What Compassionate Drug Use Means for Cancer Patients and Oncologists

How Can You Get Compassionate Drug Use of an Investigational Drug?

doctor looking at pill bottle
Understanding the compassionate use exemption for investigational new drugs. Istockphoto.com/Stock Photo©psphotograph

What is meant by compassionate drug use for cancer patients? Think about these questions:

What happens if there is only one treatment being studied that could help you, but you don't meet the clinical trial enrollment requirements? What if you have failed all alternative treatments except for a promising treatment that has not yet been approved by the FDA?  When this occurs, the FDA has a backup plan in place. This is called the compassionate use exemption or expanded access to investigational drugs.

What is the Meaning and Purpose of "Compassionate Drug Use?"

Compassionate drug use refers to the use of an investigational drug (experimental drug) or medical device (one not yet approved by the FDA) outside of a clinical trial for treatment when there is no alternative satisfactory treatment available. Prior to FDA approval, an investigational drug cannot be sold or marketed in the United States.

What is an Investigational New Drug (IND)? - Review of Clinical Trials and the Process of FDA Approval 

Before discussing compassionate drug use, it can help to back up and describe the process of a new drug or procedure as it is developed, and ultimately either rejected or approved by the FDA approval process for use by the general public. For this discussion, I'll limit the process to medications alone.

The first step researchers use when evaluating a possible medication involves non-human studies. New drugs in this setting are tested on either cancer cells grown in a dish in the lab, or on other animals, such as mice. When these studies are considered sufficiently complete, human testing then goes through 3 phases of clinical trials. Phase 1 clinical studies are done on small numbers of people, and are designed to answer the question: "Is the drug safe?" Phase 2 trials are the next step, designed to answer the question: "Does the treatment work?" The final stage before FDA approval (or rejection) is phase 3 clinical trials, trials employed to answer the question: "Does the treatment work better than standard approved treatments, or with fewer side effects?"

Using this scenario, compassionate drug use would be the use of a medication that is in one of the phases of clinical studies before FDA approval, but not as a participant in one of the clinical trials.

When Might Someone Qualify for Compassionate Drug Use (Compassionate Use Exemption)?

Ideally, cancer patients who may benefit from an investigational new drug (IND) would be enrolled in an active clinical trial which is studying that drug. That said, some people who may benefit from a drug being studied may not fit the specific criteria for enrollment in that clinical trial for reasons such as age, previous treatments, performance status, or other excluding conditions. In this case, 2 criteria must be met:

  • There must not be a satisfactory alternative therapy available to diagnose, monitor, or treat a serious disease, and
  • The probable risk to the person from the investigational drug (or procedure) is not greater than the probable risk from the disease itself.

What is Expanded Access to Investigational Drugs?

If you read through FDA documents, you may wonder what the difference is between expanded access and compassionate use exemption, or if they are used interchangeably.The answer is that there are 3 tiers of expanded access, with the first referring to use by an individual patient.These tiers include:

  • The use of investigational drugs for individual patients
  • Intermediate-sized patient populations (up to 100), and 
  • Larger groups of patients (more than 100)

Requirements for Individual Access to Investigational Drugs for Cancer

The following requirements must be met to apply for individual access:

  • The drug (or procedure) must be for the diagnosis, monitoring, or treatment of a serious illness.
  • The patient must be ineligible for any ongoing clinical trials of the drug.  
  • The FDA must determine that the compassionate use exemption will not interfere with any stage of ongoing clinical trials of the drug.
  • There must not be a satisfactory alternative or comparable treatment available for the patient, or the patient is unable to tolerate these alternative therapies.
  • A patient must have a cancer diagnosis for which the investigational drug has demonstrated activity. In other words, the FDA must determine that there is enough evidence that the drug is safe and effective to justify its use for a particular patient.
  • A patient must usually have undergone standard treatment which is not successful.
  • The drug must be used for a serious or life-threatening condition, in which the risks of the experimental treatment outweigh the risks of not receiving treatment. In other words, the risk, including death, of the experimental treatment is considered to be less than the risk of death from the disease without the treatment.
  • Obtaining the drug involves the active participation of both the physician and patient
  • The physician must be willing to manage the drug and complete the treatment monitoring 
  • The company which makes the drug must agree to provide the drug (the FDA cannot "force" the company to provide the drug.) If the company requests payment for the drug, the patient must provide this payment.
  • The FDA, after receiving the application, will make a decision on whether or not to allow a compassionate use exemption. It's important to note that while this sounds like a difficult process, since 2009 the FDA has approved the vast majority if investigational new drug applications it has received.

Application Process for Compassionate Drug Use

There are 2 types of applications for compassionate use.  These include:

  • Emergency Use - In an emergency situation a request may be made by telephone (or other rapid form of communication) and an FDA official may give authorization over the phone to begin treatment. The treating physician must follow up this verbal authorization with a written investigational drug use application within 15 days of the FDA's verbal authorization to use the drug. (If there is not enough time to obtain approval from the investigational review board (IRB) in an emergent situation, treatment may begin without IRB approval as long as the IRB is notified by the physician providing the emergency treatment within 5 working days.)
  • Compassionate Use (Single Patient Access) - Unless there is a life-threatening emergency, the treating physician must complete an investigational new drug use application. Once this application is delivered to the FDA, the FDA has a 30-day period during which the application can be reviewed and a decision made as to acceptance or denial. Keep in mind that most of the time, the decision will be shared with the treating physician as quickly as possible.

    (Since this information changes with time, check the FDA sources listed at the bottom of this article for the most up-to-date information.)

    What You Need to Know as a Patient

    There are several things to keep in mind if you are considering the use of an investigational drug. These include:

    • The drug (or procedure) may have serious risks.
    • Since the drug (or procedure) has not yet been approved by the FDA, it is not known if the investigational drug is better than or worse than the standard drug. You may not receive any added benefit from the drug.
    • The short-term side-effects and long-term side effects of the drug are still not fully known.

    Your Doctor's Responsibility in Compassionate Drug Use

    When applying for compassionate drug use, it will be your treating oncologist (the one you see yourself for care) who will be responsible for the application, administration, and documentation of treatment.

    • The treating physician must fill out an application as described above.
    • The treating physician will be responsible for submitting a treatment protocol and proving the FDA with a report of treatment outcome, summary, and any side effects.
    • The treating physician is responsible for obtaining the drug from the manufacturer/developer, and to account for any remaining drugs after treatment is done.
    • The treating physician must agree to monitor the patient throughout treatment, following all guidelines and responsibilities as she takes on the role of the investigator for this patient.
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