PB0177 - An examination of access to preventive therapies for persons with hemophilia (PWH): A pilot survey on behalf of the International Prophylaxis Study Group (IPSG)

Background: The IPSG Access Expert Working Group (EWG, N=8) aims to examine trends within haemophilia care and access, and to develop strategies achieving and maintaining optimal patient care. Information on global access to therapies is needed to address gaps. The IPSG Access EWG launched a survey to explore challenges with access to hemophilia treatment.

Aims: To gain an understanding of access to preventive therapies for PWH in 12 countries [Australia, Canada, France, Italy, New Zealand, Turkey, England, Northern Ireland, Scotland, Wales, Republic of Ireland and United States of America (USA)] with different levels of access to prophylaxis therapies. Local physician “champions” assisted with distribution.

Methods: The 18-question survey, developed by the Access EWG, was distributed electronically to Medical Directors of HTCs. Informed consent was obtained. Data were analyzed using SPSS. Results were summarized into themes: heterogeneity of responses, roles of patient advocacy groups in decision-making, and economic influences on access to preventive therapies.

Results: 155 responses (41.4%) were obtained from 374 HTCs. 77% (120/155) of responses were from non-USA countries where mainly government health agencies pay for preventive therapies, compared to a heterogenous funding system in the USA (23%, 35/155). There was variability in knowledge of respondents regarding regulatory approval decision-making and funding of therapies. Although 99% indicated awareness of a national patient advocacy group, 46% reported not knowing whether these organizations have a vote in decision-making for funding of preventive therapies. Lastly, there were inconsistencies in funding to regulatory-approved preventive therapies, with 43% reporting economic considerations impacted therapeutic choices.

Conclusion(s): There is a lack of knowledge of decision-making processes regarding access to therapies for PWH. Inclusion of representatives from national organizations, physicians, and patient advocacy groups in decision-making bodies for funding of therapies is critical. This should be instituted globally to meet the need for individualized treatment of PWH.