Department of Health and Human Services

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Expanded Access

Access to Investigational Drugs or Biologics Outside of a Clinical Trial (Expanded Access)

Expanded access, also called “compassionate use”  pathways are designed to make promising medical products available as early in the drug and device evaluation process as possible to patients without therapeutic options, either because they have exhausted or are intolerant of approved therapies, and cannot enter a clinical trial. Expanded access refers to access to investigational medical products outside of a clinical trial, where the intent is a potential treatment, rather than research.

Just as in clinical trials, these investigational drugs have not yet been approved by the FDA as safe and effective. They may be effective in the treatment of a condition, or they may not. They also may have unexpected serious side effects. It is important for you to consider the possible risks if you are interested in seeking access to an investigational drug.

FDA regulations allow access to investigational drugs and biologics for treatment purposes:

  • on a case-by-case basis for an individual patient and Emergency Investigational New Drug (IND)
  • for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial.
  • for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.

What You and Your Healthcare Provider Need to do When Requesting Access for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use

When you or your healthcare provider would like to submit an Investigational New Drug application (IND) to gain access to an unapproved drug for you or a loved one, you need to follow the following steps:

  • first ensure that the manufacturer of the unapproved drug is willing to provide the drug.
    • If the manufacturer agrees to provide the drug, then
    • your healthcare provider should submit an IND to the appropriate review division.

The IND should include the following information:

1. Statement that this is a request for an individual patient IND for treatment use

(specifying whether it is an emergency IND or individual patient IND) should be at the top of the correspondence and on the mailing cover.

2. Brief Clinical History of the patient including:

  • the diagnosis
  • the disease status
  • prior therapy
  • response to prior therapy
  • the rationale for requesting the proposed treatment, including a list of available therapeutic options that would ordinarily be tried before the investigational drug or an explanation of why use of the investigational drug is preferable to use of available therapeutic options.

 

3. Proposed Treatment Plan including:

  • the dose
  • route
  • planned duration
  •  monitoring procedures
  • modifications (e.g. dose reduction or treatment delay) for toxicity.

Reference a published protocol or journal article if appropriate.

4. Chemistry, Manufacturing, and Controls Information and Pharmacology and Toxicology Information

including a description of the manufacturing facility.

The requirement for this information may be met by providing a Letter of Authorization (LOA) to refer to this information if it has been previously submitted to FDA (for example, to an existing IND or NDA).

The treating physician should contact the sponsor of the previously submitted information for such authorization and letter.

The letter of authorization should include relevant identifying information, such as the sponsor’s relevant application (e.g., IND) number.

5. Informed Consent Statement

that states that informed consent and approval of the use by an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment.

In the case of an emergency, treatment may begin without prior IRB approval, provided the IRB is notified of the emergency treatment within 5 working days of treatment.

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.

You can download Form 1571 and other forms from the FDA Web site. For forms and instructions please see: Information for Sponsor-Investigators Submitting Investigational New Drug Applications (INDs).

8. Contact Information

to include:

  • a telephone number
  • email address
  • facsimile number.

FAQ About Expanded Access

How Can I Get Expanded Access to an Investigational Drug?

The process must begin with your healthcare provider, who should follow these steps:

  • Your healthcare provider must contact the company that manufactures the drug to make sure it is willing to provide the drug.
  • Your healthcare provider must submit an Investigational New Drug (IND) [link to section below] application to the appropriate FDA review division.
  • In an emergency situation, the request to use the drug may be made via telephone or other rapid means of communication, and authorization to ship and use the drug may be given by the FDA official over the telephone. With emergency INDs, shipment of and treatment with the drug may begin prior to FDA’s receipt of the written IND submission that is to follow the initial request.
  • In a nonemergency situation, the IND must be received by FDA before shipment of and treatment with the drug may begin. These nonemergency requests are known as individual patient INDs.

Can Any Individual Patient Qualify for Expanded Access to Any Drug?

No, to gain access to an investigational drug outside of a clinical trial:

  • You must have a serious or immediately life-threatening disease or condition and no treatment options available to you.
  • The drug maker and your healthcare provider must make special arrangements to obtain the drug for you through FDA approval. These safeguards are in place to avoid exposing patients to unnecessary risks.

If I Meet These Criteria, Will I Qualify for Expanded Access to a Drug?

Not necessarily. Even if you meet these criteria, there may still be obstacles.

  • Your healthcare provider may not be able to seek expanded access for you because of your medical history or the risks associated with taking an investigational drug. Your healthcare provider must determine that the probable risk from the drug is not greater than the probable risk from the disease.
  • Your healthcare provider may not be willing to manage the use of an investigational drug.
  • The company that makes the drug is not required to offer it outside their clinical trials, and they may not be willing or able to do so.
  • The company may not have enough of the drug available for all patients requesting expanded access. Some companies establish a lottery system to determine which patients will have treatment access. Others make the decision on a case-by-case basis.

The FDA released a draft guidance for Industry "Expanded Access to Investigational Drugs For Treatment Use - Qs & As" in May 2013

Are Costs Associated With Expanded Access?

Investigational drugs are expensive to make. It is important to know that:

  • Some companies provide the drug for free to patients.
  • Some companies charge patients costs based upon how much the drug costs to make.
  • Most insurance companies will not pay for access to an investigational drug.
  • There also may be additional costs for administration and monitoring of the investigational drug by your healthcare provider.

The FDA released a draft guidance "Charging for Investigational Drugs Under an IND - Qs & A's" on May 2013

Am I Protected From Risks?

When a drug is investigational, Federal law requires that its use be reviewed by an institutional review board (IRB). The IRB works to protect the people receiving the drug and to ensure that the risks are reasonable given the potential benefit. However, significant unknown risks may exist. The IRB will require and review an informed consent document to make sure patients are aware of potential risks and are willing to accept the level of possible risk associated with the drug.

How Can I Find Out Whether I Can Access a Particular Investigational Drug Outside of a Clinical Trial?

Some drug companies have established expanded access programs. To find them:

  • Use our clinical trials search tool or visit ClinicalTrials.gov to view a list of expanded access studies.
  • Search for specific expanded access programs through an online search engine.
  • Call a drug company directly to ask about their policies.
  • Contact patient advocacy groups to see if they have information on expanded access options.
  • Talk with your healthcare provider about your options.
  • Contact us, the FDA Office of Health and Constituent Affairs, for information and assistance at OSHI@fda.hhs.gov.

Physicians can visit Physician Request for an Individual Patient Investigational New Drug Application (IND) for Compassionate or Emergency Use.

Information about expanded access to unapproved medical devices explains emergency use, single patient or small group access, treatment use, and continued access after a controlled clinical trial under an Investigational Device Exemption has been completed.

What Are my Responsibilities if I Choose To Seek Access to an Unapproved Drug Outside of a Clinical Trial?

You can do several things:

  • Talk with your healthcare provider to see whether use of an investigational drug for your treatment is right for you. Be sure to consider how much is known about the investigational drug, the severity of your condition, and the likelihood that the therapy will be effective. You and your healthcare provider should consider the kind of illness you have, the stage of disease, other conditions you may be experiencing, and organ function (e.g., liver and kidney function), among other factors.
  • Be sure your healthcare provider is willing to oversee therapy with a drug s/he is not familiar with, and to work with the company and FDA to obtain the drug, monitor you, and file necessary paperwork.
  • Consider the cost of the investigational drugs and the medical services associated with its use that are not covered by third-party payers such as insurance or Medicare.
  • Carefully read the informed consent presented to you and make sure that you understand the risks associated with the drug before signing. Federal law requires that this form also be signed and reviewed by an IRB before access to any investigational drug can be obtained. Your healthcare provider may or may not have access to an IRB, which is usually associated with a hospital or research institution, but there are independent IRBs as well. Depending on the IRB, a cost may be associated with the review, which may be your responsibility.

 

In an emergency situation, the request to use the drug may be made via telephone or other rapid means of communication, and authorization to ship and use the drug may be given by the FDA official over the telephone.  In these situations, known as emergency INDs, shipment of and treatment with the drug may begin prior to FDA’s receipt of the written IND submission that is to follow the initial request.

In a non-emergency situation, a written request (IND) for individual patient use of an investigational drug must be received by the FDA before shipment of and treatment with the drug may begin.  These non-emergency requests are known as individual patient INDs.

A number will be assigned to the application. The IND sponsor (treating healthcare provider) should provide this IND number to the drug supplier, so the supplier may ship the drug to the treating physician. The FDA will either allow the treatment use to proceed or not allow it to proceed (put the application on clinical hold).  The IND is considered active (treatment with the drug may proceed) 30 days after FDA receives the IND submission or upon earlier notification of the physician by FDA.  If the treatment use is not allowed to proceed (i.e., a clinical hold is placed on the application), FDA will notify the physician of this decision initially via a telephone call.  The call will be followed by a written letter that provides the reasons for FDA's denial of the request.

For more information about Expanded Access