Compassionate Drug Use

What is compassionate drug use?

Compassionate drug use describes the use of investigational drugs by seriously ill people not enrolled in clinical trials. Investigational drugs are those that are being studied but have not yet been approved as safe and effective by the FDA.

Normally, investigational drugs undergo strict testing through many stages of clinical trials before they are available to the public. They are tested for safety, side effects, how well they work and at what doses, and with what conditions or diseases they are to be used. The process may take up to many years.

Compassionate drug use is legal and a fairly new practice. The FDA first approved investigational drugs to be used in this way for critically ill patients in 1987.

Who is eligible to use these drugs?

The most common means for people to access unapproved drugs is to enroll in a clinical trial. This is the safest route, as the FDA closely regulates clinical trials, and people taking new drugs are carefully watched. However, not everyone will qualify for a clinical trial. Because the use of new, unapproved drugs is risky, only certain people who are not enrolled in clinical trials can receive them. According to guidelines from the National Cancer Institute, eligible patients:

  • Have used standard treatments and found them unsuccessful

  • Are ineligible for any ongoing clinical trials

  • Have no acceptable treatment options

  • Have a cancer diagnosis for which an investigational drug has shown some benefit and is currently being studied in clinical trials

  • Are likely to have benefits that outweigh the risks involved

Accessing unapproved drugs

A patient's doctor may use a treatment investigational new drug application (Treatment IND) to apply for an investigational drug. It includes information on the patient's condition and past treatments.

The most common practices for getting investigational drugs to patients outside of clinical trials are through expanded access or special exception or single-patient exception.

  • Expanded access uses drugs that have been well-tested and are nearing FDA approval. The drug must show in studies that it is effective in treating a specific cancer. The program is offered by a drug company and the drug is given out to a group of people.

  • Special exceptions are individual requests (usually by a doctor) made to drug companies or to whomever is overseeing a program or clinical trial. The decision to give out the drug is weighed on a case-by-case basis. There must be some proof that the drug would either improve the patient's quality of life or lengthen his or her life.

Why not use an unapproved drug?

There are drawbacks to compassionate drug use -- for the patient and to the drug approval process. One is that they may interfere with clinical trials if more people seek this route rather than participating in trials. Although the NCI gathers some safety information on compassionate-use drugs, it is not as helpful in getting FDA approval of these drugs. Patients are just not watched as closely as they are in clinical trials.

Other problems include the chances of receiving no lasting benefit from the drug, undiscovered harmful side effects, and extra costs not covered by health insurance that may be associated with using the drug.

For more information

To learn more about investigational drugs and clinical trials, talk with your doctor about your options. Call NCI's Cancer Information Service at 800-4-CANCER (800-422-6237) or visit Acurian www.Acurian.com to search for clinical trials for your condition.

If you do not qualify for a clinical trial, your doctor can ask the trial's sponsor about requesting a single-patient exception. He or she may also contact the NCI or the company making the drug to find out if expanded access programs exist.

Physician request for a single patient IND for compassionate or emergency use

Source: FDA

When a physician would like to request an Investigational New Drug (IND) application to use an unapproved drug or other product for a single patient, the first step is to obtain permission from the manufacturer. Without the consent of the manufacturer, the unapproved product will not be available to the patient. After the manufacturer agrees to provide the product, the recommended procedure is to submit the following information to the appropriate review division. The request may be made by facsimile with a letter to follow.

  1. Request for a single patient IND for Compassionate or Emergency Use should be stated at the top of the correspondence.

  2. Brief Clinical History of the patient including the diagnosis, the disease status, prior therapy, response to prior therapy, and the rationale for requesting the proposed treatment.

  3. Proposed Treatment Plan including the dose, route, planned duration, monitoring procedures and modifications (e.g., dose reduction or treatment delay) for toxicity. Reference a published protocol or journal article if appropriate.

  4. Drug Supply Reference Statement naming the supplier or manufacturer and a statement that a Letter of Authorization to cross-reference an appropriate IND of the supplier or Drug Master File (DMF) of the manufacturer is included. The treating physician must contact the supplier or manufacturer for such a statement.

  5. Informed Consent Statement that states that informed consent and approval of an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment. There are some IRBs that have specific procedures for approving emergency requests.

  6. Investigator Qualification Statement that specifies the training, experience, and licensure of the treating physician. The first two pages of a Curriculum Vitae typically contain this information and are usually sufficient.

  7. FDA Form 1571 completed, with the treating physician listed as the sponsor. Form 1571 and other forms can be downloaded from the Internet.

  8. Contact telephone number and facsimile number. If the request is approved, an IND number will be issued by the FDA and the treating physician will be contacted by phone or fax with a letter to follow. The IND is considered active upon issuance of the number. The IND sponsor (treating physician) will then contact the drug supplier and provide the IND number. The supplier may then ship the drug directly to the treating physician.

For more information, please contact the appropriate Review Division:

 

Phone

Fax

CDER oncology drug products (most drugs)

301-796-2330

301-796-9845

CDER urologic and reproductive drug products (for prostate cancer hormones)

301-796-0950

301-796-9899

CDER medical imaging

301-796-2050

301-796-9848

CDER hematology products

301-796-7550

301-796-9849


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