Is IQWiG's 15% Threshold Universally Applicable in Assessing the Clinical Relevance of Patient-Reported Outcomes Changes? An ISPOR Special Interest Group Report

Schlichting, Michael; Hennig, Michael; Rüdell, Katja; McLeod, Lori; Bennett, Bryan; Shaw, James W.; Doward, Lynda; Molsen-David, Elizabeth; Chassany, Olivier

doi:10.1016/j.jval.2022.07.010

Cited by 4 publications

(4 citation statements)

References 10 publications

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“…Since there were very few established minimal clinically important thresholds in the published literature for these PROs, a 15% change in the range of scores was used based on the recent Institute for Quality and Efficiency in Health Care guidance. 23 For the PROs with a 100point range in scores, the minimal clinically important threshold was 15 points. For those PROs where higher scores reflect better HRQoL, the threshold was based on a ≥15% increase in scores.…”

Section: Discussionmentioning

confidence: 99%

“…30 Given that PwHB may benefit from EDZ in different ways, focusing While a change of 15-points or more on a 100-point scale is considered by the Institute for Quality and Efficiency in Health Care to be a plausible threshold for a minimal clinically important improvement, there is currently no widespread consensus. 23 Taking this approach allows us to apply the same threshold for minimal clinically important difference across all of the PRO instruments assessed in the HOPE-B study (Table S3). It is worthwhile to note that this is a conservative threshold relative to minimal clinically important thresholds proposed for Hem-A-QoL outcomes in other publications.…”

Section: Discussionmentioning

confidence: 99%

“…While a change of 15‐points or more on a 100‐point scale is considered by the Institute for Quality and Efficiency in Health Care to be a plausible threshold for a minimal clinically important improvement, there is currently no widespread consensus 23 . Taking this approach allows us to apply the same threshold for minimal clinically important difference across all of the PRO instruments assessed in the HOPE‐B study (Table S3).…”

Section: Discussionmentioning

confidence: 99%

“…Responder analyses were performed to assess the percentage of participants achieving minimal clinically important improvements, with exact Clopper Pearson 95% confidence intervals (CI) provided. Since there were very few established minimal clinically important thresholds in the published literature for these PROs, a 15% change in the range of scores was used based on the recent Institute for Quality and Efficiency in Health Care guidance 23 . For the PROs with a 100‐point range in scores, the minimal clinically important threshold was 15 points.…”

Section: Methodsmentioning

confidence: 99%

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Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Itzler,

Buckner,

Leebeek

et al. 2024

Haemophilia

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IntroductionFor people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health‐related quality of life (HRQoL). The pivotal phase 3 HOPE‐B trial investigating the adeno‐associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB with baseline FIX levels ≤2%.AimAssess how EDZ affects HRQoL in HOPE‐B trial participants.MethodsHRQoL was evaluated using generic and disease‐specific patient reported outcomes (PROs) including the EQ‐5D‐5L and the Hem‐A‐QoL questionnaires. Mean domain and total scores were compared 6 months pre‐ and the first 2 years post‐EDZ administration using repeated measures linear mixed models. The percentage of participants with minimal clinically important improvements in HRQoL was also evaluated.ResultsTwo years post‐EDZ, there were nominally significant increases in the least squares (LS) mean score for the EQ‐5D‐5L Index Value (.04; p = .0129), reflecting better HRQoL. Nominally significant decreases in the LS mean scores, reflecting better HRQoL, were also found for the Hem‐A‐QoL total score (−6.0; p < .0001) and the Treatment (−13.94; p < .0001), Feelings (−9.01; p < .0001), Future (−6.45; p = .0004) and Work/School (−5.21; p = .0098) domains. The percentage of participants with ≥15‐point improvement ranged from 45.83% (95% CI: 31.37%, 60.83%) for Treatment to 13.89% (95% CI: 4.67%, 29.50%) for Family Planning. Results were similar for Year 1.ConclusionIn conclusion, gene therapy with EDZ improved HRQoL in the first and second years in several Hem‐A‐QoL domains, including Treatment, Feelings, Work/School and Future domains, whereas improvement in other aspects of HRQoL were not demonstrated.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

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Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Itzler,

Buckner,

Leebeek

et al. 2024

Haemophilia

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Establishing meaningful change thresholds for European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire CLL‐specific module domain scores: An analysis based on the TRANSCEND CLL 004 study in patients with relapsed or refractory chronic lymphocytic leukaemia or small lymphocytic lymphoma

Eliason,

Fofana,

Wang

et al. 2024

eJHaem

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IntroductionThe study aimed to establish meaningful thresholds at patient and group levels for the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire CLL‐specific module (EORTC QLQ‐CLL17) domain scores in adults with relapsed or refractory (R/R) chronic lymphocytic leukaemia (CLL).Material and methodsData for the analysis were from the TRANSCEND CLL 004 study (NCT03331198). EORTC QLQ‐CLL17 and selected anchor measures were assessed at baseline and multiple postbaseline visits up to 24 months after treatment initiation. Thresholds for each of the three EORTC QLQ‐CLL17 domains were triangulated based on estimates derived from anchor‐ and distribution‐based analyses, in accordance with published guidance.ResultsThe analysis included 62 patients with 240 observations across visits. Meaningful change thresholds for improvement and deterioration, respectively, at the patient level were determined to be −11/+11 for symptom burden, −16/+16 for physical condition/fatigue and −16/+13 for worries/fears on health and functioning. The meaningful change thresholds for improvement and deterioration at the group level mostly ranged between 0.3 and 0.5 of the standard deviation of baseline domain scores.ConclusionsThese thresholds, based on EORTC QLQ‐CLL17 domain scores, could help identify patients with meaningful changes in HRQOL and interpret treatment effects in future studies of treatments for adults with R/R CLL.

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Cardiovascular safety of the class of JAK inhibitors or tocilizumab compared with TNF inhibitors in patients with rheumatoid arthritis: Systematic review and a traditional and Bayesian network meta-analysis of randomized clinical trials

Pugliesi,

Oliveira,

Filho

et al. 2024

Seminars in Arthritis and Rheumatism

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Is IQWiG's 15% Threshold Universally Applicable in Assessing the Clinical Relevance of Patient-Reported Outcomes Changes? An ISPOR Special Interest Group Report

Cited by 4 publications

References 10 publications

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Cardiovascular safety of the class of JAK inhibitors or tocilizumab compared with TNF inhibitors in patients with rheumatoid arthritis: Systematic review and a traditional and Bayesian network meta-analysis of randomized clinical trials

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