Background: Access to &quot high-cost medicines&quot under Australia's Pharmaceutical Benefits Scheme (PBS) is characterised by strict eligibility criteria. The PBS access scheme for the anti-rheumatic biologicals (etanercept, infliximab, and adalimumab) was examined for concordance with Australia?s National Medicines Policy. Methods: Semi-structured interviews with a range of stakeholders were conducted. National, aggregated prescription and expenditure data from Medicare Australia and dispensing data from the Drug Utilisation Sub-Committee were analysed. Access to biologicals was also examined from an ethical perspective. Results: Interviewees agreed that controlled access to high-cost medicines was broadly equitable and practical but specific concerns included: timeliness of access; bureaucracy of the process; contentious cases of individual patients being denied access; insufficient patient information; the quantum of resources required to administer the access scheme; inadequate stakeholder consultation. The access requirement of a history of failure of conventional anti-rheumatic drugs was supported. Recommendations included proactive review of the access criteria and outcomes; greater transparency and formal stakeholder involvement to increase public confidence in the definition of &quot target patient population" and a formal appeal mechanism to increase the fairness and accountability of the PBS. Establishment of an appeal mechanism is supported by &quot accountability for reasonableness&quot framework grounded in procedural justice. Data needed to examine the health outcomes associated with the use of biologicals on a national level was not easily available. This shortcoming is discordant with National Medicines Policy. Utilisation of biologicals over the first two years of PBS-subsidy was conservative but with considerable variability across States and Territories (an 8-fold difference between the jurisdictions), usage roughly correlating with access to rheumatologists. Introduction of PBS-subsidised biologicals did not alter the trends in utilisation of non-biological anti-rheumatic drugs. Conclusions: This research suggests that policy-makers focus upon: explicitly considering ethical principles and formally involving stakeholders when developing policies on access to high-cost medicines; improving communication and providing information based on increased transparency; and establishing formal mechanisms for review of and appeals against PBS decisions. The comprehensive evaluation of medicine use and outcomes post-subsidy is critical for the future of the PBS. The National Medicines Policy has proved a useful framework for evaluating this access scheme.

Background: Access to &quot high-cost medicines&quot under Australia's Pharmaceutical Benefits Scheme (PBS) is characterised by strict eligibility criteria. The PBS access scheme for the anti-rheumatic biologicals (etanercept, infliximab, and adalimumab) was examined for concordance with Australia?s National Medicines Policy. Methods: Semi-structured interviews with a range of stakeholders were conducted. National, aggregated prescription and expenditure data from Medicare Australia and dispensing data from the Drug Utilisation Sub-Committee were analysed. Access to biologicals was also examined from an ethical perspective. Results: Interviewees agreed that controlled access to high-cost medicines was broadly equitable and practical but specific concerns included: timeliness of access; bureaucracy of the process; contentious cases of individual patients being denied access; insufficient patient information; the quantum of resources required to administer the access scheme; inadequate stakeholder consultation. The access requirement of a history of failure of conventional anti-rheumatic drugs was supported. Recommendations included proactive review of the access criteria and outcomes; greater transparency and formal stakeholder involvement to increase public confidence in the definition of &quot target patient population" and a formal appeal mechanism to increase the fairness and accountability of the PBS. Establishment of an appeal mechanism is supported by &quot accountability for reasonableness&quot framework grounded in procedural justice. Data needed to examine the health outcomes associated with the use of biologicals on a national level was not easily available. This shortcoming is discordant with National Medicines Policy. Utilisation of biologicals over the first two years of PBS-subsidy was conservative but with considerable variability across States and Territories (an 8-fold difference between the jurisdictions), usage roughly correlating with access to rheumatologists. Introduction of PBS-subsidised biologicals did not alter the trends in utilisation of non-biological anti-rheumatic drugs. Conclusions: This research suggests that policy-makers focus upon: explicitly considering ethical principles and formally involving stakeholders when developing policies on access to high-cost medicines; improving communication and providing information based on increased transparency; and establishing formal mechanisms for review of and appeals against PBS decisions. The comprehensive evaluation of medicine use and outcomes post-subsidy is critical for the future of the PBS. The National Medicines Policy has proved a useful framework for evaluating this access scheme.