FDA In Brief: FDA provides clarity regarding evidence needed to demonstrate effectiveness for drugs and biologics

December 19, 2019

Media Inquiries

The following quote is attributed to Amy Abernethy, M.D., Ph.D., FDA principal deputy commissioner:

“As our scientific knowledge advances, and drugs and biologics with novel mechanisms are being developed to treat conditions that were previously without treatments, there is a need for additional guidance on the flexibility in the amount and type of evidence needed to establish their effectiveness. For example, while randomized trials with a placebo control are often the best way to demonstrate effectiveness, in some situations, other designs can also meet the standard for approval.

“There are circumstances where evidence generated using a variety of clinical trial designs, endpoints and statistical methodology can support effectiveness, such as in certain drug development programs for rare diseases. Today, we are issuing a draft guidance to provide additional information to industry and stakeholders on evidence that meets the statutory standard for substantial evidence of effectiveness and reflects the evolving landscape of drug and biologic development.

“It is part of our longstanding commitment to flexibility when considering types of data and clinical development approaches with the ultimate goal of getting safe and effective treatments to patients faster. ”

Related Information

Today, the FDA issued the draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products.
This guidance complements and expands on the 1998 guidance, Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products.
Although the FDA’s standard for demonstrating effectiveness has not changed since the publication of the 1998 guidance, the evolution of drug and biologic development and science has led to changes in the types of product development programs submitted to the agency.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.