REGULATORY CORNER

Let’s start by going into the way back time machine. It was in 1962 that Congress amended the Food, Drug and Cosmetic Act (FD&C Act) to require that drugs be effective as well as safe prior to approval for marketing. The law required that effectiveness of a drug was to be established by substantial evidence, defined as “adequate and well controlled investigations.” The standard FDA interpretation has been that “investigations” means more than one study. Thus, the requirement for two adequate and well-controlled studies. In 1997, Congress amended the FD&C Act to make it clear that FDA may consider data from one adequate and well-controlled study and confirmatory evidence sufficient to establish efficacy.

As many of us are well aware, while single arm trials (SATs) historically have been accepted by the US FDA as primary evidence of efficacy for marketing approval in the setting of accelerated approval (AA) of new oncology agents/indications, their acceptance in other therapeutic areas including rare diseases has been more selective.