Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report

Benjamin, Katy; Vernon, Margaret; Patrick, Donald L.; Perfetto, Elèanor M.; Nestler-Parr, Sandra; Burke, Laurie B.

doi:10.1016/j.jval.2017.05.015

Cited by 122 publications

(171 citation statements)

References 30 publications

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“…In rare conditions like EA, known challenges in patientreported outcome measurements include the development or availability of suitable instruments, recruitment difficulties, heterogeneity of outcomes, and interpretation of the findings. [50][51][52] Despite this, previous years' research involving EA patients demonstrates an increased focus on HRQoL. Furthermore, and as recommended in the literature review from 2015, 23 several advances have taken place.…”

Section: Strengths and Limitations In Hrqol Assessment Of Patients Afmentioning

confidence: 99%

Health-Related Quality of Life in Patients after Repair of Esophageal Atresia: A Review of Current Literature

2020

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Patients born with esophageal atresia (EA) require advanced reconstructive surgery and risk long-term digestive and respiratory morbidity. We describe the state of current literature on these patients' health-related quality of life (HRQoL) and give recommendations for future research. A literature search was conducted in PubMed, Cumulative Index to Nursing and Allied Health Literature (CINAHL), and PsychINFO and included articles from 2015 to December 28, 2019. Criteria for selecting articles were predefined. Seventeen articles describing HRQoL among children, adolescents, and adults were found, two of which focused on adults only. Six studies included international samples and among 15 quantitative studies sample sizes varied from 17 to 928 (median: 46). Seven different HRQoL questionnaires were used for children, three of which were specially adjusted for EA, of which one was reported validity and reliability. Four questionnaires were used for adults, one of which was generic and three symptom-specific, referring to swallowing or to the gastrointestinal tract. Hence, HRQoL outcomes were described differently. However, several studies of pediatric patients with EA revealed associations between lower HRQoL and particular congenital and surgical characteristics, which reflected disease severity. In two out of three articles, pediatric patients with EA had lower overall scores than healthy children. Digestive symptoms consistently lowered HRQoL scores in children and adults. Prior to 2015, only 12 studies were published, which illustrate a recent expansion in this field. Various HRQoL aspects and heterogeneous samples are examined, and outcomes differently reported. Current literature suggests that clinical subgroups of children with EA present with impaired HRQoL and that digestive symptomology influence HRQoL negatively. Conclusions of studies focusing on adults with EA are difficult to draw. Additional research is required.

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Section: Strengths and Limitations In Hrqol Assessment Of Patients Afmentioning

confidence: 99%

Health-Related Quality of Life in Patients after Repair of Esophageal Atresia: A Review of Current Literature

2020

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“…When assessing saturation in a rare disease there are other aspects that need to be taken into consideration. Indeed, the ISPOR taskforce highlighted that saturation in rare diseases may also require use of broader codes and confirmation from other sources, including previous literature and clinician feedback [22,23]. These approaches were used in the current study to confirm concept coverage and support confirmation of saturation in this population.…”

Section: Discussionmentioning

confidence: 93%

Symptoms and Dietary Impact in Hypertriglyceridemia-Associated Pancreatitis: Development and Content Validity of Two New Measures

Burbridge¹,

Randall

Sanchez

et al. 2019

PharmacoEconomics Open

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Background Severe hypertriglyceridemia (sHTG) is a rare condition, complicated by episodes of acute pancreatitis (AP), which can cause pain and/or life-threatening multi-organ dysfunction. Currently, there are no disease-specific patient-reported outcome (PRO) measures evaluating symptoms or dietary impact for this condition. Objective The objective of this study was to explore patient-reported symptoms and impacts of sHTG and AP and develop new measures to capture the symptoms and dietary impacts of this condition using patient language. Methods In-depth, semi-structured concept elicitation interviews were conducted with 12 US-based participants to explore their experience and identify key symptoms and impact on dietary behavior, both during and between episodes of AP. Participants had a range of AP severity with a previous triglyceride reading > 1000 mg/dL, and at least one attack of AP within the last 12 months. Transcripts were coded using thematic analysis. Results Qualitative data analysis revealed the substantial burden of AP associated with sHTG. Participants reported experiencing symptoms, especially abdominal pain, both during and between attacks of AP, and discussed considerable diet changes to prevent or minimize future attacks. A conceptual model was refined, based on patient input, and reviewed by clinical experts to determine key concepts for inclusion within two PRO measures, one evaluating symptoms and another evaluating impact on dietary behavior. Items were drafted using patient-derived language. A 19-item symptoms measure [Hypertriglyceridemia and Acute Pancreatitis Symptom Scale (HAP-SS)] and a 6-item dietary impact measure (Hypertriglyceridemia and Acute Pancreatitis Dietary Behavior (HAP-DB) measure) were developed, both with a 24-h recall period. Conclusions The qualitative analysis confirmed the substantial burden of AP associated with sHTG. This research resulted in development of two disease-specific PRO measures for use during and between attacks of AP. These measures are being utilized in a clinical trial, which will confirm content, structure, and psychometric properties.

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“…Downstream PFDD efforts, such as patient engagement during value assessments or individual treatment choice, rely on the availability of patientcentered outcomes information to inform decision making. FDA's "Roadmap to Patient-Focused Outcome Measurement in Clinical Trials," the 2009 Guidance on Patient-Reported Outcomes (PROs), and the International Society for Pharmacoeconomics and Outcomes Research's report on emerging good practices provide the blueprints for capturing relevant treatment benefit as characterized by patients themselves [9][10][11][12].…”

Section: Discussionmentioning

confidence: 99%

Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today’s Standards for Patient Engagement in Development?

et al. 2018

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For the FDA Pilot COA Compendium to fulfill its purpose of fostering PFDD, it needs fine-tuning to reflect today's standards, improving transparency and facilitating clear identification of included measures so that the level of patient engagement, among other factors, can be properly assessed. Suggested improvements include identifying clinical trials that correspond to the COA Compendium's use in drug development; more clearly identifying which measure is referred to; and including only those measures that already qualified or undergoing qualification.

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Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report

Cited by 122 publications

References 30 publications

Health-Related Quality of Life in Patients after Repair of Esophageal Atresia: A Review of Current Literature

Health-Related Quality of Life in Patients after Repair of Esophageal Atresia: A Review of Current Literature

Symptoms and Dietary Impact in Hypertriglyceridemia-Associated Pancreatitis: Development and Content Validity of Two New Measures

Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today’s Standards for Patient Engagement in Development?

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