Clinical Trials Homepage 12 Answers to Common Questions Is a Clinical Trial Right for You? Benefits and Risks Your Rights and Informed Consent Concerns for Women, Children and Genetic Privacy 10 Questions to Ask Fact or Fiction? Take the Quiz What to Expect in a Clinical Trial Glossary

The focus of clinical trials has changed in recent years. For decades, researchers mostly enrolled adult men and older women in clinical trials, but the number of studies devoted to children has grown enormously since 1997. Clinical trials that include genetic tests have also become increasingly common. These new developments have changed who enters clinical trials and the potential risks you may experience if you choose to enroll.

Younger Women

Clinical trials traditionally enrolled only men or older women because researchers feared exposing pregnant women to untested drugs. However, this meant that clinical trials rejected many women who were otherwise eligible and interested. Excluding women also prompted other concerns:

• Clinical trials should be fair. All people who meet a study's inclusion and exclusion criteria should be allowed to participate, regardless of gender.

• Many medical interventions act differently on men and women. Clinical trials that exclude women will not detect such potentially important differences.

• Some medical conditions occur more commonly in women than men. To find new ways to address these conditions, researchers must include women.

Today, clinical trials that exclude younger women must justify that decision.

Younger women considering a clinical trial need to consider some special circumstances:

• Many medications tested in clinical trials are not known to be safe for pregnant women. They may be safe, but that has not been proven in most cases. For this reason, clinical trials often require you to use certain types of birth control during the trial.

• Clinical trials of drugs that are unsafe for pregnant women may require you to take pregnancy tests during the trial.

• Similar concerns apply to breastfeeding. Nursing mothers may be ineligible for some research studies.

Children

Children are obviously a vulnerable group for research. Research on children must comply with strict federal regulations, and many studies conducted on adults cannot be approved for children.

For example, research that poses moderate or substantial risks to participants with no potential for direct benefit generally cannot be performed in children. Researchers who wish to enroll children in a clinical trial must meet all of these criteria:

• The child's parents or guardians must provide informed consent.

• Children must indicate their willingness to enter the study if they are of appropriate age (age 7 or above, according to the American Academy of Pediatrics).

• An Institutional Review Board must approve the study. IRBs alone can approve only studies that pose a minor increase in risk or that may directly benefit the children in the trial.

• For studies that pose more than minor risk, the federal government and a panel of experts must approve the study.

The American Academy of Pediatrics also has guidelines for research involving children (http://www.aap.org/policy/00655.html), but these are not mandatory.

Until 1997, clinical trials generally included only adults. Researchers wanted to avoid exposing children to the risks of research. Currently, the Food and Drug Administration encourages pharmaceutical manufacturers to conduct research with children, and many more trials involving children are under way. There are important ethical reasons to study children:

• Children deserve to have equal access to advances in medicine. This can only occur if clinical trials include children.

• Children are not "little grown-ups." They require careful dosing of medication. Because their bodies are not fully developed, they may have side effects that adults do not, such as stunted growth.

• Children differ a great deal from each other. Drugs may cause problems for children at one age, yet be very safe for children at another age.

Genetic Information

Clinical trials have recently begun to collect genetic information. Typically, this information involves certain genes that change your response to the intervention being tested. For example, clinical trials of medication for Alzheimer's disease may test participants for a gene that can increase a person's risk for the disease. As this field of medicine matures, even more clinical trials will use genetic information.

If you enter a clinical trial that collects genetic information, you should consider several issues:

• Confidentiality. Genetic information is sensitive and could be used to discriminate against people with certain genes. For example, you could have difficulty purchasing medical or life insurance or securing employment in the future if you have certain genes. Some states have enacted legislation to prevent discriminatory practices that draw on such information. Still, you should know exactly how clinical trials involving genetic research will maintain your confidentiality.

• Family. Genetic information pertains to more than just you -- your family may also be affected. Family members may have strong feelings about discovering or avoiding the results of genetic tests. Although you can decide whether to approach family members with test results, you may find these decisions difficult.

• Uncertainty. Most genes tested in clinical trials do not cause disease in everyone who has them. Rather, they simply raise your chances of getting the disease. Often, your chances of getting the disease are still low even with a gene that contributes to the disease. This degree of uncertainty may leave you uncomfortable.

• Anxiety. Finding out that you have a "bad gene" may provoke strong emotional responses, including anxiety or even depression. Counselors who help people understand genetic testing are available, but you should ask whether the clinical trial would cover the costs of such counseling before the trial starts.

• Time limits. Researchers sometimes store specimens from a clinical trial to do new tests that become available in the future. If researchers collect specimens for genetic tests, you can decide whether to restrict how long those specimens can be kept.

Published December 2002.

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