Pro32 Preference Weights for Quality-Adjusted Life-Years Estimation for Treatments of Paroxysmal Nocturnal Hemoglobinuria in Five Countries

Abstract

rash with sterile neutrophil-filled pustules. By understanding the burden of disease in this population, targeted interventions that improve patient quality of life can be developed. This study describes HCRU in patients with GPP. Methods: Patients were identified as having GPP if they had $1 inpatient or 2 outpatient ICD-10 L40.1 diagnosis codes, separated by 30 to 365 days. All analyses were conducted via the Aetion Evidence Platform TM v3.17, using Optum® Clinformatics TM Data Mart, a US administrative claims database. The study period was from October 1, 2015 to March 31, 2019, with the first diagnosis code marking the index date. A general population matched cohort (MC) of 4:1, based on age and sex, was generated for context. No formal comparisons were conducted. Patient characteristics, treatment, and all-cause HCRU calculated for each visit type (inpatient, outpatient, and emergency department [ED]) during the 12-month follow up (FU) were analyzed. Results: 1669 patients with GPP were identified at baseline, and 1014 had $12 months' FU. Compared with the MC, patients with GPP were more likely to suffer from psoriatic arthritis (GPP: 11.8% vs MC: 0.1%), anxiety (GPP: 11.6% vs MC: 5.8%), and depression (GPP: 10.7% vs MC: 5.0%) at baseline. During the 12-month FU, 449 (44.3%) were treated with a systemic therapy (biologic or non-biologic). Compared with the MC, patients with GPP had more outpatient visits (median: 21 vs 10), a greater frequency of inpatient (22.5% vs 10.4%) and ED (40.5% vs 23.0%) visits, and were hospitalized for longer (median: 8 vs 5 days) during the 12-month FU. Conclusions: This analysis suggests that patients with GPP have more HCRU than those in the MC, highlighting an unmet need among patients with GPP.

Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands

“…Study 301 and Study 302 could not show this improvement in QoL because, regardless of the infusion regimen, patients were required to attend the study center every two weeks [ 20 , 22 ]. Therefore, the QoL data for ravulizumab patients retrieved from Study 301 and Study 302 were corrected with a utility increment based on a DCE performed in the Netherlands [ 38 ]. Though the DCE used a scientific approach and has been accepted by NICE for demonstrating the effect of infusion frequency on patient QoL, the use of different sources to establish patient QoL is not ideal and may have impaired the reliability of the input data.…”

“…The design and results of the DCE survey in the Netherlands are summarized in Tables S7–S8. The utility increment of 0.070 found for the ravulizumab treatment arm was implemented in all health states of the ravulizumab treatment arm in the model [ 38 ]. The difference in utilities for the treatment arms based on the Studies 301 and 302 was assessed in a scenario analysis, referred to as model “With treatment indicator (tx)”.…”

“…Study 302 were corrected with a utility increment based on a DCE performed in the Netherlands [38]. Though the DCE used a scientific approach and has been accepted by NICE for demonstrating the effect of infusion frequency on patient QoL, the use of different sources to establish patient QoL is not ideal and may have impaired the reliability of the input data.…”