Clinical trials

The clinical trial journey determines whether a medical intervention, such as a drug, a device, or a medical procedure should move from an idea into patient care. Let’s look at an example of what happens in the research lab before clinical trials begin.

The journey to improve patient care starts in the lab with discovery science. During the discovery phase, scientists ask and answer a wide variety of research questions using computers, cells and animal models. For example, what changes in a cell lead to cancer and why isn’t the cell working normally? The goal is to eliminate unlikely hypotheses so scientists can choose a research path with the most promise.

Discovery research builds a strong foundation for future scientific advances to improve the health of patients. Let’s look at one example of how discovery science works. Dr. Hernandez leads a research lab. She and her team wants to study how cancer cells are different from normal cells. Cancer cells emerge because of damage to a gene, which changes what the cell does when it grows and when it dies. The team examines cells in the lab and creates a model to show how cancer develops. Their goal is to use this model to find new ways to treat cancer. After many years of effort, including studies on toxicity and other modeling and testing. Dr. Hernandez and her team publish a paper on their work in a scientific journal. Another researcher Dr. Petzi reads the paper and approaches Dr. Hernandez with an idea. Dr. Petzi’s lab focuses on testing drugs to find new options for cancer treatment. Dr. Petzi would like to combine forces with Dr. Hernandez to find a drug to prevent cancer tumors from growing. They share data and models. Eventually, the researchers find a new drug that shows promise in the lab. Next, they examined drug dosage in cell and animal models. The team presents their research at a conference. This leads to collaborations with other research institutions. Together they move the discovery into the next step. The next step is protocol review and approval.

Before starting clinical trials, which involve human volunteers, the researchers must develop a research plan called a protocol and send it to the US Food and Drug Administration for review and approval. They also must get approval from a group at their organization that is responsible for protecting the rights and welfare of human research participants. The review process is rigorous and not all studies get the green light to move forward. After receiving approval, the experimental treatment is ready to move out of the lab and begin the clinical trial journey.