Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards

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.

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Patient-Reported Outcomes in Metastatic Breast Cancer: A Review of Industry-Sponsored Clinical Trials

Breast Cancer Basic and Clinical Research ◽

10.4137/bcbcr.s39385 ◽

2016 ◽

Vol 10 ◽

pp. BCBCR.S39385 ◽

Cited By ~ 6

Author(s):

Meaghan Krohe ◽

Yanni Hao ◽

Roger E. Lamoureux ◽

Nina Galipeau ◽

Denise Globe ◽

...

Keyword(s):

Breast Cancer ◽

Quality Of Life ◽

Clinical Trials ◽

Metastatic Breast Cancer ◽

Metastatic Breast ◽

Patient Reported Outcome ◽

Primary Study ◽

Successful Implementation ◽

Patient Reported

Introduction Patient-reported outcome (PRO) measures serve to capture vital patient information not otherwise obtained by primary study endpoints. This paper examines how PROs are utilized as endpoints in industry-sponsored metastatic breast cancer clinical trials. Methods A search was conducted in the clinicaltrials.gov web site for trials involving common treatments for metastatic breast cancer. Thirty-eight clinical trials were identified which included a PRO endpoint in the study, and data were extracted and summarized. Results Overall, 17 unique PRO questionnaires and 14 concepts of measurement were identified as secondary or exploratory endpoints. The Functional Assessment of Cancer Therapy—Breast was the most frequently utilized questionnaire, commonly implemented to assess quality of life. The EORTC QLQ-C30 was also frequently used to measure quality of life or pain. Conclusion This review shares insights into the role of PROs in trials for metastatic breast cancer from which treatment developers and other stakeholders can enhance successful implementation of the patient voice into future trials.

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International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium

The Lancet Oncology ◽

10.1016/s1470-2045(19)30790-9 ◽

2020 ◽

Vol 21 (2) ◽

pp. e83-e96 ◽

Cited By ~ 23

Author(s):

Corneel Coens ◽

Madeline Pe ◽

Amylou C Dueck ◽

Jeff Sloan ◽

Ethan Basch ◽

...

Keyword(s):

Quality Of Life ◽

Randomised Controlled Trials ◽

Patient Reported Outcome ◽

International Standards ◽

Controlled Trials ◽

Patient Reported ◽

Randomised Controlled

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

JCO Clinical Cancer Informatics ◽

10.1200/cci.20.00100 ◽

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.

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Quality of Life and Patient-Reported Outcomes in Endometrial Cancer Clinical Trials

International Journal of Gynecological Cancer ◽

10.1097/igc.0000000000000299 ◽

2014 ◽

Vol 24 (9) ◽

pp. 1693-1699 ◽

Cited By ~ 16

Author(s):

Florence Joly ◽

Jessica McAlpine ◽

Remi Nout ◽

Elisabeth Åvall-Lundqvist ◽

Emad Shash ◽

...

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Endometrial Cancer ◽

Patient Reported Outcomes ◽

Cancer Clinical Trials ◽

Patient Reported

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Patient-Reported Outcomes for Quality of Life in SLE: Essential in Clinical Trials and Ready for Routine Care

Journal of Clinical Medicine ◽

10.3390/jcm10163754 ◽

2021 ◽

Vol 10 (16) ◽

pp. 3754

Author(s):

Matthew H. Nguyen ◽

Frank F. Huang ◽

Sean G. O’Neill

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Lupus Erythematosus ◽

Clinical Care ◽

Routine Care ◽

Patient Reported Outcome ◽

Current Evidence ◽

Patient Reported ◽

Pro Tools

Patient-reported outcome (PRO) instruments are widely used to assess quality of life in Systemic Lupus Erythematosus (SLE) research, and there is growing evidence for their use in clinical care. In this review, we evaluate the current evidence for their use in assessing quality of life in SLE in both research and clinical settings and examine the different characteristics of the commonly used PRO tools. There are now several well-validated generic and SLE-specific tools that have demonstrated utility in clinical trials and several tools that complement activity and damage measures in the clinical setting. PRO tools may help overcome physician–patient discordance in SLE and are valuable in the assessment of fibromyalgia and type 2 symptoms such as widespread pain and fatigue. Future work will identify optimal PRO tools for different settings but, despite current limitations, they are ready to be incorporated into patient care.

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[REiNS] Current Recommendations for Patient-Reported Outcome Measures Assessing Domains of Quality of Life in Neurofibromatosis Clinical Trials

Neurology ◽

10.1212/wnl.0000000000012421 ◽

2021 ◽

pp. 10.1212/WNL.0000000000012421

Author(s):

Pamela L. Wolters ◽

Ana-Maria Vranceanu ◽

Heather L. Thompson ◽

Staci Martin ◽

Vanessa Leigh Merker ◽

...

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Neurofibromatosis Type ◽

Patient Reported Outcome ◽

Self Report ◽

World Health ◽

World Health Organization Quality ◽

Patient Reported ◽

Disease Specific

Objective:To review and recommend patient-reported outcome (PRO) measures assessing multidimensional domains of quality of life (QOL) to use as clinical endpoints in medical and psychosocial trials for children and adults with neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), and schwannomatosis.Methods:The PRO working group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration used systematic methods to review, rate, and recommend existing self-report and parent-report PRO measures of generic and disease-specific QOL for neurofibromatosis (NF) clinical trials. Recommendations were based on four main criteria: patient characteristics, item content, psychometric properties, and feasibility.Results:The highest rated generic measures were 1) the Pediatric Quality of Life Inventory (PedsQL™) Generic Core Scales for NF clinical trials for children or for children through adults, 2) the Functional Assessment of Cancer Therapy-General (FACT-G) for adult medical trials, and 3) the World Health Organization Quality of Life-BREF (WHOQOL-BREF) for adult psychosocial trials. The highest rated disease-specific measures were 1) the PedsQL NF1 Module for NF1 trials, 2) the NF2 Impact on Quality of Life (NFTI-QOL) Scale for NF2 trials, and 3) the Penn Acoustic Neuroma Quality of Life (PANQOL) Scale for NF2 trials targeting vestibular schwannomas. To date, there are no disease-specific tools assessing multidimensional domains of QOL for schwannomatosis.Conclusions:The REiNS Collaboration currently recommends these generic and disease-specific PRO measures to assess multidimensional domains of QOL for NF clinical trials. Additional research is needed to further evaluate the use of these measures in both medical and psychosocial trials.

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Patient-Reported Outcomes in Clinical Trials Leading to Cancer Immunotherapy Drug Approvals From 2011 to 2018: A Systematic Review

JNCI Journal of the National Cancer Institute ◽

10.1093/jnci/djaa174 ◽

2020 ◽

Author(s):

Houssein Safa ◽

Monica Tamil ◽

Philippe E Spiess ◽

Brandon Manley ◽

Julio Pow-Sang ◽

...

Keyword(s):

Quality Of Life ◽

Clinical Trials ◽

Cancer Immunotherapy ◽

Patient Reported Outcomes ◽

Randomized Clinical Trials ◽

International Standards ◽

Type I ◽

Patient Centeredness ◽

Patient Reported

Abstract Background Patient-reported outcomes (PROs) promote patient centeredness in clinical trials; however, in the field of rapidly emerging and clinically impressive immunotherapy, data on PROs are limited. Methods We systematically identified all immunotherapy approvals from 2011 through 2018 and assessed the analytic tools and reporting quality of associated PRO reports. For randomized clinical trials (RCTs), we developed a novel 24-point scoring scale: the PRO Endpoints Analysis Score based on 24 criteria derived from the recommendations of the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium. Results We assessed 44 trial publications supporting 42 immunotherapy approvals. PROs were published for 21 of the 44 (47.7%) trial publications. Twenty-three trials (52.3%) were RCTs and 21 (47.7%) pertained to single-arm trials. The median time between primary clinical outcomes publications and their corresponding secondary PRO publications was 19 months (interquartile range = 9-29 months). Of the 21 PRO reports, 4 (19.0%) reported a specific hypothesis, and most (85.7%) used descriptive statistics. Three (3 of 21 [14.3%]) studies performed a control for type I error. As for RCTs, 14 of 23 (60.9%) published PRO data, including 13 (56.5%) that published a secondary dedicated manuscript. One-half of these 14 trials scored less than 13 points on the 24-point PRO Endpoints Analysis Score. The mean score was 12.71 (range = 5-17, SD = 3.71), and none met all the recommendations of the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium. Conclusions Suboptimal reporting of PROs occurs regularly in cancer immunotherapy trials. Increased efforts are needed to maximize the value of these data in cancer immunotherapy development and approval.

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