Patient-Reported Outcome Assessments as Endpoints in Studies in Infectious Diseases.
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ABSTRACT:
SUBMITTER: Powers JH
PROVIDER: S-EPMC5006216 | biostudies-other | 2016 Aug
REPOSITORIES: biostudies-other
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Project description:BackgroundWhen assessing the efficacy of a treatment in any clinical trial, it is recommended by the International Conference on Harmonisation to select a single meaningful endpoint. However, a single endpoint is often not sufficient to reflect the full clinical benefit of a treatment in multifaceted diseases, which is often the case in rare diseases. Therefore, the use of a combination of several clinically meaningful outcomes is preferred. Many methodologies that allow for combining outcomes in a so-called composite endpoint are however limited in a number of ways, not in the least in the number and type of outcomes that can be combined and in the poor small-sample properties. Moreover, patient reported outcomes, such as quality of life, often cannot be integrated in a composite analysis, in spite of their intrinsic value.ResultsRecently, a class of non-parametric generalized pairwise comparisons tests have been proposed, which members do allow for any number and type of outcomes, including patient reported outcomes. The class enjoys good small-sample properties. Moreover, this very flexible class of methods allows for prioritizing the outcomes by clinical severity, allows for matched designs and for adding a threshold of clinical relevance. Our aim is to introduce the generalized pairwise comparison ideas and concepts for rare disease clinical trial analysis, and demonstrate their benefit in a post-hoc analysis of a small-sample trial in epidermolysis bullosa. More precisely, we will include a patient relevant outcome (Quality of life), in a composite endpoint. This publication is part of the European Joint Programme on Rare Diseases (EJP RD) series on innovative methodologies for rare diseases clinical trials, which is based on the webinars presented within the educational activity of EJP RD. This publication covers the webinar topic on composite endpoints in rare diseases and includes participants' response to a questionnaire on this topic.ConclusionsGeneralized pairwise comparisons is a promising statistical methodology for evaluating any type of composite endpoints in rare disease trials and may allow a better evaluation of therapy efficacy including patients reported outcomes in addition to outcomes related to the diseases signs and symptoms.
| S-EPMC10474650 | biostudies-literature
Project description:BACKGROUND:Patient response burden is often raised as a human subject concern in consideration of the length or complexity of patient-reported outcome (PRO) instruments used in oncology. OBJECTIVES:To quantify patient response burden and identify its predictive factors. METHODS:Data were collected presurgically during a prospective trial that used a comprehensive symptom and health-related quality-of-life (HRQOL) PRO assessment. A subset of patients also completed HRQOL interviews. Response burden was captured using an internally developed six-item instrument. Demographic and clinical characteristics as well as HRQOL scores were examined as potential predictors using hierarchical regression. Response burden was used to predict participant dropout at the first follow-up interval. RESULTS:A total of 275 patients (mean age 67.5 years; 23.6% female) completed surveys (n = 126) or surveys in addition to interviews (n = 149). Patients experienced low response burden (mean 12.19 ± 11.65). Repetitive questions were identified by 60 patients (21.8%), whereas 31.6% indicated that additional information should be gathered; 35 patients (12.7%) identified repetitive questions and expressed a desire for additional items. Low self-reported cognitive function was a significant predictor of higher response burden (? = -0.20; t(270) = -3.38; P = 0.01; model-adjusted R2 = 0.04). Response burden was not a significant predictor of study dropout. CONCLUSIONS:Despite completing a large battery of PRO measures and interviews, patients reported minimal response burden, with nearly one-third expressing that more questions should have been asked. Patients with lower cognitive function are more likely to report higher response burden when completing PRO measures. Further examination of patient characteristics related to response burden may reveal useful pathways for tailoring patient-centered interventions.
| S-EPMC6362460 | biostudies-literature
Project description:BackgroundWith the rise of digital health technologies and telemedicine, the need for evidence-based evaluation is growing. Patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) are recommended as an essential part of the evaluation of telemedicine. For the first time, a systematic review has been conducted to investigate the use of PROMs and PREMs in the evaluation studies of telemedicine covering all application types and medical purposes.ObjectiveThis study investigates the following research questions: in which scenarios are PROMs and PREMs collected for evaluation purposes, which PROM and PREM outcome domains have been covered and how often, which outcome measurement instruments have been used and how often, does the selection and quantity of PROMs and PREMs differ between study types and application types, and has the use of PROMs and PREMs changed over time.MethodsWe conducted a systematic literature search of the MEDLINE and Embase databases and included studies published from inception until April 2, 2020. We included studies evaluating telemedicine with patients as the main users; these studies reported PROMs and PREMs within randomized controlled trials, controlled trials, noncontrolled trials, and feasibility trials in English and German.ResultsOf the identified 2671 studies, 303 (11.34%) were included; of the 303 studies, 67 (22.1%) were feasibility studies, 70 (23.1%) were noncontrolled trials, 20 (6.6%) were controlled trials, and 146 (48.2%) were randomized controlled trials. Health-related quality of life (n=310; mean 1.02, SD 1.05), emotional function (n=244; mean 0.81, SD 1.18), and adherence (n=103; mean 0.34, SD 0.53) were the most frequently assessed outcome domains. Self-developed PROMs were used in 21.4% (65/303) of the studies, and self-developed PREMs were used in 22.3% (68/303). PROMs (n=884) were assessed more frequently than PREMs (n=234). As the evidence level of the studies increased, the number of PROMs also increased (τ=-0.45), and the number of PREMs decreased (τ=0.35). Since 2000, not only has the number of studies using PROMs and PREMs increased, but the level of evidence and the number of outcome measurement instruments used have also increased, with the number of PREMs permanently remaining at a lower level.ConclusionsThere have been increasingly more studies, particularly high-evidence studies, which use PROMs and PREMs to evaluate telemedicine. PROMs have been used more frequently than PREMs. With the increasing maturity stage of telemedicine applications and higher evidence level, the use of PROMs increased in line with the recommendations of evaluation guidelines. Health-related quality of life and emotional function were measured in almost all the studies. Simultaneously, health literacy as a precondition for using the application adequately, alongside proper training and guidance, has rarely been reported. Further efforts should be pursued to standardize PROM and PREM collection in evaluation studies of telemedicine.
| S-EPMC8663685 | biostudies-literature
Project description:Background: Patient-reported outcomes (PROs) can promote person-centered biopsychosocial health care by measuring outcomes that matter to patients, including functioning and well-being. Data support feasibility and acceptability of PRO administration as part of routine clinical care, but less is known about its effects on population health, including detection of unmet healthcare needs. Our objectives were to examine differences in rates of clinically significant depression across sociodemographic groups and clinical settings from universal depression screens in a large health system, estimate the number of patients with untreated depression detected by screenings, and examine associations between biopsychosocial PROs-physical, psychological, and social health. Methods: We analyzed data from over 200,000 adult patients who completed depression screens-either PROMIS (Patient Reported Outcomes Measurement Information System) or PHQ-2/9-as part of routine outpatient care. Results: Depression screens were positive in 14.2% of the sample, with more positive screens among younger vs. older adults, women vs. men, non-White vs. White, and Hispanics vs. non-Hispanics. These same sociodemographic indicators, as well as completing screening in primary care (vs. specialty care) were also associated with greater likelihood of detected depression in the medical record. Discussion: Universal screening for depression symptoms throughout a large health system appears acceptable and has the potential to detect depression in diverse patient populations outside of behavioral health. Expanded delivery of PROs to include physical and social health as well as depression should be explored to develop a clinically-relevant model for addressing patients' biopsychosocial needs in an integrated fashion across the health system.
| S-EPMC8787088 | biostudies-literature
Project description:ObjectivesRecent focus on patient-reported outcomes (PROs) has created a new challenge as we learn how to integrate these outcomes into practice along with other quality metrics. We investigated the relationship between PROs and satisfaction among spine surgery patients. We hypothesized that there would be significant disparities between patient satisfaction and PROs at the 1-year postoperative time point.Study designRetrospective cohort study of adults undergoing elective lumbar spine surgery at 12 hospitals participating in the Spine Surgical Care and Outcomes Assessment Program.MethodsSatisfaction, pain, and function scores were collected at 1 year post operation, along with clinical information, to determine the relationship between PROs and satisfaction at the patient level.ResultsAmong 520 patients (mean age = 63 ± 13 years; 47% male), the majority of patients (82%) reported being satisfied with surgery. Satisfaction was associated with both improvement in pain (odds ratio [OR], 1.33; 95% CI, 1.17-1.51) and function (OR, 1.06; 95% CI, 1.04-1.08). However, even among patients who did not improve in pain or function, more than half (59%) reported being satisfied.ConclusionsOverall, patients undergoing elective lumbar spine surgery reported being satisfied with outcomes, but the reported responses in PROs were much more variable. As the expectations increase to include PRO measures as valid quality indicators, it is necessary to dedicate time and consideration to understanding the relationships among these measures to support meaningful translations into healthcare policy.
| S-EPMC5846490 | biostudies-literature
Project description:Patient Reported Outcome Measures (PROM) evoke measurements that allow capturing patients' perspectives on their condition. In endometriosis care, physicians' understanding of the effect of the disease and the treatment on patients is often poor. The use of PROMs in endometriosis clinical practice can facilitate patient-provider communication and the implementation of patient-centered care, improve patients' quality of life, as well as provide a tool for patients' self-management of the disease. Today, PROMs are extensively used in research and clinical trials, however they are barely used in clinical practice. The development of digital tools facilitating capturing PROMs can contribute to their use by physicians in routine endometriosis care. However, all PROMs are not adapted to be used in routine care in the context of endometriosis. The objective of this study was to present a catalogue of available PROMs for routine endometriosis care and evaluate them according to selected criteria. To do so, we explored the different PROMs currently in the literature. Consequently, 48 PROM were identified as tools used to evaluate various dimensions of the impact of endometriosis on patients. The selected PROMs were evaluated for their potential to be used as a standard in clinical practice in endometriosis. The selected catalogue of PROMs is the starting point for the integration of digital tools to capture PROMs and the development of patient-centered dashboards to be used by patients and clinicians in endometriosis care and self-management to improve care processes, patient satisfaction, quality of life, and outcomes.
| S-EPMC8585017 | biostudies-literature
Project description:Surgical procedures incorporating a cosmetic element such as septorhinoplasty and otoplasty are currently under threat in the National Health Service (NHS) as they are deemed to be procedures of 'limited clinical benefit' by many primary care providers. Patient reported outcome measures (PROMs), which assess the quality of care delivered from the patients' perspective, are becoming increasingly important in documenting the effectiveness of such procedures.The Rhinoplasty Outcomes Evaluation (ROE) questionnaire, a validated PROM tool, was used to assess patient satisfaction in 141 patients undergoing septorhinoplasty surgery over a 90-month period at the University Hospital Southampton NHS Foundation Trust.Overall, 100 patients with a mean follow-up period of 36 months completed the study. The mean ROE score was 73.3%. In addition, 75% of patients questioned were happy with the final result of their operation and 83% would undergo the procedure again if required. These benefits occurred irrespective of age, sex and primary versus revision surgery, and were maintained for up to 71 months following surgery.This study has shown that patients are generally satisfied with their functional and cosmetic outcomes following septorhinoplasty surgery. These results help support the case for septorhinoplasty surgery to continue being funded as an NHS procedure.
| S-EPMC4473903 | biostudies-other
Project description:BackgroundPatient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing and administering PROMs for rare diseases poses unique challenges because of small patient populations, disease heterogeneity, lack of natural history knowledge, and short-term studies.ObjectiveThis research aims to identify key factors to consider when using different types of PROMs in HTA for rare disease treatments (RDTs).MethodsA scoping review of scientific and grey literature was conducted, with no date or publication type restrictions. Information on the advantages of and the challenges and potential solutions when using different types of PROMs for RDTs, including psychometric properties, was extracted and synthesized.ResultsOf 79 records from PubMed, 32 were included, plus 12 records from the grey literature. PROMs for rare diseases face potential data collection and psychometric challenges resulting from small patient populations and disease heterogeneity. Generic PROMs are comparable across diseases but not sensitive to disease specificities. Disease-specific instruments are sensitive but do not exist for many rare diseases and rarely provide the utility values required by some HTA bodies. Creating new PROMs is time and resource intensive. Potential solutions include pooling data (multi-site/international data collection), using computer-assisted technology, or using generic and disease-specific PROMs in a complementary way.ConclusionsPROMs are relevant in HTA for RDTs but pose a number of difficulties. A deeper understanding of the potential advantages of and the challenges and potential solutions for each can help manage these difficulties.
| S-EPMC8357707 | biostudies-literature
Project description:Rare diseases are often not fully understood and efforts put in investigating it from patient perspective are usually met with challenges. We performed a systematic literature review (SLR) for the last 20 years in Cushing's Syndrome (CS) to illustrate Patient-Reported Outcome (PRO) challenges, and show what solutions were found.PROs and other Clinical Outcome Assessment (COA) used with CS patients were reviewed in 36 studies. Two CS-specific Health Related Quality of Life (HRQL) measures were identified (i.e., CushingQoL, Tuebingen CD-25), as well as depression and neurocognitive measures. For CS-specific HRQL measures, the CushingQoL was the most widely used measure due in part to being the first CS-specific HRQL measure developed. With algorithms mapping the CushingQoL to both the SF-6D and EQ-5D, the CushingQoL could be used to facilitate economic modelling studies in the absence of a generic HRQL measure. While the CushingQoL offers only the global scale and two subscales compared to the six subscales of the Tuebingen CD-25, there is not yet adequate statistical validation data available for the Tuebingen CD-25 to suggest it can withstand the scrutiny of review by multiple stakeholders. Results of this review indicate that the inclusion of a measure of depressive symptoms, such as the BDI-II or similar measure, would be reasonable to include given the high level of comorbidity of depression among CS patients. A brief neurocognitive performance outcome, such as Trail Making tasks A and D or Digit Symbol, could help inform the interpretation of HRQL results. Neurocognitive differences may be an unassessed mediator of HRQL outcomes, partly accounting for the persistence of depressive symptoms and HRQL deficits despite treatment. Results suggest that HRQL improvements are possible within this population. These results are limited by small sample sizes and pre/post study design.CS showcases the difficulties encountered in measuring PROs in rare diseases. A solution for this specific case was developed in the form of dedicated PRO instruments, the CushingQOL and the Tuebingen-25. However, some aspects of CS may not be fully answered or not yet validated (e.g., depressive and cognitive symptoms). Further research needs to be done to address them.
| S-EPMC6299940 | biostudies-literature
Project description:AimTo identify and appraise the quality of studies that primarily assessed the measurement properties of English language versions of multidimensional patient-reported outcome measures (PROMs) when evaluated with children with neurodisability, and to summarize this evidence.MethodMEDLINE, Embase, PsycINFO, CINAHL, AMED, and the National Health Service Economic Evaluation Database were searched. The methodological quality of the papers was assessed using the COnsensus-based Standards for selection of health Measurement INstruments checklist. Evidence of content validity, construct validity, internal consistency, test-retest reliability, proxy reliability, responsiveness, and precision was extracted and judged against standardized reference criteria.ResultsWe identified 48 studies of mostly fair to good methodological quality: 37 papers for seven generic PROMs (CHIP, CHQ, CQoL, KIDSCREEN, PedsQL, SLSS, and YQOL), seven papers for two chronic-generic PROMs (DISABKIDS and Neuro-QOL), and four papers for three preference-based measures (HUI, EQ-5D-Y, and CHSCS-PS).InterpretationOn the basis of this appraisal, the DISABKIDS appears to have more supportive evidence in samples of children with neurodisability. The overall lack of evidence for responsiveness and measurement error is a concern when using these instruments to measure change, or to interpret the findings of studies in which these PROMs have been used to assess change.
| S-EPMC5031226 | biostudies-literature