The Food and Drug Administration (FDA) today announced steps to
advance the earliest phases of clinical research in the development of
innovative medical treatments. FDA’s goal is to improve the process for
bringing safe and effective drugs for potentially serious and
life-threatening diseases, such as cancer, heart disease and
neurological disorders, to the market.
In guidance documents released today, Exploratory IND Studies and
INDs—Approaches to Complying with CGMP During Phase 1, the FDA lays out
specific approaches for researchers who are planning to conduct very
early clinical studies in people and offers approaches for performing
appropriate safety testing and producing small amounts of drugs safely.
In line with the aims of FDA’s Critical Path Initiative to modernize the
drug development process, these changes will enable U.S. medical
researchers to evaluate much more efficiently the promise of scientific
advances discovered in their laboratories.
“Currently, nine out of ten experimental drugs fail in clinical
studies because we cannot accurately predict how they will behave in
people based on laboratory and animal studies,” said Health and Human
Services Secretary Mike Leavitt. “The recommendations announced today
will help more researchers conduct earlier, more-informed studies of
promising treatments so patients have more rapid access to safer and
more effective drugs.”
The Exploratory IND Studies guidance will facilitate very early
exploratory scientific studies in people before the standard safety
studies (phase 1) begin. Because only small amounts of drugs are used in
these early studies, they represent fewer potential risks for people in
these trials. In the final version of the guidance Exploratory IND
Studies, FDA makes recommendations about safety testing, manufacturing,
and clinical approaches that can be used in these very early studies.
The guidance explains how medical researchers can take full advantage of
the flexibility built into existing regulations in the amount of data
needed when asking the FDA’s permission to proceed with such a study,
enabling more rapid delivery of innovative products to patients.
“One of the biggest barriers research and academic institutions face
is the ability to get discoveries made in the lab into clinical testing.
The new Exploratory IND guidance emphasizes the flexibility available to
researchers when conducting early clinical testing of these cutting-edge
treatments,” said Andrew von Eschenbach, MD, Acting FDA Commissioner of
Food and Drugs. “As we enter the era of personalized medicine, these
exploratory approaches enable scientists to take full advantage of new
technologies to target the development of more individualized
In related draft guidance, INDs—Approaches to Complying with CGMP
During Phase 1, the FDA outlines a suggested approach to complying with
current good manufacturing practice (CGMP) requirements for drugs
intended for use solely in phase 1 studies. With this new guidance and
an accompanying regulation, FDA formally recognizes specific standards
for the manufacture of small amounts of drug product for phase 1 studies
and formulating an approach to cGMP compliance that is appropriate for
the particular stage of drug development.
“The problem is that researchers conducting very early studies were
required to follow the same manufacturing procedures as those companies
that mass produce products for broad scale distribution," said Janet
Woodcock, MD, FDA Deputy Commissioner for Operations. "These
requirements are so burdensome for early phase 1 studies that many
leading medical research institutions have not been able to conduct
these studies of discoveries made in their laboratories. Today, for the
first time, medical researchers are getting specific advice from the FDA
about how to safely prepare products for exploratory studies."
The documents released today are part of FDA’s commitment to
modernize existing CGMP regulations to streamline clinical development.
These efforts are part of the Agency’s Critical Path Initiative,
launched in a March 2004. The goal of the Critical Path Initiative is to
reduce the time and resources expended on candidate products that are
unlikely to succeed, by creating new tools to distinguish earlier in the
process those candidates that hold promise.