New Cancer Treatments Approval Process

by National Cancer Institute

Understanding the Approval Process for New Cancer Treatments

Every year, medical researchers develop new cancer treatments or new uses for treatments already on the market. These treatments are most often drugs, chemically produced substances used to treat or prevent disease. But they may also be biologics, treatments that are made from living organisms such as vaccines or recombinant proteins.

The U.S. Food and Drug Administration (FDA) is the division of the U.S. Department of Health and Human Services charged with making sure that drugs and biologics are safe and effective before they go on the market. The FDA maintains a list of drugs and biologics approved for use with cancer.

FDA regulators face two main challenges during the approval process. First, they must make sure the treatment is safe and effective. For this, regulators rely on the results of clinical trials - research studies that test how well medical treatments or other interventions work in people.

The second challenge is to make promising treatments available as quickly as possible to the people most in need of them. Ordinarily this occurs through clinical trials. The FDA may also allow access to an unapproved investigational treatment outside of a clinical trial, if no approved therapy for the disease exists.

This guide will acquaint you with the main parts of the FDA approval process and point you to other resources for learning more about it.

The FDA's Role

One Example: Herceptin®

Before September 25, 1998, women with advanced breast cancer who wanted to take the drug Herceptin needed to enroll in a clinical trial. But after that date, they could obtain it through their doctors, like any other prescription drug. That's because Herceptin had received official approval from the U.S. Food and Drug Administration (FDA).

In the months leading up to approval, researchers had reported promising results from studies of women with advanced breast cancer whose tumor cells had extra copies of a protein called HER2. Herceptin is designed to target that protein and kill the cancer cells, leaving healthy cells alone. One group of researchers found that women who took the drug along with standard chemotherapy survived longer than those who recieved only the chemotherapy. Another group found that Herceptin alone could help some women whose cancer was not responding to chemotherapy.

A few months later, after carefully reviewing the results and weighing the benefits against the risks of side effects, the FDA approved the drug for use in women with HER2-positive, advanced breast cancer.

Approval is only one step in the process by which new treatments are developed. In fact, the FDA estimates that, on average, it takes 8.5 years to study and test a new drug before it can be approved for the general public. That includes early laboratory and animal testing, as well as the clinical trials that evaluate the treatment in humans. The FDA plays a key role at three main points in this process:

• Determining whether or not the benefits of a new treatment outweigh the risks.

• Once clinical trials begin, deciding whether or not they should continue, based on reports of the treatment's side effects and effectiveness against disease.

• When clinical trials are completed, deciding whether or not the treatment should be sold to the public and, if so, what claims the drug manufacturer can make and what the label should say about directions for use, side effects, and warnings.

To make these decisions, the FDA must review studies submitted by the drug's sponsor (which is usually the company that makes the drug), evaluate any reports of side effects or complications (called "adverse events") from preclinical studies and previous clinical trials, and review the adequacy of the chemistry and manufacturing.

This process is lengthy, but it is meant to ensure that only beneficial treatments with acceptable side effects will make their way into the hands of the public. At the same time, recent laws and streamlined procedures within the FDA have accelerated the approval of effective treatments, especially for serious illnesses such as cancer. In addition, specific provisions make some treatments available to patients with special needs even before the approval process is complete.

From Lab to Patient Care

By law, the FDA must review all test results for new treatments to ensure that products are safe and effective for specific uses. "Safe" does not mean that the treatment is free of possible adverse side effects; rather, it means that the potential benefits have been determined to outweigh any risks. The testing process begins long before the first person takes the treatment, with preliminary research and animal testing.

If a treatment proves promising in the lab, the drug company or sponsor must apply for FDA approval to test it in clinical trials with people. The application is called an Investigational New Drug (IND) application. For drugs and recombinant proteins (such as cytokines and monoclonal antibodies), sponsors submit the IND to the Center for Drug Evaluation and Research, or CDER. For other biologics, including gene therapies and vaccines, sponsors submit the IND to the Center for Biologics Evaluation and Research (CBER).

Once the IND is allowed to proceed by CDER or CBER, clinical trials can begin. If the treatment makes it through the clinical trials process - that is, if the studies show the treatment is safe and effective - the sponsor may submit to the FDA another application. For drugs, this is a New Drug Application (NDA); for biologics, it's a Biologics License Application (BLA). The application must include the following:

• The exact chemical makeup of the drug or biologic
• Results of animal studies
• Results of clinical trials
• How the drug or biologic is made, processed, and packaged
• Quality control standards

Once the FDA receives the NDA or BLA from the sponsor, the formal New Drug Application Review Process or Biologics/Product License Application Review Process begins.

About the Author

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