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

摘要

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.