Clinical Trials

Finding new treatments, and how to best use existing treatments, is evaluated by clinical trials. Researchers test the hypothesis that a certain treatment may be effective for patients, under certain conditions. Clinical trials play a vital role in bringing new treatments to patients who need them most, securing data so that FDA approval can be obtained, and new drugs can be used in widespread clinical practice. There are currently more than 25 drugs FDA-approved for the treatment of advanced prostate cancer. As an example of the importance of clinical trials, remember that nearly all of them had to go through rigorous testing in multiple trials over several years.

Although they are often perceived as being a “last resort” for patients who have exhausted all other treatment options, in fact, trials span the prostate cancer experience, from better treatments for localized prostate cancer, to life-prolonging drugs for advanced disease, to symptom management, to changes in diet and exercise which can improve patient outcomes and survivorship.

If you are considering a clinical trial, speak to your doctor about the potential benefits of participating in a trial so you can make an informed decision that is best for you.

Remember: A common misconception about clinical trials is that the “placebo” group gets no treatment at all. In fact, in earlier-phase trials, every patient gets the new medicine, and when patients are included in phase 3 randomized trials, patients in the “control” group still receive the minimum standard of care.

Patients who participate in clinical trials become citizen scientists, providing an invaluable service both to treatment science and fellow patients. During the course of treatment, you may have the opportunity to join an observational study that does not affect your treatment, but adds to the body of data. This can be a great way for people going through cancer to “give back” to the greater body of knowledge.

Frequently Asked Questions

What is a clinical trial?

A clinical trial is the investigation of a new treatment for a disease. The work might be sponsored by a pharmaceutical or biotechnology company, government, university, or a private foundation. The purpose of these studies is to find out whether a new treatment or medical technology (such as imaging or medical devices) is safe to use and effective for various diseases or medical conditions. In order to study the treatment, several questions need to be answered first. For example, what patient population or disease is a drug likely to be effective in? What criteria should be used for accepting participants into the study? What general and disease-specific information are the study doctors going to obtain?

What are the phases of the clinical trial process?

There are three primary phases an investigational drug has to go through before it can be approved by the FDA, and a fourth phase that an approved drug may enter.

Phase I

In this phase of a clinical trial, the sponsor wants to determine the maximum tolerated dose for the medication. Mode of action (how the drug exerts its effects), safety and side effects–these are some of the main issues that are of the utmost concern to drug makers at this stage of the clinical trial process. It is important to note that in Phase I studies, the overall safety of the medication in patients has not been established. Efficacy in Phase I, if observed, must be considered anecdotal until confirmed in more advanced studies.

Phase II

A drug reaches Phase II only when the FDA has reviewed the Phase I data and concludes that the drug is safe for patients, and that its clinical activity may be beneficial against a particular disease or condition. At this point, a larger group of patients are enrolled and condition-ordisease-specific rating scales are used to record data. The goals are to continue safety assessment and to determine dosage for pivotal, Phase III efficacy trials.

Phase III

This is an important turning point for drug development. The medication has already passed the rigorous testing and review process required by the FDA, and now it is ready to be studied in an even larger patient population, with even more advanced rating scales and clinical measures. In recent years, there has been a growing industry trend to not only measure clinical effectiveness at this phase, but also measure “real world” results. For example, if patients show clinical improvement after entering the study, how are their activities of daily living improving (e.g.,patients can work in the garden more often or attend their child’s games on a regular basis)? Most medications that reach Phase III will at least be considered for approval by an FDA advisory board.

Phase IV

At this phase in development, the drug has already been granted FDA approval. Phase IV studies are often performed to either identify an additional use for an already approved drug, or to gather additional safety information from a larger group of patients. In some cases, Phase IV studies are implemented to establish effectiveness in a subgroup of patients, for example, patients over age 65.

How are a trial participant’s rights and safety protected?

In the U.S., the Food and Drug Administration (FDA) is the governing agency that develops the policies and guidelines for all clinical research, regardless of manufacturer, study phase or drug type. There are also independent Institutional Review Boards (IRB) at institutions that review and approve all study-related documents, such as protocols, Informed Consent forms, physician credentials and eligibility, and patient recruitment materials, such as print advertisements and public service announcements. (See “Protocol” and “Informed Consent” Form in the “CommonTerms Used in Clinical Trials” section.)

What are some questions I may want to ask my study doctor?

• How long will this trial last?
• What if I am on other medication(s) from my regular doctor?
• What are the risks involved?
• What if I am harmed?
• What types of tests and procedures will I need to undergo?
• What should I do if I want a friend/parent to join a study?
• Will I be paid?
• Will I be reimbursed for transportation?What kinds of medical problems would prevent participation in the clinical trial?
• Do I have to talk to my health insurance company before enrolling in a study?What if I miss a dose?
• Can I drop out at any time?
• Are there placebos involved

If you decide that a clinical study may be right for you, make a list of questions and discuss them with the study doctor at your initial visit.

Last Reviewed: 12/2023