“Expanded access, also referred to as “compassionate use,” is a potential pathway for the patients with an immediately life-threatening disorder or severe illness or disease to obtain access to an investigational medical product (drug, biological or medical device) for treatment outside of clinical trials where there are no equivalent or appropriate alternative therapy choices.
Expanded access may be suitable when all the following apply:
- People with serious illness or disability, or whose lives are instantly endangered by their disease or condition.
- No equivalent or adequate alternative treatment is available for diagnosing, tracking or treating a disease or disorder.
- It is not possible to register patients to a clinical trial.
- Possible benefits to patients outweigh future medical costs.
- Providing the investigational medical product will not interfere with investigational trials that might support a medical product’s development or marketing approval for the treatment indication.
FDA has not yet licensed or cleared investigational drugs, biologics or medical devices and FDA has not considered such products safe and effective for their particular use.
In addition, the investigational medicinal drug may or may not be effective in treating the disease, and use of the medication may cause unintended severe side effects.
What are the roles and responsibilities?
Exploring alternative approved care options, available and clinical trials or determining if expanded access could be another choice.
- Licensed Physician
Agrees to supervise the patient’s treatment and works with a company (industry), files paperwork with FDA and Institutional Review Board (IRB), and is responsible for the patient care and reporting.
- Company (Industry)
Willing to provide the investigational medical substance and working with the FDA and the licensed physician of the patient to provide the details needed for the extended access request or to submit the requests to the FDA.
- Institutional Review Board (IRB)
Ensures that appropriate steps are taken to protect the rights and safety of the participating persons as participants in a research study.
The IRB reviews the extended access application for expanded access, including the informed consent to help ensure the patient knows the nature of the planned treatment’s investigational medical product.
How should we post our expanded access policy?
- 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 the expanded access public and readily available.
- Posting this information on the Reagan-Udall Foundation’s Expanded Access NavigatorExternal Link Disclaimer, including links to a company’s own web page describing its policy for the investigational drug, can be used in order to satisfy this requirement.
- If a company develops multiple investigational drugs, it will carry out specific expanded access policies for each of its investigational drugs and post them individually by name as outlined above.
- Companies developing investigational drugs should keep in mind that, among other provisions of the FDA’s investigational new drug (IND) regulations, a sponsor of an investigational drug product or an individual acting on behalf of a sponsor may not reflect, in a promotional sense, that an investigative drug is safe or successful for the purposes for which it is being investigated.
- For more information, see FDA regulations relating to INDs as 21 CFR 312.
What are the reporting requirements for industry sponsors of expanded access?
Reporting requirements differ depending on the type of investigational medical product and expanded access request.
Drugs and Biologics:
- As with any IND, sponsors are responsible for sending IND compliance reports (as required by 21 CFR 312.32) and annual reports (as required by 21 CFR 312.33 if the IND or procedure persists for 1 year or more) to FDA (see 21 CFR 312.305(c) in both cases of extended access.
- For individual patient expanded access, the regulations (21 CFR 312.310(c)(2) specify that after treatment the sponsor must have provide to FDA a written summary of the results of the expanded access use, including adverse effects.
- Sponsors must provide follow-up reports on the outcomes of extended access usage, including any harmful user consequences, for application-widened access requests. Documents should be sent within 5 days for individual patient emergency cases, as set out in 21 CFR 812.150. (a)
Additionally, IDEs used for care will meet the reporting criteria set out in 21 CFR 812.36.”