Cutting Recombinant Protein Pills?

On March 16, the NYTimes ran two stories on Cerezyme, an expensive protein-based drug used to treat a rare genetic disorder, Gaucher's disease. Both articles covered issues that might be emblematic of those that will be encountered when gene transfer applications are commercialized.

One article described uncertainties clinicians face in establishing the appropriate dose for Cerezyme. At $350K/year to treat a typical adult, lowering doses can have important economic benefits for patients. But with the drug licensed at a particular dose level, reliable clinical evidence on response at lower dose is not available. And Genzyme isn't about to run trials testing whether Gaucher's patients can get away with less of their product. Similar issues are raised by the treatment of wet age-related macular degeneration (AMD), where some ophthalmologists use Avastin off-label at a fraction of the cost of Lucentis, which is licensed for AMD. So are Gaucher's physicians practicing substandard medicine by considering their patients' pocketbooks, or are they treating the whole patient instead of the disease? (photocredit: banner from Genzyme website (www.cerezyme.com/home)

More on orphan drugs in the next few posts...