Abstract
Background: Pharmaceutical subsidy schemes are under increasing pressure to evaluate the cost effectiveness of new highly specialised and orphan drugs for universal subsidy. In the absence of longer-term outcome data, drug sponsors often present modelled data, which can carry a significant level of uncertainty over longer-term projections. Risk-sharing schemes between drug sponsor and government may provide an acceptable method of balancing the uncertainty of longer-term cost effectiveness with the public demand for equitable and timely access to new drugs.
Methods: The Bosentan Patient Registry (BPR) is an example of a unique risk-sharing model utilised in Australia aiming to provide clinical evidence to support the modelled predictions, with the registry survival outcomes linked to future price. Concomitant medication, health and vital status data was collected from clinicians, government health departments and death registries.
Results: The BPR has identified a number of issues surrounding registry governance, ethics and patient privacy, and the collection of timely and accurate data, which need to be addressed for the development of a generic registry model for systematic evaluation.
Conclusion: The success of a generic drug registry model based on the BPR will be enhanced by addressing a number of operational issues identified during the implementation of this project.
Material in this paper was presented in an oral presentation to the National Medicines Symposium: Quality Use of Medicines, 7–9 June 2006, Canberra, Australia.
Notes
*Drugs and agents used to treat, prevent or diagnose rare diseases in Australia (i.e. not affecting > 2,000 Australians at any given time) are eligible to apply for orphan drug status under the Therapeutic Goods Act (1989). The registration fees are waived to encourage sponsors to develop drugs and agents that may not be considered otherwise commercially viable under regular conditions. Further information is available on the Therapeutic Goods Administration website1.