How Does a New Treatment Get FDA Approval?

Pancreatic cancer patients who participate in clinical research have better outcomes. Every treatment available today was approved through a clinical trial. The Pancreatic Cancer Action Network strongly recommends clinical trials at diagnosis and during every treatment decision.

FDA Drug Treatment Approval Process

When a drug or treatment enters the clinical trial process, it must pass through three phases of testing before becoming eligible for Food and Drug Administration (FDA) approval. Only if the treatment proves to be safe and promising at each phase, is it allowed to proceed to the next phase of testing.

Phases of Clinical Trials

Phase I
The first step in testing a new treatment or combination of treatments in humans is done during a Phase I trial. At this point, the experimental treatment has already shown some promise in the laboratory. A potential advantage of Phase I trials is that they usually have less strict eligibility requirements with regard to prior treatment than Phase II or Phase III trials, allowing patients who have had multiple treatments to participate.

Phase II
After a new treatment has proven to be safe in a Phase I trial, it proceeds to a Phase II trial. The goal of a Phase II trial is to determine the new treatment’s effect against pancreatic cancer. At this point, the maximum tolerated dose has already been determined, so participants will receive the highest safe dose of the new treatment. After completion of a Phase II trial, a new treatment usually must go through a Phase III trial in order to obtain FDA approval.

Phase III
The goal of a Phase III study is to test how the new treatment compares with the standard treatment. At this point, the treatments being studied have already shown to be safe (in Phase I trials) and promising (in Phase II trials). If a Phase III trial shows that the new treatment is more effective than current treatments and meets safety requirements, an application for FDA approval may be submitted.

Phase IV
Phase IV trials take place after a therapy has been approved by the FDA. The treatment is observed in larger populations to determine long-term safety and cost effectiveness, and to improve the management of side effects.

Clinical Trials Resources

Frequently Asked Questions
Free Patient Education Packet: includes Clinical Trials: Understanding How Pancreatic Cancer Clinical Trials Work

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FDA Drug Treatment Approval Process

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Clinical Trials Resources