Mantovani et al.[27] |
2005 |
Physicians, patients and pharmacists |
Italy |
Treatment products in haemophilia |
Total: 305Physicians: 69Patients: 178Pharmacists: 58 |
35.9 |
Focus group (n=N/R) + Pilot (n=15) |
6 |
2–3 |
Bayer Italia S.p.A. |
Paper-based survey |
Lee et al. [28] |
2008 |
Physicians |
US |
Coagulation factor concentrates† |
Total: 30 |
N/A |
Physician engagement (n & format N/R) + testing (N/R) |
12 |
2–4 |
Novo Nordisk Inc |
Paper-based survey |
Scalone et al.[29] |
2009 |
Physicians, patients and pharmacists |
Italy |
Coagulation factor concentrates† |
Total: 101Physicians: 39Patients: 37Pharmacists: 25 |
Adults = 41.4Paedatric = 8.2 |
Focus group (n=N/R) + Pilot (n=35) |
8 |
2–3 |
Novo Nordisk Denmark |
N/R |
Brown et al.[16] |
2011 |
Patients and caregivers |
US |
Treatment products in haemophilia† |
Total: 53*Patients: 23Caregivers: 30 |
20.7 |
Used same as Lee et al. (2008)[28] |
12 |
2–4 |
Novo Nordisk Inc |
Paper-based survey |
Mohamed et al.[30] |
2011 |
Patients and caregivers |
US |
Treatment products in haemophilia A |
Total: 147Patients: 77Caregivers: 70 |
Adults = 38.7Juvenile = 12.4 |
Face-to-face interviews with adult patients and caregivers (n=8) |
6 |
2–3 |
Baxter Biosciences |
Web-based survey |
Gelhorn et al.[31] |
2013 |
Physicians |
US and EU |
Treatment products in haemophilia A† |
Total: 36Physicians EU: 17Physicians US: 19 |
N/A |
Qualitative interviews (n=4) + Pilot (n=5) |
5 |
3 |
Baxter Biosciences |
Web-based survey |
Chaugule et al.[32] |
2015 |
Patients and caregivers |
US |
Willingness to pay for treatment products in haemophilia |
Total: 79 |
40.0 |
Assistance from Haem author + Pilot (n=5) |
5 |
2–5 |
None |
iPad at conference |
Lock et al. [33] |
2016 |
Patients, caregivers and HCPs |
Multi-country |
PK-guided dosing of prophylaxis |
Total: 224HCPs: 91Patients: 114Caregivers: 19 |
38.0 |
Qualitative interviews + Pilot (n=10) |
5 |
2–3 |
Pfizer |
Paper-based survey |
Fifer et al. [34] |
2019 |
Patients and caregivers |
Multi-country |
Treatment products in haemophilia A |
Total: 54^Patients: 24Caregivers: 30 |
N/R |
Qualitative interviews (n=10) |
11 |
2–4 |
Roche |
Web-based survey |
Su et al. [35] |
2020 |
Patients and caregivers |
US |
Treatment products in haemophilia A |
Total: 209Patients: 113Caregivers: 96 |
35.5 |
Qualitative interviews (n=10)** + Pilot (n=6) |
6 |
2–4 |
Sanofi Genzyme |
Web-based survey |
Park et al. [36] |
2021 |
Patients and caregivers |
South Korea |
Treatment products in haemophilia A |
Total: 505*** |
31.5 |
N/R |
5 |
3 |
Pfizer Pharmaceuticals Korea Ltd |
Hospital-based survey |
Witkop et al. [37] |
2021 |
Patients |
US |
Haemophilia gene therapy |
Total: 183 |
38.5 |
Qualitative interviews (n=7) ** + Pilot (n=14) |
6 |
3–4 |
uniQure Inc. |
Web-based survey |