Cost to Treat Newly Diagnosed Patients with Paroxysmal Nocturnal Hemoglobinuria: A Retrospective Analysis

Background

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematologic disorder that renders affected patients’ red blood cells susceptible to premature destruction (i.e., hemolysis) and can lead to excess morbidity. Current pharmacological treatments for PNH include not only eculizumab, launched in 2007, but more recently approved treatments such as ravulizumab and pegcetacoplan. Even among patients treated with currently available therapies, suboptimal C5 inhibition or exacerbating factors such as infections may result in breakthrough hemolysis or other PNH-related complications. Given the evolving treatment landscape and challenges in treatment and diagnosis, it is unclear what the total and setting-specific costs and health care utilization are among patients newly diagnosed with PNH.

Objectives

To determine healthcare resource utilization and costs for newly diagnosed patients with PNH.

Methods

United States (US) adults with a new ICD-10 diagnosis of PNH (D59.5) from April 2016 to December 2020 identified in the OptumLabs claims data were included. The date of the first claim with a PNH diagnosis was defined as the index diagnosis date. Patients were required to have at least 6 months of continuous enrollment before the index diagnosis date to identify newly diagnosed patients and at least 6 months of continuous enrollment after the index diagnosis date for analysis. Comorbidity was measured with the National Cancer Institute (NCI) comorbidity index. All-cause healthcare resource utilization (HCRU), all-cause costs, PNH-related HCRU (defined as claims with an ICD-10 code of D59.5 in any position), and PNH-related costs were measured on a per-patient per-month (PPPM) basis for inpatient admissions, emergency department (ED) visits, office visits, and other outpatient visits for the 6-month follow-up period. The PPPM frequency of blood transfusions and utilization of bone marrow transplant were also assessed. The cost and HCRU results were age-weighted to be representative of the US population.

Results

Of the 749 patients with an ICD-10 diagnosis of PNH (D59.5) during the selected timeframe, 381 were newly diagnosed patients who met all inclusion criteria. In the unweighted sample, approximately half of the patients (50.9%) were female, with an average age of 61.3 (SD = 17.8), and the average NCI Comorbidity Index was 0.57 (SD = 0.65).

Of the 381 patients in the weighted sample, 149 (39.1%) had an inpatient admission. Of these inpatient admissions, 86 (57.7%) were PNH-related. For patients with at least one inpatient admission, the mean length of stay (LOS) was 2.2 days (SD = 3.0) and the median LOS was 1.0 days. One hundred twenty-three (32.3%) PNH patients had an emergency department (ED) visit in the first 6 months after the index diagnosis, 12 of which (9.8%) were PNH-related. Nearly all patients had office visits (97.9%) or other outpatient visits (96.6%) in the 6-month follow up period. Blood transfusions occurred in 18.6% of the PNH population, while bone marrow transplant was relatively rare (result suppressed due to small cell size). (Table 1)

The PPPM total cost for newly diagnosed patients was $10,480, or $125,758 per patient per year. Of these costs, 39.0% ($4,090 PPPM) were for pharmacy costs (including infusion costs), 33.9% ($3,551 PPPM) were for inpatient costs, 6.4% ($672 PPPM) were for office visits, 0.9% ($94 PPPM) were for ED visits, and 19.8% ($2,073 PPPM) were for other outpatient visits. The total PNH-related cost was $4,763 PPPM, accounting for 45% of the total cost. PNH-related pharmacy costs for eculizumab and ravulizumab, including administration, accounted for 60.2% ($2,868) of PNH-related costs (no pegcetacoplan use was observed since it was approved in 2021). PNH-related inpatient costs accounted for 28.0% ($1,333), office visits accounted for 3.6% ($172), and other outpatient visits accounted for 8.1% ($386) of total PNH-related costs. ED visits were rarely flagged with the PNH ICD-10 code, with less than 0.1% of PNH-related costs coming from the ED. (Figure 1)

Conclusions

Despite new treatment options available for PNH, HCRU and associated costs remain high with 39.1% of PNH patients having an inpatient visit in the first 6 months after diagnosis. Nearly one in five newly diagnosed PNH patients still require at least one blood transfusion in the follow-up period, signaling further unmet medical need in PNH.

Waheed:Novartis: Consultancy. Clayton:FTI Consulting: Current Employment. Shafrin:FTI Consulting: Current Employment. Li:Novartis: Current Employment, Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Shi:Novartis: Consultancy. Geevarghese:Novartis: Current Employment. Yen:Novartis: Current Employment, Current holder of stock options in a privately-held company.