Health-Related Quality of Life Data in Cancer Clinical Trials for Drug Registration: The Value Beyond Reimbursement

2021 ◽  
pp. 112-124
Author(s):  
Erinne Wasalski ◽  
Shashi Mehta
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Cochrane Library ◽  
Cancer Drug ◽  
Cancer Clinical Trials ◽  
Drug Registration ◽  
Hrqol Data ◽  
Patient Reported ◽  
Related Quality

PURPOSE A review of the literature was performed to evaluate how quality of life measures are collected, analyzed, and reported in cancer clinical trials intended to support drug registration.Health-related quality of life (HRQoL) data points are one of the patient-reported outcome (PRO) assessments used in clinical trials to evaluate the effects of treatments from the patient perspective. The use of PROs has gained focus in cancer clinical trials as more options become available for greater longevity of patients on treatment. Standardization of PRO data is evolving and involves unique challenges when used for assessing biologic and chemotherapeutic agents for the treatment of cancer. METHODS In this study, a review of literature published between 2009 and 2019 was conducted using PubMed, COCHRANE Library, and Medline. The research focus was on the current guidance, implementation, and reporting as well as highlighting the issues, and recommendations for the inclusion of HRQoL end points in cancer clinical trials intended for use in drug registration. RESULTS Although there exist many levels of guidance for HRQoL measures in cancer drug trials, challenges to operational implementation, the current inconsistent adherence to reporting standards, and the lack of consensus and understanding of analyses limit the value and potential of the resulting data collected. CONCLUSION The results of HRQoL data collected from cancer clinical trials can be difficult to interpret and apply to inform clinical decision making. Increased reporting and access to these data can provide opportunities for potential applications to improve translatability of HRQoL data collected in clinical trials into practice.

scholarly journals Research Objectives, Statistical Analyses and Interpretation of Health-Related Quality of Life Data in Glioma Research: A Systematic Review

Cancers ◽  
2020 ◽  
Vol 12 (12) ◽  
pp. 3502
Author(s):  
Marijke B. Coomans ◽  
Marthe C.M. Peeters ◽  
Johan A.F. Koekkoek ◽  
Jan W. Schoones ◽  
Jaap Reijneveld ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Analytical Methods ◽  
Statistical Analyses ◽  
Health Related Quality ◽  
Hrqol Data ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Background: Health-related quality of life (HRQoL) has become an increasingly important patient-reported outcome in glioma studies. Ideally, collected HRQoL data should be exploited to the full, with proper analytical methods. This systematic review aimed to provide an overview on how HRQoL data is currently evaluated in glioma studies, focusing on the research objectives and statistical analyses of HRQoL data. Methods: A systematic literature search in the databases PubMed, Embase, Web of Science and Cochrane was conducted up to 5 June 2020. Articles were selected based on predetermined inclusion criteria and information on study design, HRQoL instrument, HRQoL research objective and statistical methods were extracted. Results: A total of 170 articles describing 154 unique studies were eligible, in which 17 different HRQoL instruments were used. HRQoL was the primary outcome in 62% of the included articles, and 51% investigated ≥1 research question with respect to HRQoL, for which various analytical methods were used. In only 42% of the articles analyzing HRQoL results over time, the minimally clinical important difference was reported and interpreted. Eighty-six percent of articles reported HRQoL results at a group level only, and not at the individual patient level. Conclusion: Currently, the assessment and analysis of HRQoL outcomes in glioma studies is highly variable. Opportunities to maximize information obtained with HRQoL data include appropriate and complementary analyses at both the group and individual level, comprehensive reporting of HRQoL results in separate articles or supplementary material, and adherence to existing guidelines about the assessment, analysis and reporting of patient-reported outcomes.

scholarly journals Toward Patient-Centered Innovation

2020 ◽  
Vol 15 (10) ◽  
pp. 1522-1530
Author(s):  
Jennifer E. Flythe ◽  
Tandrea S. Hilliard ◽  
Kourtney Ikeler ◽  
San Keller ◽  
Debbie S. Gipson ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Conceptual Framework ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Health Related Quality ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incorporating patient perspectives into the total product life cycle (i.e., from product conception to postmarket surveillance) and encourages the consideration of patient-reported outcomes in regulatory-focused clinical trials when appropriate. Recognizing the significance of identifying patient-reported outcome measures (PROMs) that capture contemporary patient priorities, the Kidney Health Initiative, a public–private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a workgroup to (1) develop a conceptual framework for a health-related quality of life PROM; (2) identify and map existing PROMs to the conceptual framework, prioritizing them on the basis of their supporting evidence for use in the regulatory environment; and (3) describe next steps for identifying PROMs for use in regulatory clinical trials of transformative KRT devices. This paper summarizes the proposed health-related quality-of-life PROM conceptual framework, maps and prioritizes PROMs, and identifies gaps and future needs to advance the development of rigorous, meaningful PROMS for use in clinical trials of transformative KRT devices.

scholarly journals REiNS: Patient Report of Hearing in Neurofibromatosis Type 2: Recommendations for Clinical Trials

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012424
Author(s):  
Heather L. Thompson ◽  
Ann Blanton ◽  
Barbara Franklin ◽  
Vanessa L. Merker ◽  
Kevin H. Franck ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Neurofibromatosis Type ◽  
The Self ◽  
Self Assessment ◽  
Hearing Function ◽  
Patient Reported ◽  
Related Quality

Objective:To systematically evaluate published patient-reported outcome measures for the assessment of hearing function and hearing-related quality of life, and subsequently recommend measures selected by the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration (REiNS) as endpoints for clinical trials in Neurofibromatosis Type 2.Methods:The REiNS patient-reported outcomes working group systematically evaluated published patient-reported outcome measures of (1) hearing function and (2) hearing-related quality of life for individuals with hearing loss of various etiologies using previously published REiNS rating procedures. Ten measures of hearing functioning and 11 measures of hearing-related quality of life were reviewed. Measures were numerically scored and compared primarily on their participant characteristics (including participant age range and availability of normative data), item content, psychometric properties, and feasibility for use in clinical trials.Results:The Self-Assessment of Communication and the Self-Assessment of Communication-Adolescent were identified as most useful for adult and pediatric populations with NF2, respectively, for the measurement of both hearing function and hearing-related quality of life. Measures were selected for their strengths in participant characteristics, item content, psychometric properties, and feasibility for use in clinical trials.Conclusions:Currently, REiNS recommends the Self-Assessment of Communication adult and adolescent forms for the assessment of patient-reported hearing function and hearing-related quality of life for clinical trials for neurofibromatosis type 2. Further work is needed to demonstrate the utility of these measures in evaluating pharmacological and/or behavioral interventions.

Health-related quality-of-life in patients with cholangiocarcinoma: Results of a systematic literature review.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e15623-e15623
Author(s):  
Yohan Cho ◽  
Claire Chadwick ◽  
Scott Barrett ◽  
Kristen Bibeau ◽  
Wissam El-Hadi ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Literature Review ◽  
Novel Therapies ◽  
Health Related Quality ◽  
Duct Carcinoma ◽  
Hrqol Data ◽  
Related Quality ◽  
Health Related

e15623 Background: Cholangiocarcinoma (CCA) is a rare biliary tract cancer (BTC) with a historically poor prognosis and limited treatment options. Both quality and quantity of life can be important factors when deciding treatment, thus assessment of health-related quality-of-life (HRQoL) is needed to justify the use of novel therapies. HRQoL questionnaires are a standardized way to understand the patients’ perspective and treatment experience. The objective of this review was to examine the published literature specific to HRQoL in CCA. Methods: A systematic literature search was conducted using EMBASE and MEDLINE from Jan 2008 to June 2018 to identify abstracts with published HRQoL data in CCA patients. This review also included global conference proceedings (2015-2018) and HTA websites. Articles were considered relevant if they reported HRQoL data in patients with CCA, bile duct carcinoma, and/or hepatobiliary system cancer. Results: Of the 2,933 studies initially identified, 7 ongoing or completed studies met the scoping criteria to be included in the review. No articles in this literature review reported data from clinical trials. Multiple HRQoL measures were utilized; however within the studies, EORTC QLQ-BIL21 was the only HRQoL measure developed and validated specifically for BTC (CCA, gallbladder, or ampulla vater) identified in this review. 3/7 studies included information on CCA subtypes (intrahepatic, extrahepatic) with 2/3 studies reporting HRQoL by subtype. Patient symptoms were reported in 4/7 studies, including gastrointestinal distress, abdominal pain, fatigue, fever, itching, difficulty sleeping, anxiety, jaundice and weight loss. Conclusions: Within this literature review, few studies have been published with HRQoL data in CCA patients. Data from the existing published literature is generally from a heterogeneous patient population, and not consistently specified for CCA stage or subtype. To better understand HRQoL in CCA patients, more data needs to be generated that include clinical trials for novel therapies.

International Perspective on Health-Related Quality-of-Life Research in Cancer Clinical Trials: The European Organisation for Research and Treatment of Cancer Experience

2007 ◽  
Vol 25 (32) ◽  
pp. 5082-5086 ◽  
Author(s):  
Andrew Bottomley ◽  
Neil K. Aaronson
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
Health Related Quality ◽  
European Organisation ◽  
Related Quality ◽  
Health Related

Over recent decades, health-related quality of life (HRQOL) research has been increasingly integrated into cancer clinical trials. The purpose of this review is to examine the overall approach taken towards clinical trial–based HRQOL investigations within the European Organisation for Research and Treatment of Cancer (EORTC). This article reports a literature review of clinical trial–based HRQOL investigations and provides selective examples of HRQOL studies in phase III clinical trials in various disease sites. The findings of this review highlight that, historically, assessing HRQOL was a challenge. However, as EORTC has become more experienced in the assessment of HRQOL and has developed a portfolio of appropriate tools, HRQOL has become a more accepted end point in large-scale trials. The trials reviewed in this article show that, in general, HRQOL data do provide information that can both inform clinicians about the effectiveness of the treatments and also serve as an invaluable source of information for patients to make informed decisions regarding the treatment choice.

The Prognostic Significance of Patient-Reported Outcomes in Cancer Clinical Trials

2008 ◽  
Vol 26 (8) ◽  
pp. 1355-1363 ◽  
Author(s):  
Carolyn C. Gotay ◽  
Crissy T. Kawamoto ◽  
Andrew Bottomley ◽  
Fabio Efficace
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Prognostic Significance ◽  
Cancer Clinical Trials ◽  
Life Questionnaire ◽  
European Organization ◽  
Patient Reported ◽  
The Impact

Purpose Patient-reported outcomes (PROs), routinely collected as a part of cancer clinical trials, have been linked with survival in numerous clinical studies, but a comprehensive critical review has not been reported. This study systematically assessed the impact of PROs on patient survival after a cancer diagnosis within the context of clinical trials. Design Cancer clinical trials that assessed baseline PROs and mortality were identified through MEDLINE (through December 2006) supplemented by the Cochrane database, American Society of Clinical Oncology/European Society for Medical Oncology abstracts and hand searches. Inclusion criteria were publication in English language and use of multivariate analyses of PROs that controlled for one or more clinical factors. Two raters reviewed each study, abstracted data, and assessed study quality; two additional raters verified abstractions. Results In 36 of 39 studies (N = 13,874), at least one PRO was significantly associated with survival (P < .05) in multivariate analysis, with varying effect sizes. Studies of lung (n = 12) and breast cancer (n = 8) were most prevalent. The most commonly assessed PRO was quality of life, measured by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire C30 in 56% of studies. Clinical variables adjusted for included performance status (PS), treatment arm, stage, weight loss, and serum markers. Results indicated that PROs provide distinct prognostic information beyond standard clinical measures in cancer clinical trials. Conclusion PROs might be considered for stratification purposes in future trials, as they were often better predictors of survival than PS. Studies are needed to determine whether interventions that improve PROs also increase survival and to identify explanatory mechanisms through which PROs relate to survival.

Health related quality of life outcomes in cancer clinical trials

2005 ◽  
Vol 41 (12) ◽  
pp. 1697-1709 ◽  
Author(s):  
A. Bottomley ◽  
H. Flechtner ◽  
F. Efficace ◽  
V. Vanvoorden ◽  
C. Coens ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Cancer Clinical Trials ◽  
Health Related Quality ◽  
Life Outcomes ◽  
Quality Of Life Outcomes ◽  
Related Quality ◽  
Health Related
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