The Australian process for subsidised access to orphan drugs for rare inherited disorders of metabolism

Jack Goldblatt

    Research output: Contribution to journalReview article

    Abstract

    Introduction: In Australia, subsidised access to orphan drugs for very rare, life-threatening inherited disorders of metabolism is centrally managed through a Federal Health and Ageing Department program known as the Life Saving Drugs Program (LSDP). Areas covered: In this paper, the author reviews the process in Australia for registering orphan drugs for inherited disorders of metabolism and providing access to subsidised therapy through a centralised national program. Expert opinion: In Australia, having a centralised national scheme for delivery of therapy to patients with rare disorders of metabolism has maximised the acquisition of experience in managing these conditions, and facilitated the accumulation of data allowing for optimising individualised dosing and documenting efficacy of therapy. © 2013 Informa UK, Ltd.
    Original languageEnglish
    Pages (from-to)273-277
    JournalExpert Opinion on Orphan Drugs
    Volume1
    Issue number4
    DOIs
    Publication statusPublished - 2013

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