Issues in assessing products for the treatment of hemophilia – the intersection between efficacy, economics, and ethics

Figures & data

Table 1 Some recent studies on novel clotting factor concentrates

Product	Manufacturer	Feature	Efficacy assessment – prophylaxis versus on demand
Alprolix^Citation71	Biogen Idec	Longer half-life FIX	RCT
Eloctate^Citation72	Biogen Idec	Longer half-life FVIII	RCT
Rixubis^Citation73	Baxter	Biosimilar FIX	RCT
Kogenate FS^Citation74	Bayer	New formulation FVIII – adults	RCT
NovoEight^Citation75	Novo Nordisk	Biosimilar FVIII	Historical controls
rIX-FP^Citation76	CSL	Longer half-life FIX	Nonrandomized
Bay81-8973^Citation77	Bayer	Protein-free manufacture FVIII	RCT
Bay94-9027^Citation78	Bayer	Longer half-life FVIII	RCT
Bax855^Citation79	Baxter	Longer half-life FVIII	Nonrandomized – unmodified product comparator in prophylaxis trial
NUWIQ^Citation80	Octapharma	Human cell line derived	Nonrandomized
N8-GP^Citation81	Novo Nordisk	Longer half-life FVIII	Nonrandomized, uncontrolled
N9-GP^Citation82	Novo Nordisk	Longer half-life FVIII	Nonrandomized, uncontrolled
rVIII – single chain^Citation83	CSL	Single-chain FVIII	Single-group assignment

Abbreviation: RCT, randomized clinical trial.

Figure 1 Patient-centric delivery of hemophilia treatment products.

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