Epidemiological or population studies
Epidemiological or population studies look at a large group of people in a specific region. Researchers will observe a disease that is present and study why people have it. These are sometimes called observation studies. They find links between an exposure and a disease. Here are three common types of these studies.
Cross-sectional
Cross-sectional studies are useful in seeing how common a disease is in a certain region. Researchers may start with a cross-sectional study to decide if another type of population study is needed later on.
A group of people is gathered to observe certain information at one point in time. For example, researchers may observe 1,000 people in the state of California to see who has lung cancer on June 1, 2025. These studies are inexpensive and quick. But since they are like instant "snapshots" of people, they can't explain what led to some people getting lung cancer.
Case-control
These studies are like looking at people's diaries. They gather two groups. In one group, the people (called cases) have the disease being studied. In the other group, or control group, they do not have the disease, but they are very similar to the cases in other ways. For example, participants may be the same age, live in the same area, or have similar lifestyle habits.
Researchers then look back into the past, usually relying on the participants' memories, to see any differences between the cases and controls. The researchers look at a specific exposure or potential link to the disease. Did the two groups eat differently? Were they exposed to certain chemicals? If an exposure stands out in only the cases or only the controls, that exposure could be linked to the disease.
Cohort
This type of study looks ahead. Researchers choose an exposure and the disease (or condition) that is expected to happen because of the exposure.
A large group of people, called a cohort, is observed by researchers over a period of time to see who develops the disease and who does not. They could be followed for any length of time, such as 5 years or more than 20 years.
Clinical trials
Clinical trials are another type of human study. Clinical trials usually, but not always, look at treatments.
Cancer clinical trials work with a group of people who have something in common (for example, a type of cancer or risk factor). Researchers decide on an agent or intervention for the group that may affect the disease or problem being studied. Examples of agents are a new medicine or high-dose vitamin.
You may hear the term placebo-controlled. This is when about half the group is given an agent or intervention and the other half is given a placebo. A placebo is a fake intervention—something that looks like the agent but has no effect at all. A placebo is usually not used when there is already an effective treatment for the condition. With cancer, an agent or intervention is usually compared to a standard cancer treatment. Sometimes cancer prevention trials use placebos, such as looking at the effects of a high-dose nutrition supplement on the prevention of cancer. One group of people would get the supplement and another group the placebo.
If two groups are used (receiving either an agent or a placebo), it is important that the trial is fair and the groups are as equal as possible. So the scientists may "blind" the participants (single-blind) or even themselves (double-blind) so that no one knows who is getting the placebo or the agent. They may also randomize the participants in the beginning. This means they will use an unbiased system, often a computer, to randomly select who will get the agent being tested.
New cancer treatments develop in clinical trials through these phases:
- Phase I gives a small number of people a new treatment or medicine, observes side effects, and finds the best dose and way to give (administer) the agent. This phase tries to find the highest dose of a new treatment that can be given safely without causing severe, life-threatening side effects. People who are very sick and not responding to other treatments may enroll here.
- Phase II looks at how well the new treatment works on a specific condition or disease using a specific dose. It continues to observe side effects. People who choose to enroll here also may have used the usual (conventional) treatments, and those treatments are no longer working for them.
- Phase III uses a larger number of people. It compares the effectiveness of the new treatment or a combination of treatments to an existing or standard treatment to find out which one may be better.
- Phase IV trials evaluate FDA approved medicines that have already been proven to be effective in Phase III trials. Phase IV trials focus on long-term safety and cost.
In addition to these four phases, the American Cancer Society identifies a Phase 0 for drug trials. Phase 0 clinical trials explore how a new medicine may work. Phase 0 information is included here because more volunteers with cancer may be asked to take part in these studies in the future. The goal of this phase is to speed up the approval of a new medicine.
- Humans are used in this trial, but Phase 0 is not like the other clinical trial phases.
- The biggest difference between Phase 0 and the other phases is that the person has no chance of benefiting from this trial. People in the future are the ones who will benefit from Phase 0 research.
- Phase 0 trials are exploratory, and the medicine is given in very small doses and for a short period of time.
- Researchers look for medicine absorption, whether the medicine reaches the tumor, how the cancer cells respond, and if there are any adverse medicine actions within the body.
Phase 0 trials are very small (often with fewer than 15 people) and are not widely used. Phase 0 research is not a requirement for testing a new medicine.
Clinical trials for new cancer treatments are always emerging. Talk with your doctor about whether you may benefit from being in a clinical trial.
Visit the National Cancer Institute's Cancer Trials website to find specific clinical trials. Or call the National Cancer Institute's Cancer Information Service at 800-422-6237 for more general information about clinical trials. You can also learn more about trials on the www.clinicaltrials.gov website, a service of the National Institutes of Health.