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Letter to the Editor

Editor, – We read with interest the editorial 'Can we deny patients expensive drugs?' (Aust Prescr 2006;29:146-8). We agree with many of the author's arguments, but take exception to the suggestion that Pharmaceutical Benefits Advisory Committee (PBAC) processes be bypassed for drugs targeting rare diseases and for which no PBAC submission has been made. The authors suggest that in such cases the Pharmaceutical Benefits Scheme (PBS) 'subsidise the use of these medicines for an indication after conventional therapies have proven ineffective'. We infer that such medicine be subsidised irrespective of costs. This implies society is willing to accept a higher cost per unit of health (for example a year of life) on the basis that the disease is rare. Some things need to be clarified; rare does not mean severe and expensive does not mean better. We acknowledge that efficiency should not be the only criteria in resource allocation decisions and that equity considerations need to be taken into account also. However, the fact that a person has a rare, as opposed to a common, condition is not a good moral basis for accepting higher opportunity costs. Such a system would send all the wrong signals to the research and development community. Locally, pharmaceutical companies would stop applying for PBS funding for drugs that target rare diseases. On a global level, such a system signals our willingness to pay infinite amounts for uncertain benefits for rare conditions, at a time when we want more research and development in areas where we can make substantial gains in reducing the health burden.

Gisselle Gallego
Kees van Gool
Research Officers, Centre for Health Economics and Research Evaluation
University of Technology

Authors' comment

Ms Karen Kaye, Ms Christine Lu and Professor Richard Day, authors of the editorial, comment:

We agree that PBAC processes should not be bypassed for medicines targeting rare diseases, but in fact this often happens in our current healthcare system. Expensive treatments for severe and rare diseases that are not PBS-subsidised are instead subsidised through supply by public hospitals. The problem with this process is that it is relatively ad hoc and decisions about patients' access to such medicines vary depending on the availability of local expertise and funding. It does not promote consistency or transparency in the decision process, does not guarantee equity of access to medicines for patients with the same condition in different parts of the country, and does not facilitate national monitoring of either costs or outcomes. The current system has not resulted in adequate research or PBS submissions to date and it will not in future unless hospitals refuse to supply these medicines. This is unlikely, especially when the disease is severe and there is evidence of clinical effectiveness and other therapeutic options have been tried and failed. Such a funding approach is ethically sound; a similar ethical approach forms the basis for the PBS 'rule of rescue' and Australia's Orphan drug program. Carefully monitored supply of expensive but effective medicines via a national system would at least facilitate collation of information to inform government, clinicians, industry and the public about use of these medicines (and associated costs and outcomes) and would help ensure equity of access. Provided supply continues to be reviewed on the basis of such information, there is likely to be benefit to both patients in need and society as a whole.