Billions of health-care dollars can be saved by aggressively pursuing studies of comparative effectiveness of treatments. Drug therapy of macular degeneration provides a striking example.
Macular degeneration is a common cause of visual loss in older people. In the more severe, “wet” form of the disease, new blood vessels grow beneath the macula, the part of the retina that provides our most sensitive vision. Growth of the new vessels is accompanied by leakage of blood, which distorts the overlying cells of the macula. This leads to progressive blurring and eventual loss of vision. Scientists identified a growth factor that stimulates the growth of the new vessels.
Avastin, an anti-cancer agent already approved by the Food and Drug Administration, works by blocking the growth factor. After extensive research, retina specialists began to use Avastin “off-label” for treating macular degeneration. Once FDA approves a drug, it may be used off-label for conditions for which it was not initially approved.
Results of therapy for macular degeneration were quite good.
In 2006, Genentech, the company that also manufactures Avastin, gained FDA approval for Lucentis, a GF blocker specifically designed to treat MD. Both drugs are given by injection into the eye. In 2007, Genetech sought to block the use of Avastin for treating MD. Protests from retinal specialists led to a reversal of this restriction. The principal difference between the two drugs is cost. A dose of Lucentis costs $2000, while a similar dose of Avastin costs $50.