Important note: Information in this article was accurate in 1992. The state of the art may have changed since the publication date.
Food and Drug Administration, U.S. Department of Health and Human Services - December 8, 1992
Monica Revelle - (301) 443-4177
"These final rules will help patients who are suffering the most serious illnesses to get access to new drugs months or even years earlier than would otherwise be possible," said HHS Secreatry Louis W. Sullivan, M.D. "The effort to accelerate FDA review for these drugs has been a long-term commitment and indeed a hallmark of this administration."
These rules establish procedures for the Food and Drug Administration to approve a drug based on "surrogate endpoints" or markers. They apply when the drug provides a meaningful benefit over currently available therapies. Such endpoints could include laboratory tests or physical signs that do not in themselves constitute a clinical effect but that are judged by qualified scientists to be likely to correspond to real benefits to the patient.
Use of surrogate endpoints for measurement of drug efficacy permits approval earlier than if traditional endpoints -- such as relief of disease symptoms or prevention of disability and death from the disease -- are used.
The new rules provide for therapies to be approved as soon as safety and effectiveness, based on surrogate endpoints, can be reasonably established. The drug's sponsor will be required to agree to continue or conduct postmarketing human studies to confirm that the drug's effect on the surrogate endpoint is an indicator of its clinical effectiveness.
One new drug -- zalcitabine (also called ddC) -- was approved June 19, using a model of this process, for treating the human immunodeficiency virus, HIV, the cause of AIDS.
Accelerated approval can also be used, if necessary, when FDA determines that a drug, judged to be effective for the treatment of a disease, can be used safely only under a restricted distribution plan.
"The new rules will help streamline the drug development and review process without sacrificing good science and rigorous FDA oversight," said FDA Commissioner David A. Kessler, M.D. "While drug approval will be accomplished faster, these drugs and biological products must still meet safety and effectiveness standards required by law."
The new procedures also allow for a streamlined withdrawal process if the postmarketing studies do not verify the drug's clinical benefit, if there is new evidence that the drug product is not shown to be safe and effective, or if other specified circumstances arise that necessitate expeditious withdrawal of the drug or biologic.
FDA is a Public Health Service agency within HHS.
921208
FD921201
SOURCE: Food and Drug Administration (FDA).
AEGiS is a 501(c)3, not-for-profit, tax-exempt, educational corporation. AEGiS is made possible through unrestricted funding from Broadway Cares/Equity Fights AIDS, the Elton John AIDS Foundation, National Library of Medicine, and donations from users like you.
Always watch for outdated information. This article first appeared in 1992. This material is designed to support, not replace, the relationship that exists between you and your doctor.
AEGiS presents published material, reprinted with permission and neither endorses nor opposes any material. All information contained on this website, including information relating to health conditions, products, and treatments, is for informational purposes only. It is often presented in summary or aggregate form. It is not meant to be a substitute for the advice provided by your own physician or other medical professionals. Always discuss treatment options with a doctor who specializes in treating HIV.
Copyright ©1980, 1992. AEGiS. All materials appearing on AEGiS are protected by copyright as a collective work or compilation under U.S. copyright and other laws and are the property of AEGiS, or the party credited as the provider of the content. .