Clinical trials are medical research studies that test new ways to prevent, screen, diagnose, or treat a disease by allowing the evaluation of potential new medical and treatment options. These studies are the result of long and deliberate preclinical drug development that often spans years even before the new therapy is tested in a patient. In the United States, all new drugs must proceed through an orderly clinical trial evaluation process – Phase 1, 2 and 3 – to ensure that they have an acceptable level of safety and demonstrate a clinical benefit in helping patients with the disease they target before they can become commercially available to other patients.

Talk to your doctor about clinical trials, and whether they may be an option for you.

There are three (3) phases of clinical evaluation prior to approval: Phase 1, 2 and 3. Clinical trials are often ‘placebo controlled’, meaning that some patients receive study drug, and others don’t (usually these patients receive standard of care). This allows the safety and effectiveness of the investigational drug to be compared directly to that of the currently used standard course of treatment.

  • Phase 1 studies are usually conducted in healthy volunteers, and are utilized to determine the drug's most frequent side effects and, often, how the drug is metabolized and excreted.
  • Phase 2 studies begin to elucidate the effectiveness of a new drug. The goal is to obtain preliminary data on whether the drug works in people who have a certain disease or condition. Multiple doses of the drug are often tested in Phase 2 studies. Safety and short-term side effects continue to be evaluated.
  • Phase 3 studies, which will enroll by far the most patients in any phase of clinical study, gather more information about safety and effectiveness, often studying different populations and different dosages and using the drug in combination with other drugs.

In addition, sometimes the FDA and the sponsor agree on post-marketing studies to gather additional information about the product’s safety, efficacy, or optimal use.

The U.S. Food and Drug administration has published information for patients on all phases; they can be found at the following link: http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htm

The objective of clinical trials is to test new therapies to determine if they have an acceptable level of safety and demonstrate a clinical benefit in helping patients with the disease they targets. There are a number of reasons that you and your doctor may want to participate in a clinical trial, including:

  • The potential to benefit from a new drug
  • The potential for improved management of symptoms resulting from treatment
  • The opportunity to directly contribute to the development of new therapeutic options to benefit other patients

If a new treatment is proven to work, then patients in the study would be the first to benefit from it. That said, there is no guarantee that any clinical study will be successful.

Requirements for clinical trials are strictly managed by the Food and Drug Administration. It is important to know that patients in clinical studies are carefully monitored by healthcare professions to see how they are doing and to monitor for side effects. So, patients enrolled in clinical trials often have the opportunity to interact with their health care professionals more often and are watched more carefully than patients not involved in a trial.

In addition, companies conducting clinical studies take their obligation toward safety very seriously. Before a drug is approved for testing in humans, the FDA ensures that it has gone through strict preclinical testing, often spanning years.

Discuss with your doctor whether participating in a clinical trial is right for you.

Clinical trials evaluate the safety, tolerability and effectiveness of new therapies that are not yet commercially available to all patients. If you elect to participate, then you would be part of a study that may provide a new type of drug for your condition, instead of or in combination with the therapies that are currently commercially available to you and other patients.

If a new treatment is proven to work, then patients in the study would be the first to benefit from it. That said, there is no guarantee that any clinical study will be successful.

Requirements for clinical trials are strictly managed by the Food and Drug Administration. It is important to know that patients in clinical studies are carefully monitored by healthcare professions to see how they are doing and to monitor for side effects. In addition, companies conducting clinical studies take their obligation toward safety very seriously. Before a drug is approved for testing in humans, the FDA ensures that it has gone through strict preclinical testing, often spanning years.

Discuss with your doctor whether participating in a clinical trial is right for you.

There are risks involved with the treatment for any life-threatening illness, including treatment offered through clinical trials:

  • The treatment may not be effective.
  • There may be unexpected side effects, some of which may be severe, resulting from use of the treatment itself.

Through the process of "informed consent," your medical team will make sure you understand all the risks and benefits before your first dose of the therapy being studied. Of course, the company developing the therapy will do everything they can to minimize the risk of unwarranted side effects.

Discuss with your doctor whether participating in a clinical trial is right for you.

The length of a clinical study varies depending on what is being studied and the phase of study. Participants are told how long the study will last before enrolling.

Discuss with your doctor about whether participating in a clinical study is right for you.

Clinical studies have standards outlining who can participate, called “eligibility criteria”, which are listed in the protocol (a research plan that described the clinical study and the drug). Some research studies seek participants who have the illnesses or conditions that will be studied, other studies are looking for healthy participants, and some studies are limited to a predetermined group of people who are asked by researchers to enroll.

The factors that allow someone to participate in a clinical study are called “inclusion criteria,” and the factors that disqualify someone from participating are called “exclusion criteria.” These are based on things such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.

Anyone interested in participating in a clinical study should know as much as possible about the clinical study and feel comfortable asking the research team and your doctor questions about the study, the therapy being tested, and any expenses. The following questions provided by the U.S. National Institutes of Health (NIH) might be helpful in these discussions, but should not be considered an exhaustive list. 

  • What is being studied?
  • Why do researchers believe the new therapy being tested might be effective? Why might it not be effective?
  • Has the new therapy been tested in patients before?
  • How will it be determined if I receive the drug or a placebo (if it’s a placebo-controlled study)?
  • Who will know if I receive the study drug during the trial? Will I know? Will members of the research team know?
  • How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?
  • How often will I have to visit the hospital during the study?
  • Will hospitalization be required?
  • Who will pay for my participation?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is part of this trial?
  • If I benefit from the study drug, will I be allowed to continue receiving it after the trial ends?
  • Will results of the study be provided to me?
  • Who will oversee my medical care while I am participating in the trial?
  • What are my options if I am injured during the study?

Discuss with your doctor whether participating in a clinical trial is right for you. Additional information on clinical studies is offered by the U.S. National Institutes of Health, and can be found here: https://clinicaltrials.gov/ct2/about-studies/learn