Clinical Research 101: A Guide to Understanding Medical Trials

Clinical research is the process of studying health, illness and medical therapies in people. It's an important component of expanding the body of knowledge about human health and developing treatments for health conditions.¹

Clinical research involves a variety of stakeholder groups, including researchers, regulatory entities, healthcare providers and patients. Each of these groups plays an important role in advancing research studies.

Working in clinical research is a rewarding and valuable career path that requires scientific knowledge and skills, as well as adherence to ethical guidelines. This blog will explore the most fundamental aspects of clinical research.

Clinical research is the study of health conditions and their treatments in human participants.¹ Researchers seek to understand human health and how to improve it. In studies relating to emerging treatments, clinical trials involving human participants are typically the final step after completion of preclinical research studies aimed at determining whether a treatment could cause harm.²

There are two main types of human clinical research:¹

1. Observational studies monitor people over time without applying any interventions. Researchers convene a group of participants and gather data about them with the goal of learning more about a particular health topic. They may ask participants to fill out surveys, undergo regular health exams or otherwise share information about their health over a period of time. Researchers use this information to understand health conditions and how they affect people.
   
2. Clinical trials are research studies that test emerging medical interventions in people. Participants agree to undergo medical, surgical or behavioral treatments so researchers can evaluate how the treatments affect their health conditions. Clinical trials are often the final stage of research for new treatment methodologies.

The importance of clinical research lies in how it furthers the understanding of health conditions and develops new therapies to improve health and well-being. The aims of clinical research include:

• Finding new ways to diagnose diseases 
• Learning how to prevent the onset of health problems
• Improving quality of life for people experiencing acute disease or chronic health problems
• Discovering cures for diseases

Human trials for new treatments play a vital role in bringing new therapies to market. Clinical trials examine treatment efficacy, safety, dosage and duration. Regulatory agencies require this information before approving new treatments for use. Trials must meet strict standards and undergo expert review before the regulators will sign off on the findings and approve the treatment.

Data is the core of clinical research. Researchers may collect data through a combination of participant surveys, interviews or exams. The data collected is connected to the questions at the center of the study. For example, an observational study on the effects of diabetes on visual acuity might focus on participant reports of their vision as well as data from periodic retinal imaging.

In clinical trials, investigators track data about disease progression or regression alongside data about treatment administration. Analyzing the trends and changes is how researchers determine the efficacy of the treatment.

Data analysis in clinical research needs to be accurate and replicable.³ In practice, that means that researchers must be able to prove that the data is valid and that the analysis procedures used can be reproduced.

Machine learning and artificial intelligence are valuable tools in analyzing the vast quantities of data from clinical research. These technologies can quickly and accurately analyze data so researchers can more easily use it to demonstrate the findings of clinical studies.³

Study design is one of the key components in the success of clinical trials. Clinical trials are the final phase of medical research, usually occurring after years of research in the lab. In the United States, the Food and Drug Administration (FDA) provides oversight of clinical trials to ensure they are developed and implemented properly.⁴ Investigators are required to present a fully conceived plan for the research in order to receive approval to move forward.⁵

Each study must have a strong hypothesis. Researchers must clearly specify what they believe the human trials will demonstrate, supporting the hypothesis with evidence from their preclinical research. In addition, researchers are required to present the ways in which the stages of the trial will be conducted to ensure that it will be safe for participants.⁵

Many clinical trials compare a new product or therapy with either no treatment or an existing treatment. To do this, a portion of the participants will receive the study treatment while others will receive a placebo. In some trials, one group of participants receives an alternative treatment instead of a placebo.¹

Participants are randomly placed in groups. In a single-blind study, the researchers know which participants are receiving the new treatment, but the participants do not. In a double-blind study, neither the researchers nor the participants know.¹

Participant safety and consent are central to any clinical trial. Participants must give informed consent in order to participate. Informed consent means that they are explicitly told what the study entails, what risks it poses, what outcomes they can or cannot expect and whether or not they may receive a placebo instead of treatment. Participants are allowed to leave the study at any time. Most studies are overseen by an Institutional Review Board (IRB) to ensure that patients' rights are protected throughout the study's duration.¹

There are also ethical requirements for researchers conducting clinical trials. They must report any conflicts of interest (e.g., connections to outside corporations, trade groups or regulatory agencies).⁶ In the event of significant conflicts of interest, a researcher may be asked to withdraw from the study.

Recruiting participants for clinical research requires presenting a set of potential benefits from participation while managing participant expectations about possible outcomes. Honest, transparent communication about the risks and rewards is a critical factor during the recruitment process.

Study managers need to be prepared to answer the common questions related to the following:

• Costs: In some cases, participants or their insurance companies will need to pay for elements of treatment. Additional costs, such as travel expenses or parking fees, may also apply.
• Participant remuneration: Some clinical research projects offer a stipend to participants, but others do not. That should be clearly communicated.
• Access to the new treatment: Participants may be assigned to a control group that does not receive the new therapy.
• Side effects: Participants may have concerns about treatment side effects, so transparency is important.
• Post-trial expectations: If the therapy is successful, participants may want to continue receiving it after the trial. Continued access should be addressed in the study design.

Clinical research is essential to advancing knowledge and developing new medical treatments. It's an exciting and impactful field for scientists and clinicians who want to broaden the scope of medicine. Kent State's online Graduate Certificate in Clinical Research program is a pathway to opening up new career possibilities and leadership opportunities in this valuable area. Led by expert faculty, our program will allow you to deepen your expertise in critical topics such as research methodologies and data analysis. To learn more, connect with an admissions outreach advisor today.