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.

Thesis (Ph. D.)--Victoria University (Melbourne, Vic.), 2008. / Includes bibliographical references.

PROBLEM INVESTIGATED: Methodological issues and policy implications arising from the application of interrupted time series (ITS) analyses to assess the impact of Pharmaceutical Benefit Scheme (PBS) subsidisation policies on drug utilisation in Australia. PROCEDURES FOLLOWED: A critical review of methodological issues relating to the application and analysis of ITS designs was undertaken. This included an examination of drug utilisation data sources in Australia. The PBS policies examined were: (i) the introduction of copayments in 1990; (ii) the introduction of re-supply limits in 1994 and (iii) the introduction of a form of reference pricing in 1998. Monthly aggregate drug utilisation data was obtained from the Australian Department of Health and Ageing. Segmented regression analyses incorporating autocorrelated errors were implemented and statistical diagnostics applied to ensure correct ITS model specification. Alternative seasonal modelling approaches were compared. RESULTS OBTAINED: The copayment ITS evaluation found that while these copayments produced a reduction in the utilisation of essential and discretionary medications, this effect was stronger for discretionary drugs. An unintended policy effect was a large anticipatory increase in drug utilisation during the month prior to the copayments. Repatriation PBS data was also utilised due to the limited number of pre-intervention data points in the Community series. The re-supply limit ITS evaluation found that the 20-day rule markedly reduced the size of the seasonal increase during the month of December. However, logistic regression analyses showed that the size of this reduction attenuated over time, highlighting the need to consider alternative analysis strategies when applying a ITS approach. The reference pricing ITS evaluation found that this policy had achieved its drug utilisation objectives for H2RAs and ACE Inhibitors. However with regard to CCBs, no increase in the utilisation of benchmark priced drug was apparent, which probably reflected clinical concerns at the time about the safety of these drugs. MAJOR CONCLUSIONS: Well implemented ITS analyses provide a valuable tool for evaluating the impact of PBS subsidisation policy change on drug utilisation in Australia. As with any methodology, however, different design and data integrity issues will affect the quality of information provided.

Processes of rationalisation and restructuring within the international drug industry in the past decade have altered the conditions for governance of Australia’s pharmaceutical sector. This thesis demonstrates that the balance of power within the domestic policy network shifted to favour multinational suppliers of prescription drugs after the Government in the late 1980s embraced the objective of making the regulatory and policy environment more user-friendly. The emphasis of state activities has moved away from welfare and public interest objectives towards provision of direct support for capital accumulation under conditions of globalising capitalism. / The domain of pharmaceutical policy was historically characterised by corporatist bargaining between strong regulatory agencies within the Commonwealth Department of Health and centralised associations representing producer and professional interests. Following recent reform of these agencies and a reordering of their relative authority, the pattern of interaction within the policy network has become more open and politicised, with more active participation of groups representing consumers, patients and the research and development (R&D) community. Conversely, the capacity of Australian state agencies to manage and control sectoral change has diminished. A greater degree of pluralism at the level of interaction between the state of interest groups has evolved within the context of the principal trend towards marketisation and commodification within the drug sector. / These conclusions arise from the empirical analysis of developments in the international pharmaceutical industry, including the formation of a transnational regulatory regime, and changes in domestic policy and regulatory practices. The thesis traces the ascendance of governance through the market mechanism at the expense of direct state control or corporatist bargaining. The investigation gives particular attention to: the Pharmaceutical Benefits Scheme; the Pharmaceutical Industry Development Program introduced in 1987, notably the Factor (f) scheme (which provides notional drug price increases in exchange for expanded industry activity); and the politics of brand substitution and generic drugs. / It is shown that the Australian Government in the period under consideration, irrespective of party political composition, has pursued purposefully a policy of international integration derived from an acceptance of the imperative of retaining and attracting foreign capital. While the Factor (f) program as designed to sustain bargaining between the state and the multinational industry, it is demonstrated that the Department of Industry proved unable to maintain and generate support for strategically oriented industry policy. / The changes identified and analysed in this thesis are consistent with the hypothesis of a hollowing out of the state associated with the decline of the Fordist model of accumulation and the Keynesian welfare state. A feature of this transition is the subordination of social policy to the imperatives of innovation, flexibility and international competitiveness.

In this thesis, a microsimulation model was developed using methods which were intended to overcome the main criticism of earlier models developed in Australia - that their estimation of the distribution of health benefits1 across income groups was not accurate. To determine whether the new model � called the Person Level Model of Pharmaceutical Benefits (PLM-PB) � was more accurate, two typical means-based models were also built to replicate the most commonly used methods in Australia. A comparison of the results of the three models revealed that while they produced comparable results at the aggregate when compared with administrative data, the PLM-PB was much more accurate in capturing distributional differences by beneficiary and medication type. The PLM-PB also indicated that, as anticipated, PBS benefits were more pro-poor than earlier means-based models had suggested. The PLM-PB had another important advantage in that the method also captured the variation in the use of medication and thus the subsidy received within sub-populations. As the PLM-PB was found to be more accurate than the means-based model, a multivariate analysis of the distribution of PBS subsidy across a number of socio-economic groups was undertaken as an example application of the model. It was found that health status (defined by number of recent illnesses) and concession card type were most important in explaining the amount of PBS subsidy received. This indicates that the distribution of PBS expenditure meets the policy objectives of assisting those most in need, whether need is defined as poor health or low income. 1 Benefits refer to expenditure as transfers from government to individuals rather than the general health benefits of using medication.

Research Doctorate - Doctor of Philosophy (PhD) / In January 2005 Australia implemented the Australia-United States Free Trade Agreement (AUSFTA). The AUSFTA was a historic document because it laid out a closer trading association between Australia and its close ally, the United States (US). The Agreement was generally welcomed in both countries but it contained provisions that were controversial, perhaps none more so than those covering pharmaceuticals. In Australia there was deep concern over the effects of these provisions on the Pharmaceutical Benefits Scheme (PBS). A Senate Committee investigating the likely impact of the AUSFTA was apprehensive that the provisions would result in the PBS paying higher prices for some medicines. The many outcomes from the research reported in this thesis are preliminary but amongst them, three stood out. First, the AUSFTA instituted a number of small but significant changes to Australian pharmaceutical policies and processes. Some changes, around transparency and listing times, were positive while others, such as sponsor hearings before the PBAC, will probably have limited negative impact. Second, there is now a need to consider the AUSFTA commitments when Australian health policy is reassessed in the future, which will now make Australia’s regulatory processes more complex. Third, it is argued that the AUSFTA may have had input into major reforms of the Australian PBS. These reforms delivered on an outcome that had been sought by the US: a weakening of reference pricing as used by the Australian PBS. The benefit from the change to reference pricing for Australia is unclear, but the PBS is now more vulnerable to higher prices for new medicines in the future.