Clinical Trials 101

Clinical trial awareness starts with education.

Get answers to your questions and orient yourself to some of the most common clinical trial terminology.

Clinical Trial FAQs

Check out this great answer from the National Institute on Aging! has an excellent rundown of the steps necessary for developing and approving new drugs. We especially love the drug development funnel illustration about halfway down the page!

Drug Development Funnel graphic

There are many different ways to find a clinical trial. Carebox and Antidote are great sources to get you started, and you can also speak with your doctor if they currently run clinical trials. Here’s a great breakdown of the process from Pfizer and another from the National Institutes of Heath

There are several different factors to consider here. One is that your usual personal healthcare costs remain the same, but some lab work, X-rays, research drugs, and other medical care related specifically to the study you participate in will be covered by the sponsor of that study. has a handy explanation here

Another factor you may be wondering about is whether you’ll have to miss work and therefore miss paychecks due to participating in a clinical trial. This is certainly something to consider. Ask about the trial’s timing and the in-person commitment needed from you during sign-up. Additionally, some trials do offer compensation to participants, but not all share this practice. 

When it comes to travel to and from research sites, some trials cover these costs for patients and others don’t. Be sure to ask about this when signing up for a specific trial. 

Finally, you may be able to find a financial navigator to help you sort out costs related to your illness, including any costs associated with participating in research. Here’s an example of how a Financial Navigation Program works with cancer patients and families. 

Your primary care physician is your ally in tapping into clinical research as a care option. Some questions to ask include: 

  • Can you help me find a clinical trial that is a good fit for my health condition? 
  • Are you serving as a principal investigator in any current or upcoming clinical trials you’d like me to be aware of? 
  • What support should I count on from you and your practice during and after I become a medical studies participant? 
  • Here are some more great questions to ask from the Memorial Sloan Kettering Cancer Center… 
  • …and even more in this excellent clinical trials overview article from Patient Advocate Foundation

Yes! There are many decentralized clinical trials (those that wholly or in part rely on data that can be collected remotely) and hybrid clinical trials (those that rely on some remote data and some onsite data collection). When considering a study, you can talk to your doctor or the study enrollment team to better understand whether travel is required, and if so, how much. 

The Carebox link in question #3 is a great place to start in your case, as well! You can also check out the Pediatric Trials Network and Pfizer’s Pediatric Clinical Trials hub

As shared by the National Heart, Lung, and Blood Institute, healthy volunteers, patient volunteers, diverse volunteers, and children should all consider clinical trial participation. The NHLBI page elaborates here

Altruistic support of global wellness, research as a care option, and financial benefits are some of the reasons that people choose to participate in clinical trials. See what Johns Hopkins Medicine has to say about this

Common Clinical Trial Terminology

Controlled Study/Controlled Trial

A controlled study is a way to test if something, like a new medicine, works. There are two groups in a controlled study: the treatment group and the control group. The treatment group gets the new medicine being tested. The control group gets something else, like a fake drug (the “placebo”) or a different treatment. The researchers watch the two groups to see if the treatment group does better than the control group. If it does, then the new medicine being tested probably works. 


DCT stands for “decentralized trial”. It’s a type of clinical trial where some or all of the study happens outside of a hospital or clinic. This means that participants can do their study visits from home or another location, like their doctor’s office. For example, a company is testing a new drug for treating asthma. They want to make it easy for people with asthma to participate in the trial, so they offer the option to do study visits from home. Participants in the trial can use a special app to track their symptoms and report them to the study team. They can also do video calls with the study team if they have any questions or concerns. 

Double-Blind Study

A double-blind study is a type of scientific study where neither the people taking part (the subjects) nor the people running the study (the investigators) know who is getting the real treatment and who is getting a fake treatment (a placebo). This helps make sure the results are not influenced by people’s expectations. If people know they are getting the real treatment, they might be more likely to get better, even if the treatment doesn’t work. And if people know that they are getting a placebo, they might be less likely to get better, even if the placebo does work. Double-blind studies are some of the most reliable ways to test if a new treatment works.  

FDA (Food and Drug Administration)

The FDA stands for Food and Drug Administration. It’s a government agency in the United States that helps to make sure that food, drugs, and medical devices are safe and that they work. The FDA has the final say on whether a new drug can be sold in the United States. They do this by reviewing the drug’s safety and effectiveness (if it works). Other countries have their own food and drug agencies, like the European Medicines Agency (EMA) in Europe.  

Hybrid Trial

A hybrid trial is a type of clinical trial in which some of the study activities are done remotely, like at a patient’s home or doctor’s office, while others are done at a traditional clinical research site.  

Investigational Drug

A drug that is still being tested in people. It has not been approved by the FDA yet, so it is not available to the public. Investigational drugs are being studied to see if they are safe and effective for treating a certain disease or medical condition. They are tested in clinical trials, which are studies that look at how well a drug works and what side effects it might have. 

Medical Device Trial

A medical device trial is a clinical study that tests if a new medical device is safe and if it works. Medical devices can be anything from a bandage to an implant, like a pacemaker.  

Phase I-III Clinical Trials

Phase I-III clinical trials are studies that test new drugs or treatments in people. They are done in stages, called phases.  

Phase I trials are the first time a new drug or treatment is given to people. The goal of Phase I trials is to find the safe dose of the drug or treatment.  

Phase II trials are done to see how well the drug or treatment works. They are usually done with a small group of people who have the disease or health condition that the drug or treatment is meant to treat.  

Phase III trials are done to compare the drug or treatment to other treatments that are already available. They are usually done with a large group of people who have the disease or health condition that the drug or treatment is meant to treat  

Some trials also have a Phase 0, which is a very early stage of testing. In Phase 0, researchers give a very small amount of the drug or treatment to people to see how it works in the body.  

After a drug or treatment is approved, Phase IV trials may be done to continue to monitor its long-term effects. 

Principal Investigator

Principal Investigator, or PI, is the person in charge of a clinical study. They are responsible for designing the study, finding people to be in the study, and collecting data during the study. They also work with the sponsor of the study to make sure it follows the FDA’s rules. PIs are usually doctors or scientists with experience in clinical research.  


The protocol is a clinical trial plan. It tells what the study will do, how, and why. It includes information about the number of patients who will be in the trial, who is qualified to be in the trial, what they will be given, what tests they will receive, and when, and what information will be collected. 


Randomization is a way to make sure that the groups of people in a clinical study are as similar as possible. This is done by randomly assigning participants to different groups. For example, if a study is comparing two different treatments, the participants will be randomly assigned to either the treatment group or the control group. This means that there is an equal chance that any participant will be assigned to either group. Randomization is important because it helps to make sure that the results of the study are accurate. If the groups are not similar, then it may be difficult to tell if the difference in results is due to the treatment or to other factors. 


Reimbursement is when you get money back for something you bought. In a clinical trial, reimbursement is money that you get back for things you bought because of the trial, like bus fare, parking fees, or lost wages. 


A site is where a trial happens. This could be a doctor’s office, a hospital, a dedicated clinical research facility, or another medical facility. 


The sponsor is the person, company, or organization that pays for a clinical trial. The sponsor also has to make sure that the trial is run properly and that the participant’s rights are protected. 

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