Clinical trials are research studies that test whether a new potential therapy is safe and effective for patients. They evaluate new drugs, behaviors, or devices, and reveal whether these potential therapies work for particular illnesses or particular groups of patients. Clinical trials provide the best available data with which to make healthcare decisions, and are considered the gold standard in the development of new therapeutic options.
There are two main types of clinical trials: interventional studies and observational studies:
A trial sponsor is an organization which initiates, funds, and conducts a clinical trial. This is often a pharmaceutical company, but it can also be a university or another type of research organization. When selecting a clinical trial, it's important that potential volunteers are fully aware of and comfortable with who is sponsoring the trial.
Potential volunteers for trials can be anyone. While many people view clinical trials as a last resort only to be relied upon when all other options have been exhausted, this is not the case; research studies need a variety of participants to enroll. Some trials are looking for volunteers who have been recently diagnosed, while others may be only need those who have been living with a condition long-term. Certain studies may require patients who have had different treatment experiences in the past. Every trial is different, but with thousands of studies recruiting, there is likely a trial that is right for you.
Clinical trials begin with an idea in a lab. Researchers thoroughly test potential treatments in the lab and in animals before ever beginning trials in humans. If these lab and animal tests are successful and the therapy looks promising, the researchers will begin to conduct research studies beginning at Phase 1 and moving through to Phase IV:
It's a big decision to take part in a clinical trial, and it's important that patients and caregivers fully understand all of the benefits and risks before enrolling. We'll discuss these in the next section.
There are many reasons that people choose to volunteer for clinical research, but it's important to weigh these against potential risks. Some of the top benefits of taking part in research include:
Access to care. People who take part in a clinical trial receive access to the latest treatments in development, and for many patients looking for options, this is a real benefit. In fact, according to a CISCRP survey of 2,194 former clinical trial participants, 44% of people surveyed cited "obtain better treatment" when asked to share their top reasons for participation. In some cases, patients are able to continue taking the drug after the trial is over, as well. And, in many cases, patients receive either the standard of care or the investigational drug, removing the risk of receiving a placebo.
Quality of care. Patients in clinical trials report a high level of personalized care while participating in research. While trials may require more office visits than normal, or more tracking of symptoms, this extra time spent on health can mean a higher quality of care.
Financial benefits. Treatments administered during a clinical trial are often given at no cost to patients; in fact in many cases, volunteers are compensated for their time, travel, or participation in general. The amount of payment has to do with the phase of the trial - earlier trials such as Phase 1 pay more (nearly $2,000 on average) because the treatments being studied are less well-understood. To find a paid clinical trial, you'll likely need to find a few trials for which you may qualify, then contact the sites to learn about potential payment.
Helping advance science for future generations. The goal of clinical trials is to discover new, better treatments (and cures) for the condition being studied. This means that, if successful, patients with that condition in the future will have better options than current patients, and this is an important motivator for many patients today. In the CISCRP survey referenced above, nearly half of participants cited "help advance science, treatment of disease/condition" as one of their top reasons for participation.
On the other hand, of course, clinical trials also come with some degree of risk. There is chance that the treatment being studied might not work as expected or be a better option than the standard of care. The study drug could also cause an unexpected side effect, especially in earlier phase trials.
With that said, there are several protections in place for patients who take part in clinical trials:
Before making the decision to join a clinical trial, it can be helpful to hear from someone who's taken part in it. Emily Schaller, founder and CEO of the Rock CF Foundation, talked with Antidote about her experience participating in several clinical trials for cystic fibrosis, a progressive lung condition that she has lived with since she was 18 months old, in the below video.
How to find a clinical trial
To find a clinical trial, you can use a clinical trial search tool, such as ClinicalTrials.gov or Antidote, or ask your doctor about opportunities they know of. If you do start your own online research, you can also bring options you're interested in to your doctor.
ClinicalTrials.gov is a government website where all clinical trials are registered. It is comprehensive, but was built for researchers rather than patients. To search for clinical trials in your area, you can enter in your condition and location in their search box.
Antidote created a search tool designed with patients in mind. It pulls in listings from ClinicalTrials.gov, but organizes the trials by their inclusion and exclusion criteria so that patients can find a trial that they may qualify for just by answering a few questions. To use Antidote's match tool and find a clinical trial that may be a match for you, start by following these steps:
Emily of Let's Rock CF also shared how she found the clinical trials she participated in, and offers tips on searching for one that's right for you.
After you've expressed interest in a clinical trial by contacting the trial team or filling out a pre-screener online, what comes next?
Exact experiences vary, but your trial experience will most likely follow these steps:
Phone screening: After you've found a clinical trial you're interested in and reached out to the trial team, or completed a pre-screener online, the trial team will give you a call to ask you a few more questions and learn more about you. If they determine based on the phone screening that you may be a good fit for the trial, you'll be invited for an in-person screening at the study site.
In-person screening: At the in-person screening, doctors associated with the trial will ask you more questions, and you may be given a physical. You may also be asked to complete additional medical tests to ensure that the study is right for you. If the trial requires multiple tests, you may be asked to come in for a second visit. If the study team determines that you're qualified for the trial, you'll be invited to join.
Informed consent: Next, the trial team will answer all of your questions and go over the details of the trial. Be sure to ask any questions you'd like such as how the long the trial will last, what you can expect from each study visit, and whether you will be reimbursed for your expenses or receive additional compensation. Next, you'll be asked to sign an informed consent form to officially join the trial.
Study visits: The study visits themselves will vary based on the kind of trial it is. Your visits may be similar to regular doctor's appointments, with additional attention and questions related to the clinical trial. For some trials, site visits are conducted virtually. In those trials, you'll talk with the study team over a webcam on your computer or a device provided by the trial.
In between visits: Some trials may also ask you to keep a virtual or paper diary during the trial to track your symptoms or answer other questions related to your health, such as your exercise routine. It's important to record your diary entries as directed by the study team to ensure that the study is collecting accurate data.
After the trial: You may be able to continue taking the study drug after the end of the trial – the study team should let you know before you join, but ask if they don't mention it. You can also ask before the trial starts how you can see the results of the study after it's completed. Trial teams publish their results, so you should be able to find the study after it's complete, even if it's not directly shared.
Q: Will I be compensated for participating in a clinical trial?
A: Some clinical trials will cover your travel expenses and other expenses you may incur from participation. Other trials also offer additional compensation. Earlier stage trials are more likely to be paid, but Phase II and Phase III trials can sometimes offer compensation, too. Ask the study team before joining.
Q: What if I'm rejected from a clinical trial I'm interested in?
A: Every clinical trial has unique inclusion and exclusion criteria. If you don't qualify for one trial you're interested in, you may qualify for another in your area. You can also sign up for alerts from Antidote to hear about new trials that match your profile.
Q: What if I receive a placebo?
A: A placebo is a medication without any therapeutic effect that's used to compare against a study drug. Not all trials use placebos – for example, cancer trials never use them. Some trials measure a study drug against the standard of care for that condition, or against another treatment. Depending on the trial, you may be able to stay on your current medication, too.
Q: Will I be able to stay on the study drug after the trial is complete?
A: In some trials, you are able to continue taking the study drug at the end of the trial. Ask the study team before you join the trial.
Q: Can I quit a clinical trial once it starts?
A: Yes, even after you sign your informed consent form, you can still leave a clinical trial at any time, for any reason.
Q: How much time does it take to participate in a clinical trial?
A: Time commitment varies across clinical trials. While some trials may include weekly site visits or overnight stays, others may only require short visits every few weeks or months. Make sure you find out about time commitment as part of the informed consent process.
Medical research can't move forward without clinical trial volunteers. If you're ready to get involved, start searching for a trial that matches you below.
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