What difference might this treatment make to people?
Is it useful in the real world?
Even if a study finds that the difference between an old treatment and a new one is significantly different, it may not mean a lot of benefit for the patient.
Sometimes, a study result that shows a statistically significant difference between the treatment and control group may not make that much difference in people’s lives. For instance, a very large clinical trial can show that a treatment improves survival by an average of 10 days and that this was not by chance. Even though it’s significant statistically, for most patients, a treatment that extends life a bit more than a week may mean that the treatment may not be worth the side effects. Sometimes, it’s hard for even doctors to know how helpful a treatment may be when balancing its benefits with its side effects and other costs. Still, if you ask your doctor about a certain treatment that was found helpful in a good study, he or she may be able to talk to you about whether it may be worthwhile for you.
Of course, if a treatment is still being studied in human trials, there’s a chance that you could take part in such a study (see our document, Clinical Trials: What You Need to Know). Often this can mean you have to travel to the study site, which may cost a lot, although some study sponsors pay for part of the expense.
Keep in mind that in controlled clinical trials, you don’t get to choose whether you get the new treatment or the proven, standard treatment against which the new treatment is being tested. Also, you must meet certain criteria to qualify for the study. Common standards include being within a certain age range and having a certain type and stage of cancer. Depending on the study, you may have to have certain blood counts to be in it. Most clinical trials do not allow people who have problems such as kidney or heart failure, and other serious diseases including current infection (unless that’s part of the study). There may also be requirements about whether you’ve had certain treatments in the past.
How can I get an unapproved treatment?
The most common way to get a treatment that has not yet been approved is to take part in a clinical trial. But there are other situations where a drug may be used before the FDA approves it for general use, even if you can’t be in a clinical trial.
Sometimes a new drug has already been approved by the FDA for one purpose and is being studied for another. This means it may be available to you if your doctor agrees it might help you (see our document, Off-Label Drug Use for more on this).
Sometimes the drug can be used for certain people even before the FDA has approved the drug if “expanded access” or “compassionate use” are allowed. If this is the case, doctors can use the drug for certain problems, usually when other treatments are not working. (See Compassionate Drug Use to learn more about this option.)
Unfortunately, insurance companies will sometimes refuse to pay for treatments that are used to treat conditions for which they were not approved. One strategy you can use when your insurance won’t pay for an off-label drug is to ask your doctor whether any treatment guidelines for your condition recommend the off-label treatment. If they do, that can help your case with the insurance company.
What if I can’t find much evidence but I still want to try the treatment?
Of course, even if the evidence is not there, you may find a way to get the treatment and decide to do it anyway.
If you choose to do this, talk about it with your doctor to get his or her opinion of the treatment. See what you can find out about side effects, allergies, drug interactions, and other possible effects. Often, studies of “natural remedies” do not collect this sort of information, and it can be hard to find.
We encourage you to learn all you can before making your decision. You can always call us to see if we can help you get more information on any treatment you are considering.
Last Medical Review: 09/04/2012
Last Revised: 09/04/2012