Making Sense of Clinical Trials

Before your diagnosis, you may not have given much thought to clinical trials -- or even knew what they were. Maybe you thought clinical trials were for "guinea pigs" or people without options. In truth, people who participate in clinical trials are pioneers. Almost all of our progress in cancer research is because people have tried the latest, most novel treatments, tests, or procedures with the hope of improving outcomes for themselves, their family members and generations to follow.

Choosing to participate in a clinical trial is a serious decision. Undertsanding how they work can help you weigh your options so you can speak more confidently with your healthcare provider.


What is a Clinical Trial

A clniical trail is any research study that tests new medical approaches, medicines or treatments work in people and determines their safety and efficacy (do they work).

Private companies, educational institutions and government organizations sponsor clnical trails to find out whether there are better ways to prevent, diagnose, or treat disease. These new therapies or combination of therapies may have better outcomes than the current standard treatment. Another possible benefit is that the new medication or procedure may be as effective as the standard treatment, but have fewer side effects. Types of clinical trials include:

Most trials are conducted in phases and each phase has a different goal.

Phase I trials are the first time a medication is tested in human beings. These trials study how a medication should be given (by mouth, injection, intraveneously) and how it is absorbed. They establish a safe dose range and identify side effects. A small nuumber of people participate.

Phase II trials look at the safety of the medicine, begin to evaluate how it works and sometimes determine the most effective dose. Usually fewer than 100 people participate.

Phase III trials compare the new medication, new combination of medicines or procedure to the current standard treatment. These studies include hundreds of participants. Most medicines or procedures that reach phase III have shown promise in an earlier phase. The data from these trials are use to secure regulatory authority (e.g. FDA in the US) for approved use by clinicians for a specific intended purpose (e.g. to treat a specific stage of breast cancer).

Phase IV trials are done after a medicine or treatment is approved by the regulatory authority (Food and Drug Administration or FDA in the US). These trials evaluate side effects associated with long-term use that may not have been seen in phase III trials and include thousands of participants.


Trial Protections

To protect your rights and ensure safety, every trial is subject to FDA regulations and must be reviewed and approved by an Institutional Review Board or IRB. IRBs are made up of a diverse group of people with a variety of backgrounds and include consumers to ensure a thorough, fair review. They review and approve trials with your safety in mind.

You will be required to sign an informed consent, a document that says you understand the treatment, tests, side effects, possible risks, benefits and costs. Your physician or nurse will review this with you carefully. You have the right to withdraw your consent/participation at any time. The Health Insurance Portability and Accountability Act (HIPAA) also protects your privacy by limiting the way hospitals, insurance companies, pharmacies and others share your personal medical information.

Trials are reviewed periodically by a Data and Safety Monitoring Board, an independent group not directly involved with the trial. The DSMB ensures the trial continues to be safe by examining interim data on severe side effects and other technical aspects of the trial.

Some terms used during a trial

During the course of the trial, some terms may be used. You should become familiar with them:

Double-blind trials mean neither the patient or clinical team know whether the patient is receiving the trial medication, a placebo (sugar pill), or the standard treatment.

Single-blind trials mean the patient does not know what they are receiving (i.e. placebo, trial medication or standard treatment), but the clniical trial does.

Open-label trial is where all the participants receive the medication being tested in trial. There are no placebos.

Non-blinded trials are the opposite of Double-blind trials in that both the patient and clinical team know what they are receiving.


Making the Decision

Participating in a clinical trial is a personal decision that you make with the input of your healthcare team and your loved ones. Write down your questions or speak into a tape recorder before visiting your healthcare provider. Here are some things to consider:

Besides receiving a possibly better or less toxic treatment, participating in clinical trials can make you feel good ofor a long time after your cancer experience.


© 2006, CareLife Inc.,
Page Revision Date: 29-Jan-2006