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Exploring Novel Treatment

If you want to explore new and novel treatments that have not yet been approved for brain tumors by the Food and Drug Administration (FDA), there are a few options to consider.

Clinical Trials

Many of the treatments for brain tumors that are used today were first studied in clinical trials.

Clinical trials are research studies that involve people. They not only provide early access to potentially beneficial treatments, but they also advance science and serve an integral role in the development process for new and improved treatments for brain tumors.

In the development process, clinical trials occur after a treatment has been studied in the laboratory setting but before the treatment has been FDA-approved for a particular condition. 

Clinical trials ensure that new treatments are both safe and effective through several rounds or “phases” of research. Earlier phases like phase 1 clinical trials are used to assess safety. Later phases like phase 3 clinical trials assess whether the treatment being studied is more effective than the current treatment standard.

Clinical trials for people with brain tumors rarely use placebos. A placebo is something that is designed to look like the treatment that’s being studied without having the potential benefit of the treatment.

You will need to meet certain criteria to be eligible for various clinical trials. To be eligible for trials for people with brain tumors, that might mean being a certain age, having a certain type of brain tumor, or having specific biomarkers in your tumor. If you are interested in participating in a clinical trial, please talk to your health care team.

If you and your health care team find a trial that you’re eligible for and you choose to participate in the trial, the research team will inform you of your rights. Even if you initially choose to participate in a clinical trial, you have the right to leave the trial at any time.

Expanded Access

Often, investigational medical products (those not yet approved by regulatory bodies) may be the only option for patients. The FDA has guidelines for how patients may still be able to gain access to investigational medical products outside of a clinical trial called “Expanded Access.”

The FDA approves approximately 99% of Expanded Access requests that it receives. However, there is no guarantee that the patient’s doctor will be able to apply for expanded access from the FDA, as they first need confirmation from the manufacturer that they will provide the drug. It is up to the company on what their policy for expanded access will be, and they may deny requests.

Through the 21st Century Cures Act, pharmaceutical companies must make public and easily accessible the information regarding the company’s policy for evaluating and responding to requests for expanded access (sometimes called “Compassionate Use”). If you do not qualify for a clinical trial, you may want to discuss this option with your doctor.

Off-Label Treatments

Sometimes a therapy that may be effective for the treatment of brain tumors or related effects is already approved for another cancer type or disease. In these cases, your doctor may prescribe the therapy through a process called “off-label” use. As long as there is evidence for the use of the therapy for your brain tumor, and your doctor does not intend to change the labeling or marketing of the therapy, the doctor will not need to get FDA approval for this.

Off-label use can also describe when your doctor gives you a different dose or dosing type (such as a liquid solution instead of a pill) of a medication than what is approved by the FDA. It also describes the use of therapies approved for adults in children.

Side Effects of Experimental Treatments

Though all investigational medical products require the FDA’s Institutional Review Board (IRB) approval for use in humans, it is important to note that unapproved medical products may have serious, unexpected side effects.

You should discuss the possible side effects of any treatment with your doctor and keep your medical team informed of any side effects you experience.

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