Overview of the Patient Perspective at OMERACT 10 — Conceptualizing Methods for Developing Patient-Reported Outcomes

2011 ◽  
Vol 38 (8) ◽  
pp. 1699-1701 ◽  
Author(s):  
JOHN R. KIRWAN ◽  
PETER S. TUGWELL
Keyword(s):  
Clinical Trials ◽  
Experimental Work ◽  
Patient Reported Outcomes ◽  
Patient Perspective ◽  
Methodological Issues ◽  
Instrument Selection ◽  
Patient Reported ◽  
The Impact ◽  
Choice Of Instruments ◽  
The Way

This overview draws out the main conclusions from the 4 workshops focused on incorporating the patient perspective into outcome assessment at the 10th Outcome Measures in Rheumatology (OMERACT 10) conference. They raised methodological issues about the choice of outcome domains to include in clinical trials, the development or choice of instruments to measure these domains, and the way these instruments might capture the impact of a disease and its treatment. The need to develop a more rigorous conceptual model of quantifying the way conditions affect health, and the need to ensure patients are directly involved in the decisions about domains and instruments, emerged clearly. The OMERACT participants voted to develop guidelines for domain and instrument selection, and conceptual and experimental work will be brought forward to revise and upgrade the OMERACT Filter.

Dare voce ai pazienti nella ricerca sulle malattie rare e sui famaci orfani / Giving patients a voice in the rare diseases and orphan drugs research

2018 ◽  
Vol 67 (1) ◽  
pp. 25-40
Author(s):  
Elena Mancini ◽  
Roberta Martina Zagarella
Keyword(s):  
Clinical Trials ◽  
Rare Diseases ◽  
Patient Reported Outcomes ◽  
Narrative Medicine ◽  
Orphan Drugs ◽  
New Drugs ◽  
Patient Centered ◽  
Patient Reported ◽  
Critical Issues ◽  
The Impact

L’articolo ha l’obiettivo di mettere in luce potenzialità e criticità dell’inclusione della prospettiva dei pazienti nella ricerca sulle malattie rare e sui farmaci orfani. A tal fine, nella prima parte, si propone un’analisi epistemologica dell’utilizzo dei racconti dell’esperienza individuale della malattia nella ricerca scientifica e nei trial clinici, facendo emergere, anche attraverso gli strumenti della medicina narrativa, le sfide teoriche e operative poste dall’inclusione della soggettività del paziente e del vissuto di malattia nonché l’importanza della valorizzazione della prospettiva del paziente, sia in generale sia nella ricerca sulle malattie rare e sui farmaci orfani. Nella seconda parte, il testo analizza in particolare il ruolo degli esiti riportati dai pazienti o Patient Reported Outcomes (PROs), misure per la valutazione complessiva della salute basate sulla prospettiva dei pazienti stessi, incentrandosi sulla sperimentazione clinica nel campo delle malattie rare. In questo contesto, infatti, i racconti di malattia, raccolti e valorizzati da fonti istituzionali e associazioni di pazienti, hanno contribuito a far emergere importanti questioni critiche e difficoltà nell’impiego di outcome centrati sul paziente nello sviluppo di nuovi farmaci e trattamenti, generando una serie di documenti e raccomandazioni relative al loro utilizzo per il benessere della comunità dei malati rari. ---------- This paper aims to highlight the potentiality and criticality of including patients’ perspective in rare diseases and orphan drugs research. In the first part, we propose an epistemological analysis of individual narrations of disease experience as they are used in scientific research and clinical trials. With the help of narrative medicine approach, this analysis points out theoretical and operational challenges of a perspective that includes patient’s subjectivity and illness experience. Furthermore, it reveals the significance of patients’ standpoints in general and in rare diseases as well as in the orphan drugs research. The second part of our article focuses on the role of the Patient reported Outcomes (PROs) – which are measures for the health’s overall assessment based on patient’s perspective – by investigating the impact on clinical trials for rare diseases. In this context, illness stories, which are collected and promoted by institutional sources and patients’ associations, contribute to underline important critical issues at stake in the employment of patient-centered outcomes both in new drugs and in the treatments development. Moreover, these stories are crucial to elaborate documents and recommendations concerning the use of PROs for the rare patients’ community welfare.

Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute’s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

2016 ◽  
pp. 67-73 ◽  
Author(s):  
Paul G. Kluetz ◽  
Diana T. Chingos ◽  
Ethan M. Basch ◽  
Sandra A. Mitchell
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Patient Reported Outcomes ◽  
Patient Perspective ◽  
Cancer Clinical Trials ◽  
Cancer Therapies ◽  
Systematic Assessment ◽  
Promising Tool ◽  
Patient Reported ◽  
The Common

Systematic capture of the patient perspective can inform the development of new cancer therapies. Patient-reported outcomes (PROs) are commonly included in cancer clinical trials; however, there is heterogeneity in the constructs, measures, and analytic approaches that have been used making these endpoints challenging to interpret. There is renewed effort to identify rigorous methods to obtain high-quality and informative PRO data from cancer clinical trials. In this setting, PROs are used to address specific research objectives, and an important objective that spans the product development life cycle is the assessment of safety and tolerability. The U.S. Food and Drug Administration’s (FDA) Office of Hematology and Oncology Products (OHOP) has identified symptomatic adverse events (AEs) as a central PRO concept, and a systematic assessment of patient-reported symptomatic AEs can provide data to complement clinician reporting. The National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is being evaluated by multiple stakeholders, including the FDA, and is considered a promising tool to provide a standard yet flexible method to assess symptomatic AEs from the patient perspective. In this article, we briefly review the FDA OHOP’s perspective on PROs in cancer trials submitted to the FDA and focus on the assessment of symptomatic AEs using PRO-CTCAE. We conclude by discussing further work that must be done to broaden the use of PRO-CTCAE as a method to provide patient-centered data that can complement existing safety and tolerability assessments across cancer clinical trials.

Emerging trends and utilization of patient-reported outcomes (PROs) in clinical trials of chimeric antigen receptor (CAR) T-cell therapies.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19142-e19142
Author(s):  
Anthony John Messina ◽  
Vasily Andrianov ◽  
Daniel Mazzolenis ◽  
Liat Vidal-Fisher
Keyword(s):  
Clinical Trials ◽  
T Cell ◽  
Phase I ◽  
Patient Reported Outcomes ◽  
Utilization Rate ◽  
T Cell Therapy ◽  
Car T Cell ◽  
Patient Reported ◽  
Car T ◽  
The Impact

e19142 Background: Patient-reported outcomes (PROs) are an important tool to assess the impact of new therapies on health-related quality of life (HRQoL). This study aimed to describe if and what PRO instruments are currently being utilized in CAR-T cell therapy studies in solid and hematological malignancies while assessing the patterns of inclusion and trends of HRQoL data reporting. Methods: We used Citeline to search for clinical trials between Jan 2008 - Jan 2020, excluding planned or terminated studies, non-oncology, non-treatment, and duplicates. Reviewers extracted various parameters for included trials, then cross-matched data with EU Clinical Trials Register, Clinical trials.gov, trial protocols (when available), and Google. The reporting of PRO data was then assessed for those Closed/Completed trials that included a PRO via PubMed/MEDLINE, Sponsor, and Google. Results: A sample of 664 CAR-T trials was identified. PROs were included in only 6.17% (41/664) studies. Of the 41 trials that included a PRO, 63.41% (26/41) utilized more than one PRO, with the generic EORTC QLQ-C30 and the EQ-5D being used predominately. Median HRQoL follow-up was 5-years on most trials. No studies used PROs as primary endpoints. The majority of PROs were observed to be utilized in early phase trials (phase I, 12; Phase I/II, 17). PROs were first incorporated in CAR-T trials beginning in 2014, and the utilization rate has increased steadily, except for 2019. PROs were included in 3 first line trials, 22 second line, 5 third line, and 11 fourth line or greater. PRO utilization between solid tumor trials and hematologic malignancies was comparable (6.04% [9/149], and 6.26% [32/511]). Of the completed/closed trials, 28.57% (3/14) published PRO data and met at least eight of the CONSORT-PRO quality indicators. Conclusions: The utilization of PROs in CAR-T trials (6.17%) is under the industry average of 27%, despite the growing importance of HRQoL and its impact on value-based care. The findings from this review reflect the overall increased attention to CAR-T as a new therapeutic entity and the continued deficiency of including and reporting of PROs in trial designs.

Current Status of Patient-Reported Outcomes in Industry-Sponsored Oncology Clinical Trials and Product Labels

2007 ◽  
Vol 25 (32) ◽  
pp. 5087-5093 ◽  
Author(s):  
Kathleen Gondek ◽  
Pierre-Philippe Sagnier ◽  
Kim Gilchrist ◽  
J. Michael Woolley
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Symptom Assessment ◽  
Current Status ◽  
Cancer Therapies ◽  
Package Inserts ◽  
Patient Reported ◽  
Oncology Clinical Trials ◽  
Related Quality ◽  
The Impact

Assessing patient-reported outcomes (PROs) in clinical trials is of interest to clinicians, patients, regulators, and industry. The use and impact of PROs is a growing area of methodologic research, particularly as they relate to tumor types, biomarkers, and various patient populations and cultures. Both the US Food and Drug Administration (FDA) and European Agency for the Evaluation of Medicinal Products in recent guidance have acknowledged the need to account for treatment-related impact on patient symptoms and/or health-related quality of life (HRQOL). Clinical research likely reflects the informative value of PROs. A search of www.clinicaltrials.gov , the FDA Web site, and product package inserts was conducted to assess the inclusion of symptom assessment and HRQOL within industry-sponsored clinical trials in cancer and approved cancer therapies and their respective product labels. Overall, there were 2,704 industry-sponsored oncology trials, of which 322 (12%) included a PRO measure. Of the 70 FDA new or revised labels, only six package inserts include PRO data. Symptoms were assessed uniformly across the phases of clinical trials, whereas HRQOL assessment increased in the later phases of clinical trials. Collecting PRO data can enhance our understanding of cancer burden and the impact of interventions on patients' lives.

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.

scholarly journals Patient Reported Outcomes in Large Vessel Vasculitides

2021 ◽  
Vol 23 (2) ◽  
Author(s):  
Joanna Robson ◽  
Sarah Mackie ◽  
Catherine Hill
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Systemic Vasculitis ◽  
Clinical Care ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Patient Reported ◽  
The Impact ◽  
Large Vessel Vasculitides ◽  
Disease Specific

Abstract Purpose of Review The goal of this paper is to review current and future uses of patient-reported outcomes in large vessel vasculitis. The large vessel vasculitides comprise Giant Cell Arteritis and Takayasu arteritis; both are types of systemic vasculitis which affect the larger blood vessels. Patient-reported outcomes (PROs) capture the impact of these diseases on health-related quality of life. Recent Findings Generic PROs such as the SF-36 are currently used to compare HRQOL of people with GCA and TAK within clinical trials and observational studies and to make comparisons with the general population and HRQoL in other diseases. The development of a disease-specific PRO for GCA is currently underway. Beyond clinical trials, there is much interest in the use of PROs within routine clinical care, particularly E-PROs for remote use. Summary Further work will be needed to complete the development of disease-specific PROs for people with large vessel vasculitis and to establish feasibility, acceptability, and utility of E-PROs.

Problems with the MetaBLIND study: An examination of data on blinding patients in trials with patient-reported outcomes

2021 ◽  
pp. 135910532110593
Author(s):  
Michiel Tack
Keyword(s):  
Clinical Trials ◽  
Epidemiological Study ◽  
Study Design ◽  
Patient Reported Outcomes ◽  
Randomized Trials ◽  
Effect Sizes ◽  
Patient Reported ◽  
The Impact ◽  
Trial Participants

MetaBLIND is the largest meta-epidemiological study on the impact of blinding in randomized trials to date. We examined MetaBLIND data on the impact of blinding patients on patient-reported outcomes. 68 out of 132 included trials tested knowledge recall and had questionable relevance to clinical trials. In 17 out of 18 comparisons, the number of trials in the blinded or nonblinded group was 2 or lower. In several key trials, the blinding status was uncertain. Effect sizes compared in MetaBLIND appear to reflect random differences in study design and setting rather than the impact of blinding trial participants.

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 SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials

BMJ Open ◽  
2021 ◽  
Vol 11 (6) ◽  
pp. e045105
Author(s):  
Melanie Calvert ◽  
Madeleine King ◽  
Rebecca Mercieca-Bebber ◽  
Olalekan Aiyegbusi ◽  
Derek Kyte ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
International Consensus ◽  
Research Waste ◽  
Patient Reported ◽  
Item Checklist ◽  
Trial Protocols ◽  
Economic Analyses ◽  
The Impact

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients’ symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item’s importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.

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