PhRMA Challenge On Drug Trial Costs

Letter to the Editor:

James Love in the January 13 Marketletter doubts the accuracy of the Tufts and OTA [Office of Technology Assessment] estimates of the cost of new drug development. His basis for questioning the estimates is the discrepancy he finds between the Tufts/OTA estimate and the average cost of developing an orphan drug, as reflected in data from Orphan Drug Tax Credit payments.

The difference in data is easily explained. Clinical trials for orphan drug candidates nearly always involve far fewer patients. The reason, of course, is there are fewer patients available with rare diseases and they are often geographically dispersed. Also, it is clear that FDA [the US Food and Drug Administration] applies a more lenient standard to approval of orphan drugs, in some cases, granting approval on the basis of existing literature.