Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia

Abstract

Background Extended half‐life (EHL) factor VIII (FVIII) and IX (FIX) products are intended to decrease the burden of prophylaxis for patients with haemophilia A or B. Whether these newer concentrates have led to meaningful clinical practice change remains vague. Aim To characterize the longitudinal use of standard (SHL) and EHL factor concentrates at haemophilia treatment centres (HTCs), using the ATHNdataset, a US database of 138 ATHN‐affiliated HTCs. Methods Factor concentrate use among moderate and severe haemophilia A and B patients without inhibitors was analysed at three time points over 18 months. Results Use of EHL concentrates rose from 10% of patients to 22% during this study. EHL FVIII prophylaxis is prescribed to the minority of patients, 28%; EHL FIX now predominates for prophylaxis, 52%. Rates of prescribed EHL products varied significantly by age group and HTC region. Median prescribed prophylaxis for SHL compared to EHL products was FVIII 6240 and 5200 and FIX 6968 and FIX 3900 IU/kg/y, respectively. On‐demand EHL use has grown but has minimal contribution to overall usage (2%). Conclusion Haemophilia treatment centre region and patient age impact the rate of adoption of EHL products; however, EHL prescribing continues to rise nationally, particularly for EHL FIX. Careful attention to annual cost of prophylaxis is imperative as the decrease in median EHL prophylaxis consumption is not offset by the higher unit cost of these products. It is unclear how further growth in use of EHLs will be impacted by emerging non‐factor replacement and gene therapies.

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

“…Furthermore, the clinical management between treatment arms was not considered to be meaningfully different and therefore was not included in the model. We assume that individuals in the comparator arm use three weekly doses of exogenous FVIII protein at $65.33 per kg per dose (based on a median prescribed dose of 40 IU/kg cost of standard half-life FVIII prophylaxis, with a median wholesale acquisition cost [WAC] of $1.63 per IU) 9 . Both joint and nonjoint bleeds were assumed to be treated on-demand, with two doses of FVIII at the aforementioned cost of standard prophylaxis for a dose of 50 IU/kg of standard FVIII per bleed event 25 .…”

“…Chronic bleeds over time induce inflammation and joint degeneration, leading to chronic pain, limitations in mobility, and joint replacement surgery 6 . Consequently, patients with hemophilia A typically endure significant comorbidities, increased mortality, poorer health-related quality of life (HRQoL), and greater work-related impairment than the general population 7,8 . In the US, the current standard of care is prophylactically administered recombinant FVIII protein 1,9 . Compared to ondemand FVIII replacement, prophylactic administration has been associated with improved HRQoL in some patients; however, its clinical and economic burden of treatment is substantial 10 .…”

“…Compared to ondemand FVIII replacement, prophylactic administration has been associated with improved HRQoL in some patients; however, its clinical and economic burden of treatment is substantial 10 . Prophylactic treatments can require regular intravenous infusions, up to 2-3 times per week, and can cost hundreds of thousands of dollars per year 1,9,11 . Adherence to prophylactic therapy regimens can be challenging due to the frequency of infusions: one analysis of pooled trial data estimated that approximately 25% of patients do not follow frequency guidelines 10 .…”

“…The life-long management of hemophilia A places significant economic burden on patients, their families, and the health care system. In a recent analysis, the projected annual drug cost of prophylactic treatment in the US ranged from nearly $700,000 to $750,000 for standard and extended halflife FVIII products, respectively 9 . Separate studies have shown patients with hemophilia A incur higher costs due to greater rates of hospitalization, ER visits, office visits, medical procedures, and laboratory tests compared with the general population 7,14,15 .…”

“…Key cost estimates. Croteau et al9 Based on a median dose of 40 IU/kg, thrice weekly Cost per unit USD per kg per dose 65 33. Croteau et al9 Based on a median prescribed dose of 40 IU/kg cost of standard half-life FVIII prophylaxis, with a median WAC of $1.63…”