Research in which people, or data or samples of tissue from people, are studied to understand health and disease. Clinical research helps find new and better ways to detect, diagnose, treat, and prevent disease. Types of clinical research include clinical trials, which test new treatments for a disease, and natural history studies, which collect health information to understand how a disease develops and progresses over time.

What type of research is clinical?

Clinical research is the study of health and illness in people. There are two main types of clinical research: observational studies and clinical trials.

Types of clinical trials

1. Pilot studies and feasibility studies.
2. Prevention trials.
3. Screening trials.
4. Treatment trials.
5. Multi-arm multi-stage (MAMS) trials.
6. Cohort studies.
7. Case control studies.
8. Cross sectional studies.

What is clinical research role?

The primary roles and responsibilities of clinical research associates include conducting regular site visits and managing data accumulated during trials. They also review study progress, identify and mitigate trial risks and report any issues to the sponsor.

What is the main goal of clinical research?

The goal of clinical research is to develop knowledge that improves human health or increases understanding of human biology. People who take part in clinical research make it possible for this to occur.

How does a clinical trial work ?

Clinical trials occur in four phases and each phase has a different purpose.

1. Phase I → Focus on safety and the proper dose. → 15 to 50 patients
2. Phase II → Focus on effectiveness and side effects. → less than 100 patients
3. Phase III → Compares the new treatment to existing treatment → Hundreds of people
4. Phase IV → Treatment is approved and Available. Long-term effects are observed → Thousands of people

1. Phase I Trials

Test if a new treatment is safe in people. Doctors also find the best way to give the treatment.

The goal of a Phase I trial is to:

• Find out if a new treatment is safe.
• Find the best way to give the new treatment, such as by mouth or by vein.
• See if there are signs that cancer responds to the new treatment.

Phase I trials usually include 15 to 50 patients who are divided into small groups. These groups are called cohorts. The first cohort receives a dose of the new drug. Doctors may collect blood or urine samples to measure drug levels in the patients.

If the first cohort does not have any severe side effects, then a new cohort receives a higher dose of the same drug. The dose increases with each new cohort until the doctors find the best dose for future testing. With each increasing dose, doctors test each patient to see if he or she is responding to the treatment. If the doctors find that the treatment is safe, then it will move forward to be studied in a Phase II trial.

2. Phase II Trials

Test if a new treatment works in one type of cancer.

Fewer than 100 patients usually join a Phase II trial. Even though the main goal is to see if the treatment works, doctors still closely watch patients’ side effects. If the new treatment works, doctors may go on to study it in a Phase III trial.

3. Phase III Trials

Test if a new treatment is better than standard treatment.

Phase III trials may include hundreds to thousands of patients around the country or world. Each patient enrolled in a Phase III clinical trial has a chance of being in one of the following groups:

• Control group – the group that gets the standard treatment
• Study group – the group that gets the new treatment being tested

Doctors do not know if the new treatment is better than the standard treatment, but they believe it is as good and may be better.

After the Phase III trial, the FDA reviews the clinical trial results to make sure the treatment is safe and effective for people to use. The FDA decides whether to approve the treatment so that it is available for all patients.

There are many frequently asked questions about Phase III trials. These include:

How are patients put into groups?

A computer decides which patients are in the control group and which patients are in the study group. Patients have a chance of being in either group. The patient and doctor do not decide. It is random and due to chance alone. This helps to avoid bias in the clinical trial. (Bias happens when human choices affect a study’s results.)

Would my doctor know which group I am in?

In single blind studies, patients do not know whether they are in the control or study group, but the doctor does. In double blind studies, neither the patients nor the doctors know which patients are in each group. (In case of an emergency, doctors can find this information in the study file.)

Would I be given a placebo?

A placebo is something that looks like medicine, but is not. If a placebo is used, it is given together with the best standard treatment. This allows doctors to compare standard treatment alone to standard treatment with a new drug. If there isn’t a standard treatment, then the placebo may be given alone, but this is not common in cancer trials.

4. Phase IV Trials

Find more information about long-term side effects.

In Phase IV trials, doctors study treatments that the FDA has already approved. The goal of Phase IV trials is to continue studying side effects of a new treatment