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
YET ONLY
12%
(N=5,897)
USED AN EVERY-4-WEEK FVIII MIMETIC1,a
WHY AREN'T MORE PEOPLE UTILIZING AN EVERY-4-WEEK DOSING REGIMEN DESPITE IT BEING AVAILABLE?
aBased on qualitative survey data captured from Q1-Q3 2024.1
ACCORDING TO World Federation of Hemophilia,
PATIENTS MAY TRADE
ADMINISTRATION BURDEN
FOR BLEED PROTECTION4
HIGH INJECTION VOLUME2
Large needles for injections and infusions (23G to 27G)5-7
Fear of needles8
Time-consuming administration9
Injection site reactions1
Potential need for central venous access devices8
Requires multiple steps, including mixing product9
Number and quality of accessible veins8
Storage restrictions10
Concerns carrying medication and supplies when going out10
INJECTIONS AND
INFUSIONS CAN BE TOUGH
In a real world study of 472 patients with hemophilia that were surveyed reported the following1
with FVIII mimetics (N=85) and FVIII prophylaxis (N=104), respectively, at least sometimes1
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: not at all/never, a little/rarely, somewhat/sometimes, very/often, or extremely/always.1
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.
The objective of this real-world, observational survey was to assess the nature of treatment burden and annualized bleed rates (ABR) for people with hemophilia A and B in the United States. From June 6th 2020 to June 15th 2022, 472 patients with hemophilia responded to a survey that captured concerns around protection from bleeding episodes. Additional cross-sectional data on treatment burden were collected via a validated questionnaire (Hemophilia Treatment Experience Measure [Hemo-TEM]) by PicnicHealth. PicnicHealth collects and extracts medical records on behalf of patients and invites them to participate in recurring outcome surveys. Patients were recruited to the PicnicHealth research platform using digital marketing, direct referrals, and community and affiliate partnerships1
TREATMENTS MAY DISRUPT SOME PATIENTS' LIFESTYLES1
In a real-world observational study of 472 patients with hemophilia1
16%
(N=85)
On FVIII
MIMETIC
26%
(N=104)
ON FVIII
PROPHYLAXIS
REPORTED CURRENT TREATMENT INTERFERED WITH...
TRAVEL AND VACATIONS
...at least sometimes1
SOME PATIENTS ALSO REPORTED THAT CURRENT TREATMENT INTERFERED WITH SOCIAL, DAILY ACTIVITIES, WORK AND 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, work, or school]?" Patients responded on a 5-point scale: not at all/never, a little/rarely, somewhat/sometimes, very/often, or extremely/always.1
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.1
The objective of this real-world, observational survey was to assess the nature of treatment burden and annualized bleed rates (ABR) for people with hemophilia A and B in the United States. From June 6th 2020 to June 15th 2022, 472 patients with hemophilia responded to a survey that captured concerns around protection from bleeding episodes. Additional cross-sectional data on treatment burden were collected via a validated questionnaire (Hemophilia Treatment Experience Measure [Hemo-TEM]) by PicnicHealth. PicnicHealth collects and extracts medical records on behalf of patients and invites them to participate in recurring outcome surveys. Patients were recruited to the PicnicHealth research platform using digital marketing, direct referrals, and community and affiliate partnerships.1
Luke lives with Hemophilia A
Are patients
missing doses?
In a real-world observational study of 472 patients with hemophilia1
15%
(N=85)
On FVIII
MIMETIC
32%
(N=104)
ON FVIII
PROPHYLAXIS
STATED THEY
postponed,
delayed or
missed a dose
AT LEAST SOMETIMES.1
Response to survey question: “How often do you postpone, delay or miss a treatment on purpose?” Patients responded on a 5-point scale: not at all, a little, somewhat, very, or extremely.1
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.1
The objective of this real-world, observational survey was to assess the nature of treatment burden and annualized bleed rates (ABR) for people with hemophilia A and B in the United States. From June 6th 2020 to June 15th 2022, 472 patients with hemophilia responded to a survey that captured concerns around protection from bleeding episodes. Additional cross-sectional data on treatment burden were collected via a validated questionnaire (Hemophilia Treatment Experience Measure [Hemo-TEM]) by PicnicHealth. PicnicHealth collects and extracts medical records on behalf of patients and invites them to participate in recurring outcome surveys. Patients were recruited to the PicnicHealth research platform using digital marketing, direct referrals, and community and affiliate partnerships1
Nikola lives with hemophilia A
References
- Data on file. Novo Nordisk Inc; Plainsboro, NJ.
- 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.
- Park YS, Hwang TJ, Cho GJ, et al. Patients' and parents' satisfaction with, and preference for, haemophilia A treatments: a cross-sectional, multicentre, observational study. Haemophilia. 2021;27(4):563-573.
- 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.
- 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.
- 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.
- 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.
