The expanded access navigator: Helping patients who need potentially life-saving drug treatments

Jonathan P. Jarow

by Jonathan P. Jarow, MD

You just diagnosed a patient with a serious condition for which there is no FDA-approved drug therapy.

Now what?

After the hard conversation that no physician wants to have with a patient and his or her caregivers and loved ones, you may be left feeling helpless and hopeless.

You know there may be investigational therapies in development but not FDA approved that could help your patient, but you don’t know where to begin.

Enter the Expanded Access Navigator, a new resource that can walk you through the often complex options of enrolling a patient in a clinical trial or obtaining an investigational product from the manufacturer through what is known as expanded access.

The navigator — available online at navigator.reaganudall.org — is a product of a collaboration among the Reagan-Udall Foundation, FDA, patient advocacy groups, the pharmaceutical industry and other government agencies.

The navigator was developed primarily for physicians and patients. From our conversations with physicians, we understood that the navigator also needed to be a resource for your staff.

Share this new resource with your staff and your patients. The result will be knowledgeable advocates who can better navigate the clinical trials and expanded access process.

Enrolling in a clinical trial is the preferred method of providing a patient with access to an investigational therapy. Clinical trials ensure adequate patient protection and are the best mechanism through which to collect the evidence of safety and efficacy needed before a drug maker can apply for FDA approval.

The navigator provides extensive information about finding an appropriate trial and how to apply for enrollment. You and your staff can access the ClinicalTrials.gov User Guide, which explains how to search for clinical trials recruiting in your area.

Each clinical trial in ClinicalTrials.gov describes the criteria patients must meet to be eligible for enrollment. There are times, however, when there are no actively recruiting trials for the investigational drug, your patient does not meet the enrollment criteria, or they cannot participate due to other reasons.

If your patient cannot participate in a trial, the navigator helps both you and your patient understand the requirements of the FDA’s expanded access program, including how to obtain an investigational drug directly from the manufacturer.

Expanded access allows the product’s manufacturer — with the authorization of the FDA — to provide an investigational drug to an individual patient, even though the drug is still in development.

FDA’s role primarily is to review expanded access submissions to ensure the safety of the patient. Since 2012, we have authorized more than 99% of requests for access to investigational products. The median time for FDA review is 4 days for nonemergencies and within several hours for emergency requests.

At FDA, we have heard from many physicians who are overwhelmed while exploring the expanded access process. Here are some examples of the questions received for which the navigator can provide solutions:

  • What is the first step to starting an expanded access request?
  • Is there a form that I need to submit to the FDA?
  • What if I don’t have access to an institutional review board?
  • How do I find a company’s policy on providing expanded access?
  • If my patient experiences a side effect, do I need to report it?
  • What questions can I anticipate from my patients about expanded access?
  • How do I close expanded access once the patient completes treatment?
  • Who can I call at the FDA if I have questions?

You can reach FDA’s Division of Drug Information (DDI) by calling (855) 543-3784 or by sending email to druginfo@fda.hhs.gov.

DDI’s staff is available around the clock, 7 days a week, to help you get started with your expanded access request and answer any questions you have after you review the navigator website. DDI also handles emergency expanded access requests and can help get your request in the right hands quickly.

Use of an experimental medication always carries risks to the patient, and it is wise to follow procedures employed to minimize those risks as much as possible. But the last thing a patient, his or her loved ones, and the physician needs is an overly burdensome process for access to treatment. The navigator helps to reduce that burden.

For more information:

Jonathan P. Jarow, MD, is senior medical adviser to the center director and chair of the medical policy council in FDA’s Center for Drug Evaluation and Research. He can be reached at Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993.

Disclosure: Jarow reports no relevant financial disclosures.

Jonathan P. Jarow

by Jonathan P. Jarow, MD

You just diagnosed a patient with a serious condition for which there is no FDA-approved drug therapy.

Now what?

After the hard conversation that no physician wants to have with a patient and his or her caregivers and loved ones, you may be left feeling helpless and hopeless.

You know there may be investigational therapies in development but not FDA approved that could help your patient, but you don’t know where to begin.

Enter the Expanded Access Navigator, a new resource that can walk you through the often complex options of enrolling a patient in a clinical trial or obtaining an investigational product from the manufacturer through what is known as expanded access.

The navigator — available online at navigator.reaganudall.org — is a product of a collaboration among the Reagan-Udall Foundation, FDA, patient advocacy groups, the pharmaceutical industry and other government agencies.

The navigator was developed primarily for physicians and patients. From our conversations with physicians, we understood that the navigator also needed to be a resource for your staff.

Share this new resource with your staff and your patients. The result will be knowledgeable advocates who can better navigate the clinical trials and expanded access process.

Enrolling in a clinical trial is the preferred method of providing a patient with access to an investigational therapy. Clinical trials ensure adequate patient protection and are the best mechanism through which to collect the evidence of safety and efficacy needed before a drug maker can apply for FDA approval.

The navigator provides extensive information about finding an appropriate trial and how to apply for enrollment. You and your staff can access the ClinicalTrials.gov User Guide, which explains how to search for clinical trials recruiting in your area.

Each clinical trial in ClinicalTrials.gov describes the criteria patients must meet to be eligible for enrollment. There are times, however, when there are no actively recruiting trials for the investigational drug, your patient does not meet the enrollment criteria, or they cannot participate due to other reasons.

If your patient cannot participate in a trial, the navigator helps both you and your patient understand the requirements of the FDA’s expanded access program, including how to obtain an investigational drug directly from the manufacturer.

Expanded access allows the product’s manufacturer — with the authorization of the FDA — to provide an investigational drug to an individual patient, even though the drug is still in development.

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FDA’s role primarily is to review expanded access submissions to ensure the safety of the patient. Since 2012, we have authorized more than 99% of requests for access to investigational products. The median time for FDA review is 4 days for nonemergencies and within several hours for emergency requests.

At FDA, we have heard from many physicians who are overwhelmed while exploring the expanded access process. Here are some examples of the questions received for which the navigator can provide solutions:

  • What is the first step to starting an expanded access request?
  • Is there a form that I need to submit to the FDA?
  • What if I don’t have access to an institutional review board?
  • How do I find a company’s policy on providing expanded access?
  • If my patient experiences a side effect, do I need to report it?
  • What questions can I anticipate from my patients about expanded access?
  • How do I close expanded access once the patient completes treatment?
  • Who can I call at the FDA if I have questions?

You can reach FDA’s Division of Drug Information (DDI) by calling (855) 543-3784 or by sending email to druginfo@fda.hhs.gov.

DDI’s staff is available around the clock, 7 days a week, to help you get started with your expanded access request and answer any questions you have after you review the navigator website. DDI also handles emergency expanded access requests and can help get your request in the right hands quickly.

Use of an experimental medication always carries risks to the patient, and it is wise to follow procedures employed to minimize those risks as much as possible. But the last thing a patient, his or her loved ones, and the physician needs is an overly burdensome process for access to treatment. The navigator helps to reduce that burden.

For more information:

Jonathan P. Jarow, MD, is senior medical adviser to the center director and chair of the medical policy council in FDA’s Center for Drug Evaluation and Research. He can be reached at Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993.

Disclosure: Jarow reports no relevant financial disclosures.

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