Extended half-life clotting factor concentrates for the treatment of haemophilia A and B
Milverton, J, Vogan, A, Newton, S, Parsons, J, Merlin, T
Record ID 32018000548
Original Title: Application 1511
Authors' results and conclusions: Comparative Safety - Haemophilia A - No studies directly compared the safety of standard half-life (SHL) products and extended half-life (EHL) products. However, the overall rate of adverse events due to EHL products was low, and there were no data to suggest that EHL products are associated with a higher rate of adverse events than SHL products. Non-comparative studies reported a total of five serious adverse events related to the EHL product occurred in adults and nine in children - the majority being hypersensitivity. Haemophilia B - There were no data to suggest that EHL products are associated with a higher rate of adverse events than SHL products. Comparative effectiveness - Haemophilia A - The primary clinical outcome of interest was annualised bleeding rate (ABR). Four EHL products (BAX 855, Bay 81-8973, N8-GP and rFVIIIFc) provided historical data on bleeding rates of patients when they were receiving SHL factors. In these studies, the ABRs in the patients receiving prophylactic treatment with EHL products were between 11-83% of the rates of patients receiving SHL prophylaxis. Haemophilia B - The primary clinical outcome of interest was ABR. All studies comparing prophylaxis with SHL factor IX products (historical data) with prophylaxis with EHL factor IX products (trial data) in adolescents and adults reported that bleeding rates were reduced through the use of EHL products. Likewise, bleeding rates in those treated on-demand with EHL products were reduced compared to historical bleeding rates in those treated on-demand with SHL products.
Authors' recomendations: After considering the strength of the available evidence in relation to comparative safety, clinical effectiveness and cost effectiveness, Medical Services Advisory Committee (MSAC) supported the inclusion of extended half-life clotting factor concentrates (factor VIII and IX) in the National Products Price List maintained by the National Blood Authority (NBA).
Project Status: Completed
Year Published: 2018
URL for published report: https://www.google.com/url?sa=t&rct=j&q=&esrc=s&source=web&cd=&cad=rja&uact=8&ved=2ahUKEwi8kPrYm-LqAhVKwjgGHZS0BEQQFjABegQIBBAB&url=http%3A%2F%2Fwww.msac.gov.au%2Finternet%2Fmsac%2Fpublishing.nsf%2FContent%2FC3B4D8FB345D71C4CA2581BD00141E25%2F%24File%2F1511%2520-%2520Final%2520PSD.pdf&usg=AOvVaw2iKBvm57iY4INBCNGnDeAh
English language abstract: An English language summary is available
Publication Type: Other
- Blood Coagulation Factors
- Hemophilia A
- Hemophilia B
- Factor VIII
- Factor IX
Organisation Name: Adelaide Health Technology Assessment
Contact Address: School of Public Health, Mail Drop 545, University of Adelaide, Adelaide SA 5005, AUSTRALIA, Tel: +61 8 8313 4617
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