diseases, has not been well studied. OBJECTIVES: To synthesize evidence about engagement of patients and other stakeholders in research on rare diseases, including the role of rare disease organizations in facilitating patient-centered research. METHODS/RESEARCH DESIGN: A systematic review and gray literature search were guided by a technical expert panel composed of patient representatives, clinicians, and researchers. English-language studies that engaged patients or other stakeholders in research on rare diseases or evaluated engagement were included. Studies were assessed on how well key research questions were answered, based on the level of detail describing engagement activities and whether outcomes from engagement were assessed. RESULTS: Thirty-five studies were included, although many reported minimal information on engagement. Patients and other stakeholders were most commonly engaged to identify patient-centered research agendas, to select which study outcomes were important to patients, to provide input on study design, and to identify strategies for increasing enrollment in trials. Rare disease organizations mainly helped provide access to patients and communicated research opportunities and findings. They also helped promote collaborative networks and provided financial support for research infrastructures. Although authors reported benefits of engagement and identified changes to their research processes, no empirical assessments of engagement practices and their effectiveness were found. CONCLUSIONS: Researchers studying rare diseases can obtain patient input regarding which research questions and health outcomes to study; however, the most effective approaches to engagement have not been well defined.

Congestive heart failure self-management among US veterans: The role of personal and professional advocates.

OBJECTIVE: Understand patients' experiences with primary care services for congestive heart failure (CHF) and explore the relationship between health services and self-management. METHODS: We conducted semi-structured interviews with thirty-nine patients with CHF receiving care at one Veterans Affairs Medical Center (VA). We analyzed data using thematic content analysis. RESULTS: Participants acknowledged the importance of ongoing engagement in the plan of care for CHF. They attributed success in this effort to be greatly influenced by personal advocates. The advocates included both members of the healthcare team with whom they had a continuity relationship and friends or family members who assisted on a daily basis. Participants also identified psychological symptoms as a major barrier to carrying out self-care. CONCLUSION: Patients identify relationships with health care workers, help from family and friends, and mental health problems as major influences on the ability to manage their CHF. PRACTICE IMPLICATIONS: Efforts to optimize CHF self-management should attend to health system and psychosocial barriers to care.

Pressure ulcer treatment strategies: a systematic comparative effectiveness review.

BACKGROUND: Pressure ulcers affect as many as 3 million Americans and are major sources of morbidity, mortality, and health care costs. PURPOSE: To summarize evidence comparing the effectiveness and safety of treatment strategies for adults with pressure ulcers. DATA SOURCES: MEDLINE, EMBASE, CINAHL, Evidence-Based Medicine Reviews, Cochrane Central Register of Controlled Trials, Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects, and Health Technology Assessment Database for English- or foreign-language studies; reference lists; gray literature; and individual product packets from manufacturers (January 1985 to October 2012). STUDY SELECTION: Randomized trials and comparative observational studies of treatments for pressure ulcers in adults and noncomparative intervention series (n > 50) for surgical interventions and evaluation of harms. DATA EXTRACTION: Data were extracted and evaluated for accuracy of the extraction, quality of included studies, and strength of evidence. DATA SYNTHESIS: 174 studies met inclusion criteria and 92 evaluated complete wound healing. In comparison with standard care, placebo, or sham interventions, moderate-strength evidence showed that air-fluidized beds (5 studies [n = 908]; high consistency), protein-containing nutritional supplements (12 studies [n = 562]; high consistency), radiant heat dressings (4 studies [n = 160]; moderate consistency), and electrical stimulation (9 studies [n = 397]; moderate consistency) improved healing of pressure ulcers. Low-strength evidence showed that alternating-pressure surfaces, hydrocolloid dressings, platelet-derived growth factor, and light therapy improved healing of pressure ulcers. The evidence about harms was limited. LIMITATION: Applicability of results is limited by study quality, heterogeneity in methods and outcomes, and inadequate duration to assess complete wound healing. CONCLUSION: Moderate-strength evidence shows that healing of pressure ulcers in adults is improved with the use of air-fluidized beds, protein supplementation, radiant heat dressings, and electrical stimulation.

Abdominal bloating in employed adults: prevalence, risk factors, and association with other bowel disorders.

UNLABELLED: BACKGROUND Bloating is common, but its significance as a marker of underlying disease has not been defined. AND AIMS: We report on risk factors for bloating, its relationship to physical activity and quality of life (QOL), and its predictive value for functional bowel disorders. METHODS: This is a cross-sectional population-based study of 1,069 employees of the Veterans Affairs Black Hills Health Care System. The validated Bowel Disease Questionnaire was used to identify subjects with abdominal bloating and other bowel disorders. The association of bloating with QOL was assessed using the SF36 (Short-Form 36) questionnaire. Physical activity was assessed using the modified Baecke questionnaire. RESULTS: The response rate was 72% (723 of 1,069). Bloating was reported by 21% of all subjects (95% confidence interval [CI] 17.7-23.7), 64% with irritable bowel syndrome (IBS), 35% with non-IBS constipation, 23% with non-IBS diarrhea, and 42% with dyspepsia. Functional bloating (i.e., bloating in the absence of other bowel disorders) was reported by 7% of subjects (95% CI 5.2-9.0). Of those with bloating, 28% had IBS, 25% non-IBS constipation, 8% non-IBS diarrhea, and 30% dyspepsia. The positive and negative predictive values of bloating in the diagnosis of functional bowel disorder were 66% and 87%, respectively. The only risk factors were smoking and high-dose aspirin. Bloating was not associated with physical activity. QOL on all subscales of SF36 was lower in subjects with bloating than those without bloating. CONCLUSIONS: Bloating is a common symptom in otherwise healthy adults, and is often associated with but not predictive of functional bowel disorders. Smoking and high-dose aspirin are associated with bloating while physical activity is not.

Depression decision support in primary care: a cluster randomized trial.

BACKGROUND: Intensive collaborative interventions improve depression outcomes, but the benefit of less intensive interventions is not clear. OBJECTIVE: To determine whether decision support improves outcomes for patients with depression. DESIGN: Clinician-level, cluster randomized, controlled trial. SETTING: 5 primary care clinics of 1 Veterans Affairs medical center. PARTICIPANTS: 41 primary care clinicians, and 375 patients with depression (Patient Health Questionnaire [PHQ-9] depression scores of 10 to 25 or Hopkins Symptom Checklist-20 [SCL-20] scores > or = 1.0). MEASUREMENTS: The primary outcome was change in depression score (SCL-20) at 6 and 12 months. Secondary outcomes were health-related quality-of-life (36-item Short Form for Veterans [SF-36V] score), patient satisfaction, antidepressant use, and health care utilization. INTERVENTION: Clinicians received depression education and were randomly assigned to depression decision support or usual care. The depression decision support team, which consisted of a psychiatrist and nurse, provided 1 early patient educational contact and depression monitoring with feedback to clinicians over 12 months. RESULTS: Although SCL-20 depression scores improved in both groups, the intervention had no effect compared with usual care. The difference in slopes comparing intervention and control over 12 months was 0.20 (95% CI, -0.37 to 0.78; P = 0.49), which was neither clinically nor statistically significant. Changes in SF-36V scores also did not differ between groups. At 12 months, intervention patients reported greater satisfaction (P = 0.002) and were more likely to have had at least 1 mental health specialty appointment (41.1% vs. 27.2%; P = 0.025), to have received any antidepressant (79.3% vs. 69.3%; P = 0.041), and to have received antidepressants for 90 days or more (76.2% vs. 61.6%; P = 0.008). LIMITATIONS: Usual care clinicians received depression education and had on-site mental health support, which may have mitigated intervention effectiveness. CONCLUSIONS: Decision support improved processes of care but not depression outcomes. More intensive care management or specialty treatment may be needed to improve depression outcomes.

Diagnosis, access and outcomes: Update of a systematic review of telemedicine services.

Telemedicine services are being increasingly used. Although insurers and other payers are covering some services in the USA, the rationale for these coverage decisions is not always evidence-based. We reviewed the literature for telemedicine services that substitute for face-to-face medical diagnosis and treatment. We focused on three types of telemedicine services: store-and-forward, home-based and office/hospital-based services. Studies were included if they were relevant to at least one of the three study areas, addressed at least one key question and contained reported results. We excluded articles that did not study a service requiring face-to-face encounters (i.e. teleradiology was excluded). Our search initially identified 4083 citations. After review, 597 were judged to be potentially relevant at the title/abstract level. Following a full-text review, 106 studies were included. Store-and-forward services have been studied in many specialties, the most common being dermatology, wound care and ophthalmology. The evidence for their efficacy is mixed. Several limited studies showed the benefits of home-based telemedicine interventions in chronic diseases. Studies of office/hospital-based telemedicine suggest that telemedicine is most effective for verbal interactions, e.g. videoconferencing for diagnosis and treatment in specialties like neurology and psychiatry. There are still significant gaps in the evidence base between where telemedicine is used and where its use is supported by high-quality evidence. Further well-designed research is necessary to understand how best to deploy telemedicine services in health care.

Survival, mortality, and location of death for patients seen by a hospital-based palliative care team.

BACKGROUND: Little is known about patient outcomes after discharge planning by inpatient palliative care teams. A major difficulty is that successful discharge planning often effectively limits or ends the team's relationship with the patient and family. The goal of this study was to gather a clearer picture of what happened to our palliative care consult patients after discharge. METHODS: This was a longitudinal survey of all patients seen over a one year period by the inpatient palliative care team at Oregon Health & Science University (OHSU). Data were recorded by team members at the time of consultation and supplemented by data from administrative databases and death certificates. RESULTS: The team provided consults to 292 unique patients: 60% were younger than age 65, 39% were female, and 16% were members of an ethnic or racial minority. Almost three quarters of patients carried a non-cancer diagnosis. Of the 292 patients, 37% died in hospital and 63% were discharged alive, either to home (54%), nursing facilities (20%), or inpatient hospice (26%). Of the 183 patients discharged alive, 38% died within 2 weeks, 32% died between 2 weeks and 6 months, 25% were alive at 6 months, and 4% were unknown. Of note, only 10% of patients seen by the consult service were readmitted to OSHU within 30 days, and only 5% of those discharged alive from OHSU ultimately died in an acute care hospital. DISCUSSION: We characterized patient outcomes following inpatient palliative care consultation: where patients are discharged, how long they live, and where they die. Two thirds of patients were able to be discharged, even when death occurred within two weeks. The low rates of readmission and death in an acute care hospital support that the decision to discharge the patients was reasonable and the discharge plan was adequate. Hospital based palliative care teams can play an important and unique role in discharge planning--allowing even patients very near death to leave the hospital if they wish.

Telemedicine for the medicare population: update.

CONTEXT: Telemedicine services are increasingly utilized by patients, clinicians, and institutions. Although private and Federal insurers are covering some telemedicine services, the rationale for these coverage decisions is not always evidence-based. OBJECTIVES: The goal of this report was to assess the peer-reviewed literature for telemedicine services that substitute for face-to-face medical diagnosis and treatment that may apply to the Medicare population. We focused on three distinct areas: store-and-forward, home-based, and office/hospital-based services. We also sought to identify what progress had been made in expanding the evidence base since the publication of our initial report in 2001 (AHRQ Publication No. 01-E012). DATA SOURCES: Ovid MEDLINE, reference lists of included studies, and non-indexed materials recommended by telemedicine experts. STUDY SELECTION: Included studies had to be relevant to at least one of the three study areas, address at least one key question, and contain reported results. We excluded articles that did not study the Medicare population (e.g., children and pregnant adults) or used a service that does not require face-to-face encounters (e.g., radiology or pathology diagnosis). DATA EXTRACTION: Our literature searches initially identified 4,083 citations. Using a dual-review process, 597 of these were judged to be potentially relevant to our study at the title/abstract level. Following a full-text review, 97 studies were identified that met our inclusion criteria and were subsequently included in the report's evidence tables. DATA SYNTHESIS: Store-and-forward services have been studied in many specialties, the most prominent being dermatology, wound care, and ophthalmology. The evidence for their efficacy is mixed, and in most areas, there are not corresponding studies on outcomes or improved access to care. Several limited studies showed the benefits of home-based telemedicine interventions in chronic diseases. These interventions appear to enhance communication with health care providers and provide closer monitoring of general health, but the studies of these techniques were conducted in settings that required additional resources and dedicated staff. Studies of office/hospital-based telemedicine suggest that telemedicine is most effective for verbal interactions, e.g., videoconferencing for diagnosis and treatment in specialties like neurology and psychiatry. CONCLUSIONS: There are still significant gaps in the evidence base between where telemedicine is used and where its use is supported by high-quality evidence. Further well-designed and targeted research that provides high-quality data will provide a strong contribution to understanding how best to deploy technological resources in health care.

The role of doctor's opinion in shared decision making: what does shared decision making really mean when considering invasive medical procedures?

OBJECTIVE: The goal of this study was to gain understanding about patients' perspectives on decision making in the context of invasive medical interventions and whether patients' decision-making preferences influenced the type of information they desired to be provided by physicians. DESIGN: Questionnaire study of consecutive patients in a university-based general medicine clinic. INTERVENTIONS: Patients were presented with a randomized list of three types of information that physicians could provide (risk, benefit and physician's opinion on whether they should undergo the procedure). Patients were asked whether they preferred patient-based, physician-based, or shared decision making and then were asked to select which one or combination of these three information types was most important to them in their own decision making. Patients were also asked to self-report on how many invasive procedures they had undergone in their own lives. PARTICIPANTS: A total of 202 consecutive patients (mean age = 65.1 years, SD = 12.3, range 28-88; mean education 13.3 years, SD 2.9, range 2-23). MAIN OUTCOME MEASURES: Patient reports. RESULTS: Of the 202 patients, two patients reported no decision-making preference. These two patients were excluded from the analysis. Of the 200 remaining patients, 62.5% (125/200) preferred shared, 22.5%(45/200) preferred physician-based, and 15.5% (31/200) preferred patient-based decision making. More than half of all subjects chose physician opinion as the most important type of information for decision making. Older patients (odds ratio 1.028; confidence interval 1.003-1.053) were more likely to have ranked the doctor's opinion as the most important in their decision making for invasive medical interventions. CONCLUSIONS: Although most patients want to share decision making with their physicians regarding invasive procedures, the majority of these patients report relying on the doctor's opinion on whether to undergo the procedure as the most important information in their own decision making.

Is constipation associated with decreased physical activity in normally active subjects?

BACKGROUND: The effectiveness of physical activity in the management of constipation remains controversial. We examined the associations among physical activity, constipation, and quality of life (QoL) in a population of employed adults to determine whether the risk of constipation is related to physical activity. METHODS: A total of 1,069 employees (age range 24-77) of the Veterans Affairs (VA) Black Hills Health Care System were mailed validated questionnaires (response rate 72%), inquiring about bowel habits, QoL (SF 36), and physical activity (modified Baecke questionnaire). Constipation was defined using the Rome I criteria. RESULTS: One hundred and forty (19.4%, 95% CI 16.2-22.4) employees reported constipation. The average total physical activity and all subscales of physical activity were not significantly different in subjects with and without constipation (all p > or = 0.2). Subjects with constipation had lower QoL scores than subjects without constipation, and physical activity was positively correlated with physical functioning and health perception. CONCLUSION: Physical activity appears to be unrelated to the risk of constipation in employed adults, but higher physical activity was associated with improved QoL. Recommendations to increase physical activity may not alter symptoms of constipation but may improve overall well-being.

Explaining low ratings of patient satisfaction among Asian-Americans.

Asian-Americans report lower levels of satisfaction with health care than other racial or ethnic groups. To determine potential reasons for this observation, we analyzed data from a national household telephone survey examining various aspects of health and health care across racial or ethnic groups. As in previous studies, Asians reported lower satisfaction with their physicians and health care services than other racial or ethnic groups, even after adjusting for demographic and health-related variables. However, Asians also reported lower satisfaction with their health insurance and with life in general and were less likely than non-Asians to have changed physicians due to dissatisfaction. We conclude that lower satisfaction ratings among Asians may be due to different response tendencies rather than to differences in quality of care. Further research is needed to explain this phenomenon and assess its potential impact on quality-of-care evaluations for clinicians and health care organizations providing care for large numbers of Asian-Americans.