Clinical Trials 101: The Ultimate Guide to Understanding Clinical Research
You have probably heard about clinical trials, but you may have many questions about what they are, how they work, and how to get started as a participant. Are they just for people with cancer? Are they safe? Am I eligible? How would I find one? There is a lot to ask about—and you are not alone. We have created Clinical Trials 101 to answer your questions about clinical trials so you can have a clear understanding of trial participation is the right fit for you.
What are clinical trials?
Clinical trials are research studies that evaluate new—and existing—medicines, vaccines, and devices for effectiveness and safety. In many clinical trials, the new treatment is compared to the current standard treatment to learn if the new treatment is more effective and/or has fewer side effects.
What are clinical trial phases?
- Phase I trials involve a small number of people (often less than 100) to evaluate the dosage amount, side effects, and safety.
- Phase II trials enroll more participants (usually 100 to 300) to evaluate effectiveness. The researchers want to see if the new treatment or device works for the condition or disease, and phase II may last for two or three years.
- Phase III includes more patients, from several hundred to 3,000, to try different doses in a larger group. The effectiveness of the new treatment is the number one priority, followed by its safety and side effects. Phase III often compares the new treatment to the standard treatment. If results are encouraging, researchers will ask for FDA approval.
- Phase IV is conducted after approval to study the benefits and safety.
Why are clinical trials important?
Clinical trials are the reason why new—and sometimes life-saving—medicines, vaccines, and devices are available to the world. It is hard to completely underscore the importance enough. From the Advil you have on your bathroom shelves to the chemo that could save someone’s life, all those drugs once started out in clinical research with participants like you or someone you know. Even now after COVID-19, we are seeing the global effects of clinical research: over five million people participated in trials that went to help the world at large.
Clinical trials are also offered as an additional care option to many who face an illness or disease. That is why we believe in connecting as many patients and physicians to available clinical research opportunities as possible—everyone deserves to know what their options are and what is out there.
Why do people join clinical trials?
Everyone has different reasons for joining a trial. Some of the most common reasons people decide to join are:
- Access to a new treatment option
- Personalized and attentive monitoring throughout the trial, often improving the patient-provider relationship
- Advance research for other people and bring new medicines, vaccines, and devices to the world
- Advance our global understanding of a condition
- Gain access to support groups and helpful resources
- Access to a low- or no-cost care option. In most cases, trial participants do not have to pay out-of-pocket when participating in a trial. Research-related costs are typically covered by the clinical trial sponsor.
How to get started with clinical research
A familiar quote comes to mind as anyone starts with clinical research and joins a clinical trial: "Sometimes the hardest thing to do is to take the first step." You will probably have more questions than answers, but eventually, you will see a clearer path and the direction you want to go.
If you're considering a clinical trial, talking to your doctor is a good first step. A trial might be conducted at their facility that would be appropriate for you, and they may be able to direct you where to start. If your physician isn't aware of any clinical trials, it may be up to you to be proactive and take charge of finding a suitable clinical trial. That’s what TrialJourney is for. Here, you can find any available clinical trial and filter by criteria such as your location, disease or ailment, or top-rated research sites. You can also see what other participants have said about their experiences which can help you make a truly informed decision about where to go.
How do you know if a clinical trial is right for you?
Before enrolling in a particular trial, you will usually meet with someone from the clinical trial research team who will explain all the aspects of the trial. They may also ask some basic screening questions to ensure you meet the criteria to participate in the study.
This is also the perfect time to ask questions to help you make your decision. Write down questions before meeting with the research team and take notes about the answers to your questions. Then you, and anyone who may help inform your decision, can help you determine if a clinical trial is a good option. It is worth noting, participants in clinical trials can leave the study at any time and for any reason.
What should you expect when participating in a clinical trial?
If you choose to enroll in a clinical trial and you are eligible,you may hear about a clinical trial protocol. A clinical trial protocol is a document that details how the trial will be conducted. The protocol includes the purpose/objective, the details, the methods to be used, and how the statistics/outcomes will be calculated.
- Purpose of a protocol: A trial protocol helps assure patient safety. The document helps ensure the integrity of the data collected and the ethical interpretation of the study.
- Inclusion/Exclusion criteria: every clinical trial has criteria for eligibility of a particular trial. This criteria is very specific and may include age, gender, ethnicity, smoking status, BMI, and other chronic medical conditions. The criteria are important to increase the chances that the study will produce accurate, reproducible, and reliable results.
Who is involved in the clinical trial process?
Throughout your time in a clinical trial, there will be key people involved, some of whom are always in the background, while others are patient-facing.
- Research Coordinator. The researcher coordinator utilizes the trial protocol as their guidance — they recruit and screen patients and ensure the safety and security of trial medications. They oversee the collection of all patients' data, tests, side effects, and outcomes. The coordinator pays particular attention to any adverse events that patients experience.
- Research Assistant. The research assistant helps the coordinator fulfill the protocol requirements — from recruiting patients to patient interviews and preparing mid-trial reports to help prepare final reports.
- Principal Investigator. A principal investigator, or PI, is a physician who coordinates the medical side of the trial — ensuring the patient's access to potentially more effective treatment while watching for adverse events. The investigator interfaces with the drug company as they evaluate the efficacy and safety of a new treatment.
- Sponsors. Sponsors help ensure the necessary funding for the clinical trial and direct the ongoing process of the trial. The sponsor communicates with the FDA, other regulatory agencies, and ethics boards over any concerns about the treatment's safety or benefits. Sponsors include government agencies, biotech, medical device, pharmaceutical companies, healthcare entities, or private individuals or entities.
- CROs. CRO stands for Contract Research Organization, which provides clinical trial management services for biotech, medical device, and pharmaceutical companies. A CRO has extensive experience in the day-to-day operation of clinical trials to maintain a well-run trial. You may hear about a CRO but never interact with them directly.
Participant protection and safety measures
Patient safety is the most important thing throughout every clinical trial. Each trial goes through an exacting review, and the staff is supervised—including the doctors, research team, and procedures. Potential participants receive one-on-one counseling about the trial and the potential risks and benefits. You will have a chance to ask questions to ensure that you understand the risks and the extensive measures taken by the trial team and oversight organizations to ensure your safety. As mentioned earlier, participants are always allowed to stop participating in the trial at any time for any reason.
Patients receive a printed document containing information with sufficient details of the trial for patients to decide. Each patient meets with the doctor, research staff, or both to answer any questions you may have. The information provided includes:
- Your treatment options, including deciding not to join
- The clinical trial treatment and how it differs from the standardized treatment
- The potential benefits and predictable risks, including how the risks may be different from the risks of standard treatment
- How many doctor visits, the frequency of visits, and medical tests
- The costs that you may have to pay
- How your privacy and personal information is protected
- If compensation is available for travel and/or overnight expenses
- How to contact the staff
Participants are given time to take the consent form home to discuss with their family members or trusted friends. Should you decide to join a trial, you will receive a signed copy to keep as part of your permanent records. In some trials, this process is done electronically and may include videos, quizzes, or electronic signatures (also known as eConsent).
IRB (Institutional Review Board)
The Institutional Review Board comprises several physicians, a scientist, a non-scientist, and a representative from the community where the trial is located. An IRB is required for every research project that involves humans as study participants.
They serve as an ethics review to ensure that the rights of research participants are protected—and protect the rights of future patients who might benefit from this trial research. The IRB ensures that informed consent forms are provided to all clinical trial participants and that they have been properly signed.
Office of Human Research Protections (OHRP)
OHRP is an organization that furnishes leadership, clarification, and guidance for clinical trials and other biomedical and behavioral research. In other words, OHRP safeguards the rights, well-being, and safety of participants. The Federal Drug Administration (FDA) oversees trials to ensure that each one is designed, managed, evaluated, and presented according to good clinical practice (GCP) regulations and federal law.
The oversight from OHRP ensures that rigorous medical research is conducted while ensuring the safety of patients.
Food and Drug Administration (FDA)
The Food and Drug Administration is charged with overseeing both the effectiveness and safety of treatments that either cure, control symptoms, or delay the progression of diseases in humans. Treatments include drugs, medical devices (like insulin pumps), or biologics (vaccines, blood products, or gene therapy). Not every treatment is completely effective for every patient, so the search continues for new and more effective treatments with fewer side effects.
While a new treatment may appear "promising" in the laboratory, it is impossible to know how effective or safe it will be until it is used within humans. The goal of the FDA is to protect human participants from excessive risks while allowing clinical trial patients the opportunity to benefit from a new treatment and help advance the cause of science.
Where can I find resources for clinical trials?
There are many clinical trial resources, but you will have to learn which ones are "trusted" resources; with so many options, it may seem overwhelming. You may not find a trial at first, but do not be discouraged—new trials begin regularly, so continue looking until you find a trial that is right for you.
Clinicaltrials.gov is one of the most known resources for clinical trial participation, though it may not be the most helpful in your search.
ClinicalTrials.gov is the largest repository of clinical trials globally, and over 325,000 trials from 209 countries are listed.
While the concept that "bigger is better" sounds good, this concept may not be necessarily true for ClinicalTrials.gov. Its large size limits its usefulness to patients searching for a clinical trial that is actively enrolling, nearby, and might be right for them. And there are three other areas where it is lacking:
- Non-registration of some trials
- Registration of trials with incomplete information
- Non-reporting of trial results
Get started with TrialJourney
TrialJourney was founded by physicians, nurses, researchers, and computer scientists who realized the difficulties and frustrations patients faced when searching for a clinical trial.
While information is abundant online, it is not always well-organized or easy to use. The terminology used is difficult for many patients to understand—TrialJourney provides a user-friendly search method.
How to use TrialJourney
The most important feature of TrialJourney is the Find a Clinical Trial tool. It is easy to use, with the ability to search nearby cities; you can also extend your search area to a wider distance, the 100-mile or 300-mile radius, to include suburbs or centers in adjoining states. You can narrow your search by closest location or highest ratings, by gender, or by age. Be sure to check back regularly because clinical trial research facilities add new trials throughout the year.
Why are site reviews and ratings important?
Patient reviews are extremely valuable to patients considering a clinical trial. Knowing that someone like you has a good/positive experience is encouraging and uplifting. Seeing a negative review alerts a potential patient to ask lots of questions to help ensure that they are making a wise decision.
What information is available?
Every research center in the United States that has registered a clinical trial on ClinicalTrials.gov is included in our database. TrialJourney offers its website visitors a variety of resources:
- What You Should Know Before Participating in a Clinical Trial
- Frequently Asked Questions
- The clinical trials landscape series
So, what is the next step?
If you think you are ready to participate, we thank you! If you qualify, you will be helping to bring new treatment options and learnings to the world. You can think about the kind of trial that you may be looking for, and then look through trial opportunities near you. It is also recommended that you consult your provider for their recommendations.
If you still need more time or understanding before you feel ready to participate, that is okay too! Clinical research is not for everyone, and there is so much to learn. Read through our Nest hub to keep learning about how clinical research works, and it is always worth looking through available opportunities in your area to see if anything could be the right fit in the future.