Should You Take Part in Clinical Trials?

Clinical trials help researchers answer questions about specific illnesses or collect information they hope will have either immediate or long-term benefits in caring for patients. If you have hemophilia, you may be eligible to take part in clinical research trials, many sponsored at hemophilia treatment centers. (In the United States, there are more than 100 federally funded treatment centers; you can see a list of them on the Centers for Disease Control and Prevention’s Web site at www.cdc.gov).

Also known as clinical studies or research studies, clinical trials are important because they can advance knowledge that may not be possible to attain in the laboratory or through animal research. This might include, for instance, testing the effectiveness and safety of newly developed drugs.

For the medical community to better understand hemophilia and such complications as joint disease and the development of inhibitors, government agencies and public and private organizations conduct research that can be used to develop programs, medications, and therapies that can help reduce complications and improve the quality of life.

Universal Data Collection (UDC) is one such clinical study. About 85 percent of patients who are treated at a hemophilia treatment center participate in the UDC. Through questionnaires and exams such as blood tests and joint measurements, patients provide data that help researchers determine the frequency and severity of complications of hemophilia, describe treatment and patterns of care, assess quality of life, and determine health issues that need further study. Other areas of clinical research on hemophilia include a study to look at why some people develop inhibitors, an evaluation of healthy weight programs, and an evaluation of the U.S. National Hemophilia Foundation’s National Prevention Program.

Clinical trials have four research phases:

Phase I: A Phase I trial studies the first use of a new medicine or a new application of an existing medicine in humans. The drug is tested for safety and side effects, and to determine how it should be given. The number of patients accepted into Phase I trials is limited.

Phase II: If a drug is determined to be safe during a Phase I trial, it moves into Phase II where researchers investigate the most effective dosing of the drug. During Phase II, the medication is given to a larger number of patients so researchers can see how well it works, and whether there are possible side effects.

Phase III: In a Phase III trial, researchers compare experimental treatments to a placebo (an inactive substance that resembles the experimental treatment) or common or standard therapies. Patients who participate in Phase III trials are randomly placed (randomized) in a control group that receives the placebo or standard therapy, or in the experimental group. The objective of a Phase III trial is to find out whether patients who are given the experimental therapy have better, worse or equal results than those in the control group.

Phase IV: Sometimes a drug or treatment that has already been approved by a regulatory agency is studied further to evaluate its safety and effectiveness or to investigate a new use. This is known as a Phase IV clinical trial.

There are many things to consider when deciding whether to enroll in a clinical trial. On the plus side, not only will you receive top-quality care, but by taking part, you may also help others by increasing the medical community’s knowledge about a disease and its potential treatments. As a clinical trial participant, you may also be one of the first patients to see positive results from a new therapy.

It should be remembered, however, that clinical trials are sometimes unsuccessful. Patients can naturally invest a great deal of hope and expectation in a study, and it can be deeply disappointing if the end result is not an approved therapy. You should try to be clear-eyed about the possibility of an unsuccessful study outcome before you enroll.

Further, there is also a chance that you may experience unexpected side effects from the treatment, or that you could be placed in a control group that receives a placebo or standard care instead of the new treatment being studied.

In general, clinical trials are designed to minimize patient risks, and are overseen by review boards that ensure that the research being conducted is ethical and patient rights are protected. Either the review board or the regulatory agency can stop a study that may be too risky.

When deciding whether to enroll in a clinical study, you should discuss the pros and cons with your doctor and your family, of course. Support groups may also help you make up your mind, as can talking to patients who have taken part in clinical trials themselves.

In some clinical trials, patients are reimbursed for such expenses as travel and meals. Medical care is often provided at no cost, but if you are responsible for routine costs, such as lab tests, your health insurance may cover those.

Your doctor may refer you to a clinical trial, or you can explore the options available on your own by going online to visit the Web site of your country’s regulatory agency.

Ultimately, only you can decide if a clinical trial is right for you. If you do decide to participate, however, you could be making a valuable contribution to the health of future generations—just as previous generations made possible the treatments you receive today.