What is a clinical trial?

UnknownHave you ever wondered whether participating in a clinical trial of a medication is right for you? Whether you are a candidate for a trial, and what kind of trial may be best for you, may depend on your disease, treatment history, symptoms, and overall health.

A clinical trial is a research study that involves human subjects (studies that involve animals are referred to as pre-clinical), and advances in modern medicine would not be possible without human studies. Clinical trials are required before any new medication (drug) can receive approval by the Food and Drug Administration (FDA) for sale in the United States. Even after reaching the market, many more studies of each drug are required before doctors know how best to prescribe the new medication.

If your doctor offers you the opportunity to participate in a clinical drug study, you should ask your doctor the following 8 questions (at least).

Whose trial is it? In other words, who is the “Sponsor” of the trial? The Sponsor starts and is responsible for the study, and may be a person (usually a doctor), institution (for example, an academic university), group (such as the government), or pharmaceutical company (Pharma). Sponsors can introduce their own bias, whether intentional or not, into the design of a trial, but each trial is very carefully monitored to assure the data presented is scientifically based and clearly unbiased.

What kind of treatment(s) is included in the study? Does the study involve standard of care drugs that are already on the market, but perhaps at new dose(s), or in a new combination? Or does the study involve an experimental (not on the market yet) drug, either alone or perhaps in combination with a marketed drug(s)?

What is the purpose of the study?
Why do researchers believe the study is necessary, and how will they know if it is working?
What is the Phase of Study? In general, there are 4 phases of clinical studies: Phase 1 trials are designed to define the side effects of a new drug, or new combination of drugs, and to determine the best and safest dose(s). Phase 1 studies are not focused on how effective a new drug or combination of drugs is against a particular disease. Phase 2 studies focus on how effective a new drug or combination of drugs is against a particular disease. Phase 3 studies usually compare the safety and efficacy of the new drug or combination of drugs to the current standard of care for the disease in question. Phase 3 studies are often large, randomized studies that may involve a placebo. Given their size and expense, Phase 3 studies are usually sponsored by the Government (for example the National Institutes of Health) or by Pharma. Phase 4 studies take place after a drug is on the market, and may provide additional details on a drug’s safety and efficacy.

What are the expected side effects, especially for me?

How many times do I have to travel to the clinical study site, and for what purpose, for example, to get blood drawn? Have a sample of tissue taken?

What costs will I be responsible for?

Why do you think this specific trial is right for me?

Enrolling into a clinical trial can offer advantages:

May provide access to promising novel therapies
Usually involves close monitoring of YOU by the research team
May cover the cost of care
Helps scientists learn more about your condition
May benefit you and/or future patients

However, you also need to think about the potential disadvantages:

May require more visits to the clinic than you want to do
May require extra procedures
Side effects may not be known (this is often the case)
Some costs (for example, a hotel for an overnight stay, or parking) may not be covered.

Before you agree to enroll into any trial, be sure that ALL your questions are answered, and to your satisfaction. It may be helpful to bring a family member or friend in to your doctor’s office to discuss the trial. You may also want to get a second opinion.

***This comprehensive explanation of clinical trials along with the appropriate questions patients need to ask was written by a Pharm.D. (Doctor of Pharmacy), Jeanne Noe, (Jeanne@PDstat.com).  She is a consultant who writes and designs clinical trials.

*Next blog:  A patient’s perspective of in a clinical trial.

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