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If you have exhausted standard treatments and want to turn to experimental therapies, consider the following guidelines:

Step 1: Look at all the available Clinical Trials to see if there is one that fits your need and for which you can qualify.

Step 2: If you don't qualify for a particular study, there are two other options for obtaining the drugs:

    • Expanded access programs
    • So-called individual "compassionate use" or Individual Special Exemptions

Each of these alternatives are discussed in other sections of this article.

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Expanded Access Programs

If the FDA approves, pharmaceutical companies can set up a formal expanded access program for drugs that have been studied and are in the approval process. These programs permit access to the drug before FDA approval, but outside of a clinical trial.

When a pharmaceutical company sets up an Expanded Access Program, it submits a protocol to the FDA including criteria for people to qualify. If you fit within those criteria, nothing further needs to be done to permit you access to the drug. If you do not fit within those criteria, an alternative way to obtain the drug is through the Individual Special Exemption discussed in the next section.

To locate an Expanded Access Program (EAP) that works for you:

  • Ask your specialist.
  • Look at the website of the manufacturer of a particular drug in which you are interested
  • Speak with patient advocacy organizations that specialize in your disease.
  • If you have cancer: a Web page that lists expanded-access programs for people living with cancer is offsite link. Cancer Action Now is a nonprofit that helps patients learn if they can gain access to experimental drugs.
  • Look at offsite link The site provides regularly updated information about federally and privately supported clinical research in human volunteers, including information about a trial's purpose, who may participate, locations, and phone numbers. Or call 800.INFO.FDA.

Compassionate Use - Individual Special Exemptions

If you don't qualify for a clinical trial or an Expanded Access Program, you can still request an individual special exemption which gives you permission to take a drug on a "compassionate use" or "compassionate exception" basis.

If the drug is in a clinical trial for which you don't qualify, you can attempt to get permission from the people involved in the clinical trial or apply to the FDA for an individual exemption.

Drug makers often provide drugs to individuals on a so-called "compassionate use" basis. The criteria for enlisting in these programs differs from company to company. Some companies have no policy and give approval on a case-by-case basis.

The FDA will consider each request on a case-by-case basis, to decide whether it agrees that there is a reasonable chance the drug will prolong survival or improve a person's quality of life. The FDA usually makes this decision within a few days after receiving the request.

To find such a drug:

  • Ask your specialist.
  • Speak with patient advocacy organizations that specialize in your health condition.
  • Search chat rooms on the internet of groups of people with your health condition.

In order to receive an individual special exemption you will need the cooperation of your doctor at least to obtain your treatment history.

How To Obtain Compassionate Use Of A Drug After The End Of A Clinical Trial

There is no guarantee that you will be able to continue to take a drug or treatment during the period after a clinical trial ends until the drug or treatment is approved by the FDA. As noted elsewhere in this document, you may be able to obtain the drug as a "compassionate use."

The investigator, the Institutional Review Board (IRB) that oversees the clinical trial, and the drug maker must agree to provide you the drug.

The place to start is with the drug maker. Either the drug maker or your doctor will then get the approval of the investigator and the IRB. Either the drug maker or your doctor will then approach the FDA about your particular situation and ask for a "single patient IND" granting permission to treat you with the drug.Once the drug maker agrees, it will

Your doctor is the appropriate person to contact the drug maker or person running the clinical trial (the "investigator) to request a compassionate use of a drug or treatment. Your doctor will also be asked to provide medical and treatment history.

Do whatever you can to help your doctor in this process. For example:

  • Some patients do the leg work to schedule a time for the doctor to call the correct person at the drug maker.
  • If your doctor won't take charge of helping you get a compassionate use of a drug, at least ask for a letter describing:
    • Your condition 
    • The treatments that have been tried
    • Why the drug you are requesting is so important to you and your health.
  • It may be up to you to find the person at the drug maker who decides who receives single-patient access and who doesn't. Generally this is the medical director for clinical development.

The Procedure To Apply To The FDA For A Single Patient Exception

Your doctor applies for you for a single patient exception.

If you'd like to see the process for your doctor to apply for a single-patient exception, see the FDA's web site at offsite link.