Part 1 of 2
Enrolling in a clinical trial is not something that should be done lightly or without a good deal of thought. While there may be advantages to you, your family and to society, there are likely to be risks as well. People enroll in clinical trials for a variety of reasons. Some are seeking out new treatments for themselves or family members. Others have an interest in a particular disease. Many people find out about clinical trials from their physician. Others are identified through ads in local newspapers, television and radio announcements and posters. Regardless of why you may be considering enrolling in a clinical trail or how you found out about it, you will need some additional information before making a decision.
Clinical trials are studies done in humans that are designed to answer very specific questions. Examples of kinds of issues that clinical trials might examine include the use of a new treatment or a new diagnostic test, the impact of the environment on a disease, or how genetics determine clinical outcomes. When done correctly, clinical trials can add to our understanding of health, disease, diagnosis and treatments. The overwhelming majority of clinical trials are done responsibly. However, this was not always the case. As a result, a series of regulations have been established that define the ways in which studies should be done and provide for both government and institutional oversight.
All clinical trials done in the United States fall under a complex and sometimes bewildering set of state and federal regulations. The regulations are designed to protect the people who participate in clinical trials. Although they require a bit of interpretation, the regulations are actually well constructed and are based on a series of time-honored principles. The foundations for the regulations lie in two documents produced by the National Institutes of Health: the Nuremberg Code and the Belmont Report. Simply put, the regulations ensure:
To make sure that these principles are carried out, the federal government in 1974 mandated the formation of an oversight committee at every institution that conducts clinical trials. The committees, called institutional review boards (IRBs), were given the responsibility of ensuring the protection of people participating in clinical trials. The IRBs must have people from the community serving as full members in addition to experts from the medical profession and other professional staff. The activities of the IRBs are regulated by both the federal and in some cases state governments. In addition to meeting standards set by the government, IRBs can seek to be accredited by well-respected national organizations such as the Association for the Accreditation of Human Research Protection Programs and others.
Most clinical trials fall into one of three general categories:
From the standpoint of risks, interventional studies usually are at the top of the list, with non-interventional and observational studies ranking second and third, respectively.
Observational Clinical Trials:
Observational clinical trials involve the collection of information. There are no study procedures or laboratory tests. No new drugs or diagnostics are involved. Your involvement is usually limited to allowing the collection of:
The major risks of being part of an observational study are risks to privacy. Observational studies can involve the collection of a great deal of information about you and your family.
Non-Interventional Clinical Trials
Interventional Clinical Trials
Interventional trials are studies that do involve some action performed solely for study purposes. The intervention can include:
Interventional studies usually include some form of risk. The risks may be directly related to the drug or test or procedure. There is also the risk of not receiving the standard drug, test or procedure if you are assigned to the experimental group and the experimental action turns out to be less effective.
Interventional trials involving new drugs are usually conducted in a stepwise fashion (called phases). Each phase has a different purpose and helps answer different questions:
Next month, in Part 2, I will discuss how you can participate in a clinical trial, and what questions you should ask when making the decision.
Harold J. DeMonaco, M.S. is senior clinical associate in the Decision Support and Quality Management Unit at the Massachusetts General Hospital and is currently a Visiting Scholar at the MIT Sloan School of Management. He is author of over 20 publications in the pharmacy and medical literature and routinely reviews manuscript submissions for eight medical journals.