Should You Try a Clinical Trial as a Treatment Option? What to Know

You may want to consider a clinical trial when exploring new treatment options. They can involve cutting-edge treatments and may even offer compensation, but it’s important to know the pros and cons.

If you’ve recently received a diagnosis or are interested in exploring different treatment options for your condition, one option to look into is clinical trials.

Clinical trials are an opportunity to participate in the early stages of new treatments that may not be available through your regular care team.

However, the decision to participate in a clinical trial is highly personal. Read on to learn more about clinical trials and whether they’re the right option for you.

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What is a clinical trial?

Clinical trials study interventions like medications, lifestyle tweaks, procedures, or medical devices, says Alison Stopeck, MD, a clinical researcher specializing in breast cancer.

Stopeck explains that clinical trials aim to determine whether an intervention improves outcomes or is equivalent to the current standard of care.

“A clinical trial is essentially any study where some kind of therapy is being tested in humans,” says neuroscientist and researcher Tommy Wood, PhD. “The word ‘clinical’ essentially just separates from preclinical, which is where therapies are tested in animal models or test-tube models.”

What should I expect when participating in a clinical trial?

It’s challenging to explain step by step what it’s like to participate in a clinical trial because it depends on what’s being tested.

However, you can usually expect:

1. a selection process
2. testing to understand your “baseline”
3. treatment assignment
4. additional testing and monitoring

The selection process involves setting specific criteria based on:

• health condition
• demographics
• health-related factors, such as not having a preexisting condition
• age

To determine whether the treatment produced a specific outcome, like an improvement in blood sugar, scientists need to understand where people started. This baseline testing might include questionnaires about symptoms or bloodwork.

“Generally, a clinical trial entails taking an additional drug, imaging, and bloodwork,” Stopeck says. “It usually requires you to do something different than you normally would. It might be as simple as drawing extra blood when doctors would normally be drawing blood anyway.”

Cost

Usually, there’s no cost to participate in a clinical trial, Stopeck says. You may even receive some form of financial assistance and benefit.

“You’ll usually be compensated for your travel plus your time, so participating in a clinical trial won’t usually cost you anything,” Wood says.

What are the phases of a clinical trial?

Clinical trials typically have four phases.

Phase 1

“Phase 1 is small and assesses how a drug acts on the body and how the body affects the drug, as well as safety,” says neurologist Peter McAllister, MD.

According to the Food and Drug Administration (FDA), phase 1 usually involves about 20–100 healthy volunteer participants and can last several months. About 70% of drugs move on to phase 2.

Phase 2

“Phase 2 is medium-sized and looks at a range of doses for efficacy, safety, and tolerability,” McAllister says.

This phase includes up to several hundred people with a specific condition and can take up to 2 years to complete. The FDA reports that about one-third of drugs advance to the next phase.

Phase 3

“Phase 3 trials test the new drug or device against the current standard of care, [which is the] existing approved treatment,” says Caroline Melendez Daigle, who connects people with clinical trials via Carebox.

The FDA reports these trials also test for efficacy and potential negative side effects and can have 300–3,000 participants.

The studies are typically placebo-controlled and double-blind:

• Placebo-controlled means some participants will receive a sugar pill, or placebo.
• Double-blind means the participant and study doctors won’t know whether a particular participant receives the actual intervention being tested or the placebo.

Phase 3 trials typically last 1–4 years. From there, 25–30% of tested interventions move to the fourth and final phase.

Phase 4

“Phase 4 trials take place after approval is received to monitor long-term effects,” Daigle says.

These trials involve thousands of participants with the condition in question and can last several years.

What are the pros and cons of participating in a clinical trial?

Participating in a clinical trial is a highly personal decision.

“You can also change your mind at any time,” Wood says.

Weighing the pros and cons before participating can increase your comfort level and help manage expectations.

Pros of clinical trials

Clinical trials may appeal to people with chronic conditions for many reasons, including:

• access to new therapies
• higher level of care
• financial benefits
• desire to contribute to science

Access to new therapies

Clinical trials allow people to try potential, new therapies before anyone else, which may appeal to some.

“Maybe you’ve tried therapies that are already available, and they haven’t had the impact that you wanted,” Wood says. “Maybe for the condition you have, there isn’t a good standard therapy, and this is an opportunity to help develop or find an actual therapy that may work for this condition.”

Higher level of care

Some people may welcome the additional care that clinical trials provide, like extra doctors’ appointments, bloodwork, and imaging.

“While in a trial, patients also receive a higher level of care as they’re monitored much more closely than when receiving standard of care or the approved treatments,” Daigle says.

Financial benefits

Some people may find the financial benefits appealing. These can include coverage for travel costs, gift cards, and compensation for participating.

Scientific contribution

Finally, some people participate in trials altruistically.

“Enrolling in a clinical trial also allows participants to contribute to science and thus help others with their particular disease,” McAllister says.

Cons of clinical trials

Clinical trials aren’t for everyone. You may want to consider these potential drawbacks before you decide:

• safety concerns
• contraindications for your condition
• data and privacy concerns
• time commitment

Safety

Experts share that clinical trials are generally safe and monitored.

“Most trials in the U.S. must be approved by an institutional review board (IRB) before starting recruitment,” Daigle says. “The IRB is made up of researchers, doctors, and lay people not associated with the trial. Their job is essentially oversight.”

The IRB also must approve any changes to the trial, such as the dosage of a medication.

However, like any intervention, they are not without risk. These risks can vary depending on what is being trialed.

Eligibility

Wood suggests asking both the people running the trial and the doctor for your condition about the risks and benefits so you can weigh them.

“It’s an important point because some therapies that you’re currently taking for your condition may exclude you from a new trial,” Wood says.

Data and privacy concerns

The additional tracking and associated data put your information in more people’s hands. Wood says the risk of a data leak is rare because protections are in place, but it’s a possibility to be aware of.

“There will have been an ethical review of the study making sure that your data are going to be handled well and discreetly and not shared with anybody else,” Wood says. “There’s always some risk that somebody will have access to your data, and something could happen with that.”

Extra time and effort

While the extra monitoring may be a benefit for some, others may not like it.

“For example, the travel burden may be too great or perhaps the cost of child care that would be required for them to attend necessary study visits,” Daigle says.

Questions to ask before enrolling in a clinical trial

Asking yourself, your doctor, and the clinical trial team questions before participating can help you make an informed decision you feel comfortable with.

Consider asking:

• What are the most common risks and outcomes?
• How often do I have to come in?
• What additional tests do I have to undergo, such as biopsies or blood tests, and how many?
• What is the expected outcome?
• Is it going to increase my quality of life?
• Is it going to help me learn more about my disease?

It’s a good idea to ask whether a trial will interfere with your ability to undergo other treatment options or personal plans, like travel, especially if the trial is slated to last for several months or years.

“It’s always fine to decline or say no,” Wood says. “With any clinical trial, you’re always free to remove yourself at any time.”

How do you find a clinical trial?

You can search for a clinical trial online through databases like:

• World Health Organization (WHO)
• ClinicalTrials.gov
• Research Match
• Center for Information and Study on Clinical Research Participation (CISCRP)

Condition-specific clinical trial databases include:

• Headache and Migraine Foundation
• National Cancer Institute
• American Lung Association
• American Diabetes Association
• Anxiety and Depression Association of America
• National Multiple Sclerosis Society
• Arthritis Foundation
• National Psoriasis Foundation
• Crohn’s and Colitis Foundation

If you don’t see your condition listed above, try a Google search with your condition name plus “clinical trials.”

Takeaway

A clinical trial involves testing a new medical intervention, like a drug or lifestyle tweak. Participation is usually free and may involve compensation.

Clinical trials can be a chance to receive new treatments, learn more about your condition, and contribute to science in the process.

However, they’re not for everyone. Talk with your healthcare team and weigh the benefits and risks before making a decision you feel good about.