Patient-reported outcome measures (PROMs) as proof of treatment efficacy

Kluzek, Stefan; Dean, Benjamin; Wartolowska, Karolina

doi:10.1136/bmjebm-2020-111573

Cited by 83 publications

(59 citation statements)

References 23 publications

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“…Combining PROMs with objective measures is crucial to insight into treatment effects and patients’ well-being. 41 , 42 …”

Section: Introductionmentioning

confidence: 99%

Patient-Reported Outcomes in Rheumatoid Arthritis: A Key Consideration for Evaluating Biosimilar Uptake?

Horta-Baas¹

2022

PROM

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Purpose This review aims to provide an overview of the impact of TNFis biosimilars, with marketing authorization, in patient-reported outcome measures (PROMs) scores and explore how PROMs endpoints might add value in biosimilars uptake in RA patients. Patients and Methods A comprehensive search of Medline, Scopus, Lilacs, and CINAHL databases was performed for papers published between January 2012 and December 2021. For inclusion, studies had to be prospective, published in a peer-reviewed journal, published in English or Spanish language; studies using PROMs as an outcome measure. After screening title and abstracts and assessing the remaining full texts fulfilling the inclusion criteria, 31 papers were used in this narrative review. Results PROMs were used as secondary outcomes in included studies. The most frequently employed domains to assess biosimilar efficacy include physical function, patient global assessment (PtGA), health-related quality of life (HRQoL), and fatigue. The results of randomized clinical trials uniformly showed that mean change in PROMs scores is comparable between biosimilar and reference biologic treatment groups. However, open-label and real-world studies revealed high rates of discontinuation of therapy, mainly for subjective worsening of disease activity or non-specific adverse events. Even without objective clinical evidence of inflammation, patients who are considered to have active disease (higher scores on PtGA) have higher discontinuation rates of biosimilars. The available information suggests that the nocebo effect is the most likely cause for the discontinuation of biosimilars. Conclusion There is scarce literature surrounding the impact of biosimilars in PROMs, especially in open-label studies. In real-life studies, biosimilars have a higher discontinuation rate than reference products. TNFis biosimilars treatment efficacy in RA depends on disease activity and other factors such as PtGA and fatigue. The nocebo effect is the best explanation for biosimilar’s discontinuation.

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“…Combining PROMs with objective measures is crucial to insight into treatment effects and patients’ well-being. 41 , 42 …”

Section: Introductionmentioning

confidence: 99%

Patient-Reported Outcomes in Rheumatoid Arthritis: A Key Consideration for Evaluating Biosimilar Uptake?

Horta-Baas¹

2022

PROM

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“…The 5-Dimensional European Quality of Life Scale (EQ-5D-5L) was already applied and validated as a patient-reported outcome measure (PROMs) in patients with stroke ( 4 ). The PROMs was recognized by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) as a measure of treatment efficacy ( 5 ). The EQ-5D-5L anxiety/depression dimension was used to analyze the patient-reported feeling of anxiety/depression in our study.…”

Section: Introductionmentioning

confidence: 99%

Patient-Reported Anxiety/Depression After Endovascular Thrombectomy: A post-hoc Analysis of Direct-MT Trial

et al. 2022

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ObjectiveTo analyze the effect of endovascular thrombectomy (EVT) alone vs. EVT after an intravenous (IV) alteplase of ischemic stroke on a patient-reported anxiety/depression, and to identify predictors of patient-reported anxiety/depression by analyzing data from Direct Intraarterial Thrombectomy in Order to Revascularize the patients with Acute Ischemic Stroke with a Large Vessel Occlusion Efficiently in Chinese Tertiary Hospitals: a Multicenter Randomized Clinical Trial (DIRECT-MT).MethodsPatients with acute ischemic stroke (AIS), triggered by a large-vessel occlusion in the anterior circulation, were randomly allocated to undergo an EVT after IV alteplase (combination-therapy group) or an EVT alone (EVT-alone group) at a 1:1 ratio in DIRECT-MT. Patients in both groups were followed up for 90 days (±14 days) after stroke using a structured modified Ranking Scale (mRS), a Barthel Index (BI), and a 5-Dimensional European Quality of Life Scale (EQ-5D-5L). Patients who returned EQ-5D-5L were included. The EQ-5D-5L anxiety/depression dimension was used to analyze the patient-reported anxiety/depression. First, differences in patient-reported anxiety/depression were compared between the combination-therapy group and the EVT-alone group. Then, the baseline and influencing factors between the anxiety/depression group and no anxiety/depression group were analyzed using univariate regression analysis. Finally, variables with p < 0.1 in univariate regression were subjected to multivariable binary regression analysis to screen independent predictors for patient-reported anxiety /depression after ischemic stroke.Results: Five hundred fifteen patients returned the EQ-5D-5L in Direct-MT. Of these patients, 226 (43.88%) reported a level of anxiety/depression, and about 7% reported a severe or extremely severe anxiety/depression. The patient-reported anxiety/depression in the EVT-alone group was significantly higher than that in the combination-therapy group (48.26% vs. 39.45%, p = 0.04). The clinical outcomes were significantly different between the no Anxiety/Depression Group and the anxiety/depression group (mRS at 90 days:2 vs 3, p < 0.001; BI of 95 or 100 at 90 days: 73.36% vs 42.04%, p < 0.001; EQ-5D-5l utility indexes at 90 days:0.96 vs.57, p < 0.001). Logistic regression analysis showed that allocation to thrombolysis before EVT strategy was inversely associated with anxiety/depression [0.61(0.40, 0.94), p = 0.03], an insular cortex ischemia, and National Institute of Health Strocke Scale (NIHSS) at 7 days were positively associated with anxiety/depression [2.04(1.07, 3.90), p = 0.03; 1.07(1.03, 1.12), p < 0.001].ConclusionsPatient-reported anxiety/depression may suggest that there is a benefit to administering intravenous alteplase before EVT. It may also indicate that it is better to provide IV alteplase before EVT, rather than EVT alone according to patient-reported anxiety/depression. Future research should consider not only the motor function impairments but also the patient-reported mental problems as measures of treatment efficacy in patients with stroke (DIRECT-MT ClinicalTrials.gov number, NCT03469206).

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“…Patient‐reported outcome measures (PROMs) and patient‐reported experience measures (PREMs) are increasingly acknowledged as providing valuable and essential information for assessing effectiveness of interventions, for shared decision making, and for achieving health system goals 1,2 3–5 .…”

Section: Introductionmentioning

confidence: 99%

“…Patient-reported outcome measures (PROMs) and patientreported experience measures (PREMs) are increasingly acknowledged as providing valuable and essential information for assessing effectiveness of interventions, for shared decision making, and for achieving health system goals. 1,2 The European Medicines Agency, the Food and Drug Administration, and the Institute of Medicine have recognized PROMs as measures of treatment efficacy. [3][4][5] Satisfaction with treatment is an important, patientreported construct which is distinct from quality of life and health status assessments and is associated with adherence to treatment, perception of health status and perception of effectiveness of interventions.…”

Section: Introductionmentioning

confidence: 99%

Validity and reliability of global ratings of satisfaction with epilepsy surgery

et al. 2022

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Objective We aimed to assess the reliability and validity of single‐item global ratings (GR) of satisfaction with epilepsy surgery. Methods We recruited 240 patients from four centers in Canada and Sweden who underwent epilepsy surgery ≥1 year earlier. Participants completed a validated questionnaire on satisfaction with epilepsy surgery (the ESSQ‐19), plus a single‐item GR of satisfaction with epilepsy surgery twice, 4–6 weeks apart. They also completed validated questionnaires on quality of life, depression, health state utilities, epilepsy severity and disability, medical treatment satisfaction and social desirability. Test‐retest reliability of the GR was assessed with the intra‐class correlation coefficient (ICC). Construct and criterion validity were examined with polyserial correlations between the GR measure of satisfaction and validated questionnaires and with the ESSQ‐19 summary score. Non‐parametric rank tests evaluated levels of satisfaction, and ROC analysis assessed the ability of GRs to distinguish among clinically different patient groups. Results Median age and time since surgery were 42 years (IQR 32–54) and 5 years (IQR 2–8), respectively. The GR demonstrated good to excellent test‐retest reliability (ICC = 0.76; 95% CI 0.67–0.84) and criterion validity (0.85; 95% CI 0.81–0.89), and moderate correlations in the expected direction with instruments assessing quality of life (0.59; 95% CI 0.51–0.63), health utilities (0.55; 95% CI 0.45–0.65), disability (−0.51; 95% CI −0.41, −0.61), depression (−0.48; 95% CI −0.38, −0.58), and epilepsy severity (−0.48; 95% CI −0.38, −0.58). As expected, correlations were lower for social desirability (0.40; 95% CI 0.28–0.52) and medical treatment satisfaction (0.33; 95% CI 0.21–0.45). The GR distinguished participants who were seizure‐free (AUC 0.75; 95% CI 0.67–0.82), depressed (AUC 0.75; 95% CI 0.67–0.83), and self‐rated as having more severe epilepsy (AUC 0.78; 95% CI 0.71–0.85) and being more disabled (AUC 0.82; 95% CI 0.74–0.90). Significance The GR of epilepsy surgery satisfaction showed good measurement properties, distinguished among clinically different patient groups, and appears well‐suited for use in clinical practice and research.

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Patient-reported outcome measures (PROMs) as proof of treatment efficacy

Cited by 83 publications

References 23 publications

Patient-Reported Outcomes in Rheumatoid Arthritis: A Key Consideration for Evaluating Biosimilar Uptake?

Patient-Reported Outcomes in Rheumatoid Arthritis: A Key Consideration for Evaluating Biosimilar Uptake?

Patient-Reported Anxiety/Depression After Endovascular Thrombectomy: A post-hoc Analysis of Direct-MT Trial

Validity and reliability of global ratings of satisfaction with epilepsy surgery

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