Experimental Treatments: A Difficult Decision, Is The Risk Worth It?

by Food and Drug Administration (FDA)

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Companies sometimes say that they cannot make the drug available to a patient because FDA won't allow it, but that is rarely true. FDA only denies access when there is evidence that the risk of using the experimental drug clearly outweighs any potential benefit to the patient.

If a drug is frequently used in single-patient INDs, FDA streamlines the process for obtaining permission. One example is thalidomide, a drug initially associated with birth defects in the 1950s but now being used experimentally to treat cancer. (FDA approved thalidomide in 1998 to treat leprosy.)

FDA has similar rules that give patients access to investigational new medical devices.

A Difficult Decision

All things being equal, is it worth it for a patient to get access to an experimental medication?

For society the additional safety information about the new drug may prove useful. And sometimes it does make a difference for individual patients. For example, people with AIDS who participated in the clinical trials for a category of drugs called protease inhibitors probably benefited because this class of drugs proved so dramatically effective. But for many other INDs, the success rates are much less impressive, such as tacrine (Cognex) for the treatment of Alzheimer's disease.

Even if access does not change long-term survival, it may provide for the patient and the family a sense that they are doing something and are not simply victims of some serious disease. Biomedical research advances rapidly and breakthroughs come from unexpected places, all feeding the hope that the next experimental drug will be the one that cures our ills.

Is The Risk Worth It?

No matter how promising a clinical trial or investigational new drug seems, there is no way to know about all the risks before the study begins. While the hope is that the study will produce a cure, it's important to recognize that risks can prove significant. For example, in 1992, tests for a promising hepatitis B drug severely damaged the liver in 10 patients. Some died and others required liver transplants.

Because of these inherent uncertainties the health-care professionals conducting the study must ensure that the patient understands the risks as well as the benefits beforehand and is willing to proceed.

Here are some questions patients might want to ask to make sure they understand the consequences of entering a study or using an investigational new drug:

1. What are the potential benefits from the treatment being studied? What have the animal or other human studies shown about the effectiveness of the drug?

2. What are the potential dangers from using this drug? Again, what do other animal and human studies show about the side effects?

3. In what phase is this clinical trial?

Clinical trials are generally performed in three phases. A phase 1 trial is primarily designed to assess the safety profile in a small number of patients. Phase 2 tests the effectiveness of the treatment in a relatively small number of patients. Many drugs never progress beyond phase 2 because they are not effective. In phase 3, a large number of patients receive the drug to substantiate that the effectiveness seen in phase 2 is real and to work out the details of its use. Individual patients are most likely to benefit from drugs in the later phases of development.

4. Will there be a control group?

For a clinical trial to produce useful information, the group of patients receiving the new treatment needs to be compared with patients who receive something — or nothing — else. Often, patients in the control group receive whatever is the current standard therapy for the disease. Sometimes, the control group patients will receive a placebo — so-called sugar pills that produce no therapeutic benefit. In a clinical study, patients are randomly assigned to either the group treated with the experimental drug or to a group receiving the standard therapy or placebo.

5. How do I know if I am eligible to be in the study?

Every trial has a set of criteria to select the people that will be included in the study. These criteria generally relate to general health, stage of disease, and prior treatments and are designed to produce useful scientific information.

6. Do I have to pay to be in a clinical study?

Generally, studies funded by the federal government are free for the patient. Many studies funded by drug companies also do not cost anything. Some costs, however, may be paid by a patient's health insurance or managed-care plan.

7. So I'm just a guinea pig, right?

By the time most studies reach the stage where the new drug is being tested in people, a great deal is known about how it affects the body. While there is always the chance that something could go wrong, the safety of most drugs being studied is well understood. It is true, however, that researchers do not know if a treatment being studied works better than current therapies or not.

What FDA Does Not Do

Although FDA is responsible for overseeing the field of drug development, there are a number of services the agency cannot provide to individual patients. For one thing, it cannot give out the name of drugs in development, a common request from patients who call the agency. Unless the company publicly releases information about the experimental treatment, FDA is currently forbidden to even acknowledge that it knows about the drug.

Along the same lines, FDA cannot make the drug available to individual patients or physicians. The agency simply does not have the product; only the company that is developing the drug has a supply. And FDA has no authority to require that the company make its drug available outside of the clinical trial.

FDA, itself, does not conduct any clinical trials or drug studies. The agency carries out its drug review and approval responsibilities by examining clinical and other data generated by the drug company.

And lastly, FDA does not give advice. While staff from the Office of Special Health Issues and the Center for Drug Evaluation and Research's drug information branch will often provide detailed information and explain the process for getting access to an experimental medication, the agency does not steer patients in one direction or the other. Information is provided so patients, in consultation with their physicians, can make their own informed decisions.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.