Food and Drug Administration, U.S. Department of Health
The Food and Drug Administration today announced immediate
implementation of a plan to reduce the time required for human
testing of drugs for life-threatening and severely debilitating
The following is the text of today's statement by FDA
Commissioner Frank E. Young, M.D., Ph.D.:
Today, we at the Food and Drug Administration are announcing
immediate implementation of a plan to reduce the time required
for human testing of drugs for life-threatening and severely
debilitating diseases... conditions such as AIDS, Parkinson's,
deadly cancers and blindness.
This plan was developed at the request of the President's Task
Force on Regulatory Relief, chaired by Vice President George
Bush. The Vice President asked FDA to take into account the
urgent needs of people with life-threatening diseases, such as
AIDS, in developing research requirements.
The procedures spelled out in the plan reflect a recognition
that physicians and patients are generally willing to accept
greater risks or side effects from products that treat
life-threatening or severely debilitating diseases. The
procedures also reflect a recognition that the benefits of a
drug need to be evaluated in light of the severity of the
disease being treated.
The resulting plan has one primary aim: To move promising
therapies through research more quickly and make those
therapies available earlier to our patients. The plan
encourages researchers to accelerate and condense their
studies... assures that FDA will realistically balance the
risks and benefits of promising drugs in light of the severity
of these conditions.
The thrust of the plan involves FDA working with drug sponsors
early in the process to agree on the most time-efficient animal
and human studies necessary to answer safety and efficacy
questions. By this cooperation, we hope to enable sponsors of
drugs for life-threatening diseases to eliminate a major phase
of their human studies, Phase Three. Where this and other
aspects of the plan work fully, we hope this can cut up to
one-third to one-half of the time required for most standard
studies in humans today.
You know that FDA does not itself conduct clinical trials and
it is not FDA's review, but research by drug sponsors in human
volunteers, that is the most time-consuming part of drug
development. Thus, to reduce the time it takes to move a
chemical to the point where it can be approved as a drug, you
have to attack not only FDA review time but the research time
required of the drug sponsor doing animal and human studies.
(Refer to Chart 1) Scientists at a drug company, university or
government facility ordinarily take three to seven years for
tests of drugs in humans, following laboratory and animal work.
For many products, such a research pace may be cost-effective
and appropriate. But for treatments for AIDS and the
AIDS-associated complications and opportunistic infections, for
example, as well as for other life-threatening conditions, we
want to reduce that time to the bare minimum. That's why AZT
or zidovudine, the only proven drug for AIDS itself available
anywhere in the world, got such special treatment here --
including 1) availability, a week after the study was
terminated, for 4,000 AIDS patients under a special
pre-approval treatment IND program, and 2) final approval for
thousands more in three and one-half months after receipt of
the drug sponsor's application.
I think it is important to note that if a drug is going to fail
to be useful or is too risky, researchers generally learn this
early on. (Refer to Chart 2.) You see that the dropout rate
is highest in the early stages.
(Refer to Chart 3) The new plan deals not only with FDA's
review of a drug, but with the time-consuming work of
researchers BEFORE application is made to FDA. Here's the key:
Under the plan, drug researchers could intensify their efforts
early in a drug's development and thereby eliminate -- for
life-threatening diseases -- the last of the three traditional
phases of human drug research.
In determining if a drug can then be approved, FDA will
consider if the benefits of the drug outweigh its known and
potential risks, including the questions remaining about the
drug, and also take into consideration the severity of the
disease and the absence of satisfactory alternative therapies,
as well as the statutory criteria for approval.
Thus, a treatment could be approved even if some questions
remain to be answered by researchers. There might be questions
about the very best way to use a drug -- the lowest dose that
was effective, for example.
Such questions could be studied after the drug was approved --
and would not delay its approval.
To reduce or eliminate some of the delays that have occurred in
the past, FDA:
-- Will offer to meet with drug sponsors to devise the kind of
animal tests that can get a drug into clinical testing, in
humans, with safety, in the minimum time.
-- Will offer to meet with the drug sponsor after the very
first human testing, in small numbers of people, to help devise
Phase Two clinical testing that provides sufficient data for
approval without the need for the traditional Phase Three.
-- Will actively monitor and evaluate clinical trials with an
eye toward facilitating them.
-- Will continue to safeguard patient safety via a variety of
mechanisms by incorporating informed consent and institutional
review board approval during the clinical trial stage, as well
as adverse drug reaction reporting throughout the research,
approval and post-marketing stages.
-- If preliminary analysis of the Phase Two results appears
promising, will provide a treatment protocol so groups of
desperate patients can receive a drug before its approval for
-- While statutory standards of safety and effectiveness apply
to all drugs, will review drugs for life-threatening conditions
on the basis of risk-benefit analysis, keeping in mind the
severity of the disease.
-- When appropriate, approve a drug and seek an agreement from
the sponsor to conduct certain postmarketing studies providing
additional information on risks, benefits and optimal uses and
-- Focus FDA's regulatory research on issues that may help
products be evaluated earlier.
The early release of experimental drugs to patients in
life-threatening situations -- the "Treatment IND" -- meshes
with today's policy. It was announced initially on May 22,
1987, also at the Task Force's request, and took effect a month
FDA has long given AIDS-related products "1-AA" status. This
means, for example, that our drug reviewers and staffs give
highest priority to potential AIDS therapies.
This new plan, and our previous steps, show, I believe, that
FDA is not "part of the problem" but an integral, aggressive
part of the solution to AIDS and other diseases. In this plan,
we have worked to promote the best science -- in the best time.
This plan is another step, a particularly significant step,
along the path toward providing therapies quickly for the
desperately ill. It recognizes that that short pathway
involves well-controlled trials designed to answer questions
I don't want to over-promise, however. This procedure won't
create breakthroughs where they don't exist. It won't create
genius, or sudden insights into the workings of a disease. It
won't eliminate the need for brainpower, money and hard work --
and, I might add, prayer -- but it can help medical scientists
capitalize more quickly on any breakthroughs we realize.
I hope it will help ensure that some key drugs and biologics go
through human research and FDA's review in the minimum time. I
hope it will let scientists seize upon promising therapies and
move them more quickly to where they count, our patients.