The role of patient-reported outcome measures in the continuum of cancer clinical care: ESMO Clinical Practice Guideline

Maïo, Massimo Di; Basch, Ethan; Denis, Fabrice; Fallowfield, Lesley; Ganz, Patricia A.; Howell, Doris; Kowalski, Christoph; Perrone, Francesco; Stover, Angela M.; Sundaresan, Puma; Warrington, Lorraine; Zhang, L.; Apostolidis, K.; Freeman-Daily, J.; Ripamonti, Carla; Santini, Daniele

doi:10.1016/j.annonc.2022.04.007

Cited by 231 publications

(131 citation statements)

References 106 publications

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“…Evaluating the level of clinical evidence of a solution is an important step for the acceleration of its use in the medical world and possible reimbursement by health authorities. In 2022, this evaluation was a major criterion for obtaining a favorable recommendation from the ESMO for use [ 6 ]. We noted a great heterogeneity in the clinical evaluation criteria of the solutions.…”

Section: Discussionmentioning

confidence: 99%

“…Our prescreening score was developed based on recent and updated recommendations. It is based, among other things, on the recommendations of the HAS guides [ 7 ], as well as the recommendations of the ESMO released in 2022 [ 6 ]. However, the values of the different parameters will evolve according to new standards, recommendations, and data from the literature.…”

Section: Discussionmentioning

confidence: 99%

“…The design of the MDS scoring tool was based on the 2021 HAS Solution Evaluation Criteria [ 7 ], the HAS Good Practices Framework on Solutions and Connected Objects in Health (eHealth or mobile Health) of 2016 [ 14 ], and the European Society of Medical Oncology (ESMO) recommendations of 2022 [ 6 ].…”

Section: Methodsmentioning

confidence: 99%

“…These prescreening tools must be based on good clinical practice guides and recommendations. Many standards and scoring methods already exist, and the first international recommendations for remote monitoring in oncology are now available [ 6 ].…”

Section: Introductionmentioning

confidence: 99%

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Assessing a New Prescreening Score for the Simplified Evaluation of the Clinical Quality and Relevance of eHealth Apps: Instrument Validation Study

Wagneur¹,

Callier²,

Zeitoun³

et al. 2022

J Med Internet Res

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Background In 2020, more than 250 eHealth solutions were added to app stores each day, or 90,000 in the year; however, the vast majority of these solutions have not undergone clinical validation, their quality is unknown, and the user does not know if they are effective and safe. We sought to develop a simple prescreening scoring method that would assess the quality and clinical relevance of each app. We designed this tool with 3 health care stakeholder groups in mind: eHealth solution designers seeking to evaluate a potential competitor or their own tool, investors considering a fundraising candidate, and a hospital clinician or IT department wishing to evaluate a current or potential eHealth solution. Objective We built and tested a novel prescreening scoring tool (the Medical Digital Solution scoring tool). The tool, which consists of 26 questions that enable the quick assessment and comparison of the clinical relevance and quality of eHealth apps, was tested on 68 eHealth solutions. Methods The Medical Digital Solution scoring tool is based on the 2021 evaluation criteria of the French National Health Authority, the 2022 European Society of Medical Oncology recommendations, and other provided scores. We built the scoring tool with patient association and eHealth experts and submitted it to eHealth app creators, who evaluated their apps via the web-based form in January 2022. After completing the evaluation criteria, their apps obtained an overall score and 4 categories of subscores. These criteria evaluated the type of solution and domain, the solution’s targeted population size, the level of clinical assessment, and information about the provider. Results In total, 68 eHealth solutions were evaluated with the scoring tool. Oncology apps (22%, 20/90) and general health solutions (23%, 21/90) were the most represented. Of the 68 apps, 32 (47%) were involved in remote monitoring by health professionals. Regarding clinical outcomes, 5% (9/169) of the apps assessed overall survival. Randomized studies had been conducted for 21% (23/110) of the apps to assess their benefit. Of the 68 providers, 38 (56%) declared the objective of obtaining reimbursement, and 7 (18%) out of the 38 solutions seeking reimbursement were assessed as having a high probability of reimbursement. The median global score was 11.2 (range 4.7-17.4) out of 20 and the distribution of the scores followed a normal distribution pattern (Shapiro-Wilk test: P =.33). Conclusions This multidomain prescreening scoring tool is simple, fast, and can be deployed on a large scale to initiate an assessment of the clinical relevance and quality of a clinical eHealth app. This simple tool can help a decision-maker determine which aspects of the app require further analysis and improvement.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Assessing a New Prescreening Score for the Simplified Evaluation of the Clinical Quality and Relevance of eHealth Apps: Instrument Validation Study

Wagneur¹,

Callier²,

Zeitoun³

et al. 2022

J Med Internet Res

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“…Surrogate measures are laboratory values or radiologic measurements used in place of direct clinical outcomes, such as how long a patient survives, feels or functions (quality of life (QoL), patient-reported outcomes (PROs)) [ 25 , 26 , 27 ]. The use of surrogate measures–such as progression-free survival (PFS), disease-free survival (DFS), objective response rate (ORR), minimal residual disease (MRD) and many others–in clinical trials of cancer medicines has been widespread [ 28 , 29 ].…”

Section: Challenges Relating To Clinical Trial Datamentioning

confidence: 99%

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

et al. 2022

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Background: Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment of health technology. This paper reviews the challenges in conducting HTA for cancer medicines arising from oncology trial designs and uncertainties of safety-efficacy data. Methods: Multiple databases (PubMed, Scopus and Google Scholar) and grey literature (public health agencies and governmental reports) were searched to inform this policy narrative review. Results: A lack of robust efficacy-safety data from clinical trials and other relevant sources of evidence has made HTA for cancer medicines challenging. The approval of cancer medicines through expedited pathways has increased in recent years, in which surrogate endpoints or biomarkers for patient selection have been widely used. Using these surrogate endpoints has created uncertainties in translating surrogate measures into patient-centric clinically (survival and quality of life) and economically (cost-effectiveness and budget impact) meaningful outcomes, with potential effects on diverting scarce health resources to low-value or detrimental interventions. Potential solutions include policy harmonization between regulatory and HTA authorities, commitment to generating robust post-marketing efficacy-safety data, managing uncertainties through risk-sharing agreements, and using value frameworks. Conclusion: A lack of robust efficacy-safety data is a central problem for conducting HTA of cancer medicines, potentially resulting in misinformed resource allocation.

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Validity of Patient-Reported Outcome Measures in Evaluating Nerve Damage Following Chemotherapy

Li,

Timmins,

Mahfouz

et al. 2024

JAMA Netw Open

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ImportanceChemotherapy-induced peripheral neuropathy (CIPN) is a substantial adverse effect of anticancer treatments. As such, the assessment of CIPN remains critically important in both research and clinic settings.ObjectiveTo compare the validity of various patient-reported outcome measures (PROMs) with neurophysiological and sensory functional measures as the optimal method of CIPN assessment.Design, Setting, and ParticipantsThis cohort study evaluated participants treated with neurotoxic chemotherapy across 2 cohorts using a dual-study design. Participants commencing treatment were assessed prospectively at beginning of neurotoxic treatment, midtreatment, and at the end of treatment. Participants who completed treatment up to 5 years prior were assessed cross-sectionally and completed a single assessment time point. Participants were recruited from oncology centers in Australia from August 2015 to November 2022. Data analysis occurred from February to November 2023.ExposuresNeurotoxic cancer treatment including taxanes, platinums, vinca-alkaloids, proteasome inhibitors, and thalidomide.Main Outcomes and MeasuresCIPN was assessed via PROMs (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire [EORTC-CIPN20], Functional Assessment of Cancer Therapy/Gynecological Cancer Group Neurotoxicity Questionnaire (FACT/GOG-Ntx), and the patient-reported outcomes version of the Common Terminology Criteria for Adverse Events [PRO-CTCAE]), neurological and neurophysiological assessment (Total Neuropathy Score and sural and tibial compound nerve amplitudes), and sensory measures (Grating orientation, Von Frey monofilament, and 2-point discrimination tasks). Core measurement properties of CIPN outcome measures were evaluated. Convergent and known-groups validity was assessed cross-sectionally following treatment completion, and responsiveness was evaluated prospectively during treatment. Neurological, neurophysiological, and sensory outcome measure scores were compared between those who reported high and low levels of CIPN symptoms using linear regressions.ResultsA total of 1033 participants (median [IQR] age, 61 [50-59] years; 676 female [65.4%]) were recruited to this study, incorporating 1623 assessments. PROMs demonstrated best ability to accurately assess CIPN (convergent validity), especially the PRO-CTCAE composite score (r = 0.85; P &lt; .001) and EORTC-CIPN20 (r = 0.79; P &lt; .001). PROMS also demonstrated the best ability to discriminate between CIPN severity (known-groups validity) and to detect changes at onset of CIPN development (responsiveness), especially for EORTC-CIPN20 (d = 0.67; 95% CI, 0.52-0.83), FACT/GOG-Ntx (d = 0.65; 95% CI, 0.49-0.81) and the PRO-CTCAE (d = 0.83; 95% CI, 0.64-1.02). Other measures did not achieve threshold for convergent validity (α &lt; 0.7). Neurophysiological and sensory measures did not demonstrate acceptable responsiveness. In regression models, neurological, neurophysiological, and sensory outcome measures were significantly impaired in participants who reported high levels of CIPN symptoms compared with those who reported low levels of CIPN symptoms.Conclusions and RelevanceIn this cohort study of 1033 cancer patients, PROMs were the only measures to satisfy all 3 core measurement property criteria (convergent validity, known-groups validity, and responsiveness). These findings suggest that adoption of PROMs in clinical practice can equip clinicians with valuable information in assessing CIPN morbidity.

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The role of patient-reported outcome measures in the continuum of cancer clinical care: ESMO Clinical Practice Guideline

Cited by 231 publications

References 106 publications

Assessing a New Prescreening Score for the Simplified Evaluation of the Clinical Quality and Relevance of eHealth Apps: Instrument Validation Study

Assessing a New Prescreening Score for the Simplified Evaluation of the Clinical Quality and Relevance of eHealth Apps: Instrument Validation Study

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

Validity of Patient-Reported Outcome Measures in Evaluating Nerve Damage Following Chemotherapy

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