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

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.

Keywords::

• Australia
• inherited disorders of metabolism
• Life Saving Drugs Program
• orphan drugs
• subsidised access

Acknowledgement

The author is grateful to the patients and their treating doctors for permission to use their de-identified data, and the staff of the LSDP for administering the program. The analysis which forms the basis of this paper was conducted by the author without the use or disclosure of personal information of any patient of the LSDP. Any views expressed or conclusions drawn in the paper are entirely those of the author and do not reflect any views of the Federal Department of Health and Ageing or the Australian Government.

Notes

This box summarises key points contained in the article.