scholarly journals Role of Patient-Reported Outcome Evaluation in the Approval of Orphan Drugs: A Review of 15 Year Approvals by the FDA and the EMA

2018 ◽  
Vol 21 ◽  
pp. S257
Author(s):  
B Arnould ◽  
C Acquadro ◽  
S Lanar ◽  
I Savre
Keyword(s):  
Orphan Drugs ◽  
Patient Reported Outcome ◽  
Outcome Evaluation ◽  
Patient Reported

scholarly journals The role of response domain and scale label in the quantitative interpretation of patient-reported outcome measure response options

2021 ◽  
Author(s):  
Tessa Peasgood ◽  
Jen-Yu Chang ◽  
Robina Mir ◽  
Clara Mukuria ◽  
Philip A. Powell
Keyword(s):  
First Language ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Response Options ◽  
The Public ◽  
Online Panel ◽  
Patient Reported ◽  
Common Response ◽  
Chi Squared

Abstract Purpose Uncertainties exist in how respondents interpret response options in patient-reported outcome measures (PROMs), particularly across different domains and for different scale labels. The current study assessed how respondents quantitatively interpret common response options. Methods Members of the general public were recruited to this study via an online panel, stratified by age, gender, and having English as a first language. Participants completed background questions and were randomised to answer questions on one of three domains (i.e. loneliness (negatively phrased), happiness or activities (positively phrased)). Participants were asked to provide quantitative interpretations of response options (e.g. how many times per week is equal to “often”) and to order several common response options (e.g. occasionally, sometimes) on a 0–100 slider scale. Chi-squared tests and regression analyses were used to assess whether response options were interpreted consistently across domains and respondent characteristics. Results Data from 1377 participants were analysed. There was general consistency in quantifying the number of times over the last 7 days to which each response option referred. Response options were consistently assigned a lower value in the loneliness than happiness and activities domains. Individual differences, such as age and English as a second language, explained some significant variation in responses, but less than domain. Conclusion Members of the public quantify common response options in a similar way, but their quantification is not equivalent across domains or every type of respondent. Recommendations for the use of certain scale labels over others in PROM development are provided.

scholarly journals Autonomic conditioning therapy reduces fatigue and improves global impression of change in individuals with post-acute COVID-19 syndrome

2021 ◽  
Author(s):  
David Putrino ◽  
Laura Tabacof ◽  
Jenna Tosto-Mancuso ◽  
Jamie Wood ◽  
Mar Cortes ◽  
...  
Keyword(s):  
Physical Therapy ◽  
Patient Reported Outcome ◽  
Therapy Program ◽  
Patient Global Impression ◽  
Autonomic Conditioning ◽  
Patient Reported ◽  
Conditioning Therapy ◽  
Physical Therapy Program ◽  
Global Impression Of Change

Abstract Post-acute COVID-19 syndrome (PACS) is a collection of persistent and debilitating symptoms lasting weeks to months after acute COVID-19 infection, with fatigue most commonly reported. There is controversy surrounding the role of exercise programs for this condition, due to concerns over the potential to worsen fatigue. We developed a novel physical therapy program known as Autonomic Conditioning Therapy (ACT) for PACS, and report on the preliminary patient-reported outcome (PRO) data from individuals who completed ACT for PACS, compared with those who did not. Seventy-eight (55 [71%] female, median [range] age 43 [12 to 78]) met the inclusion criteria and consented to have their data included in the analyses. A total of 31 (40%) individuals completed ACT for PACS. There was within-group improvement in fatigue in individuals who completed ACT for PACS (mean difference [95% CI] -14 [-27 to -1], p = 0.03), as well as greater between-group impression of change measured on the Patient Global Impression of Change scale (ACT for PACS median [range] 5 [1 to 7], no ACT for PACS 4 [1 to 7], p < 0.01). ACT for PACS is a novel physical therapy program that can reduce fatigue in individuals with PACS.

scholarly journals Outcome evaluation by patient reported outcome measures in stroke clinical practice (EPOS) protocol for a prospective observation and implementation study

2019 ◽  
Vol 1 (1) ◽  
Author(s):  
D. Leander Rimmele ◽  
Lisa Lebherz ◽  
Marc Frese ◽  
Hannes Appelbohm ◽  
Hans-Jürgen Bartz ◽  
...  
Keyword(s):  
Everyday Life ◽  
Outcome Measures ◽  
Predictive Factors ◽  
Patient Reported Outcomes ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Outcome Evaluation ◽  
Link Type ◽  
Patient Reported ◽  
Stroke Center

Abstract Introduction The impact of stroke-related impairment on activities of daily living may vary between patients, and can only be estimated by applying patient-reported outcome measures. The International Consortium for Health Outcome Measurement has developed a standard set of instruments that combine clinical and longitudinal patient-reported outcome measures for stroke. The present study was designed (1) to implement and evaluate the feasibility of the use of it as a consistent outcome measure in clinical routine at the stroke center of a German university hospital, (2) to characterize impairment in everyday life caused by stroke, and (3) to identify predictive factors associated with patient-relevant outcomes. Methods We plan to enroll 1040 consecutive patients with the diagnosis of acute ischemic stroke, transient ischemic attack, or intracerebral hemorrhage in a prospective observational study. Demographics, cardiovascular risk factors, and living situation are assessed at inpatient surveillance. At 90 days and 12 months after inclusion, follow-up assessments take place including the Patient-reported Outcomes Measurement Information System 10-Question Short Form (PROMIS-10), the Patient- Health Questionnaire-4, and the simplified modified Ranking Scale questionnaire. The acceptance and feasibility (1) will be assessed by a process evaluation through qualitative semi-structured interviews with clinical staff and patients and quantitative analyses of the data quality evaluating practicability, acceptance, adoption, and fidelity to protocol. The primary outcome of objective 2 and 3 is health-related quality of life measured with the PROMIS-10. Additional outcomes are depressive and anxiety symptoms and patient participation in their social roles. Patient-reported outcomes will be assessed in their longitudinal course using (generalized) mixed regressions. Exploratory descriptive and inference statistical analyses will be used to find patterns of patient characteristics and predictive factors of the outcome domains. Perspective The results will describe and further establish the evaluation of stroke patients of a stroke center by standardized PROMs in everyday life. Trial registration The trial is registered at ClinicalTrials.gov (NCT03795948). Approval of the local ethics committee (Ethik-Kommission der Ärztekammer Hamburg) has been obtained.

scholarly journals Short form development for oral health patient-reported outcome evaluation in children and adolescents

2018 ◽  
Vol 27 (6) ◽  
pp. 1599-1611 ◽  
Author(s):  
Honghu Liu ◽  
Ron Hays ◽  
Yan Wang ◽  
Marvin Marcus ◽  
Carl Maida ◽  
...  
Keyword(s):  
Oral Health ◽  
Children And Adolescents ◽  
Short Form ◽  
Patient Reported Outcome ◽  
Outcome Evaluation ◽  
Patient Reported ◽  
Form Development

scholarly journals Barriers to early mobilisation within Musculoskeletal and Colorectal Enhanced Recovery

2020 ◽  
Author(s):  
Emma Jones ◽  
Thomas Wainwright ◽  
Sarah Davies ◽  
Paul Porter ◽  
Nader Francis
Keyword(s):  
Enhanced Recovery ◽  
Knowledge Exchange ◽  
Implementation Process ◽  
Patient Reported Outcome ◽  
Data Sets ◽  
Early Mobilisation ◽  
Patient Reported ◽  
Staffing Patterns ◽  
Multiple Challenges

Introduction Enhanced Recovery After Surgery (ERAS) protocols emphasise the importance of early mobilisation after surgery. When applied in combination with pre operative and intra-operative ERAS elements early mobilisation is known to improve clinical and patient reported outcome. However, although early mobilisation is a well established element of ERAS its implementation can be hampered by clinical, organisational and emotive factors. The aim of this research is to survey staff opinion on the implementation process and challenges to early mobilisation across Musculoskeletal (MSK) and Colorectal (CR) wards at Yeovil Hospital. Methods MSK and CR staff (nursing, health care assistant and therapy) were issued with paper surveys addressing: i) timing of mobilisation, ii) competency to mobilise day one post operative patients and iii) barriers in July 2013. Qualitative data was subjected top thematic analysis and shared and disparate views were considered across the two data sets. Themes were cross checked by SD. Results 32 staff completed the survey; 17 MSK and 15 CR of a cohort of 40. CR staff reported unanimously that mobilising patients before breakfast could impact on length of stay while 53% of MSK staff believed that the timing of first mobilisation does not alter it, with 35% stipulating this should occur after breakfast. All staff felt competent and identified no training issues. The most common barriers identified were; staff availability (88% of MSK) and low blood pressure (60% of CR and MSK). Other factors included nausea, pain and anaesthetic/analgesia type especially after knee surgery (figure 1). MSK staff reported that organisational multi-disciplinary changes should be implemented to overcome barriers (fig 2). MSK staff emphasise the role of the therapists to drive mobilisation while CR staff emphasised the role of nurses. Discussion Differences between MSK and CR staff opinion on the timing of mobilisation and barriers to mobilisation could be underpinned by: staffing patterns, beliefs, ERAS tradition, clinical reasoning and interdisciplinary working. This survey has demonstrated that greater knowledge exchange between specialities could be important. Further analysis is required to evaluate whether the timing of first mobilisation improves outcomes. Conclusion There is a difference in culture between Musculoskeletal and Colorectal staff in the timing of early mobilisation, and a divided opinion on whether this impacts LOS. Multiple challenges to early mobilisation can be overcome by a multi-disciplinary approach across specialities.

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