Case study | HAS appraisal | NICE appraisal | IQWiG and G-BA appraisals |
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Treatment: DUPIXENT (dupilumab) Indication: atopic dermatitis Instrument: DLQI | Comparative study between treatment (n = 107) and placebo (n = 108) groups. Significant impact on the DLQI was demonstrated in the trial and was used as secondary judgement criteria by CT | NICE assessed similar data as that reviewed by HAS. The committee agreed that the product improved both efficacy and quality of life as measured by DLQI at week 16 | IQWiG and G-BA also assessed the CHRONOS study (see HAS column). G-BA determined a considerable added benefit for dupilumab, based on significant improvements in morbidity (itch, sleep, eczema severity) and QoL (DLQI) |
Treatment: TEGSEDI (inotersen) Indication: polyneuropathy Instrument: NORFOLK QoL-DN (questionnaire) and SF-36 (questionnaire) | Comparative study between treatment (n = 113) and placebo (n = 60) groups. Modest impact on the NORFOLK QoL-DN was demonstrated in the trial and was used as secondary judgement criteria by CT | NICE reviewed the data from the NEURO-TTR study. A statistically significant difference in favour of inotersen was seen on the mNIS + 7 but not on the Norfolk QoL-DN score The committee concluded that the evidence showed that inotersen had considerable benefit in slowing disease progression, but it did not stop progression | IQWiG and G-BA assessed the NEURO-TTR study and found a non-quantifiable added benefit for inotersen. This was driven by the functional domain of the SF-36. While the overall results on NORFOLK QoL-DN and SF36 were statistically significant, G-BA did not find the magnitude of the effect clinically relevant. No benefits were found on mortality and morbidity |
Treatment: SCENESSE (afamelanotide) Indication: erythropoietic protoporphyria Instrument: DLQI and EPP-QOL | RWE data collection study following 117 patients over 2 years and evaluating the change in QoL over time. The EPP-QOL score had a mean QoL was significantly better in the treatment group. CT used these RWE findings | NICE considered the data emanating from the EPP-QoL questionnaire, as well as from the DLQI questionnaire, but noted some serious reservations about the collection method The committee concluded that the there was no marked improvement in the quality of life of patients who had treatment beyond the duration of the controlled clinical trials | IQWiG and G-BA found a non-quantifiable added benefit based on the “time spent in sunlight” as a morbidity end-point from the CUV039. G-BA reviewed the QoL data but concluded that the instruments were not validated in this disease and criticized that different versions of the instrument were used in the study |