FDA Approval Process for Cancer Drugs


The U.S. Food and Drug Administration (FDA) is an agency within the U.S. Department of Health and Human Services. The FDA works to protect the public health by regulating food, medications, supplements, and cosmetics by making sure they are properly labeled and tested before being sold.

The FDA is made up of five centers, each with special responsibilities. The center responsible for monitoring drug development is the FDA’s Center for Drug Evaluation and Research (CDER).  Within this center, the Office of Hematology and Oncology Drug Products works specifically on cancer drugs and treatments.

After researchers develop a potential new cancer drug, the first step is laboratory testing on animals whose cancer cells behave like humans do. This allows researchers to determine if the drug is safe to test on humans and if it works well enough to move forward. The rest of the process can become very costly, so researchers usually have a sponsor to help with the expenses of the research process. Pharmaceutical companies, government agencies, and health care organizations all sponsor trials.

After gathering data from animal research, the sponsor of the trial submits an Investigational New Drug (IND) application to the FDA. The IND application includes detailed information on the drug including

  • What the drug’s ingredients are
  • How the drug acts in the body
  • How the drug is made and by whom
  • Who the principal investigators (main researchers) are in the study
  • The results from animal testing
  • Detailed plans, including a very specific outline of how the trial will be conducted (the trial’s protocol)

If the FDA determines that the benefits of the trial outweigh the risks, the trial may begin recruiting participants. Within 21 days of enrolling the first participant, the sponsor must register the trial with ClinicalTrials.gov. ClinicalTrials.gov is run by the National Institutes of Health (NIH). (Note: The information in My PearlPoint’s clinical trial locator is directly pulled from the NIH’s database.)

Once clinical trials begin, there are three phases of testing.

Phase I: The first step is to determine the safety of the drug and the potential side effects. Phase I trials usually recruit fewer than 100 participants. These are also known as “first in man” trials.

Phase II: Next, researchers determine if the drug works for its intended purpose in a group of several hundred participants.

Phase III: Finally, trials gather more information about safety and effectiveness in a wider population of 1,000-3,000 participants.

After collecting enough information from clinical trials to prove that the treatment is safe and effective, the sponsor completes and submits a New Drug Application (NDA).  The NDA has to provide enough information for the FDA to know the following:

  • The drug is safe and effective for its intended use.
  • The labeling and instructions to accompany the drug are appropriate.
  • The manufacturing methods for the drug will ensure the drug’s quality.

If the NDA proves the above factors, the FDA approves the drug.  The FDA regularly provides updates on approved drugs on the FDA website. If not approved, the FDA sends a response letter to the sponsor rejecting the application.

After the FDA approves a drug, the sponsor is still required to report safety updates to the FDA as needed. The updates often come from Phase IV clinical trials. If patients or healthcare providers notice an unlisted side effect, they can also report the event through the FDA’s MEdWatch program. If new side effects are discovered, the drug’s labeling is changed and the public is informed. If a new side effect is deemed too dangerous, the FDA revokes approval.

During the approval process of a new drug, a drug may be given a special designation. Some of these designations include

Fast Track Designation: Speeds up the review of drugs that fulfill an unmet need to treat a serious condition

Breakthrough Therapy Designation: Speeds up the review of drugs that offer benefits over existing treatments for serious or life-threatening illnesses

Orphan Drug Designation: Given to a drug for the treatment of a condition that affects fewer than 200,000 people in the United States yearly, and the drug offers a significant advantage over existing treatments

There are also other ways a drug’s approval may be sped up, including

Accelerated Approval: A program designed to speed that approval of promising drugs that treat serious or life-threatening illness and provide benefit over current treatments. This program is mostly used for drugs to treat chronic diseases or diseases that can last a long time. It may take a long time to evaluate the long-term  clinical value. Even after the drug is approved, the sponsor may have to submit more information to the FDA to prove the long term benefits.

Priority Review: The standard review timeline is 10 months. Drugs granted priority review have a timeline of only 6 months.


U.S. Food and Drug Administration (FDA)


Related Resources

All About Cancer Research

Why Should I Know About Clinical Trials?

Next Steps: How To Talk To Your Doctor About the Latest Research

Why Should I Consider a Clinical Trial?