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Expanded Access: Information for Industry

Information for Industry

What is expanded access?

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

When seeking expanded access to an investigational medical product, it is critical that the patient and his/her licensed physician consider all possible risks. Investigational medical products have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

Whenever possible, an investigational medical product should be used as part of a clinical trial. However, if patient enrollment is not possible (e.g., patient ineligibility, lack of ongoing clinical trials) or enrollment in a clinical trial is not feasible (e.g., distance to a trial precludes access), expanded access offers a possible route for gaining access to an investigational medical product.

FDA is committed to increasing awareness about its expanded access process and the procedures for obtaining access to investigational drugs, biologics, and medical devices.

Learn more about expanded access categories for drugs and biologics.

Learn more about expanded access categories for medical devices.

  1. Patient
    Consults with licensed physician to explore and decide about alternative options.
  2. Licensed Physician
    Agrees to oversee the patient's treatment and works with industry (e.g., medical product developer), files paperwork with FDA and IRB (for many expanded access request types), and is responsible for patient care and reporting.
  3. Industry (Company)
    Willing to provide the investigational medical product and either submits the expanded access request to FDA, allows the FDA to cross-reference their IND (for drugs and biologics) or IDE (medical devices) on behalf of the expanded access sponsor-investigator through the use of a letter of authorization, or provides the necessary investigational medical product information for the sponsor-investigator to submit to support an expanded access request.
  4. Investigational Review Board (IRB)
    Reviews expanded access protocol and consent to ensure that the patient is informed about the nature of the treatment.
  5. FDA
    Reviews the expanded access request and determines if the treatment may proceed.

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  • The 21st Century Cures Act requires that a company developing investigational drugs (including biologics) shall make its policy regarding evaluating and responding to requests for expanded access public and readily available. Posting this information on the Reagan-Udall Foundation’s Expanded Access Navigator, including links to a company’s own web page describing its policy for the investigational drug, can be used to satisfy this requirement.
  • When a company is developing multiple investigational drugs, they may establish different expanded access policies for each of their investigational drugs and post these individually by name as outlined above.
  • See FDA guidance: Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers Q31 for more information on the elements included in this requirement.
  • Companies developing investigational drugs should keep in mind that, among other requirements from FDA’s investigational new drug (IND) regulations, a sponsor, or a person acting on behalf of a sponsor, of an investigational drug product may not represent in a promotional context that an investigational drug is safe or effective for the purposes for which it is under investigation. For more information, see FDA regulations relating to INDs as 21 CFR 312.

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Reporting requirements vary depending on the type of investigational medical product and expanded access request.

Drugs and Biologics:

Medical devices:

  • For device expanded access requests, sponsors must submit follow-up reports concerning the outcomes of the expanded access use, including any adverse device effects. For individual patient emergency situations, reports should be submitted within 5 days as specified in 21 CFR 812.150(a)(4). Additionally, treatment use IDEs should follow the reporting requirements outlined in 21 CFR 812.36. Visit the medical device expanded access page to learn more about the reporting requirements for each type of device expanded access request.

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Drugs or Biologics: Visit the How to Submit a Request (Forms) page for step-by-step instructions.

Medical Devices: Visit the medical devices expanded access page.

If you are not familiar with the process for submitting a request for expanded access, you may want to contact the appropriate FDA organization or review division before submitting an expanded access request.

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From a public health perspective, early identification of important adverse events is beneficial. For example, a relatively rare adverse event might be detected during expanded access use, or such use might contribute safety information for a population not exposed to the drug in clinical trials. FDA is aware of a small number of cases in which clinical safety data from expanded access treatment was used to help assess the risks and benefits of the drug. In a very small number of cases, adverse event information from expanded access has contributed to safety information reflected in the FDA-approved labeling for a drug product. FDA is not aware of instances in which adverse event information from expanded access has prevented FDA from approving a drug. FDA reviewers of these adverse event data understand the context in which the expanded access use was performed and will evaluate any adverse event data obtained from an expanded access submission within that context. For example, FDA reviewers recognize that:

  1. expanded access treatment generally occurs outside a controlled clinical trial setting;
  2. patients who receive a drug through expanded access may suffer from a more advanced stage of the disease or condition than patients participating in a clinical trial;
  3. patients who receive a drug through expanded access may be receiving other therapies for their disease or condition at the same time as the drug they are receiving through expanded access; and
  4. patients who receive a drug through expanded access may suffer from one or more comorbidities.

All of these factors make it difficult to link an expanded access treatment to a particular adverse event. Moreover, it is extremely rare for FDA to place an IND on clinical hold due to adverse events observed in expanded access treatment.

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Additional resources

21 CFR 312 Subpart I
Learn more about FDA’s current expanded access regulations for investigational drugs (including biologics).

21 CFR 812.35 - 21 CFR 812.36
Learn more about FDA’s regulations supporting investigational device expanded access.

21 CFR 312.8 - 21 CFR 812.7
Learn more about FDA’s regulations on charging for expanded access to investigational medical products.

Form and Letter Resources
Guidance Documents
FDA Expanded Access Program Data
Articles and Reports
  1. Jarow, J. P., Lemery, S., Bugin, K., Khozin, S., & Moscicki, R. (2016). Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period. Therapeutic Innovation & Regulatory Science, 50(6), 705–709. Retrieved from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5135086/
  2. Jarow, J. P., Lemery, S., Bugin, K., & Lowy, N. (2017). Ten-Year Experience for the Center for Drug Evaluation and Research, Part 2: FDA’s Role in Ensuring Patient Safety. Therapeutic Innovation & Regulatory Science, 51(2), 246–249. Retrieved from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443559/
  3. Report from the 2018 external assessment of the Expanded Access Program

 

 

 

 

Page Last Updated: 11/08/2018
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