Τρίτη, 2 Ιουλίου 2019

The Patient - Patient-Centered Outcomes Research

Correction to: Important Treatment Outcomes for Patients with Psoriatic Arthritis: A Multisite Qualitative Study

The Open Access license, which previously read.



Towards Meaningful Engagement for the Patient Voice


10th Meeting "International Academy of Health Preference Research" Basel, Switzerland, 13–14 July, 2019


Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score)

Abstract

Background and Objective

For people with cystic fibrosis, validated patient-reported outcome measures for the assessment of the complex abdominal involvement are lacking. The objective of this study was to examine whether the CFAbd-Score, a novel questionnaire consisting of 28 items, meets the essential requirements (validity and reliability) for a patient-reported outcome measure according to US Food and Drug Administration recommendations.

Methods

Content validity was assessed by recording the frequencies and severity of symptoms that occurred during the prior 2 weeks in patients with cystic fibrosis (n = 116; aged ≥ 6 years). Comparing the CFAbd-Score results obtained from patients with cystic fibrosis and healthy controls (n = 88), we determined known-groups validity. To explore the structure of the patient-reported outcome measure, a factor analysis was conducted. Internal consistency of the five extracted score domains was assessed using Cronbach's alpha. For test–retest reliability, a subgroup of patients (n = 43) was reevaluated and intra-class correlation coefficients were determined.

Results

The CFAbd-Score differentiated patients with cystic fibrosis from healthy controls with a large effect size (17.3 ± 1.1 vs. 8.0 ± 0.7 points; p < 0.001; Cohen's d = 0.85). Items, domains, and scores reflected the relevance to patients with cystic fibrosis and allowed a differentiation between subgroups of patients with cystic fibrosis (e.g., patients with and without abdominal pain, pancreatic sufficiency, and age groups). High item-domain loadings as well as good to excellent internal consistency and reproducibility (Cronbach's α = 0.70–0.92; intra-class correlation coefficient = 0.932, 95% confidence interval 0.874–0.963) indicated construct validity and reliability.

Conclusions

The CFAbd-Score has successfully passed through key steps of the iterative process of patient-reported outcome measure development. Prospectively, the CFAbd-Score is proposed as a patient-centered instrument for monitoring abdominal symptoms and, most interestingly, for evaluating changes in symptoms with novel treatments such as cystic fibrosis transmembrane regulator modulators.

Trail Registration

ClinicalTrials.gov: NCT03052283.



Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review

Abstract

Background

Adult-onset myotonic dystrophy type 1 (DM1) is a chronic, multisystem disorder that leads to disability and premature death.

Objectives

The objective of our study was to conduct a systematic literature review of the health-related quality of life (HRQoL) of patients with DM1.

Methods

We searched Embase, Web of Science, and PubMed for English language full-text articles reporting results from studies of HRQoL in patients with adult-onset DM1 published between 1 January 2000 and 21 February 2018. We excluded reviews, editorials, and studies reporting results for a sample with fewer than five patients (to allow for meaningful inference).

Results

The search identified 266 unique publications. Of these, 231 were excluded following title and abstract screening and 16 after full-text review, leaving 19 articles for data synthesis. We found 15 articles measuring the HRQoL of patients with adult-onset DM1 using the 36-Item Short Form Health Survey (SF-36), six using the Individualized Neuromuscular Quality of Life Questionnaire (INQoL), and one using Cantril's Ladder. Available evidence shows that patient HRQoL is impaired in DM1, mainly due to compromised physical health, but also reveals that substantial heterogeneity exists in estimates across studies.

Conclusions

HRQoL in adult-onset DM1 has been extensively studied using the SF-36 and the INQoL, but current estimates are inconclusive, and little is known of the impact of the disease as measured using other instruments. Our data synthesis should help characterize the patient burden of DM1 and inform future studies of HRQoL in this indication.



Patient and Oncologist Preferences for the Treatment of Adults with Advanced Soft Tissue Sarcoma: A Discrete Choice Experiment

Abstract

Background

There has been no single standard-of-care treatment of patients with advanced/metastatic soft tissue sarcoma (STS). This study was designed to understand patient and oncologist preferences in the advanced/metastatic setting.

Methods

Adult patients diagnosed with STS and oncologists treating patients with STS completed discrete choice experiment surveys. Study participants chose between pairs of hypothetical treatment profiles for advanced STS characterized by varying levels of overall survival (14, 20, or 26 months), progression-free survival (3, 5, or 7 months), objective tumor response rate (12, 18, or 26%), risk of hospitalization due to side effects (12, 30, or 46%), and days/month to administer treatment (1, 2, or 4 days). A hierarchical Bayes model was used to estimate preferences and relative importance of attributes.

Results

Seventy-six patients (23.7% male, mean age 52.8 years) and 160 oncologists (73.8% male, mean 16.9 years in practice) completed the surveys. Among patients, overall survival had the highest relative importance (39.5%, standard deviation [SD] 18.2%), followed by response rate (21.2%, SD 10.4%), and hospitalization (19.8%, SD 12.5%). Among oncologists, overall survival had the highest relative importance (44.6%, SD 16.0%), followed by hospitalization (18.4%, SD 8.3%).

Conclusions

Both patients with STS and oncologists preferred a treatment that maximizes the life of patients while avoiding hospitalizations.



EQ-5D-5L is More Responsive than EQ-5D-3L to Treatment Benefit of Cataract Surgery

Abstract

Background

It is not clear whether 5-level EQ-5D (EQ-5D-5L) utilities based on recently developed value sets are more responsive than 3-level EQ-5D (EQ-5D-3L) utilities.

Objectives

The study aims were to compare (1) the responsiveness of EQ-5D-5L and EQ-5D-3L utilities and (2) the responsiveness of these utilities with the Short Form–6 Dimension (SF-6D) and Health Utilities Index Mark 3 (HUI3) utilities to the treatment benefit of cataract surgery.

Methods

A total of 148 patients were interviewed before and after their cataract surgery using EQ-5D-3L, EQ-5D-5L, SF-6D, and HUI3. Responsiveness was assessed for all measures using the mean change (post-treatment—pre-treatment), standardized effect size (SES), standardized response mean (SRM), and F-statistic.

Results

Using the Singapore value sets, mean change for EQ-5D-3L and EQ-5D-5L utilities was 0.016 and 0.028, SES was 0.097 and 0.199; SRM was 0.091 and 0.196; and F-statistic was 1.2 and 5.7, respectively. Similar trends were observed using the UK/England EQ-5D value sets, although the magnitude was slightly smaller. The mean change, SES, SRM and F-statistics for SF-6D (UK value set) were 0.020, 0.234, 0.249, and 9.2, respectively. The values of mean change, SES, SRM and F-statistics for HUI3 (Canada value set) were 0.080, 0.472, 0.474, and 33.3, respectively.

Conclusions

The EQ-5D-5L utilities tend to be more responsive than the EQ-5D-3L utilities to treatment benefits of cataract surgery. The HUI3 utilities are more responsive than both the EQ-5D-5L and SF-6D, and SF-6D utilities may be slightly more responsive than the EQ-5D-5L for assessing patients undergoing cataract surgery.



Measuring the Burden of Schizophrenia Using Clinician and Patient-Reported Measures: An Exploratory Analysis of Construct Validity

Abstract

Background

Various self-reported or clinician-reported (as a proxy) measures exist to quantify the burden of schizophrenia on patients. Evidence of the psychometric relationship between these measures to inform their practical use is limited.

Objectives

Our objective was to conduct an exploratory analysis of the construct validity of patient-reported (EQ-5D, SF-6D, WEMWBS, SQLS subscales of Psychosocial, Motivation, Symptoms) versus clinician-reported measures (PANSS, CGI-SCH, NSA-4, HoNOS-PbR) to inform future use of patient-reported measures for burden-of-illness assessment and/or economic evaluation.

Methods

In an adult patient population with schizophrenia, construct validity was assessed in relation to convergent and known-group validity. Convergent validity was assessed using Spearman's rank absolute correlation strength (ACS: weak ≤ 0.3, moderate = 0.3 < 0.5, strong ≥ 0.5) and graphically using locally weighted scatterplot smoothing (LOWESS) techniques. Known-group validity was assessed using Cohen's d absolute effect size (AES: small ≤ 0.5, moderate = 0.5 < 0.8, large ≥ 0.8). Floor and ceiling effects were assessed as a proxy of sensitivity in this cross-sectional study. Statistical significance was assessed at the 5% threshold level (p < 0.05). Across head-to-head assessments, the frequency of producing the strongest ACS, largest AES, and statistically significant results determined the best overall construct validity.

Results

Overall, 304 patients consented to the study. In relation to statistically significant results, the SF-6D most frequently exhibited the strongest ACS and largest AES against the clinician-reported measure scores (ACS range 0.084–0.436; AES range 0.043–0.746), and the SQLS Motivation subscale most frequently exhibited the weakest/smallest values (ACS range 0.009–0.157; AES range 0.002–0.397), although these results were mixed according to the clinician-reported measure used for comparative analysis (ACS range 0.009–0.529; AES range 0.002–0.934).

Conclusion

The SF-6D indicated the best (mostly moderate) construct validity but still missed the negative symptoms of the condition. Although further evidence is required to confirm or refute these exploratory results, compared with the EQ-5D, the SF-6D can be self-reported to better capture generic health-related quality-of-life aspects of schizophrenia for the purpose of economic evaluation. The lack of construct validity for SQLS Motivation and Symptoms subscales were hypothesized post-hoc to be representative of the complementary information elicited by the subscales not captured by the clinician-reported measures. Therefore, the SQLS can be self-reported to capture complementary (i.e., additional) information relative to clinician-reported measures.



Patient-Community Perspectives on Real-World Evidence: Enhancing Engagement, Understanding, and Trust

Abstract

Background

Healthcare stakeholders have pronounced both enthusiasm and apprehension over the expanding use of real-world evidence (RWE). The patient community—those who benefit from new treatments but are vulnerable to potential safety risks and whose routine medical encounters are used to generate RWE—has been less vocal. Understanding patient perspectives on the use of RWE to guide clinical decision making and inform regulatory decisions and value assessments is imperative.

Methods

We convened a day-long, multi-stakeholder roundtable in Washington D.C., USA, on 31 July 2017 to gather patient-community views on RWE and related concerns and the communications, information and tools needed by patients to understand, trust, and use RWE. Participants included a convenience sample of National Health Council (NHC) members primarily representing patient groups as well as non-patient members with an interest in RWE. Participants were organized into small, pre-assigned groups, ensuring representativeness across stakeholders and patient leadership. Discussions, including storyboards, notes, and illustrative examples were captured and later analyzed thematically by NHC staff.

Results

Ten RWE themes emerged: (1) most patients were unaware of RWE and its actual or potential uses, (2) common definitions for real-world data and RWE are needed, (3) patient organizations need RWE skills and tools, (4) patient–scientist partnerships can help differentiate high-quality RWE, (5) RWE should inform decision making, (6) clinician support is needed for RWE uptake in patient decision making, (7) communications to patients should be balanced and empowering, (8) context of use impacts RWE acceptability/trust, (9) privacy/data ownership require clarity, and (10) patient-generated data are also real-world data (RWD).

Conclusion

Patients see great possibility in using RWE to understand how a treatment works—to find someone that "looks like me" as assurance of how a treatment might benefit them personally. Patient groups will play a critical role in helping to educate constituents on understanding, contributing to, and using RWE. To maximize patient uptake and the co-development and application of RWE, patient groups require education and tools.



Support Tools for Preference-Sensitive Decisions in Healthcare: Where Are We? Where Do We Go? How Do We Get There?


Alexandros Sfakianakis
Anapafseos 5 . Agios Nikolaos
Crete.Greece.72100
2841026182
6948891480

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