322.

DISORDERS OF COAGULATION OR FIBRINOLYSIS: POSTER II | DECEMBER 07, 2017

Real-World Expenditures Associated with Treatment

of Hemophilia a and B Patients with Standard and
Extended Half-Life Factor Replacement Products in the US
*,1 *,1 *,2 *,3
Amit Chhabra, MBBS, MPH, José Alvir, DrPH, Dean Spurden, PhD, Patrick F. Fogarty, MD,
4 *,5 *,6
Bartholomew Tortella, MDMBA, Margaret McDonald, PhD, Andreas M Pleil, PhD
1
Pfizer Inc, New York, NY
2
Pfizer Limited, Surrey, United Kingdom
3
Pfizer Inc, Collegeville, PA
4
Pfizer, Inc., Collegeville, PA
5
Pfizer Inc, New York, NJ
6
Pfizer Inc, San Diego, CA

Blood blood (2017) 130 (Suppl_1) : 2351.

http://doi.org/10.1182/blood.V130.Suppl_1.2351.2351

Abstract
Introduction:

Management of Hemophilia A and B require intravenous factor VIII (FVIII) and factor IX (FIX) infusions
respectively to replenish missing coagulation factor. We compared real world expenditures in the US for
clotting factor concentrates associated with treatment of Hemophilia A and B patients with EHL products
versus recombinant standard half-life (SHL) products.

Methods:

De-identified claims data from the Truven Health MarketScan® Research US claims databases were
used to identify male patients with either hemophilia A who received FVIII replacement from August, 2014
(month first EHL FVIII product dispensed) to April 2017; or hemophilia B who received FIX replacement
from June 2014 (month first EHL FIX product dispensed) to April 2017 and had data for at least 3 months
of dispensation. The two groups of patients (SHL vs EHL) were compared. Key outcome measures were
direct expenditures and factor IUs dispensed for factor replacement products. Expenditures and IUs
dispensed were measured over quarterly (3-month) increments for 10 quarters in each group (SHL &
EHL). Descriptive statistics were used to analyze results. Medians for expenditures and IUs were used to
accommodate for the skewness of data distribution.

Results:

Hemophilia A

538 Hemophilia A patients were included in the analysis (437 SHL; 104 EHL), including patients who
switched from one class to the other. Quarterly expenditures and IUs dispensed were analyzed for these
patients up to 30 months.

The median quarterly study period factor per patient expenditure was $72,466 higher (2.15 times) in the
EHL cohort ($135,392; IQR: $101,690-$189,501) compared with the SHL cohort ($62,927; IQR: $19,448-
$121,192). Median quarterly study period per patient IU dispensation was also higher in the EHL cohort
(68,736 IUs; IQR: 49,687-100,883) vs SHL users (48,473 IUs; IQR: 13,396-91,102).

Median total Hemophilia A related expenditures were $137,210 and $66,713 in the EHL and SHL cohorts
indicating that the total expenditures were chiefly due to factor replacement.

In the switch analysis, 35 patients had switched from one of three SHL FVIII products to one of two EHL
FVIII products. The total median IU dispensation per calendar quarter increased following the switch
from 60,396 IU (pre-switch, SHL) to 78,295 IU (post-switch, EHL; 30% increase), as did the factor-related
expenditure ($78,945, SHL, versus $160,008, EHL; 103% increase).

Hemophilia B

104 Hemophilia B patients that had at least 3 months data were included in the analysis (79 SHL; 25
EHL). Quarterly expenditures and IUs dispensed were analyzed for these patients over up to 30 months.

The median quarterly study period factor per patient expenditure was $106,441 higher (2.8 times) in the
EHL cohort ($165,022; IQR: $97,444-$260,839) compared with the SHL cohort ($58,580; IQR: $26,864
- $113,959). Median quarterly study period per patient metric IU dispensation was also higher in the EHL
cohort (68,541 IUs; IQR: 33,941-97,098) vs SHL users (39,180 IUs; IQR: 19,000-87,550).

Median total Hemophilia B related expenditures were $165,188 and $60,713 in the EHL and SHL cohorts
indicating that the total expenditures were chiefly due to factor replacement.

In the switch analysis, 15 patients with hemophilia B switched from SHL to EHL. Median quarterly per-
patient factor expenditure for up to 30 months pre/post-switch was $73,862 (IQR: $54,717-$103,798)
in the period pre-switch, and $209,795 (IQR: $124,418-$330,650) after the switch. Median quarterly
per-patient metric IUs utilization pre-switch was 62,325 (IQR: 41,223-88,396) and 68,541 (IQR:
26,345-113,144) IUs post-switch.

Conclusion:

This real-world data analysis, unadjusted for treatment regimen or disease severity, showed a higher
expenditure associated with EHL use in both Hemophilia A and Hemophilia B treatment in the US.
Median quarterly study period per patient IU dispensation was also higher in the EHL cohort for both
Hemophilia A and B. These real-world data may challenge assumptions regarding typical factor usage
and expenditures associated with EHL products in patients with hemophilia A and B. Further analyses,
incorporating essential clinical characteristics, should be explored.
Disclosures
Chhabra: Pfizer Inc: Employment, Other: Stockholder. Alvir: Pfizer Inc: Employment, Other: Stockholder.
Spurden: Pfizer Limited: Employment, Other: Stockholder. Fogarty: Pfizer Inc: Employment, Other:
Stockholder. Tortella: Pfizer Inc: Employment, Other: Stockholder. McDonald: Pfizer Inc: Employment,
Other: Stockholder. Pleil: Pfizer: Employment.

Author notes
*Asterisk with author names denotes non-ASH members.

© 2017 by the American Society of Hematology