Integrating Patient-Reported Outcomes Into Cancer Symptom Management Clinical Trials Supported by the National Cancer Institute–Sponsored Clinical Trials Networks

2007 ◽  
Vol 25 (32) ◽  
pp. 5070-5077 ◽  
Author(s):  
Jeff A. Sloan ◽  
Lawrence Berk ◽  
Joseph Roscoe ◽  
Michael J. Fisch ◽  
Edward G. Shaw ◽  
...  
Keyword(s):  
Clinical Trials ◽  
National Cancer Institute ◽  
Symptom Management ◽  
Patient Reported Outcomes ◽  
Draft Guidance ◽  
End Point ◽  
Patient Reported ◽  
The Us ◽  
Cancer Symptom Management ◽  
Made In

Patient-reported outcomes (PROs) are often the primary end point in symptom management trials. The scientific field of PROs is evolving, as evidenced by the US Food and Drug Administration's February 2007 release of a draft guidance for using PROs in effectiveness claims for drug labeling. This article presents issues encountered during use of PROs in National Cancer Institute–sponsored symptom management trials. Selected trials are presented that exemplify the challenges often seen in symptom management trials, and solutions are described. The examples presented include defining the appropriate end point, selecting and validating assessments, and answering the research questions through statistical analysis and interpretation. Progress has been made in addressing some of the unique challenges of PRO-based symptom management research. Many challenges still remain, but a foundational body of work now exists for more consistent and rigorous application of PROs into symptom management trials. There remains a need for more research in several methodologic aspects of design, analysis, and interpretation of symptom management trials.

Patient-Reported Outcomes in Phase II Cancer Clinical Trials: Lessons Learned and Future Directions

2007 ◽  
Vol 25 (32) ◽  
pp. 5058-5062 ◽  
Author(s):  
Lynne I. Wagner ◽  
Lari Wenzel ◽  
Edward Shaw ◽  
David Cella
Keyword(s):  
Clinical Trials ◽  
Phase Ii ◽  
Symptom Management ◽  
Patient Reported Outcomes ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
Phase Ii Trials ◽  
Patient Reported ◽  
Phase Iii Trials ◽  
The Impact

With increasing limits on the resources available to conduct cancer clinical trials, the inclusion of patient-reported outcomes (PROs) in treatment and symptom management trials must be prioritized. Although it has been suggested on occasion that phase III trials should take precedence over phase II trials, we argue that there is a clear and important role for PRO assessment in phase II trials going forward. To illustrate the value realized from including PROs in phase II trials, we provide case examples from cancer treatment and supportive care. The benefits of including PROs in symptom management intervention research are exemplified using phase II trials targeting cognitive impairment. The inclusion of PROs in phase II cancer clinical trials adds important information about the impact of treatment in health-related quality of life, and advances the science of PRO measurement. These contributions significantly enhance the design of phase III trials, ultimately leading to the efficient utilization of clinical trial resources.

scholarly journals Software for Administering the National Cancer Institute�s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events: Usability Study (Preprint)

2018 ◽  
Author(s):  
Martin W Schoen ◽  
Ethan Basch ◽  
Lori L Hudson ◽  
Arlene E Chung ◽  
Tito R Mendoza ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
National Cancer Institute ◽  
Patient Reported Outcomes ◽  
Task Completion ◽  
Heuristic Evaluation ◽  
Web Based ◽  
System Usability Scale ◽  
Patient Reported ◽  
The Common

BACKGROUND The US National Cancer Institute (NCI) developed software to gather symptomatic adverse events directly from patients participating in clinical trials. The software administers surveys to patients using items from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) through Web-based or automated telephone interfaces and facilitates the management of survey administration and the resultant data by professionals (clinicians and research associates). OBJECTIVE The purpose of this study was to iteratively evaluate and improve the usability of the PRO-CTCAE software. METHODS Heuristic evaluation of the software functionality was followed by semiscripted, think-aloud protocols in two consecutive rounds of usability testing among patients with cancer, clinicians, and research associates at 3 cancer centers. We conducted testing with patients both in clinics and at home (remotely) for both Web-based and telephone interfaces. Furthermore, we refined the software between rounds and retested. RESULTS Heuristic evaluation identified deviations from the best practices across 10 standardized categories, which informed initial software improvement. Subsequently, we conducted user-based testing among 169 patients and 47 professionals. Software modifications between rounds addressed identified issues, including difficulty using radio buttons, absence of survey progress indicators, and login problems (for patients) as well as scheduling of patient surveys (for professionals). The initial System Usability Scale (SUS) score for the patient Web-based interface was 86 and 82 (P=.22) before and after modifications, respectively, whereas the task completion score was 4.47, which improved to 4.58 (P=.39) after modifications. Following modifications for professional users, the SUS scores improved from 71 to 75 (P=.47), and the mean task performance improved significantly (4.40 vs 4.02; P=.001). CONCLUSIONS Software modifications, informed by rigorous assessment, rendered a usable system, which is currently used in multiple NCI-sponsored multicenter cancer clinical trials. CLINICALTRIAL ClinicalTrials.gov NCT01031641; https://clinicaltrials.gov/ct2/show/NCT01031641 (Archived by WebCite at http://www.webcitation.org/708hTjlTl)

Abstract TP292: Patient Reported Outcomes in Stroke Clinical Trials

Stroke ◽  
2017 ◽  
Vol 48 (suppl_1) ◽  
Author(s):  
Christopher Carr ◽  
Laya Reddy ◽  
Azad Hussain ◽  
Sean Murray ◽  
Neha Vazirani ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Randomized Clinical Trials ◽  
Depression Scale ◽  
Large Degree ◽  
Cerebrovascular Diseases ◽  
Neurological Deficits ◽  
Health Profile ◽  
Patient Reported ◽  
The Us

Stroke is the 5 th -leading cause of death in the US and the major cause of disability. Many patients who have suffered from strokes are left with permanent neurological deficits, new occupational and social difficulties, and reductions in quality of life that may be idiosyncratic or vary considerably from patient to patient. Solicitation of patient reported outcomes (PROs) in the treatment and recovery from strokes is therefore imperative. We examined a list compiled in 2013 for Stroke by Magin et al. of representative stroke-related, randomized clinical trials published in 10 high-impact journals between 2002 and 2012 to assess whether PROs were collected and which constructs were reported. We categorized PRO measures as stroke-specific (e.g. Stroke Impact Scale), health profile and utility scales (e.g. Beck Depression Scale), or general (e.g. pain visual analog scale). Two reviewers independently abstracted PRO measures from each article and disagreements were resolved by consensus. Fisher’s exact test was used for statistical analyses. Of the 99 articles that met study inclusion criteria, 20% concerned prevention, 22% acute treatment, and 58% rehabilitation. A plurality of trials were European (43%) followed by studies based in the US (25%), Asian countries (10%), and Australia (10%). Altogether, 37% of studies used a PRO of any kind. Stroke-specific PROs were collected in 17% of studies, health profile and utility scales were used in 17%, and general PROs were used in 23%. There were no significant differences in PRO use with regards to year of publication or study location. Health profile and utility scales (p=0.01) and unclassified PROs (p<0.001) were most-commonly reported in rehabilitation trials. Stroke-specific PROs were most commonly published in the journals Brain and Journal of Stroke and Cerebrovascular Diseases (p=0.001). Overall, our review and analysis detected a low prevalence and a large degree of heterogeneity of PRO measures reported in stroke-related clinical trials. Future stroke research must routinely incorporate PROs into the study design to help patients, caregivers, and providers make informed decisions about stroke prevention, treatment, and rehabilitation options that yield outcomes of greatest importance to them.

scholarly journals Potential of patient-reported outcomes as nonprimary endpoints in clinical trials

2013 ◽  
Vol 11 (1) ◽  
pp. 83 ◽  
Author(s):  
Ari Gnanasakthy ◽  
Sandra Lewis ◽  
Marci Clark ◽  
Margaret Mordin ◽  
Carla DeMuro
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Patient Reported

scholarly journals SAT0121 PAIN AND OTHER PATIENT-REPORTED OUTCOMES IN PATIENTS WITH RHEUMATOID ARTHRITIS WHO DID OR DID NOT ACHIEVE TREATMENT RESPONSE BASED ON IMPROVEMENT IN SWOLLEN JOINTS IN TOCILIZUMAB CLINICAL TRIALS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 994.2-995
Author(s):  
A. Sebba ◽  
J. Han ◽  
S. Mohan
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trials ◽  
Pain Score ◽  
Patient Reported Outcomes ◽  
Component Score ◽  
Link Type ◽  
Pain Scores ◽  
Sf 36 ◽  
Patient Reported ◽  
Nonresponder Group

Background:Significant improvements in pain and other patient-reported outcomes (PROs) have been shown in large clinical trials in patients with rheumatoid arthritis (RA) who receive tocilizumab (TCZ) compared with placebo (PBO). Recent data suggest that pain in RA may be noninflammatory as well as inflammatory, and improvement in pain scores and other PROs may be seen in patients who do not respond to treatment based on disease activity measures that evaluate inflammation.Objectives:To assess changes in pain scores and other PROs in patients with RA who did or did not achieve ≥ 20% improvement in SJC in TCZ clinical trials.Methods:Data from patients with active RA who received intravenous TCZ 8 mg/kg + MTX or PBO + MTX in 3 Phase III studies (OPTION [NCT00106548], TOWARD [NCT00106574] and LITHE [NCT00109408]) were included. All patients had moderate to severe RA with an inadequate response or intolerance of MTX (OPTION, LITHE) or conventional synthetic disease-modifying antirheumatic drugs (csDMARDs; TOWARD). Changes in pain (visual analog scale [VAS], 0-100 mm), Health Assessment Questionnaire Disability Index (HAQ-DI, 0-3), 36-Item Short Form Survey (SF-36) physical component score (PCS) and mental component score (MCS; 0-50) and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score (0-52) from baseline to Week 24 were evaluated. Results were compared between patients receiving TCZ + MTX and those receiving PBO + MTX in both patients who achieved ≥ 20% improvement in SJC (responders) and those who did not (nonresponders). The changes from baseline were analyzed using a mixed model with repeated measures, including the following covariates and interactions: treatment, visit, baseline of endpoint, region, baseline DAS28 and interactions of visit with treatment and baseline of endpoint.Results:Data from 1254 responders (TCZ + MTX, n = 831; PBO + MTX, n = 423) and 620 nonresponders (TCZ + MTX, n = 225; PBO + MTX, n = 395) were included. Patients receiving TCZ + MTX had significantly greater improvement in pain scores and HAQ-DI compared with PBO + MTX in the responder group (–27.19 vs –16.77 and –0.55 vs –0.34, respectively;P< 0.0001 for both) and nonresponder group (–9.59 vs 2.53 and –0.20 vs 0.01;P< 0.0001 for both) at Week 24 (Figure 1). Similar results were seen at Week 16 in the nonresponder group (–11.06 vs –2.38 and –0.23 vs –0.04;P< 0.0001 for both) prior to initiation of rescue treatment. At Week 24 in the responder group, patients receiving TCZ + MTX had significantly greater improvements compared with PBO + MTX in SF-36 PCS and MCS (9.16 vs 5.71 and 6.55 vs 3.79, respectively;P< 0.0001 for both) (Figure 2) and FACIT-Fatigue (8.39 vs 5.11;P< 0.0001). In the nonresponder group, patients receiving TCZ + MTX had significantly greater improvements compared with PBO + MTX in SF-36 PCS at Week 16 (3.81 vs 1.65;P= 0.0006) and Week 24 (4.42 vs 1.01;P< 0.0001) (Figure 2) and FACIT-Fatigue at Week 16 (3.82 vs 1.32;P= 0.0039) and Week 24 (3.90 vs 1.40;P= 0.0111).Conclusion:Patients with RA who received TCZ + MTX had significantly greater improvements in pain score and other PROs than those who received PBO + MTX regardless of whether they achieved ≥ 20% improvement in SJC. Clinical outcome at Week 24 correlated well with PROs, with a relatively larger improvement in pain score and other PROs in the responder group than in the nonresponder group; relative to PBO + MTX, these improvements appear numerically similar in the responder and nonresponder groups with consistently smaller difference between the groups in TCZ-treated arms. The consistent effect of TCZ on PROs in both responder and nonresponder groups warrants further study on the impact of TCZ on sources of pain independent of that caused by joint inflammation.Figure:Acknowledgments:This study was sponsored by Genentech, Inc. Support for third-party writing assistance, furnished by Health Interactions, Inc, was provided by Genentech, Inc.Disclosure of Interests:Anthony Sebba Consultant of: Genentech, Gilead, Lilly, Regeneron Pharmaceuticals Inc., Sanofi, Speakers bureau: Lilly, Roche, Sanofi, Jian Han Shareholder of: Genentech, Inc., Employee of: Genentech, Inc., Shalini Mohan Shareholder of: Genentech, Inc., Employee of: Genentech, Inc.

scholarly journals Longitudinal Analysis of Patient-Reported Outcomes in Clinical Trials: Applications of Multilevel and Multidimensional Item Response Theory

Psychometrika ◽  
2021 ◽  
Author(s):  
Li Cai ◽  
Carrie R. Houts
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Latent Variable ◽  
Patient Reported Outcomes ◽  
Multilevel Models ◽  
Latent Variable Models ◽  
Trial Data ◽  
Regulatory Science ◽  
Measurement Information ◽  
Patient Reported

AbstractWith decades of advance research and recent developments in the drug and medical device regulatory approval process, patient-reported outcomes (PROs) are becoming increasingly important in clinical trials. While clinical trial analyses typically treat scores from PROs as observed variables, the potential to use latent variable models when analyzing patient responses in clinical trial data presents novel opportunities for both psychometrics and regulatory science. An accessible overview of analyses commonly used to analyze longitudinal trial data and statistical models familiar in both psychometrics and biometrics, such as growth models, multilevel models, and latent variable models, is provided to call attention to connections and common themes among these models that have found use across many research areas. Additionally, examples using empirical data from a randomized clinical trial provide concrete demonstrations of the implementation of these models. The increasing availability of high-quality, psychometrically rigorous assessment instruments in clinical trials, of which the Patient-Reported Outcomes Measurement Information System (PROMIS®) is a prominent example, provides rare possibilities for psychometrics to help improve the statistical tools used in regulatory science.

scholarly journals Linguistic validation of the simplified Chinese version of the US National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE™)

BMC Cancer ◽  
2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Cheng KKF ◽  
S. A. Mitchell ◽  
N. Chan ◽  
E. Ang ◽  
W. Tam ◽  
...  
Keyword(s):  
Adverse Events ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
A Priori ◽  
Cognitive Interviews ◽  
Simplified Chinese ◽  
Patient Reported ◽  
The Us ◽  
The Common ◽  
Response Choices

Abstract Background The aim of this study was to translate and linguistically validate the U.S. National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™) into Simplified Chinese for use in Singapore. Methods All 124 items of the English source PRO-CTCAE item library were translated into Simplified Chinese using internationally established translation procedures. Two rounds of cognitive interviews were conducted with 96 cancer patients undergoing adjuvant treatment to determine if the translations adequately captured the PRO-CTCAE source concepts, and to evaluate comprehension, clarity and ease of judgement. Interview probes addressed the 78 PRO-CTCAE symptom terms (e.g. fatigue), as well as the attributes (e.g. severity), response choices, and phrasing of ‘at its worst’. Items that met the a priori threshold of ≥20% of participants with comprehension difficulties were considered for rephrasing and retesting. Items where < 20% of the sample experienced comprehension difficulties were also considered for rephrasing if better phrasing options were available. Results A majority of PRO-CTCAE-Simplified Chinese items were well comprehended by participants in Round 1. One item posed difficulties in ≥20% and was revised. Two items presented difficulties in < 20% but were revised as there were preferred alternative phrasings. Twenty-four items presented difficulties in < 10% of respondents. Of these, eleven items were revised to an alternative preferred phrasing, four items were revised to include synonyms. Revised items were tested in Round 2 and demonstrated satisfactory comprehension. Conclusions PRO-CTCAE-Simplified Chinese has been successfully developed and linguistically validated in a sample of cancer patients residing in Singapore.

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