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 diseases.

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 marketing.

-- 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 dosages.

-- 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 later.

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 speedily.

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.