how Drugs Are Approved

It takes, on average, about 12 years and typically costs more than $350 million for a promising treatment to journey from “bench to bedside,” a path that ensures that it is safe and effective for humans before it is approved for routine clinical care. The journey involves:

The first studies take place in a laboratory, where researchers study how the drug works in test tubes or cell cultures. These are followed by studies in  animals—typically mice or rats that have been bred to develop cancer or are injected with cancer cells. These studies further illuminate how a drug works and also identify potential toxic side effects. It typically takes about three and half years for scientists to conduct the studies and to collect the data they will need to get permission from the U.S. Food and Drug Administration to begin testing the drug in humans.

The first studies take place in a laboratory, where researchers study how the drug works in test tubes or cell cultures. These are followed by studies in  animals—typically mice or rats that have been bred to develop cancer or are injected with cancer cells. These studies further illuminate how a drug works and also identify potential toxic side effects. It typically takes about three and half years for scientists to conduct the studies and to collect the data they will need to get permission from the phases, each successively enrolling an increasing number of participants. These are the studies that show whether a drug that appeared safe and effective in the laboratory is indeed effective and safe in humans.

Phase I Trials
Phase I trials mark the first time a new treatment is tried in humans. These trials are designed to determine if the new drug (or drug combination) is safe. This means that the primary focus—in addition to seeing if the treatment has any effect on the tumor—is on whether the drug might hurt the kidney, liver, or other organs.

Phase II Trials
If a phase I trial is successful, the researchers will initiate a phase II trial, which will look at the drug’s safety and effectiveness in a larger number of people. If the phase II trial indicates that the drug is effective, the next step is to conduct a phase III randomized trial.

Phase III Trials
Phase III trials are the final step in the research process. They include hundreds or even thousands of patients who are randomly assigned to either the new drug (or drug combination) or the standard of care. Phase III trials allow researchers to see if the drug’s ability to slow or stop tumor growth that was observed in the small phase II trial is real or may have just have occurred by chance. It also allows the researchers to see if the new drug (or new drug combination) is more effective than the current standard of care, which it is being compared to in the study.

Seeking Approval
Scientists know that one study is never enough to prove something is true. A study’s finding must be replicated and confirmed (often many times) before the results are considered to be definitive. That’s why frequently cancer scientists in other laboratories will try to reproduce the results of cell or animal studies, or clinical trial investigators will test a drug in new populations of cancer patients. This ensures that the studies were done right, that the results were not a fluke, and that they hold true for different groups of people. It also enables a broader assessment of risks and benefits. When replication leads to conflicting results, even more studies are done.

After all of the studies are completed, scientists present their findings to the FDA, which must determine if the drug is safe, effective, and can be approved as a breast cancer treatment. You can learn more about how the FDA’s role in the approval of new cancer treatments here.

As you can see, it would not be possible for new treatments to be approved without research volunteers. To ensure the safety of these volunteers, there are many rules and regulations that researchers must follow. For example, researchers are not permitted to recruit volunteers for a study until they have received approval from an Institutional Review Board (IRB).

An IRB is an independent committee that approves, monitors, and reviews research involving human subjects in order to protect research subjects’ rights and welfare. After the study gets underway, the IRB continues to monitor the trial to be sure the researchers are recruiting volunteers appropriately and conducting the trial properly. The IRB also periodically reviews the data collected by the study to determine if the trial should be stopped at any point, for any reason.