An evidence-driven proposal for a change in the treatment paradigm for Fabry disease to ensure timely and equitable access to treatment after confirmatory diagnosis.
In Australia access to available treatments is managed via the Life Saving Drug Program (LSDP) Committee and each patient must meet pre-specified diagnostic and eligibility criteria in order to access treatment. Despite significant progress in the understanding of Fabry disease and its management, these LSDP criteria have not been reviewed for more than 15 years. The Australian patient organisation, Fabry Australia, and its Medical Advisory Committee of six experts who manage the majority of the known cases of Fabry disease in Australia, drew on the published literature and current clinical practices to provide contemporary recommendations to guide clinical management of patients diagnosed with Fabry disease. It is hoped that these recommendations will help to inform updated treatment access for patients with Fabry disease in Australia.
This paper was authored by the Fabry Australia Medical Advisory Committee, with the assistance of a medical writer, Hazel Palmer. Thanks to industry groups Amicus Therapeutics, Sanofi Aventis Australia Pty Ltd, and Takeda Pharmaceuticals Australia, for assisting with grant funding for this important project.