Part 2: Questions About the Future
“It’s tough to make predictions, especially about the future”.
This quote, attributed to baseball legend Yogi Berra, nicely captures the challenge faced by clinical researchers.
Research is about creating new knowledge and insights. Clinical research is about creating new knowledge and insights focused on health. That means identifying, diagnosing, predicting, preventing, and treating disease. The sorts of questions that clinical researchers try to answer are either descriptive – which focuses on how things are, or predictive – which focuses on what might happen and how to change it.
Descriptive research yields findings like: 1 in 10 adults have diabetes; the chance of getting diabetes increases with age; or people of South Asian ancestry have higher rates of diabetes than people of European ancestry.
Predictive research yields findings like: People with diabetes and small amounts of protein in their urine (also called albuminuria), are at risk of future kidney problems; SGLT2 inhibitor drugs reduce the likelihood of developing kidney problems; or nerve damage in the feet increase the chance of a foot ulcer.
The rest of this blog focuses on predictive research.
As you can see from the examples I gave, predictive research seeks the answer to two types of questions.
- How do we predict the future? For example, can a particular measurement in a person – like a glucose level or a blood pressure, or a urine test – tell you whether they are more or less likely to develop a problem in the future? This is usually best done by taking measurements in large groups of people, and then determining whether those with an abnormal measurement (high or low) are more likely to develop some problem than those with a normal measurement. The official term for a study like this is a prospective observational study or a cohort study.
- How do we change the future? For example, does treatment A prevent or reduce the likelihood of some future problem compared to treatment B or no treatment? Studies like this are not observational; they are interventional or experimental. In other words, the researcher is doing an experiment which involves doing or giving something to one or more individuals and then observing them to see what happens. When this research involves at least 2 groups who are, on average, identical, and something unique is given or done to each group, the study is called a randomized clinical trial.
Before we explore randomized trials in detail, we will see how clinical researchers figure out what they want to study and what treatments or interventions they want to test.
See you in Part 3.
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About the author
Hertzel Gerstein
Hertzel is an endocrinologist and professor at McMaster University who is in high demand as a speaker, advocate, and educator on diabetes-related topics. His research focuses on using large, international randomized trials to identify and test new ways of preventing type 2 diabetes, reducing serious health outcomes like strokes and death, and achieving type 2 diabetes remissions.
