Behind the Research is an ongoing series to peek behind the research bench and learn more about the words and phrases that scientists use. We hope that through a better understanding of the science of disease and how clinical research works, you will be a more empowered and confident participant in your care.
Just Aliquot That
My husband is a scientist like me, and I remember distinctly just after we started dating, he had purchased a large package of meat at the grocery store. Since he lived by himself, he told me he was “just going to aliquot it and put it in the freezer.” Scientists use that term all the time — aliquot — but I’ve never heard anyone else use it. To aliquot something is just to divide it into smaller portions. He could have easily said he planned to “divide it up and put it in the freezer,” but the fact is, as scientists that’s a word we use so much, we think it’s part of everyone’s basic vocabulary. At the time, I thought it was cute the way he used such a science-y word in such an everyday way, but now I make a conscious effort to avoid those kinds of terms when I write. Unfortunately, sometimes there isn’t a good alternative, so I’ve created this series to help shine a light on what research terms actually mean.
- Adverse event: This is an easy one! Adverse event is basically FDA-speak for side effect. It’s something bad (adverse) that happens when a patient is taking a drug. In a clinical trial, it is important to determine which adverse events happen because of the drug — those are the true side effects that patients will need to be aware of when they are making treatment decisions. This is why it’s so important to have a control group who doesn’t get the investigational drug — it helps the researchers determine which adverse events are from the drug and are not just symptoms of the disease or unrelated ailments.
- Drug Development and Validation: Before a drug is approved for routine use in patients, it must go through the lengthy process of drug development and validation. After researchers decide how they are going to try to target the disease, they must screen many compounds (usually in the thousands) to choose one which will ultimately be refined over and over again to ensure that the formulation and composition of the drug is optimal for patient safety and efficacy. They must also ensure it is optimal for manufacturing. The validation studies show regulators that the drug that is produced is the correct substance, in the correct amount, for the intended purpose. A manufacturer needs to be able to show that their protocol produces the same product every time it is used in that lab, by a number of different scientists using different equipment over a period of time, as well as by different scientists in a different lab. This provides the assurance that all patients will receive the same drug at the appropriate dose, regardless of where or when it is produced.
- Informed Consent: Every time my kid goes to a trampoline park, rock climbing, or even to an escape room, I have to sign another waiver. Most people probably think of an informed consent form as a type of waiver, and there are a lot of similarities — lots of words, lots of legalese, and a few places that you have to sign in order to participate. Simply put, the informed consent form fully explains the procedures, risks, and possible benefits of participation in a clinical research study. Ensuring that all these conditions are met is much more complicated, which is why these documents are often very long. While the sheer volume of information in this document can overwhelm many patients, it is important for the patient to be sure they fully understand the research study before they sign the document. Because fully understanding the risks is crucial to protecting a patient’s welfare, rights, and privacy, the Institutional Review Board (which is tasked with maintaining patient protections) spends a significant amount of time studying the informed consent form while they are deciding whether the researcher has permission to start the study.
- Placebo: Commonly referred to as a sugar pill, a placebo is a harmless substance that looks like the real drug in a clinical trial. It is given to some of the patients instead of the investigative drug to make sure that the drug is having a real effect, and that patients aren’t just imagining that the drug is helpful. It also helps researchers determine if the adverse events a patient experiences are really a result of the drug. It’s important to remember that not all trials use placebos! If you are in a clinical trial for a life-threatening disease where there is already an approved treatment, that treatment will not be withheld from you. For example, in clinical trials for cancer drugs, the two treatment groups that are compared are often the standard of care group and a second group that receives the standard of care and the investigative drug.
- Protocol: A recipe is the kitchen equivalent of a protocol. It gives specific steps that need to be followed, including amounts of ingredients and timing of procedures. A clinical trial protocol is the same thing — it sets up rules for doses, timing of procedures, and which equipment should be used. Just like the recipe for your favorite chocolate chip cookie, it’s important for the protocol to be followed closely so that the investigators can be sure that the results predict what will happen when the drug is given to patients in the future.
- Safety and efficacy: This is a phrase I use a lot and it can’t really be avoided. It is a term that is used by the FDA in the approval process. It means that a drug is relatively safe for use in humans, and it does a good job of treating the disease for which it is indicated. It does not mean that the drug is completely free of side effects, but rather that the benefits outweigh the risks.
- Standard of Care: This is also sometimes described as “usual care,” and means pretty much what it sounds like! When conditions have a pretty standard treatment plan that is widely accepted by healthcare professionals, it is referred to as standard of care.
This started as an idea for a single post, but it quickly became evident that the best plan would be to aliquot the idea into multiple posts to form a series (see what I did there?). If there’s a word, phrase, or concept you’d like me to tackle in the future, please leave a comment and I’ll add it into my queue!