scholarly journals Fundamental Outcome Measurement: Selecting Patient Reported Outcome Instruments and Interpreting the Data they Produce

2021 ◽  
Vol 12 (2) ◽  
pp. 17
Author(s):  
Stephen P McKenna ◽  
Alice Heaney ◽  
Paul C Langley
Keyword(s):  
Outcome Measurement ◽  
Therapy Response ◽  
Simulation Models ◽  
Patient Reported Outcome ◽  
Specific Patient ◽  
Patient Reported ◽  
The Many ◽  
Reported Data ◽  
The Impact ◽  
Pro Instruments

Over the past 40 years literally thousands of generic and disease specific patient reported outcome (PRO) instruments have been developed. While most were developed for a specific study and were never used again, there is still the question of how manufacturers and others should select a PRO instrument for a study. These studies may be clinical pivotal trials or observational tracking studies to support therapy response. Formulary committees also need to be able to interpret PRO data to make decisions about whether to accept claims for therapy response. It is possible to argue that the many different approaches to outcome measurement have resulted from the lack of agreed methodologies. However, a more likely explanation is that the authors have failed to apply the axioms of fundamental measurement when creating their measures. The result is a plethora of ordinal PRO instruments that inform little about the impact of interventions. Clinical trials rarely report PRO data. Where they do, analyses are generally restricted to (for example) changes in the experimental group’s scores. Comparisons between the treatment and placebo groups or between active groups are infrequently reported, most likely due to the failure of the instrument to show differences or changes in outcome. This is unfortunate as it means no assessment is made of the value that patients gain from the intervention. This commentary is intended to make researchers and formulary committees aware of the issues that need to be addressed when selecting PRO instruments for a study or evaluating publications and claims for therapy response. The latter is crucial as reported data influence the selection of medicines and healthcare products. In the latter case a particular concern is with PRO claims embedded in simulation models.

scholarly journals Understanding the patient experience in hepatocellular carcinoma: a qualitative patient interview study

2021 ◽  
Author(s):  
Nikunj Patel ◽  
Joshua Maher ◽  
Xandra Lie ◽  
Chad Gwaltney ◽  
Afsaneh Barzi ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Drug Development ◽  
Conceptual Model ◽  
Patient Experience ◽  
Outcome Measurement ◽  
Qualitative Interviews ◽  
Patient Reported Outcome ◽  
Weight Changes ◽  
Patient Reported ◽  
Pro Instruments

Abstract Purpose This study aimed to elucidate the patient experience of hepatocellular carcinoma (HCC) to guide patient-centered outcome measurement in drug development. Methods Patients with HCC participated in qualitative interviews to elicit disease-related signs/symptoms and impacts, using discussion guides developed from literature searches and discussions with oncologists. Interview participants rated the disturbance of their experiences (0–10 scale). A conceptual model was developed and mapped against patient-reported outcome (PRO) instruments identified from database reviews. Results Interviews were conducted with 25 individuals with HCC (68% were men; median age: 63 years; 12% Barcelona clinic liver cancer (BCLC) stage A; 32% stage B; and 56% stage C) in the USA. Fifty-one HCC-related concepts were identified from the interviews and were grouped into eight sign/symptom categories (eating behavior/weight changes; extremities [arms, legs]; fatigue and strength; gastrointestinal; pain; sensory; skin; other) and four impact categories (emotional; physical; cognitive function; other) for the conceptual model. The most prevalent and disturbing experiences across the disease stages were fatigue/lack of energy and emotional impacts such as frustration, fear, and depression. Abdominal pain and skin-related issues were particularly common and disturbing in individuals with HCC stage C. The EORTC QLQ-C30 and HCC18 were identified as commonly used PRO instruments in HCC studies and captured the relevant signs/symptoms associated with the patient experience. Conclusion Patients with HCC reported a range of signs/symptoms and impacts that negatively affect daily functioning and quality of life. Including PRO measures in HCC clinical trials can provide meaningful patient perspectives during drug development.

scholarly journals Development of a patient-reported outcome measure for neck pain in military aircrew: qualitative interviews to inform design and content

BMJ Open ◽  
2021 ◽  
Vol 11 (2) ◽  
pp. e039488
Author(s):  
Anna Dowling ◽  
Ellen Slungaard ◽  
Nicola R Heneghan
Keyword(s):  
Neck Pain ◽  
Outcome Measure ◽  
Physical Symptoms ◽  
Patient Reported Outcome ◽  
Direct Access ◽  
Specific Patient ◽  
Concept Elicitation ◽  
Patient Reported Outcome Measure ◽  
Patient Reported ◽  
The Impact

IntroductionThe prevalence of flight-related neck pain is 70% in UK fast jet pilots; much higher than the general population. The Aircrew Conditioning Programme and direct access physiotherapy exist to minimise the impact on military capability, but a population specific patient-reported outcome measure (PROM) is required to investigate the effectiveness of these. We aimed to explore the experiences of flight-related neck pain to inform the content validity and development of a population specific PROM.MethodsQualitative semistructured interviews combining phenomenological and grounded theory methods, reported using Consolidated criteria for Reporting Qualitative research guidelines. A purposive sample of 10 fast jet pilots with neck pain was recruited. Concept elicitation interviews were audio recorded, transcribed verbatim along with field notes. Data analysis involved subject and methodological expertise used a concept elicitation approach.ResultsParticipants included 10 male fast jet pilots, age 34.7 years. Identified themes included: (1) physical symptoms associated with flying activities; (2) occupational effects revealed modifications of flying, or ‘suboptimal’ performance owing to neck pain; (3) psychological effects revealed feelings or worry and (4) social and activity effects showed impact on out of work time.ConclusionPopulation-specific occupational, psychological and social factors should be considered alongside physical symptoms when managing neck pain in military aircrew. Findings support the development of a PROM specifically designed for military aircrew with neck pain.

scholarly journals A systematic review of patient-reported outcome measures in paediatric otolaryngology

2017 ◽  
Vol 132 (1) ◽  
pp. 2-7 ◽  
Author(s):  
J Powell ◽  
S Powell ◽  
A Robson
Keyword(s):  
Outcome Measures ◽  
English Language ◽  
Health Assessment ◽  
Paediatric Population ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Specific Patient ◽  
Patient Reported ◽  
The Impact

AbstractBackground:Recently, there has been increased emphasis on the development and application of patient-reported outcome measures. This drive to assess the impact of illness or interventions, from the patient's perspective, has resulted in a greater number of available questionnaires. The importance of selecting an appropriate patient-reported outcome measure is specifically emphasised in the paediatric population. The literature on patient-reported outcome measures used in paediatric otolaryngology was reviewed.Methods:A comprehensive literature search was conducted using the databases Medline, Embase, Cumulative Index to Nursing and Allied Health Literature, and PsycInfo, using the terms: ‘health assessment questionnaire’, ‘structured questionnaire’, ‘questionnaire’, ‘patient reported outcome measures’, ‘PROM’, ‘quality of life’ or ‘survey’, and ‘children’ or ‘otolaryngology’. The search was limited to English-language articles published between 1996 and 2016.Results:The search yielded 656 articles, of which 63 were considered relevant. This included general paediatric patient-reported outcome measures applied to otolaryngology, and paediatric otolaryngology disease-specific patient-reported outcome measures.Conclusion:A large collection of patient-reported outcome measures are described in the paediatric otolaryngology literature. Greater standardisation of the patient-reported outcome measures used in paediatric otolaryngology would assist in pooling of data and increase the validation of tools used.

scholarly journals Development of the Near Vision Presbyopia Task-based Questionnaire for use in evaluating the impact of presbyopia

2021 ◽  
Vol 5 (1) ◽  
Author(s):  
Elaheh Shirneshan ◽  
Cheryl D. Coon ◽  
Nathan Johnson ◽  
Jonathan Stokes ◽  
Ted Wells ◽  
...  
Keyword(s):  
Reading Ability ◽  
Alternative Assessment ◽  
Patient Reported Outcome ◽  
Good Test ◽  
Specific Patient ◽  
Near Vision ◽  
Regulatory Guidance ◽  
Patient Reported ◽  
The Impact ◽  
Domain Scores

Abstract Background Presbyopia is a progressive condition that reduces the eye’s ability to focus on near objects with increasing age. After a systematic literature review identified no existing presbyopia-specific patient-reported outcome (PRO) instruments meeting regulatory guidance, a new PRO instrument, the Near Vision Presbyopia Task-based Questionnaire (NVPTQ), was developed. Results To explore the patient experience with presbyopia, concept elicitation interviews were conducted with 20 presbyopic participants. The most frequently reported impacts were difficulty with reading menus/books/newspapers/magazines, reading on a cell phone/caller ID, and reading small print. Based on these results, a task-based PRO instrument (the NVPTQ) was developed instructing participants to complete four near-vision, paper-based reading tasks (book, newspaper, nutrition label, menu) under standardized settings, and subsequently assess their vision-related reading ability and associated satisfaction. The draft NVPTQ was cognitively debriefed with a sample of 20 presbyopes, which demonstrated that most participants interpreted the items as intended and endorsed the relevance of the concepts being assessed. After the qualitative research, the draft instrument was psychometrically tested using data from a Phase 2 study. Based on item-level analyses, all items in the NVPTQ demonstrated expected response option patterns and lacked substantial floor or ceiling effects. The reliability, validity, and responsiveness of the NVPTQ Performance and Satisfaction domain scores were assessed. All domains scores had large Cronbach’s coefficient α values and good test–retest statistics, indicating that the scores are internally consistent and produce stable values over time. The pattern of correlations with a concurrent measure of visual functioning (National Eye Institute Visual Function Questionnaire 25) demonstrated that the NVPTQ domain scores were related to an alternative assessment of near-vision activities. The NVPTQ domain scores were able to distinguish between groups that were known to differ on the clinical outcome of uncorrected near visual acuity, supporting the construct validity of these scores. The NVPTQ domain scores showed evidence of responsiveness to change by being able to distinguish between groups defined as improved and not improved based on patient-reported and clinical outcomes. Conclusions This research has resulted in a content-valid and psychometrically sound instrument designed to evaluate vision-related reading ability and satisfaction with vision-related reading ability. Trial registration: ClinicalTrials.gov NCT02780115. Registered 23 May 2016, https://www.clinicaltrials.gov/ct2/show/NCT02780115?term=NCT02780115&draw=2&rank=1.

scholarly journals Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

2022 ◽  
Vol 17 (1) ◽  
Author(s):  
Deborah Elstein ◽  
Nadia Belmatoug ◽  
Patrick Deegan ◽  
Özlem Göker-Alpan ◽  
Derralynn A. Hughes ◽  
...  
Keyword(s):  
Gaucher Disease ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Specific Patient ◽  
Patient Reported ◽  
Test Retest Reliability ◽  
Development And Validation ◽  
The Impact

Abstract Background Disease-specific patient-reported outcome measures (PROMs) are fundamental to understanding the impact on, and expectations of, patients with genetic disorders, and can facilitate constructive and educated conversations about treatments and outcomes. However, generic PROMs may fail to capture disease-specific concerns. Here we report the development and validation of a Gaucher disease (GD)-specific PROM for patients with type 1 Gaucher disease (GD1) a lysosomal storage disorder characterized by hepatosplenomegaly, thrombocytopenia, anemia, bruising, bone disease, and fatigue. Results and discussion The questionnaire was initially developed with input from 85 patients or parents of patients with GD1 or GD3 in Israel. Owing to few participating patients with GD3, content validity was assessed for patients with GD1 only. Content validity of the revised questionnaire was assessed in 33 patients in the US, France, and Israel according to US Food and Drug Administration standards, with input from a panel of six GD experts and one patient advocate representative. Concept elicitation interviews explored patient experience of symptoms and treatments, and a cognitive debriefing exercise explored patients’ understanding and relevance of instructions, items, response scales, and recall period. Two versions of the questionnaire were subsequently developed: a 24-item version for routine monitoring in clinical practice (rmGD1-PROM), and a 17-item version for use in clinical trials (ctGD1-PROM). Psychometric validation of the ctGD1-PROM was assessed in 46 adult patients with GD1 and re-administered two weeks later to examine test–retest reliability. Findings from the psychometric validation study revealed excellent internal consistency and strong evidence of convergent validity of the ctGD1-PROM based on correlations with the 36-item Short Form Health Survey. Most items were found to show moderate, good, or excellent test–retest reliability. Conclusions Development of the ctGD1-PROM represents an important step forward for researchers measuring the impact of GD and its respective treatment.

Patient-reported outcome measurements (PROMs) and provider assessment in mental health: a systematic review of the context of implementation

2019 ◽  
Author(s):  
David Roe ◽  
Yael Mazor ◽  
Marc Gelkopf
Keyword(s):  
Mental Health ◽  
Mental Health Services ◽  
Health Services ◽  
Outcome Measurement ◽  
Data Extraction ◽  
National Policy ◽  
Patient Reported Outcome ◽  
Self Report ◽  
Patient Reported ◽  
The Impact

Abstract Purpose To review and integrate the vast amount of literature yielded by recent growing interest in patient-reported outcome measurement and routine outcome measures (PROMs/ROMs), in order to suggest options and improvements for implementation. PROMs are the systematic assessment of service users’ health using standardized self-report measures. Specifically, for ROMs, it includes routine provider or caretaker assessment measures. Both are administered to ascertain routinely, the impact of treatment in mental health settings and to improve care. A review is needed because of the large differences in setting, conceptualization, practice and implementation. Here, we examine the different major projects worldwide. Data sources Psych INFO and PubMed including Medline, Biomed Central, EMBASE Psychiatry and Elsevier Science’s Direct. Study selection We conducted a systematized review of the literature published from 2000 to 2018 on the implementation and sustainability of PROMs and ROMs in mental health services for adults. Data extraction, synthesis and Results We described and characterized the programs in different countries worldwide. We identified 103 articles that met the inclusion criteria, representing over 80 PROMs/ROMs initiatives in 15 countries. National policy and structure of mental health services were found to be major factors in implementation. We discuss the great variability in PROMs/ROMs models in different countries, making suggestions for their streamlining and improvement. Conclusion We extracted valuable information on the different characteristics of the numerous PROMs/ROMs initiatives worldwide. However, in the absence of a strong nationwide policy effort and support, implementation seems scattered and irregular. Thus, development of the implementation of PROMs/ROMs is left to groups of enthusiastic clinicians and researchers, making sustainability problematic.

Affordance-Based Assessment Cuts Across The Dichotomy Of Subjective-Objective Outcome Measure At The Knee Joint

2019 ◽  
pp. 1-4
Author(s):  
Wangdo Kim ◽  
Keyword(s):  
Outcome Measurement ◽  
Patient Reported Outcome ◽  
Assessment Process ◽  
Active Sets ◽  
Patient Reported ◽  
Objective Outcomes ◽  
Objective Outcome ◽  
Subjective Outcomes ◽  
Pro Instruments ◽  
Inseparable Pair

There is increasing awareness of the need to include patient-reported outcome (PRO) instruments in evaluating the measurement of clinical outcomes, with an increasing focus placed on the patients’ perspective. Scientists have tried to link PROs with objective outcomes, providing unique information for managing patient care. Traditionally, objective and patient-reported outcomes (such as the Knee Injury and Osteoarthritis Outcome Score (KOOS)) are considered two distinct constructs, which cannot serve as a direct proxy for each other. Can Gibson´s affordance theory specify more about objective outcome measurements vs. subjective outcomes measurement and unify them? The present article develops a theoretical framework called entrainment of touch and posture that advocates the vis viva (living force) as the proper gauge for the dynamical action of a force, and that could explain “possibilities for action or affordance” during outcome measurement. We found that active touch and posture refer to what is ordinarily called touchingvariations in skin stimulation caused by surfaces are altered together by motor activity variations. This affordance of “walk-on-able” is worth noting because it is often neglected that locomotion and its surfaces form an inseparable pair. The assessment process can be viewed in terms of action possibilities provided by the active sets of organs residing that can obtain and utilize information about the tissue environments in which the grafts are to be located.

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