What Are Clinical Trials?

Clinical trials are research studies to test new drugs or other treatments involving participants who meet specific admission criteria. All new medications are required by the federal Food and Drug Administration to undergo clinical trials, which are designed to prove safety and efficacy.Because clinical trials are conducted in four phases, you may consider participation in any phase. Be aware, however, that admission criteria and the approved sites to conduct these trials vary. More often than not, Phase I and II trials are conducted in large research facilities, which may limit your access to a trial if you live far away. Phase III trials, on the other hand, are often more widely available and might even be offered in your own community. Phase III trials typically compare promising new therapies or combinations of therapies with standard (current) approaches; this not only helps scientists and clinicians know whether the treatments and the techniques are safe and effective but also whether they are better than the existing standard of care.

Currently, a very small percentage of eligible patients actually participate in clinical trials. Reasons for this low participation rate are varied and complex. Patients may choose not to participate because they lack basic knowledge about clinical trials and the potential risks and benefits. In some cases a physician will not recommend a trial for a patient; in other cases a patient’s cultural background might not support participation. Some patients may not know how to locate a clinical trial or get information about participating, whereas others express concern about the severity of investigational treatment or about the process of randomly assigning drugs. Additional concerns include travel time, transportation logistics, and increased number of clinic visits. And while these concerns weigh heavily on patients considering clinical trials, the biggest deterrent is often whether insurance will cover the costs associated with participation.

Some costs of a clinical trial may be covered by the trial’s sponsor, which can be a government agency, a pharmaceutical company, or even the National Cancer Institute (NCI). This information can be found in the informed-consent document that you’ll sign when you are accepted into the clinical trial. The informed-consent document will describe the study in detail and explain the possible risks and benefits of participation. In addition, it describes how medications are randomly assigned (if applicable), side effects, testing schedule, follow-up, and reporting procedures. Clinical trials by design collect a wide variety of information; however, your personal health privacy is protected. Privacy disclosures may be discussed in the informed-consent document as well as with your physician or the study coordinator.

The Patient Advocate Foundation (PAF) has authored a full-length publication titled Your Guide to the Appeals Process, which is available in English and Spanish. You can download this guide from www.patientadvocate.org or request a copy by calling PAF directly. This easy-to-read guide features sample appeal letters, specific strategies to use when negotiating the internal and external appeals processes, and precise instructions to follow when all appeals have been exhausted and it’s time for the next step. It offers insight into the principles that PAF case managers use to overturn coverage denials. The PAF publications and services are provided at no cost to patients with chronic, life-threatening, or debilitating conditions. If you need assistance with issues related to clinical trial participation or would like a copy of any of the PAF publications, call PAF toll-free at (800) 532-5274.

By Donna Sternberg, RN, OCN, Vice President of Patient Services and
Connie Slayton, BSN, Senior Case Manager; with
Nancy Davenport Ennis, President and CEO, Patient Advocate Foundation