USA – FDA Drafts Guidance on Demonstrating Substantial Evidence of Effectiveness

The US Food and Drug Administration (FDA) on Thursday issued draft guidance on demonstrating “substantial evidence” of effectiveness for drugs and biologics, expanding on decades old guidance issued to address statutory changes brought on by the Food and Drug Administration Modernization Act of 1997 (FDAMA).
 
Specifically, the 18-page draft guidance builds on FDA’s 1998 guidance Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products, which detailed FDA’s expectations for evidence to support the approval of new drugs and biologics or applications for supplemental indications in light of FDAMA’s clarification that a single adequate and well-controlled clinical trial and confirmatory evidence can be used to support approval.
 
While FDA says its “standard for demonstrating effectiveness has not changed” since the 1998 guidance, the agency says that new guidance is necessary to keep up with scientific advancements and changes in drug development over the last two decades…