10 Questions to Ask about Clinical Trials

When patients and their families are considering enrolling in a clinical trial, they often have lots of questions — from “how does a clinical trial work?” to “what are clinical trial eligibility criteria?” — that need to be answered.

At myTomorrows, we have helped more than 10,000 patients around the world search for clinical trials. As part of a free service that we offer to help patients and their physicians find clinical trials, each patient who reaches out to myTomorrows is assigned a Patient Navigator.

Patient Navigators not only help patients find clinical trials, but they also have medical backgrounds and are trained to explain clinical trials and medical information in a way that is easier for patients to understand.

We thought about some of the common questions to ask before joining a clinical trial, and we made this list of 10 questions to ask before participating in a clinical trial.

What is a clinical trial?

Clinical trials are research studies that evaluate treatments or drugs that are being developed. When a new drug or treatment is in development, it must be tested extensively to evaluate its safety and effectiveness for patients.

Clinical trials may give patients the opportunity to get access to a new drug before it is available to the public. However, there also may be risks involved with participating in a trial, such as the risk of stopping your current medication or the possibility of experiencing side effects.

Watch our video to learn more about clinical trials.

What is the purpose of a trial?

The purpose of a clinical trial is to evaluate the safety and efficacy of a drug, treatment or medical device.

Clinical trials are an essential part of developing new drugs and ultimately making new drugs available to treat patients with unmet medical need. Participating in a clinical trial helps advance medicine for future patients by helping researchers understand how a medication works (or doesn’t work) for patients.

How does a clinical trial work?

Clinical trials gather data about the effectiveness and potential side effects of the drug or treatment being tested, and then researchers compare this data to standard treatments or placebo. This helps researchers figure out if the new treatment is safe and effective for specific patients — and if it is more effective than the standard treatment options currently available to patients.

Randomized controlled trials are considered the gold standard in measuring the effectiveness of a new drug or treatment compared to a standard treatment or placebo.

“Randomized controlled” refers to the way patients are randomly assigned to either receive the new treatment (the “experimental arm” of the trial) or the placebo/standard treatment (known as the “control arm” of a clinical trial). This is often done by a computerized or automated randomization system.

Randomized controlled trials may also be “double-blind,” which means that neither the patients nor the researchers conducting the trial know which patients are receiving the treatment being tested and which are receiving standard treatment (or the placebo). Blinding is done in order to help reduce any potential bias when researchers are comparing the new drug to the standard treatment or placebo.

Will I get a placebo?

Clinical trials for cancer or other serious conditions usually do not use a placebo. Instead, they often give patients the standard, approved treatment that is already available. In most cases, cancer clinical trials do not use a placebo, as it is often considered medically unethical to give a cancer patient a placebo if there is a standard treatment available. This may be one of the questions to ask about cancer clinical trials when patients are discussing their possible options with their oncologist.

However, placebos are used in clinical trials when there is no standard treatment available for a condition, such as a very rare disease. A placebo is designed to look the same as the drug being tested so that physicians and patients cannot tell the difference (but it does not contain any medicine).

Who can enroll in this clinical trial?

Clinical trials have eligibility criteria that explain who can take part in the clinical trial. For example, a trial may be seeking patients with certain types of mutations and a specific age range, while they may exclude patients with other unrelated health conditions (such as diabetes or mental health conditions) from taking part. This is known as inclusion and exclusion criteria.

Clinical trial eligibility criteria are designed to ensure that the patients participating in a trial are similar enough that researchers can be sure that the results they measure are due to the investigational drug being studied (and not affected by other factors such as age or other health conditions). Clinical trial eligibility criteria are also designed to keep patients safe.

What are the possible side effects or risks of the drug being tested?

Side effects are unintended effects that may be related to a drug that a patient has taken. Side effects can range from minor issues, such as a headache or an upset stomach, to life-threatening or even fatal incidents. Side effects are sometimes also called adverse reactions.

It’s important to discuss possible side effects and whether the potential benefits outweigh the potential risks of participating in a specific clinical trial with your physician.

What will I be expected to do during this clinical trial, and what type of time commitment is involved?

Each trial will require a different level of involvement and time commitment. It’s important to ask about the type of monitoring involved and the number of visits, blood tests, diagnostic procedures, etc.

It’s also essential to consider whether there is travel involved. Furthermore, patients and families should consider the impact of time away from school and work.

What does the clinical trial phase mean (and how long does a clinical trial take)?

There are four phases of clinical trials, from Phase 1 to Phase 4. Prior to these phases, something called “preclinical studies” are conducted. Preclinical studies are research studies that use animals to evaluate a drug — before any testing in humans ever takes place.

Here is a brief description of each phase, from Phase 1 to Phase 4, and an explanation of what each phase aims to study.

Phase 1 Clinical Trial: These early trials test a drug on a small group of people (usually about 10 to 30 people) in order to study its safety and side effects. Phase 1 clinical trials are also used to evaluate dosages and determine the correct dosage. These trials typically last several months to a year.

Phase 2 Clinical Trial: A Phase 2 clinical trial is focused on studying a drug’s effectiveness. These trials, which usually last about two years, gather preliminary data on whether a drug works in patients with a specific disease. Phase 2 clinical trials also continue to study safety and any possible side effects.

Phase 3 Clinical Trial: These are larger trials, typically ranging from several hundred patients to 3,000 patients. Phase 3 clinical trials, which usually last from a year to four years, are focused on studying how a drug works in diverse populations at various dosages.

Phase 4 Clinical Trial: Phase 4 clinical trials are conducted after a drug or has received regulatory approval (e.g., approved by the European Medicines Agency or the U.S. Food and Drug Administration). These trials monitor a drug’s safety and effectiveness and in larger and more diverse populations than earlier trials did.

On average, the process of evaluating a drug or treatment through these phases of clinical trials takes six to seven years. Overall, it takes at least ten years from the time a new drug is discovered until the time when that drug goes on the market and becomes available to patients.

Are there any potential costs involved for patients?

Patients usually do not have to pay for costs involved in a clinical trial. Drug manufacturers typically supply the drug used in a clinical trial free of charge, as well as pay for any required blood tests, diagnostics and extra doctor visits. Patients and their families should discuss any possible costs with the clinical trial staff and their insurance provider when considering enrolling in a clinical trial.

For example, in the United States, federal law requires most health insurers to cover the routine costs of care of patients who enroll in clinical trials as long as they meet certain conditions. These conditions state that the patient must be eligible for the clinical trial and the trial must be an approved clinical trial, and that the trial must not involve out-of-network physicians or hospitals if your insurance plan does not cover out-of-network care.

Further, it’s important to also consider any other non-medical costs that may be involved in participating in a clinical trial. Most clinical trials will help reimburse patients for expenses such as hotels, parking, tolls, gas, food, etc. However, there may be other costs such as childcare or the cost of lost wages from taking time off work that may not be reimbursed.

What will happen when the clinical trial ends?

When a clinical trial ends, sometimes the drug or treatment will be approved by regulatory authorities, such as the European Medicines Agency or the U.S. Food and Drug Administration, and it will go on the market and become available to patients. However, sometimes a clinical trial ends because the BioPharma company stops it due to poor results, inadequate enrollment, funding challenges or regulatory issues.

Sometimes there is also a window of time between when the trial ends and when the drug becomes widely available. In some instances, BioPharma companies may offer something called “post-trial access,” which allows clinical trial participants to continue to access a drug after a clinical trial ends. If you or a family member decides to enroll in a clinical trial, you may want to ask the principal investigator about whether there are any protocols for post-trial access.

To learn more about possible clinical trials and get help finding clinical trials, schedule a call with a Patient Navigator.

The information in this blog is not intended as a substitute for a medical consultation. Always consult a doctor before receiving a diagnosis or treatment.

The myTomorrows team
Anthony Fokkerweg 61-2
1059CP Amsterdam
The Netherlands