What impact does ‘conventional’ economic evaluation have on patient access to new orphan medicines? A comparative study of their reimbursement in Australia (2005–2012)

Abstract

Background: Orphan medicines used to treat patients with rare diseases often come at high costs with lower levels of clinical evidence. We compared the likelihood and timeliness of reimbursement for orphan medicines with non-orphan medicines in Australia between 2005 and 2012. Methods: We developed two key assessment metrics to compare submissions and outcomes for new orphan medicines with those for new non-orphan medicines, viz., the likelihood of submissions being recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) (success rate) and the time from Therapeutic Goods Administration registration to reimbursement (overall timeliness). Results: Thirty eight out of 95 outcomes for orphan medicines (40%) received a PBAC recommendation compared to 257/481 (53%) for non-orphan medicines (p = 0.17). The PBAC recommendations that resulted in listings were comparable between the orphan and non-orphan categories (33/38 [87%] vs 218/257 [85%], respectively, p = 0.74). Conclusion: The results suggested orphan medicines were not accorded any special status for reimbursement.

Keywords:

• Australia
• decision-making
• equity
• health technology assessment
• orphan drug

Acknowledgements

The authors thank Laurent Billot for his assistance with the statistical analyses and Michael Drummond for reviewing and commenting on the draft manuscript.

Financial & competing interests disclosure

Data not readily available from documents in the public domain were obtained from industry colleagues and the LSDP Secretariat. Novartis provided funding for the statistical analyses of the data. G Chin is a full-time employee of Novartis Pharmaceuticals and is involved in the preparation of submissions to the PBAC for orphan and non-orphan medicines. The views expressed in this article are entirely those of the authors and do not necessarily reflect the views or policies of their current or past employers.

Key issues

• Few studies have examined the level of reimbursement of new orphan medicines compared to new medicines used to treat patients with more prevalent (non-orphan) diseases; we conducted a systematic review in Australia over a 7-year period.

• We developed two metrics to compare the submissions and outcomes for new orphan medicines with those for new non-orphan medicines, viz., the likelihood of submissions being recommended by the Pharmaceutical Benefits Advisory Committee (success rate) and the time from Therapeutic Goods Administration registration to Pharmaceutical Benefits Scheme/Life Saving Drugs Program listing (overall timeliness).

• The rate of recommendations by the Pharmaceutical Benefits Advisory Committee for the orphan category was 40% compared to 53% for the non-orphan category over the study period.

• While orphan submissions, on average, achieved a listing 7–9 months sooner than non-orphan submissions, the difference was not statistically significant and is mainly driven by sponsors of orphan drug submitting earlier to the PBAC rather than any preferential treatment.

Notes

LSDP: Life Saving Drugs Program; PBAC: Pharmaceutical Benefits Advisory Committee; PBS: Pharmaceutical Benefits Scheme; TGA: Therapeutic Goods Administration.

^†t-test and log-rank test.

LSDP: Life Saving Drugs Program; PBAC: Pharmaceutical Benefits Advisory Committee; PBS: Pharmaceutical Benefits Scheme; TGA: Therapeutic Goods Administration.