This white paper evaluates access to rare disease medicines in Australia between 2018 and 2025, examining the journey from orphan drug designation to reimbursement through the Pharmaceutical Benefits Scheme (PBS). IQVIA found that only 39% of orphan-designated medicines achieved reimbursement, with an average wait of 22 months from registration and most requiring multiple Health Technology Assessment submissions before approval. Benchmarking Australia against 14 comparable countries revealed it ranks near the bottom for funding rare disease medicines. Through policy analysis and case studies, the report highlights barriers to timely patient access and calls for streamlined reimbursement pathways, faster assessments, and stronger support mechanisms to improve treatment availability for the

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