Cost and outcome: comparisons of two alternative bypassing agents for persons with haemophilia A complicated by an inhibitor.
The development of inhibitory antibodies to factor VIII is a serious complication of haemophilia. Two haemostatic agents with different bypassing mechanisms have been used in the treatment of patients with inhibitors: activated prothrombin complex concentrate (aPCC) and recombinant factor VIIa (rFVIIa). The objective was to compare cost and outcome of aPCC and rFVIIa in the treatment of joint bleeds. The analyses were based on the FENOC (FEIBA NovoSeven Comparative Study) crossover study where 48 patients used aPCC and rFVIIa to treat two joint bleeds. Incremental cost-effectiveness ratios were calculated for three outcome measures and the variation in cost was analyzed using two alternative regression methods. Results were subjected to sensitivity analyses. Key determinants of cost were prescribed dose, bodyweight and treatment in addition to protocol. The cost of aPCC was on average lower than rFVIIa. At all but one time point, patients rated slightly higher (but not statistically significantly) percentages of treatment efficacy and stopping of the bleed by aPCC. The reported reduction in pain from start of treatment up to 48 hours varied considerably among individuals. The different relative prices in the US, Turkey and Sweden mattered, but did not reverse the main results. In conclusion, the cost per episode was significantly lower for aPCC. The large individual-level variation in reduction of pain supports decisions that consider the individual patient's experience and that accept trade-offs between cost and reduction in pain rather than focusing on cost only.
Steen Carlsson
Katarina
K
Lund University Centre for Health Economics, LUCHE, Lund, Sweden. katarina.steen@luche.lu.se
Astermark
Jan
J
Donfield
Sharyne
S
Berntorp
Erik
E
D000293:Adolescent; D000328:Adult; D018771:Arthralgia / Q000188:drug therapy / Q000191:economics / Q000209:etiology; D001323:Autoantibodies / Q000097:blood*; D001779:Blood Coagulation Factors / Q000191:economics* / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003362:Cost-Benefit Analysis; D018592:Cross-Over Studies; D016527:Drug Costs; D005169:Factor VIII / Q000276:immunology*; D015942:Factor VIIa / Q000191:economics* / Q000627:therapeutic use; D006395:Hemarthrosis / Q000188:drug therapy / Q000191:economics* / Q000209:etiology; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy / Q000191:economics* / Q000276:immunology; D006490:Hemostatics / Q000191:economics* / Q000627:therapeutic use; D006801:Humans; D008875:Middle Aged; D010147:Pain Measurement; D017060:Patient Satisfaction; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use; D013548:Sweden; D016896:Treatment Outcome; D014421:Turkey; D014481:United States
D001323:Autoantibodies; D001779:Blood Coagulation Factors; D006490:Hemostatics; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; D005169:Factor VIII; C103587:recombinant FVIIa; D015942:Factor VIIa