/ Clinical trial guides

What are clinical trials and who should participate in one?

Clinical trials are research studies where volunteers help researchers look at new ways to prevent, detect, or treat conditions. The results of clinical trials help doctors find new treatments that are safe and effective for patients.

Why are clinical trials done?

Before a treatment can be approved for everyday use and made accessible to most patients, the U.S. Food and Drug Administration (FDA) requires that a potential therapy’s safety and efficacy be widely evaluated in a large group of human volunteers. These studies, called clinical trials, are the last stage in the clinical research process before a treatment, such as a new drug, can be made available to patients.

Clinical trials follow specific plans (protocols) developed by medical specialists to answer questions like:

  • Does the treatment cause fewer side effects than the treatments used today without being any less effective?
  • Does it improve the quality of life for patients with the condition?
  • Does the therapy work for people who aren’t being helped by the options that are currently available?

While the entire process to approve a new treatment lasts at least 10-15 years on average, participating in a trial can give patients access to therapies earlier than they would normally be available.

How does a treatment qualify to be used in a clinical trial?

drug_development_process

Before any clinical trial starts, the organization funding the study, also known as the trial's sponsor, is required to submit an Investigational New Drug (IND) application to the FDA. This application includes the data they've collected from animal studies to show that the researchers have done the needed steps before starting a clinical trial. It also includes information about how the drug is made, what's in it, and how the clinical trials will be done.

With new drugs, for example, the trials are started only after treatments discovered in labs have gone successfully through years of chemical and biological studies. Throughout the trial, the FDA and other organizations continue to make sure patient safety is the highest priority. Of the many potential treatments tested in these early stages, very few are promising enough to enter into a clinical trial.

Yet, clinical trials can (and often do) fail because not enough people volunteer to participate, which increases the time it takes for new treatments to become available. That’s why volunteers who participate in trials play an extremely important role in allowing new treatments to reach patients.

The different types of clinical trials

Clinical trials, also known as interventional studies, directly evaluate the impacts of a specific treatment or preventative measure on a disease. At least one group of participants in the study will receive a treatment (also called an "intervention") so that the researcher can evaluate its effects. There are many different types of studies, each unique in their treatments and goals.

Trials are run in multiple steps, called phases, that build on one another. Each phase helps answer different questions, but the treatment’s safety and efficacy is monitored throughout each phase. What phase a clinical trial is in gives an estimate for how much is known about the treatment being studied.

There are a total of four phases:
Trial-Phases-1

  • A phase 1 trial is done to find if a new treatment is safe for people
  • Phase 2 trials tell doctors more about how well the treatment works
  • In a phase 3 trial, the new treatment is compared with a standard treatment that is given to patients today

After the first three phases, if the treatment is determined to be safe and effective, the FDA approves it for clinical use and continues to monitor its effects. Phase 4 studies look at treatments that have already been approved by the FDA.

There are a variety of new treatments being studied for different conditions, but some common types include:

  • New drugs or surgeries
  • Different ways of using existing treatments, such as testing if a drug that treats one condition works well for treating a different condition
  • Medical devices (such as a pacemaker)
  • Natural treatments, such as herbal remedies
  • Changes in daily life, such as exercise, diets, or counseling

Observational studies

Observational studies are clinical research studies that don’t test a new treatment, but instead follow a patient’s health condition and medical history to learn how it might be treated. These studies have a wide variety of goals, like discovering risk factors for disease development or progression (called natural history studies), geographic or genetic correlations to diseases, or improving current treatment routines. Patients will usually continue their normal care routine and treatments when participating in an observational study.

Expanded access trials

Lastly, there are trials known as ‘expanded access’ studies. Expanded access, also known as “compassionate use,” provides a way for patients with a serious medical condition to access a treatment being tested in a clinical trial that they would not be eligible to participate in otherwise.

Participating in a clinical trial

A clinical trial can provide the best treatment option for you as you manage your medical condition. There are clinical trials available for almost every condition, in many different places around the world. If you take part in a clinical trial, you may get tests or treatments in a hospital, clinic, or doctor's office.

Trials can be the right choice for a variety of people, at a variety of levels of health. They can be right for people who have yet to find a treatment that works to treat their condition, or those looking for a treatment that might work even better than current options. They can be right for people who want to consider all available treatment options, or for people who have no options from formally approved drugs alone. Trials can be right for a perfectly healthy person, a patient suffering with a serious diagnosis, or any state of health in between.

What’s most important when deciding if participation in a trial is the right choice is understanding all the potential risks and rewards, and talking to your entire care team to choose the best course of action.

Here are the main possible benefits and risks of participating in a clinical trial:

Possible Benefits

  • Since the entire clinical trial process lasts 10-15 years on average, participating in a trial can give patients access to the newest treatments earlier than they would normally be available.
  • These treatments are held to higher standards, since the ultimate goal is to make them better than current treatment options. For example, psoriasis treatments currently in trials phases aim to completely eliminate all signs of the condition, whereas most of the currently available options aim to simply reduce the number of symptoms experienced.
  • Many patients report that the experience of enrolling in a clinical trial was similar to what they would have received without participating, but they got better care and received closer attention from the trial team.
  • The cost of the treatment is typically covered by the study’s sponsor, and they may also provide additional compensation to participants.
  • The trial may help scientists learn more about what treatments work, and help people in the future.

Possible Risks

  • The new treatment may not work better than the standard treatment does for you.
  • New treatments may have side effects that doctors do not expect or that are worse than those of the standard treatment.
  • You might be required to make more visits to the doctor than if you were receiving standard treatment.
  • Health insurance may not cover all patient care costs in a trial - this should be discussed with the study team before you decide to participate.

With over 50,000 clinical studies worldwide, participating in a trial is an opportunity to be part of medical breakthroughs that can change the lives of other patients in the future.

At Clara, we understand that clinical trials can seem daunting. Though we believe that the biggest barrier is simply accessing enough information to feel comfortable taking your health care into your own hands. That’s why we created Clara Guides – to walk you through every step of the journey. Have a specific question? Search it in our guides to find answers, or email us directly at hello@clarahealth.com!