Trends in patient‐reported outcome use in early phase dose‐finding oncology trials – an analysis of ClinicalTrials.gov

Lai-Kwon, Julia; Minchom, Anna; Yap, Christina

doi:10.1002/cam4.4307

Cited by 11 publications

(12 citation statements)

References 75 publications

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“…Lower COA use rates in early phase clinical trials reiterate previously reported concerns about phase 1 cancer trials, 15,26 and likely reflect challenges of incorporating COA instruments into small, non‐randomized trials 11 often assessing heterogeneous patient populations or multiple tumor types 27,28 . The importance of COAs in patient‐focused toxicity assessment 29 and the drive to include medical benefit outcomes in early phase cancer trials 30 call for incorporation of meaningful COA outcomes to supplement or strengthen early phase studies as advocated by groups including the NIH Office of Patient‐Centered Outcomes Research 31,32 .…”

Section: Discussionmentioning

confidence: 94%

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

et al. 2023

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BackgroundClinical outcome assessments (COAs) are key to patient‐centered evaluation of novel interventions and supportive care. COAs are particularly informative in oncology where a focus on how patients feel and function is paramount, but their incorporation in trial outcomes have lagged that of traditional survival and tumor responses. To understand the trends of COA use in oncology and the impact of landmark efforts to promote COA use, we computationally surveyed oncology clinical trials in ClinicalTrials.gov comparing them to the rest of the clinical research landscape.MethodsOncology trials were identified using medical subject heading neoplasm terms. Trials were searched for COA instrument names obtained from PROQOLID. Regression analyses assessed chronological and design‐related trends.ResultsEighteen percent of oncology interventional trials initiated 1985–2020 (N = 35,415) reported using one or more of 655 COA instruments. Eighty‐four percent of the COA‐using trials utilized patient‐reported outcomes, with other COA categories used in 4–27% of these trials. Likelihood of COA use increased with progressing trial phase (OR = 1.30, p < 0.001), randomization (OR = 2.32, p < 0.001), use of data monitoring committees (OR = 1.26, p < 0.001), study of non‐FDA‐regulated interventions (OR = 1.23, p = 0.001), and in supportive care versus treatment‐focused trials (OR = 2.94, p < 0.001). Twenty‐six percent of non‐oncology trials initiated 1985–2020 (N = 244,440) reported COA use; they had similar COA‐use predictive factors as oncology trials. COA use increased linearly over time (R = 0.98, p < 0.001), with significant increases following several individual regulatory events.ConclusionWhile COA use across clinical research has increased over time, there remains a need to further promote COA use particularly in early phase and treatment‐focused oncology trials.

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Section: Discussionmentioning

confidence: 94%

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

et al. 2023

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“…Of course, the selection of the most appropriate PRO assessment strategy should be guided by a specific research question and a combination of existing PRO validated questionnaires and ad‐hoc items is also a valid option. A trend of improvement over time regarding the inclusion of PROs in early‐phase cancer trials was recently observed, 71 and it is hoped this trend will continue considering the importance of the unique patient's perspective in the drug development process 72 . An advantage of including PRO measures in early‐phase trials could also be that of using preliminary results to better inform the PRO design of later phase trials.…”

Section: Discussionmentioning

confidence: 99%

Chimeric Antigen Receptor T-cell Therapy in Hematologic Malignancies and Patient-reported Outcomes: A Scoping Review

et al. 2022

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The inclusion of patient-reported outcome (PRO) measures in chimeric antigen receptor (CAR) T-cell therapy research is critical for understanding the impact of this novel approach from a unique patient standpoint. We performed a scoping review to map the available literature on the use of PRO measures in CAR T-cell therapy studies of patients with hematologic malignancies published between January 2015 and July 2022. Fourteen studies were identified, of which 7 (50%) were investigational early-phase trials, 6 (42.9%) were observational studies, and 1 (7.1%) was a pilot study. The EQ-5D and the PROMIS-29 were the 2 most frequently used PRO measures, being included in 6 (42.9%) and 5 (35.7%) studies, respectively. Despite differences in study designs, there seems to be evidence of improvements over time since CAR T-cell infusion in important domains such as physical functioning and fatigue, at least in patients who respond to therapy. Overall, the studies identified in our review have shown the added value of PRO assessment in CAR T-cell therapy research by providing novel information that complements the knowledge on safety and efficacy. However, there are several questions which remain to be answered in future research. For example, limited evidence exists regarding patient experience during important phases of the disease trajectory as only 4 (28.6%) and 5 (35.7%) studies provided information on PROs during the first 2 weeks from CAR T-cell infusion and after the first year, respectively. Time is ripe for a more systematic implementation of high-quality PRO assessment in future clinical trials and in real-life settings of patients treated with CAR T-cell therapy.

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“…The 35-question survey focused on prior experience in designing, conducting, and reporting DFOT involving a PRO endpoint, attitudes toward potential benefits and barriers to PRO use, and attitudes toward using PROs to define tolerable doses ( Supplementary Text ). Questions were informed by prior work examining trends in PRO use in DFOT 19 and other studies examining PRO use in DFOT. 20 , 21 The survey was hosted by a commercial survey tool provider (JISC Online Surveys) and used adaptive questioning ( Supplementary Fig.…”

Section: Methodsmentioning

confidence: 99%

“…We conducted a review of DFOT registered on ClinicalTrials.gov between 2007 and 2020. 19 PRO endpoints were identified in more studies (an increase of 2.3 studies/ year, 95% CI, 1.6-2.9) from an increasing variety of countries over time. However, overall use remained limited, with only 29 studies in 2019 including a PRO endpoint.…”

Section: Introductionmentioning

confidence: 99%

Using Patient-Reported Outcomes in Dose-Finding Oncology Trials: Surveys of Key Stakeholders and the National Cancer Research Institute Consumer Forum

et al. 2022

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Background Patient-reported adverse events may be a useful adjunct for assessing a drug’s tolerability in dose-finding oncology trials (DFOT). We conducted surveys of international stakeholders and the National Cancer Research Institute (NCRI) Consumer Forum to understand attitudes about patient-reported outcome (PRO) use in DFOT. Methods A 35-question survey of clinicians, trial managers, statisticians, funders, and regulators of DFOT was distributed via professional bodies examining experience using PROs, benefits/barriers, and their potential role in defining tolerable doses. An 8-question survey of the NCRI Consumer Forum explored similar themes. Results International survey: 112 responses from 15 September–30 November 2020; 103 trialists [48 clinicians (42.9%), 38 statisticians (34.0%), 17 trial managers (15.2%)], 7 regulators (6.3%), 2 funders (1.8%)]. Most trialists had no experience designing (73, 70.9%), conducting (52, 50.5%), or reporting (88, 85.4%) PROs in DFOT. Most agreed that PROs could identify new toxicities (75, 67.0%) and provide data on the frequency (86, 76.8%) and duration (81, 72.3%) of toxicities. The top 3 barriers were lack of guidance regarding PRO selection (73/103, 70.9%), missing PRO data (71/103, 68.9%), and overburdening staff (68/103, 66.0%). NCRI survey: 57 responses on 21 March 2021. A total of 28 (49.1%) were willing to spend <15 min/day completing PROs. Most (55, 96.5%) preferred to complete PROs online. 61 (54.5%) trialists and 57 (100%) consumers agreed that patient-reported adverse events should be used to inform dose-escalation decisions. Conclusion Stakeholders reported minimal experience using PROs in DFOT but broadly supported their use. Guidelines are needed to standardize PRO selection, analysis, and reporting in DFOT.

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Trends in patient‐reported outcome use in early phase dose‐finding oncology trials – an analysis of ClinicalTrials.gov

Cited by 11 publications

References 75 publications

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

Chimeric Antigen Receptor T-cell Therapy in Hematologic Malignancies and Patient-reported Outcomes: A Scoping Review

Using Patient-Reported Outcomes in Dose-Finding Oncology Trials: Surveys of Key Stakeholders and the National Cancer Research Institute Consumer Forum

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