ederal Health Minister Nicola Roxon recently met with an alliance of consumer, industry and other stakeholders to justify the government’s plan to indefinitely delay the listing of seven new medicines on the Pharmaceutical Benefits Scheme (PBS). She argued that, after considering the advice of the Pharmaceutical Benefits Advisory Committee (PBAC), it was the government’s responsibility to decide whether or not to list a new drug, taking into account other priorities across the health portfolio and current fiscal circumstances. 1 Clearly, the cost of the PBS must be sustainable. However, there are other ways of reducing its cost apart from delaying the listing of drugs recommended by PBAC as cost-effective. The treatment of macular degeneration provides an illustrative example. Age-related macular degeneration (AMD) is responsible for almost half of all cases of blindness in Australia. 2 In neovascular (or “wet”) macular degeneration, vision loss results from the abnormal growth and leakage of blood vessels in the macula. Ranibizumab (Lucentis), developed by Genentech and marketed by Novartis in Australia, is currently the only drug approved by the Therapeutic Goods Administration (TGA) and available on the PBS to treat wet AMD. It is administered as an intravitreal injection, usually 4–8-weekly, for 12 to 18 months or longer. The PBS-listed price of each injection is $1967. Ranibizumab is the antigen-binding fragment of a recombinant, humanised, monoclonal antibody that binds to vascular endothelial growth factor A (VEGF-A), the cytokine primarily responsible for blood vessel growth. The inhibition of VEGF-A reduces the permeability and growth of the neovascular vessels. Blindness is prevented in most patients, and the majority of treated patients go on to have some improvement in vision. 3-5 Bevacizumab (Avastin) is an anti-VEGF-A humanised, monoclonal antibody (also developed by Genentech, and marketed in Australia by Roche; Genentech is a wholly owned subsidiary of Roche) that has been approved by the TGA for the systemic treatment of certain cancers. It was successfully used “off-label” for the treatment of wet AMD before ranibizumab became available, but has also been used to some degree since the availability of ranibizumab, especially in the United States, where patients bear more of the costs of drugs. 5-7 It is prepared for ophthalmic use in a sterile pharmacy by taking a dose used in chemotherapy and splitting it for use in treating wet AMD for up to 25 patients. The cost of its off-label use has been significantly less than that of ranibizumab (around a 40th of the cost, at $50 per dose). Ranibizumab received PBS listing for use in treating wet AMD in 2007 and has since largely replaced bevacizumab for the treatment of AMD in Australia. Although therapy with ranibizumab has been successful, its PBS listing has come at great expense, costing taxpayers $237 million in 2010 (second only to atorvastatin and rosuvastatin). It is consistent with many of the principles of quality use of medicines (QUM), outlined in the National Medicines Policy, 8,9 for ophthalmologists to select a PBS-listed therapy that has been demonstrated to be safe and effective. But advocates of QUM also emphasise the importance of choosing medicines that are costeffective for individuals and the community. A recent study by the US National Eye Institute 10 has raised the question of whether use of ranibizumab can be justified economically. The study compared bevacizumab and ranibizumab for the treatment of wet AMD, administered either monthly or as needed, in 1208 randomly assigned patients. At 1 year, bevacizumab and ranibizumab had equivalent effects on visual acuity, when administered according to the same schedule. Ranibizumab given as needed, with monthly evaluation, had effects on vision that were equivalent to those of ranibizumab administered monthly. The comparison between bevacizumab as needed and monthly bevacizumab was inconclusive. Differences in rates of serious adverse events were higher with bevacizumab but did not reach statistical significance, and require further study. Results from the second year of this study and from other comparative trials and experiential databases will provide additional information.