Table 2.

Current unmet needs in haemophilia treatment

Unmet need	Impact
Treatment convenience	Lifetime treatment, frequent injections.65, 66 Prophylaxis is time‐consuming, contributing to poor adherence 67
Joint damage despite factor prophylaxis	Indicates that prophylaxis is failing to control some subclinical bleeding 55, 68
Inhibitor development	Occurs in approximately one‐third of patients with severe haemophilia A and <5% of those with haemophilia B and increases treatment cost and morbidity risks 69
High lifetime‐treatment costs	High factor concentrate costs,1, 70, 71, 72 means availability of factor prophylaxis is limited in many countries
Pain	See Table 1
Limits on activity and social participation	See Table 1