Patient-reported outcomes in PD-1/PD-L1 inhibitor registration trials: FDA analysis of data submitted and future directions.

2018 ◽  
Vol 36 (5_suppl) ◽  
pp. 134-134 ◽  
Author(s):  
Lynn Jackson Howie ◽  
Harpreet Singh ◽  
Bellinda King-Kallimanis ◽  
Jessica Roydhouse ◽  
Marc Robert Theoret ◽  
...  
Keyword(s):  
Physical Function ◽  
Patient Experience ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Symptom Scale ◽  
Open Label ◽  
Benefit Risk Assessment ◽  
Patient Reported ◽  
Lung Cancer Symptom Scale ◽  
Eortc Qlq

134 Background: Patient-reported outcome measures (PROs) can capture the patient’s experience with disease and treatment. Anti-PD-1/PD-L1 therapies have unique symptomatic side effects; PRO data can help to better understand the patient experience on therapy. Health-related quality of life (HRQL) components most impacted by therapy include disease symptoms, symptomatic toxicity and physical function. Methods: We reviewed FDA registration trials for 5 immunotherapy agents (anti-PD-1/PD-L1) to evaluate trial design and PRO assessment. We assessed whether the PRO strategy assessed physical function and symptomatic immune-related adverse events (irAEs) by reviewing whether trials used a well-defined physical function domain and 8 symptoms related to irAEs reported in product labels (fatigue, diarrhea, cough, shortness of breath, musculoskeletal pain, rash, pruritis and fever). Results: Data from 25 trials across 7 disease types and 1 tumor agnostic indication were evaluated. Of these, 13 were randomized and 22 were open label. Eighteen of 25 contained PRO assessments and all 18 used > 1 instrument. The most common instruments were the EQ-5D (N = 17), followed by EORTC QLQ-C30 (N = 15). Disease-specific PRO tools were included in 8 trials (5 lung, 1 head and neck, 1 melanoma and 1 renal cell), consisting of modules or scales from EORTC (N = 5), FACIT (N = 2) or the Lung Cancer Symptom Scale (N = 1). Sixty percent of the trials (15/25) used an instrument that contained a well-defined physical function (PF) domain. No trial used a PRO strategy assessing all 8 selected symptoms related to irAEs. Conclusions: Collection of PRO data in anti-PD-1/PD-L1 trials submitted to FDA was variable, and did not consistently assess treatment related symptoms and physical function. Use of a HRQL tool with well-defined functional scales supplemented by item banks or libraries to incorporate symptoms associated with irAEs may improve understanding of the patient experience while receiving anti-PD-1/PD-L1 treatment. These data, along with other important clinical data such as hospitalizations, ER visits and supportive care medications can inform the benefit risk assessment for regulatory purposes.

Patient-Reported Outcome Measures in Colorectal Surgery: Construction of Core Measures Using Open-Source Research Method

2021 ◽  
pp. 155335062199887
Author(s):  
Alaa El-Hussuna ◽  
Ines Rubio-Perez ◽  
Monica Millan ◽  
Gianluca Pellino ◽  
Ionut Negoi ◽  
...  
Keyword(s):  
Colorectal Surgery ◽  
Outcome Measures ◽  
Short Form ◽  
Wide Spectrum ◽  
Core Outcome Set ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Eortc Qlq

Purpose. The primary aim of the study was to review the existing literature about patient-reported outcome measures (PROMs) in colorectal cancer and IBD. The secondary aim was to present a road map to develop a core outcome set via opinion gathering using social media. Method. This study is the first step of a three-step project aimed at constructing simple, applicable PROMs in colorectal surgery. This article was written in a collaborative manner with authors invited both through Twitter via the #OpenSourceResearch hashtag. The 5 most used PROMs were presented and discussed as slides/images on Twitter. Inputs from a wide spectrum of participants including researchers, surgeons, physicians, nurses, patients, and patients’ organizations were collected and analyzed. The final draft was emailed to all contributors and 6 patients’ representatives for proofreading and approval. Results. Five PROM sets were identified and discussed: EORTC QLQ-CR29, IBDQ short health questionnaire, EORTC QLQ-C30, ED-Q5-5L, and Short Form-36. There were 315 tweets posted by 50 tweeters with 1458 retweets. Awareness about PROMs was generally limited. The general psycho-physical well-being score (GPP) was suggested and discussed, and then a survey was conducted in which more than 2/3 of voters agreed that GPP covers the most important aspects in PROMs. Conclusion. Despite the limitations of this exploratory study, it offered a new method to conduct clinical research with opportunity to engage patients. The general psycho-physical well-being score suggested as simple, applicable PROMs to be eventually combined procedure-specific, disease-specific, or symptom-specific PROMs if needed.

scholarly journals Implementation of patient-reported outcome measures and patient-reported experience measures in melanoma clinical quality registries: a systematic review

BMJ Open ◽  
2021 ◽  
Vol 11 (2) ◽  
pp. e040751
Author(s):  
Zachary Blood ◽  
Anh Tran ◽  
Lauren Caleo ◽  
Robyn Saw ◽  
Mbathio Dieng ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cost Effectiveness ◽  
Outcome Measures ◽  
Cutaneous Melanoma ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Secondary Outcome ◽  
Clinical Quality ◽  
Patient Reported ◽  
Eortc Qlq

ObjectivesTo identify patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) in clinical quality registries, for people with cutaneous melanoma, to inform a new Australian Melanoma Clinical Outcomes Registry; and describe opportunities and challenges of routine PROM/PREM collection, especially in primary care.DesignSystematic review.Primary and secondary outcome measuresWhich PROMs and PREMs are used in clinical quality registries for people with cutaneous melanoma, how they are collected, frequency of collection, participant recruitment methods and funding models for each registry.Results1134 studies were identified from MEDLINE, PreMEDLINE, Embase, PsychInfo, Cochrane Database of Abstracts of Reviews of Effects databases and TUFTS Cost-Effectiveness Analysis Registry, alongside grey literature, from database inception to 5th February 2020. Following screening, 14 studies were included, identifying four relevant registries: Dutch Melanoma Registry, Adelphi Real-World Disease-Specific Programme (Melanoma), Patient-Reported Outcomes Following Initial treatment and Long-term Evaluation of Survivorship Registry, and Cancer Experience Registry. These used seven PROMs: EuroQol-5 Dimensions, Functional Assessment of Cancer-General (FACT-G) and FACT-Melanoma (FACT-M), European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Cancer 30 (EORTC QLQ-C30), Fatigue Assessment Scale Hospital Anxiety and Depression Scale, Patient-Reported Outcome Measures Information System-29 and one PREM; EORTC QLQ-Information Module 26. PROMs/PREMs in registries were reported to improve transparency of care; facilitate clinical auditing for quality assessment; enable cost-effectiveness analyses and create large-scale research platforms. Challenges included resource burden for data entry and potential collection bias toward younger, more affluent respondents. Feedback from patients with melanoma highlighted the relevance of PROMs/PREMs in assessing patient outcomes and patient experiences.ConclusionsClinical registries indicate PROMs/PREMs for melanoma care can be incorporated and address important gaps, however cost and collection bias may limit generalisability.PROSPERO registration numberCRD42018086737.

scholarly journals Patient-Reported Outcome Measures for Post-mastectomy Breast Reconstruction: A Systematic Review of Development and Measurement Properties

2020 ◽  
Vol 28 (1) ◽  
pp. 386-404 ◽  
Author(s):  
C. F. Davies ◽  
R. Macefield ◽  
K. Avery ◽  
J. M. Blazeby ◽  
S. Potter
Keyword(s):  
Systematic Review ◽  
Breast Reconstruction ◽  
Content Validity ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
Future Studies ◽  
Patient Reported ◽  
Eortc Qlq

Abstract Background Breast reconstruction (BR) is performed to improve outcomes for patients undergoing mastectomy. A recently developed core outcome set for BR includes six patient-reported outcomes that should be measured and reported in all future studies. It is vital that any instrument used to measure these outcomes as part of a core measurement set be robustly developed and validated so data are reliable and accurate. The aim of this systematic review is to evaluate the development and measurement properties of existing BR patient-reported outcome measures (PROMs) to inform instrument selection for future studies. Methods A PRISMA-compliant systematic review of development and validation studies of BR PROMs was conducted to assess their measurement properties. PROMs with adequate content validity were assessed using three steps: (1) the methodological quality of each identified study was assessed using the COSMIN Risk of Bias checklist; (2) criteria were applied for assessing good measurement properties; and (3) evidence was summarized and the quality of evidence assessed using a modified GRADE approach. Results Fourteen articles reported the development and measurement properties of six PROMs. Of these, only three (BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23) were considered to have adequate content validity and proceeded to full evaluation. This showed that all three PROMs had been robustly developed and validated and demonstrated adequate quality. Conclusions BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23 have been well-developed and demonstrate adequate measurement properties. Work with key stakeholders is now needed to generate consensus regarding which PROM should be recommended for inclusion in a core measurement set.

Does physical function impact the acceptability of routine patient-reported outcome surveys in cancer patients?

2016 ◽  
Vol 34 (7_suppl) ◽  
pp. 67-67
Author(s):  
Lauren Wong ◽  
Catherine Brown ◽  
Andrea Perez-Cosio ◽  
Yvonne Leung ◽  
Mindy Liang ◽  
...  
Keyword(s):  
Physical Function ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Clinic Visit ◽  
Routine Screening ◽  
Limiting Factors ◽  
Related Symptom ◽  
Patient Reported ◽  
Routine Basis ◽  
Caucasian Patients

67 Background: Patient-reported outcome measures (PROMs) are used to collect cancer-related symptom and symptomatic toxicity data. Understanding factors that affect the acceptability of PROMs is useful for improving the participation of such routine screening practices. We investigated the association between patient’s functional status on their acceptance and perception of usefulness of tablet technology to routine collection of PROM data in the clinical setting. Methods: 279 adult cancer outpatients at Toronto’s Princess Margaret Cancer Centre completed two physical function surveys, the HAQ-DI and WHODAS, using touchscreen tablet technology. Patient socio-demographics and their willingness of completing PROMs on a routine basis were also collected. Results: The median age was 62 (range: 20–94); 50% male, and 68% Caucasian. Patients were recruited from breast (10.4%), gastrointestinal (10.4%), genitourinary (14%), gynecology (14.3%), head & neck (17.9%), hematology (17.6%), and thoracic (15.4%) cancer clinics. 87% were willing to complete the survey on a touchscreen tablet, 74% did not find it time-consuming, and 4% found completion of the survey made their clinic visit more difficult. Only 1% found the questions upsetting or distressful; however, less than 60% were willing to complete similar surveys at every visit. Being Caucasian (p = 0.015) and Canadian-born (p = 0.021) were significantly associated with acceptability of the survey on a regular basis. However, HAQ-DI and WHODAS are not significantly associated with willingness to complete PROMs regularly (p = 0.30 & p = 0.16 respectively). Conclusions: Physical function status is not associated with acceptability; however, ethnicity does play a role where Canadian-born and Caucasian patients are more likely to complete PROMs regularly. Despite this, over 40% of patients were not willing to fill in the PROMs at each visit. Socio-cultural factors may impact on patients’ perceptions of the value of routine screening; however, assessment burden may also limit willingness to complete surveys on an ongoing basis. Further research is underway to gain more insight into the limiting factors of patients to complete the survey routinely.

Patient experience with patient-reported outcome measures in neurologic practice

Neurology ◽  
2018 ◽  
Vol 91 (12) ◽  
pp. e1135-e1151 ◽  
Author(s):  
Brittany Lapin ◽  
Belinda Udeh ◽  
Jocelyn F. Bautista ◽  
Irene L. Katzan
Keyword(s):  
Outcome Measures ◽  
Patient Experience ◽  
Patient Characteristics ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Successful Implementation ◽  
Patient Centered ◽  
Care Clinic ◽  
Patient Reported ◽  
And Control

ObjectiveTo quantify the neurologic patient experience with patient-reported outcome measures (PROMs) and identify factors associated with a positive PROMs experience.MethodsThis retrospective study included all patients seen in 6 neurologic clinics who completed patient experience questions at least once between October 2015 and September 2016. Questions assessed overall satisfaction with PROMs, as well as 4 facets of the PROM experience: usefulness of questions, ease of understanding, effect on communication with provider, and effect on control of their own care. Clinic and patient characteristics were summarized across questions and predictors of response were identified using multivariable proportional odds models.ResultsA total of 16,157 patients answered generic and condition-specific PROMs, as well as questions on their experience with completing PROMs. The majority of patients agreed/strongly agreed questions were easy to understand (96%), useful (83%), and improved communication (78%) and control (71%). After adjustment for other factors, being younger, black, or depressed, or having lower household income, were independent predictors of high satisfaction with PROMs. Patients who indicated the system improved communication and control of care were more often male, black, and lower income. Variability in responses was shown by clinic.ConclusionGiven the growing importance of patient satisfaction in health care, the patient experience with PROMs is a critical component of their successful implementation and utilization. Findings from this study support the feasibility of collecting PROMs in neurologic practice and the potential as a tool to optimize patient-centered neurologic care.

scholarly journals Sex differences in postural orientation errors and association with objective and patient-reported function in patients with ACL injury: an exploratory cross-sectional study

2021 ◽  
Vol 7 (2) ◽  
pp. e001045
Author(s):  
Jenny Nae ◽  
Mark W Creaby ◽  
Anna Cronström ◽  
Eva Ageberg
Keyword(s):  
Sex Differences ◽  
Physical Function ◽  
Cruciate Ligament ◽  
Visual Assessment ◽  
Patient Reported Outcome Measures ◽  
Cross Sectional Study ◽  
Patient Reported Outcome ◽  
Cross Sectional ◽  
Patient Reported ◽  
Postural Orientation

ObjectivesThere is limited research on sex differences in postural orientation (ie, alignment between body segments) in people with knee injury measured with a clinically applicable method. An understanding of the relationship between postural orientation and physical function may help guide decision making in rehabilitation. The aims were to evaluate (1) sex differences in visual assessment of Postural Orientation Errors (POEs) and (2) the association between POEs and objective and patient-reported physical function, in men and women with anterior cruciate ligament reconstruction (ACLR).MethodsTwenty-four women and 29 men (mean 26.7 (SD 6.5) years) with ACLR were included. Six POEs (lower extremity and trunk) were scored from a video of five tasks with varying difficulty to compute POE scores (total and subscores). Objective physical function was evaluated with the single-leg hop for distance and side hop. Patient-reported physical function was evaluated using patient-reported outcome measures (PROMs).ResultsWomen had significantly more POEs than men (median difference 5.5–25, p≤0.028). More POEs were associated with shorter hop distance and fewer side hops in women (rs= −0.425 to −0.518, p<0.038), but not in men (rs<0.301, p>0.05). No associations were found between POE scores and PROMs, in either sex (rs< –0.246, p>0.05).ConclusionsWomen with ACLR seem to have more POEs compared with men, indicating worse postural orientation. More POEs were associated with worse hop performance, suggesting that POE scores may be used as criteria for rehabilitation progression. The lack of associations between POE scores and PROMs indicate that these measures complement each other.

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