A Short Tour

What is the U. S. Food and Drug Administration (FDA)?

The FDA is a Federal agency within the Department of Health and Human Services with the responsibility to regulate and evaluate products for human and animal use that are applied to or taken into the body.

What are the basic principles and mandate of the FDA?

The FDA evolved during the 20th century on the basis of three principles, each mandated by Acts of Congress. They are that products should be labeled with the ingredients, that the product should be safe, and that the product should be effective. These principles have evolved into an organization based on scientific principles that:  

• performs risk analysis and assessment to monitor studies on unapproved products,
• evaluates claims about products for safety and efficacy to grant marketing licenses (the approval process), and
• monitors products after approval for continued risk assessment.

For more information about FDA, please visit the history of the FDA, the history of the Center for Drug Evaluation and Research, frequently asked questions addressed to CDER, and general information about the FDA.

What is the basis of FDA authority?

Authority for the FDA comes from laws from the Legislative branch and regulations, which are interpretations of laws, from the Executive branch of the Federal government. The basic law is found in the Food, Drug and Cosmetic Act and the regulations are found in the Code of Federal Regulations. The FDA web site also contains a more complete list of laws and regulations that pertain to the FDA.

How are studies conducted and regulated?

A product must first be investigated in humans to decide if it is safe to use.   Then the efficacy of the drug, its effectiveness in treating a particular disease, is studied.  In order to perform studies in humans, an applicant must file for an Investigational New Drug Application (IND).  Both the FDA and local authorities, in the form of an Institutional Review Board (IRB), must review the proposed study to assess the appropriateness and ethical issues involved in it.   There is more information on this web site about the general process for filing an IND for a drug, filing an IND for a biological, or general information about IRBs. 

In addition to proposed human studies, an IND application will contain information based on non-clinical data, usually in two species of animals, a proposed study plan or protocol, and a rationale for the projected dose and schedule. We have included a flow chart to help select the starting dose for cytotoxic drugs in oncology.

How are Investigational New Drug (IND) applications reviewed?

An FDA multidisciplinary team of scientists conducts a review of a proposed study, by law, within 30 days of receipt.  This team determines whether it is safe to proceed with the proposed study, that is whether it has adequate safeguards to protect study subjects.  Clinical study proposals or protocols are evaluated on the basis of safety and the likelihood of providing an answer to a scientific question.   There is no endorsement implied about the efficacy of the drug under study.

Permitting a study to proceed is not the same as approving a claim for marketing a product.  The law prohibits charging for or promoting a drug that has not been approved for marketing.  Reimbursement is permitted, however, for the costs of administering an investigational drug and for general patient care, including treatment for any toxicity that may result.

Oncology Tools includes some of the assessment tools for conducting clinical investigations, such as a staging manual for different types of cancers, scales for assessing patient performance status, standard tables to describe adverse events (toxicity), and calculators for human and animal dosing and human fluid requirements.   For more specific information, you may wish to contact an FDA review division.

How is access to investigational drugs obtained?

Investigational drugs are typically used after conventional approved therapy has been attempted and there is further progression of the disease.  In some circumstances, when there is no effective approved therapy, immediately enrolling in a study of an investigational drug may be appropriate.  This site contains some general advice on how to obtain access to investigational drugs.  More detail about some ongoing studies in oncology are covered under the Clinical Trial Listings here.  Although the listing from the National Cancer Institute is extensive, it does not contain all ongoing studies; no FDA endorsement is implied of those that are listed. 

What are the steps to approval?

If there are sufficient data to consider a product safe and effective for a particular use, the information is summarized and assembled into a New Drug Application (NDA), or the equivalent for biologicals and devices, with a proposed claim about its use.  A multidisciplinary team of FDA scientists conducts a review, by law, within either 6 months (priority) or 10 months (standard) of receipt of the NDA.  The team determines whether the proposed claim can be granted a marketing license based on the potential risks and benefits.  The CDER web site describes the general process for filing an NDA for a drug and, in the Drug Application Approval Process, on preparing and submitting drug applications.  For an overview of new drug development, see the CDER Handbook.

In some cases, the FDA seeks the advice of a panel of experts, known as an advisory committee, to help decide whether a claim should be approved.  The FDA web site provides general information about FDA advisory committees.  Another Oncology Tools web page has detailed information about the regulatory process as it applies to oncology, including guidance documents, forms, contact information, details about the Oncologic Drugs Advisory Committee with a calendar and an index of their meeting transcripts, and much more.

What does FDA approval mean?

It is important to recognize that it is never a drug or other product that is approved or not approved, but a claim about the use of the drug or product. The claim is granted a marketing license, which is, in effect, a product label.  The FDA has approved about 90 drugs for use in oncology.  Oncology Tools has detailed information about approved oncology drugs, including uses, product labels, and approval summaries. 

There are a number of reasons the FDA may not approve a product claim, including:

• insufficient evidence to support the claim;
• the risks are considered unacceptable; or
• the FDA and the sponsor cannot come to agreement about the scope or wording of the claim.

The majority of oncology drugs that are not approved on the basis of an initial claim are subsequently approved upon resubmission of either more data or a revised claim.

Neither the FDA nor the Federal government regulate the practice of medicine. Any approved product may be used by a licensed practitioner for uses other than those stated in the product label. Off-label use is not illegal, but means that the data to support that use have not been independently reviewed by the FDA.

There is an obligation to report adverse events, particularly those not previously described in the product label or that result in hospitalization or death.   There is more information here about reporting adverse events.

We are interested in any comments you may have about the Oncology Tools web page.

FDA/Center for Drug Evaluation and Research
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