Research and Clinical Trials: An Overview

What are the different kinds of research?

A lot of research is conducted to learn more about diseases that only happen in children, or that have a different course in children than in adults. Doctors, nurses, and other health professionals also study children’s mental and physical development in research that can ultimately benefit all children.

Different types of research studies include:

  • “chart reviews,” which look at children’s existing hospital records to study what happened before, during and after treatment of a disease
  • questionnaires, surveys, and interviews, used to collect information on disease outcomes, behaviour, opinions, quality of life, or patient satisfaction
  • standard research on sick patients using physical examinations, imaging, and specimen collection to study diseases or diagnostic methods
  • intervention studies, which look at a therapy such as a drug, medical device, surgery, or physical therapy. Intervention studies include clinical trials.

What is a clinical trial?

A clinical trial is a research study that examines the use of drugs or other therapies in treatment. It asks a specific question to try and find better ways of preventing, diagnosing, or treating different medical conditions. For example, clinical trials could study new drugs or new combinations of known drugs.

Usually, there are four different phases of clinical drug trials. Phase I and Phase II trials determine the safety of a drug. By the time a treatment reaches a phase III clinical trial, it has been well studied, deemed safe for humans. Phase III trials are conducted by an organizing group of experts and under a set of strict rules called a protocol.

Why is research important?

Research is the only way of improving the success of treatments. To show how dramatic the effect of research can be, consider the case of immature lungs in premature babies. In the 1960s, premature babies often went on to develop severe and permanent lung problems as adults. This was because their lungs had not developed enough before they began to be used for breathing. Through research, it was found that premature babies given surfactant therapy went on to develop healthier lungs. This research took place in the form of clinical trials. Each time a trial showed an improvement with treatment methods with surfactant, this new treatment could be used more effectively with more children.

How are new drugs developed?

Drug development usually takes a lot of time and research. Researchers, knowing the properties of certain molecules and the working of the body, speculate that a compound may have some benefit. This benefit and the safety of the drug in general are then demonstrated in animals. This stage is called preclinical research. After a drug has shown promise, it will be tested in a clinical trial with humans. Clinical trials move through various phases.

After each clinical trial, the researchers will write about their findings. They will describe the procedure, the participants, treatment results, side effects, and their understanding of the results. The researchers will send their report to a scientific journal. Other experts who are not connected to the study will read and analyze this report. This is called peer review. If these experts agree that the trial was of high quality, the report will be published.

After reading the report, other researchers in different cities and countries should be able to repeat similar trials to see if they get the same results. Larger and larger numbers of patients will participate.

After a number of trials that show its effectiveness, a drug or treatment is approved by a national regulatory body, such as Health Canada, the U.S. Food and Drug Administration, or the Medicines Control Agency in the United Kingdom. Usually, it is first approved for a very particular disease and situation. Later trials help pave the way for the drug to be approved for use with different diseases or age groups.

This long process shows why the discovery of “miracle cures” in science is so rare. Scientific discovery moves in small steps. Each of these steps involves a clinical trial that takes a great deal of time, effort, money, collaboration, and thought by many different researchers.

This scientific process is not always perfect but it is the best way to make sure that a new treatment is safe and effective before being introduced into routine clinical care.

Research and treatment protocols

What is a protocol?

A formal protocol is a written plan that describes in detail how to treat a particular illness. It describes the drugs, dosages, and tests that are needed. It tells the treatment team how often and when each drug should be given based on a schedule. Each protocol is based on research that shows which treatments are more effective.

Experts from many hospitals work together to develop protocols. The results from each hospital are gathered and studied. Researchers have developed international groups to oversee and coordinate these efforts. Protocols can change if new research shows there are other drugs or combinations of drugs that are more effective. Drug companies, while involved in research protocols that initially establish the safety, dosage limits, and efficacy of drugs, are not involved in organizing treatment protocols.

What is randomization?

Randomization is used in studies that involve two or more groups. Each group gets a different treatment, and patients have to be placed in one of the groups. Randomization means that the patients are placed in a group by chance. This makes sure the results are not biased. A bias may be possible if a person decides who goes in which group. Usually a computer will assign a patient to a group. Most research protocols need randomization at diagnosis, or shortly after diagnosis.

Key points

  • There are many kinds of health research, ranging from chart reviews all the way up to clinical trials.
  • Research is the only way to improve medical treatment and make sure that new treatments are safe and effective.

Jonathan Hellmann, MBBCh, MHSc,  FCP(SA), FRCPC

Jonathan Hellmann, MBBCh, MHSc,  FCP(SA), FRCPC