Clinical Trials: Part 2. Identifying and Participating in Clinical Research Studies

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At some point in our lives, all of us could find ourselves or friends and colleagues faced with a disease or medical condition for which existing, fully approved drug treatments are not effective. We may have difficulty lowering blood pressure, controlling diabetes, attaining healthy cholesterol levels, combatting arthritis pain, or sending cancer into remission. Perhaps you or a loved one has been diagnosed with a rare disease with few, if any, therapies available on the market. A clinical trial may be your only option.

Making a decision about whether or not to enroll in a clinical trial is not necessarily an easy one. Too frequently, we are bombarded with news reports about study results that have been falsified by investigators affiliated with universities, hospitals, or pharmaceutical companies. Or, we may read about the withholding of significant side effect data that could have influenced a decision to participate in a clinical trial. When we hear about data that has been kept secret or manipulated to misrepresent trial results, we have to wonder if profit motives are sometimes more important than patient safety to those conducting and reporting on these studies.

So, what factors should we consider when determining whether or not to enroll in a clinical trial? Following up on my last column ("Clinical Trials Part 1. How Prescription Drugs Are Developed, Tested, and Approved," September 2012, pp. 42-46)), I now plan to identify ethical and safety issues one should take into account before seeking out a clinical trial and to provide resources for locating treatment trials focusing on specific diseases.

Types of Clinical Trials

Clinical trials are conducted for a number of reasons. We know that regulatory agencies, such as the U.S. Food & Drug Administration (FDA), require every new drug to undergo clinical study in order to be tested for safety and effectiveness. However, clinical trials may also be initiated to examine combinations of marketed drugs; to explore new ways to use or compare approved treatments; to evaluate therapies in different populations, such as children; to explore prevention options, such as vitamins, vaccines, or lifestyle changes; to investigate diagnostic and/or screening tools; and to improve the quality of life and comfort of those with serious chronic conditions.

Each sponsor of a clinical trial must develop a plan, called a protocol, which is approved by the FDA or the corresponding regulatory body in countries outside the U.S. The protocol describes what types of patients may enter the study. For example, age or gender restrictions and any other inclusion/exclusion criteria are written into the protocol, as well as descriptions of tests and procedures to be undertaken and scheduled, drugs and dosages to be studied, length of study, and outcomes to be measured.

Clinical trial sponsors must also disclose the risks and potential benefits of participating in a study. Trial participants must sign an informed consent document acknowledging they understand the risks and benefits of the trial. However, "signing the document and providing consent is not a contract. Participants may withdraw from a study at any time, even if the study is not over" ("Learn About Clinical Trials," ClinicalTrials.gov [http://clinicaltrials.gov/ct2/about-studies/learn#HowAreParticipants]). In other words, participating in a clinical trial is voluntary and one can leave a study at any time.

Ensuring Participant Safety

Each January, the Tufts Center for the Study of Drug Development (Tufts CSDD) publishes the "Tufts CSDD Outlook," highlighting the Center's "view on near-term pharmaceutical and biopharmaceutical drug development trends." The most recent 2012 "Outlook" is available at no cost at the following URL: http://csdd.tufts.edu/files/uploads/outlook-2012.pdf.

Although Tufts' latest report noted that clinical trial sponsors are conducting about 60% of their studies outside North America and Europe to lower costs, "that trend may be reversing, as coordinating trials in dozens of countries --and working with many regulatory authorities--imposes complexity and costs. …