Clinical Trials: With Informed Samaritans, a Key Way to Advance Science and Medicine, Benefit Others -- and Receive Leading-Edge Therapies

10/22/2012 11:51 am ET | Updated Dec 22, 2012

Thousands of clinical trials take place every day in this country and around the world as part of modern science's best attempt to find answers to medicine's most pressing questions. How can we extend quality life among cancer patients? Which drugs work best to control blood pressure? Can we find a method of slowing the progression of Alzheimer's disease? How can we help prevent breast cancer in women we know to be genetically vulnerable?

We all have benefitted from informed patients willing to volunteer for medical research. Clinical trials paved the way for the Salk vaccine to erase the terror of polio in the 1950s; they have proven that drugs that lower blood pressure and cholesterol can reduce deaths from heart disease; and more recently they have allowed revolutionary new drugs like Gleevec, a targeted anti-cancer drug, to give many patients with a type of leukemia a new lease on life. For patients with serious disease that is unresponsive to conventional treatment, clinical trials can offer hope and help.

But people volunteering for medical trials must know what they're getting into. And, according to a nonprofit organization dedicated to educating the public as well as professionals about clinical research, people have much to learn. A survey by the Center for Information and Study on Clinical Research Information found that, while 94 percent of people understand that clinical research is important in advancing the goals of science, 75 percent of the general public say they don't know much about clinical trials or how to participate.

What's a Clinical Trial?

Since more than a million children lined up for vaccinations against polio in 1954, the rules that researchers follow in testing vaccines, drugs, medical devices and procedures have been refined to most efficiently advance science while protecting the lives and well-being of volunteers.

Before a clinical trial is even planned laboratory research is run first in test tubes and petri dishes, followed with further research on laboratory mice or other animals. All of this nonhuman research takes about a decade before a drug or treatment even can be tested in the first human being.

Medical scientists may conduct other kinds of research that do not involve clinical tests. Epidemiologic studies, or observational studies, compare two similar groups of humans who are as much alike as possible except for one factor. They ask questions about past behavior. Or they observe people over a long period of time, such as has been done for more than 60 years in the Framingham Heart Study. By asking questions or observing, these studies show associations between a behavior and an outcome -- but they don't provide definitive proof. Such studies helped scientists figure out that smoking, for example, leads to heart disease and lung cancer and that high-fat diets lead to diabetes.

Out of those necessary and preliminary studies emerge hypotheses, or theories, worth putting to the gold standard of scientific proof -- a clinical trial. In such trials, groups of similar patients undergo either a new form of treatment, the standard treatment of the time or a placebo treatment. In cases of serious disease, such as cancer, patients get either the new treatment or the current standard of care.

What You Need to Know

If you or a loved one decides to participate in a clinical trial, you'll fill out informed consent forms. Someone closely involved in the trial will talk with you and answer all your questions. Beyond that, make sure you understand what questions researchers hope to answer. Do they hope to find out whether a new drug works better than an existing drug? Are they testing whether giving a therapy before surgery, say, works better than giving the same therapy after surgery?

Find out how many people have already been tested. If you are among the first humans to try a new drug, the trial will be a Phase I trial, with the goal of determining if the treatment is safe. Phase I trials enroll small numbers of patients, and while they may prove beneficial in rare cases, it is only safety that is being explored. Phase II trials test dosage levels and begin to explore whether the treatment is effective as well as safe. Phase III trials are much larger, involving multiple researchers in hospitals and clinics across the country or around the world. In those trials, a patient may receive the new treatment, the old standard of care, or a placebo -- and what each patient gets is randomized. Even the researchers don't know which patients are receiving which treatments -- which is why such trials are called "double blinded."

As a patient, you may withdraw from a trial at any time for any reason, or for no reason at all. And before you sign up, it's perfectly appropriate to get a second opinion from a physician unconnected to the study.

High Cost of Low Participation

Most people agree in theory that scientific research in general, and clinical trials in particular, are good things. But many trials are delayed because not enough people enroll. In fact, the average clinical trial is delayed 4.6 months because of slow enrollment. One analysis looked at 114 clinical trials and found that less than a third of them signed up enough volunteers within the time allotted for recruitment. About half of those trials were granted more time to sign up volunteers, but that means added costs and more time passing before any potential new treatment is available to the public.

As time-consuming as they are, clinical trials still provide the safest and -- given the important potential for improving health for millions -- the fastest way to keep medicine moving forward.

A key informational element that will be provided to you if you wish to participate is who is running a clinical study and who is funding it. The main researcher, dubbed the principal investigator, typically will be a physician or possibly a Ph.D. or RN; there may be others in the research team, scientists, nurses, social workers and others with roles in health care. It's a key part of informed consent that you know who is supporting the research -- parties that may include: drug companies; universities, academic medical centers and other institutions; organizations, especially those focused on specific diseases and conditions; and, of course, government agencies.

If you're uncomfortable with the researchers or their funders you should not participate in their clinical trials, though it's well worth your time to make a thoughtful analysis of your possible objections. You will be voluntarily participating and will do so only on an informed basis, so you should never think of yourself as someone who is ill, helpless and about to be exploited or experimented upon, solely for the benefit, say, of some big corporation or giant government agency or institution. As discussed, the clinical trial represents a part of a long, sometimes costly commitment and investment to improving care and benefiting many people; you're a prospective partner in this big challenge.

It's also worth noting that studies can include payment or financial assistance, particularly early on in research. These aim to address the time and discomfort involved for voluntary participants. You're not going to get wealthy off these sums and details of how, how much and why you will receive them will be clearly spelled out for you. You also should feel free to inquire of those involved in research if they may have declared any financial conflicts of interest, for example, in referring patients for particular studies.

Finding a Clinical Trial

The National Institutes of Health has made it easier to sort through the 132,616 trials it has listed on its web site ( Disease by disease, you can locate clinical trials sponsored by the NIH or private industry. You can find out where they're being carried out, if they are enrolling patients, what phase the trials are in and which patients are eligible for the trial. If the study has been completed, you can see a summary of the results, including any adverse events.

If you live near a specific academic medical center such as my own, you can go directly to that center's web site for a list of trials currently under way at a particular institution.

Your own physician or the hospital or medical center where you're receiving care also may play a role in referring you to a clinical trial. Medicine, it is true, is a business and often a competitive enterprise. But I can tell you with great confidence that for most physicians, your best health outcome trumps all, and, if your options are dwindling and someone else at another place can be helpful to you, the MDs I know won't hesitate to send you there. Talk to your trusted physician, of course, about any clinical trials that may be appropriate, and you may need his or her assistance in helping you to enroll in a trial.

Eligibility, Insurance

While clinical trials can offer experimental therapies otherwise unavailable to those with serious and late-stage disease, researchers must design and control their studies carefully to ensure they're finding scientific results that can be documented and repeated. They work under rigorous internal and external scrutiny about their human testing. This also means that patients must be screened and they must meet clinical eligibility requirements that are clearly specified. Certain aspects of your individual health or history may disqualify you. You may need to undergo preliminary exams, tests or procedures to ensure you're the right candidate for a clinical trial. If you've kept your current physician, specialist or hospital in the loop, you may be able to skip repeat tests that can be uncomfortable and costly.

That last element -- cost -- also cannot, unfortunately, be ignored. Periodically stories pop up in news pages and broadcasts about patients with dire illnesses who may be helped by therapies still in the test or clinical trial phase. The obstacle to this crucial care is typically depicted as an obstinate, hard-hearted insurer. And certainly, patients not enrolled in clinical trials should not be given unproven drugs and expect insurers to cover the costs. But in rare cases, the Food and Drug Administration allows drug companies to provide experimental drugs to patients outside of clinical trials in an exception called "compassionate use."

Even within a clinical trial, insurance coverage can provide challenges for your participation. Insurers may deem the therapy under testing to be "experimental" and decline to pay for parts of the charges because of your being in a clinical trial, though you can still expect insurance payment for aspects, like lab tests and conventional therapies that would be part of conventional treatment. Your "home" physician and hospital may prove helpful in resolving such issues, as can the research team and its support staff at the institution running the clinical trial. Before you build your hopes and get too excited about joining a clinical trial, investigate your eligibility and coverage and find as many allies as you can.

Why Volunteer?

For clinical trial participants, the outcomes simply are not known. Along with hope for a cure or other benefit goes risk of unanticipated side effects. Researchers are ethically bound to point out any known risks but the very nature of experimental treatments means a lot is not known. So why do it?

In some cases, the reason is that patients have run out of other medical options, nothing so far has worked and a clinical trial can give them access to a treatment that is otherwise unavailable.

But often their reasons are even more generous. Some people, stricken out of the blue with a tragic disease, or perhaps part of a long genetic line of people susceptible to a disease, simply want to be part of science's hopeful reach into the future. Many clinical trial participants, regardless of their reasons, are genuine altruists, and we need many, many more of them. We should be grateful to all who participate.