WHAT'S HOLDING PATIENTS BACK?
Administration challenges and dosing frequency associated with managing hemophilia A1,2
Bentlee lives with hemophilia A.
Many patients prefer less frequent dosing...3,a
YET ONLY
12%
(N=6187)
USED AN EVERY-4-WEEK FVIII MIMETIC4,b
WHY AREN'T MORE PEOPLE FOLLOWING AN EVERY-4-WEEK DOSING REGIMEN DESPITE IT BEING AVAILABLE?
aA web-based survey containing a discrete-choice experiment was completed by adults (≥18 years old) and caregivers of children (aged 8-17 years) with hemophilia in the United States and United Kingdom. Participants were asked to complete 10 choice tasks, choosing their preferred prophylaxis from two hypothetical profiles defined by seven attributes. The primary analysis employed a mixed logit model, and subgroup analyses explored preference heterogeneity. Participants included 194 adults with hemophilia (US: 150; UK: 44) and 169 caregivers (US: 150; UK: 19). Avoiding daily treatment administration had the highest attribute importance among both adults and caregivers, followed by changes in annual bleeds.3
bBased on qualitative survey data captured as of Q2 2025.4
ACCORDING TO World Federation of Hemophilia,
PATIENTS MAY TRADE
ADMINISTRATION BURDEN FOR BLEED PROTECTION5
HIGH INJECTION VOLUME2
Large needles for injections and infusions (23G to 27G)6-8
Fear of needles9
Time-consuming administration10
Injection site reactions1
Potential need for central venous access devices9
Requires multiple steps, including mixing product10
Number and quality of accessible veins9
Storage restrictions11
Concerns carrying medication and supplies when going out11
PATIENTS MAY EXPERIENCE
CHALLENGES WITH INJECTIONS AND INFUSIONS
In a real-world study, 354 patients with hemophilia A who were surveyed reported the following:4,12
with FVIII mimetics (N=85) and FVIII prophylaxis (N=104), respectively, at least sometimes4,12
Response to survey questions: "Due to your treatment (while injecting or after), how often do you have [soreness, physical discomfort, pain, bruising, blown or ruptured veins, or problems due to scarring or scar tissue]?" Patients responded on a 5-point scale: never, rarely, sometimes, often, or always.
Survey responses were collected in a cross-sectional study design. Responses in individuals over time may vary and may not account for continuous patient participation.
Healthcare providers may refer to individual product labels for product-specific efficacy and other information. Data are descriptive, not head-to-head.
Data from an observational study including 354 patients with hemophilia A, conducted from 2020 to 2022, were collected through online surveys as part of the Hemo-TEM questionnaire with PicnicHealth. Patients responded to questions using a 5-point scale: never, rarely, sometimes, often, or always. Limitations may include selection bias due to the cross-sectional study design, variability in individual responses over time, and does not account for continuous patient participation.
BURDEN OF ADMINISTRATION CAN IMPACT
LIFESTYLE4,12
In a real-world observational study of 354 patients with hemophilia A4,12
16%
(N=85)
On FVIII
MIMETIC
AND
26%
(N=104)
ON FVIII
PROPHYLAXIS
REPORTED CURRENT TREATMENT INTERFERED WITH...
TRAVEL AND VACATIONS
...at least somewhat4,12
Some patients also reported that current treatment interfered with social activities, daily activities, and work/school at least sometimes.
Response to survey questions: “How much does taking your current treatment interfere with [your travel or vacations, social activities, daily activities, and work/school]?” Patients responded on a 5-point scale: not at all, a little, somewhat, very, extremely.4
Survey responses were collected in a cross-sectional study design. Responses in individuals over time may vary and may not account for continuous patient participation.4
Luke lives with hemophilia A.
Healthcare providers may refer to individual product labels for product-specific efficacy and other information. Data are descriptive, not head-to-head.
Data from an observational study including 354 patients with hemophilia A, conducted from 2020 to 2022, were collected through online surveys as part of the Hemo-TEM questionnaire with PicnicHealth. Patients responded to questions using a 5-point scale: not at all, a little, somewhat, very, extremely. Limitations may include selection bias due to the cross-sectional study design, variability in individual responses over time, and does not account for continuous patient participation.
Luke lives with hemophilia A.
Are patients
missing doses?
In an analysis of secondary data from a real-world study of 431 adults with hemophilia A4
55%
(N=44)
On FVIII
MIMETIC
AND
67%
(N=257)
ON FVIII
PROPHYLAXIS
STATED THEY
postponed,
delayed or
missed a dose
AT LEAST SOMETIMES.1
Response to survey question: “How many infusions (approximately) do you miss according to your hemophilia regimen?” The options were never, one treatment every week, one treatment every 2 weeks, one treatment every month, one treatment every 3 months, not applicable.
Healthcare providers may refer to individual product labels for product-specific efficacy and other information. Data are descriptive, not head-to-head.
Analysis of secondary data collected as part of a repeated cross-sectional, retrospective observational study of 431 adult patients with hemophilia A in the United States. Data collected as part of the CHESS US data platform were used from 2019, 2023, and 2024. Information was collected using two questionnaire forms: a web-based ‘case record form’ (CRF), completed by physicians, and a ‘patient and public involvement element’ (PPIE) completed by patients. Limitations included the retrospective, non-interventional design and voluntary participation, which may introduce selection and recall bias.
Top patient-reported reasons FOR
non-adherence4
FVIII mimetic4
Forgetfulness
Difficulty following treatment regimen
Needle phobia
Interference with daily/social life
Difficulty attending appointments
FVIII prophylaxis4
Venous access issues
Side effects experienced
Forgetfulness
Difficulty attending appointments
Too frequent
Response to survey questions: Patients were asked to identify the reason or reasons for missed doses or being non-adherent; the most common are summarized here by treatment type.
Healthcare providers may refer to individual product labels for product-specific efficacy and other information. Data are descriptive, not head-to-head.
Analysis of secondary data collected as part of a repeated cross-sectional, retrospective observational study of 431 adult patients with hemophilia A in the United States. Data collected as part of the CHESS US data platform were used from 2019, 2023, and 2024. Information was collected using two questionnaire forms: a web-based ‘case record form’ (CRF), completed by physicians, and a ‘patient and public involvement element’ (PPIE) completed by patients. Limitations included the retrospective, non-interventional design and voluntary participation, which may introduce selection and recall bias.
Nikola lives with hemophilia A.
CHESS US=Cost of Severe Hemophilia across the US: a Socioeconomic Survey; FVIII=Factor VIII.
References
- Brod M, Bushnell DM, Neergaard JS, Waldman LT, Busk AK. Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM). J Patient Rep Outcomes. 2023;7(1):17.
- Usach I, Martinez R, Festini T, Peris JE. Subcutaneous injection of drugs: literature review of factors influencing pain sensation at the injection site. Adv Ther. 2019;36(11):2986-2996.
- Lu H, Gould T, Colavecchia C, et al. Patient and caregiver preferences for long‐term prophylactic treatment of bleeds in haemophilia: a discrete‐choice experiment. Haemophilia. 2026;32(1):116-130.
- Data on file. Novo Nordisk Inc; Plainsboro, NJ.
- Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition [published correction appears in Haemophilia. 2021;27(4):699]. Haemophilia. 2020;26(suppl 6):1-158.
- HEMLIBRA® [package insert]. South San Francisco, CA: Genentech, Inc; 2024.
- Arendt-Nielsen L, Egekvist H, Bjerring P. Pain following controlled cutaneous insertion of needles with different diameters. Somatosens Mot Res. 2006;23(1-2):37-43.
- World Federation of Hemophilia. Protocols for the treatment of hemophilia and von Willebrand disease. WFH. April 2008. Accessed January 9, 2025. https://www1.wfh.org/publication/files/pdf-1137.pdf
- Mannucci PM. Benefits and limitations of extended plasma half-life factor VIII products in hemophilia A. Expert Opin Investig Drugs. 2020;29(3):303-309.
- Wiley RE, Khoury CP, Snihur AWK, et al. From the voices of people with haemophilia A and their caregivers: challenges with current treatment, their impact on quality of life and desired improvements in future therapies. Haemophilia. 2019;25(3):433-440.
- Hakimi Z, Ghelani R, Bystrická L, et al. Patient experience of living with hemophilia A: a conceptual model of humanistic and symptomatic experience in adolescents, adults, and children. J Health Econ Outcomes Res. 2024;11(2):95-102.
- Wheeler AP, Cibelli E, Hanson Get al. Treatment and disease burden in a cohort of people with haemophilia without inhibitors in the United States. Haemophilia. 2025;31(5):912-921.
