Demystifying Clinical Trials – Part 1

In honor of Clinical Trials day on May 20th, we’re kicking off a series that's designed to shed some light on the world of clinical research. Follow us on Facebook, Twitter, and Instagram and stay tuned for posts over the coming months about commonly confusing terms, questions to ask before enrolling in a clinical trial, where to find research results, interviews with members of our research team about their roles, and more!

What is a clinical research trial?

A clinical research trial, also called a research study, is a process designed to test whether a new treatment or drug is safe and effective. There are strict regulations and guidelines, from both the Food & Drug Administration (FDA) and the institution conducting the research, which all researchers must follow to ensure that a trial is conducted safely and reliably.

Why is clinical research important?

As researchers learn more about science and health, they develop ideas, called hypotheses, about new treatments that might help people. However, before these new treatments can be used for everyone, they need to be tested.

The process to test a new drug or treatment starts by giving it to a small number of people and watching closely to make sure it’s safe. The next step is to give it to more people and compare them to people who didn’t get the new treatment. If a clinical trial shows that a new treatment is both safe and effective, researchers share their results publically so doctors know they can use the new treatment regularly.

Who conducts clinical research?

Most major hospitals and universities around the world participate in clinical trial research. Health care device and pharmaceutical companies also conduct trials. All researchers in the US must follow strict rules designed by the National Institutes of Health, FDA, and the institution conducting the research to make sure that participants are kept safe and that their data is kept confidential.

A research team composed of a lead investigator, several co-investigators, research coordinators, statisticians, and sometimes laboratory staff work together to conduct the trial. One of the ways patients are kept safe is by having the research teams follow clear guidelines for how the study will be conducted. In addition, having pre-determined criteria for which patients can enroll ensures everyone is healthy enough to participate.

When should I consider participating in research?

Most clinical trial research involves testing a new procedure or drug that’s intended to treat a specific medical condition. Some studies are observational and are only designed to naturally watch what happens to help researchers better understand a condition. Your doctor may discuss clinical trials that are available if you have been diagnosed with a specific disease or disorder and are healthy enough to participate.

You may also consider participating in a research study if you are healthy and want to help researchers compare data between healthy and unhealthy groups of people. Studies looking for healthy volunteers are often advertised around hospital and university campuses or online.

How is clinical research conducted?

Today’s clinical trials follow steps that are based on the Scientific Method that was developed in the 17th century.  Let’s walk through the process using a new cholesterol-lowering drug as an example.

Hypothesis – Dr. Jane Smith studied the results of other scientists, as well as her own previous research, and developed a theory about a new drug that might lower cholesterol.

Protocol – Dr. Smith writes a long, detailed plan called a protocol. This document explains why she thinks this drug will work better than the current ones, which people will be able to enroll in the study, exactly how and when data will be collected to test the drug, how the data will be measured, and how she will know for sure if it’s safe and effective.

Data Collection and Analysis – Dr. Smith and her research team tell patients with high cholesterol about the new drug and why it might be better than current ones. Those who are eligible and want to enroll are treated according to the plan in the protocol. The research team collects data on their health and outcomes, sometimes over several years, to ensure everyone is safe. Once the data has been collected, the team analyzes the results to see whether or not the drug lowered cholesterol better than the standard.

Publication – The research team then writes a brief paper (usually 6-10 pages) explaining the details of their hypothesis, how they conducted the study, and their results. Just like there are many magazines on a variety of specialized topics, there are many scientific journals that publish papers written by research teams. Dr. Smith would choose a journal related to the topic of their study and submit it for review. All reliable journals have what’s called a “peer-review process” in which experts in the field volunteer to review papers scientifically (both the research team and reviewers are anonymous). If a group of reviewers all agree that Dr. Smith’s study was conducted well and the results are valid, the paper will be published in the journal. Others in the field can then read about it and design similar studies to confirm or contradict Dr. Smith’s results.

Changing Clinical Practice – Once several studies have found that the new drug is safe and effective, doctors everywhere can start using it in regular clinical practice. This means that all patients can get it without needing to enroll on a clinical trial at a particular hospital. The point of clinical research is to continually find safer and more effective treatments for all patients.

Where can I find clinical trials and learn more?

• ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world.
• ResearchMatch.org is a free, non-profit site that helps match volunteers to medical research studies.
• NIH Policies & Regulations – Human Subjects – https://grants.nih.gov/policy/humansubjects/policies-and-regulations.htm
• FDA Clinical Trials and Human Subject Protection – https://www.fda.gov/science-research/science-and-research-special-topics/clinical-trials-and-human-subject-protection

Many local hospitals and universities also have information on their websites about available trials and who to contact.

The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of finding solutions for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science to diagnostic imaging to regenerative therapies. Email the program at HLHS@mayo.edu to learn more.