- The patient has a life-threatening or serious disease or condition;
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; and.
- Potential patient benefit justifies the potential risks of the investigational device.
What is the FDA’s approval process for Compassionate Use?
After a compassionate use request is received, the FDA will either approve, approve with conditions, or disapprove the request. When there is an IDE for the device, compassionate use request IDE supplements have the same statutory 30-day review cycle as other IDE submissions. However, the patient need is considered when reviewing these requests.
Can you use compassionate drugs in clinical trials?
These drugs are normally available only to people who are taking part in a clinical trial. Being able to use one of these drugs when you are not in a clinical trial is most commonly referred to as compassionate drug use. Is compassionate drug use legal? Compassionate drug use is legal, but it’s limited to people who meet certain conditions.
When to use compassionate use criteria and procedures?
The above compassionate use criteria and procedures can also be applied when a physician wishes to treat a few patients rather than an individual patient suffering from a serious disease or condition for which no alternative therapy adequately meets the patient’s medical need.
Who can benefit from compassionate use of unapproved drugs?
Patients with serious or life-threatening conditions who aren't able to get access to an unapproved drug through a clinical trial might benefit from compassionate use, if it's available.
What is the difference between compassionate use and emergency use?
Compassionate use means that the use does not meet the criteria for emergency use, and therefore prior FDA approval (IDE) is required before the device can be used. Standard IRB review and approval (like a research study) is required for neither emergency nor compassionate use.
Is compassionate use the same as expanded access?
Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside ...
What is a compassionate use patient?
Listen to pronunciation. (kum-PA-shuh-nut yoos) A way to provide an investigational therapy to a patient who is not eligible to receive that therapy in a clinical trial, but who has a serious or life-threatening illness for which other treatments are not available.
What is compassionate in clinical trials?
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 ...
What is FDA compassionate use?
The compassionate use provision provides a path to accessing investigational devices that have not received FDA approval or clearance for patients for whom the treating physician believes the device may provide a benefit in diagnosing, monitoring, or treating their disease or condition.
How do you get compassionate drug use?
To be eligible for Right to Try, a person must:Be diagnosed with a life-threatening disease or condition.Have tried all approved treatment options for the disease or condition.Have a doctor certify that they are unable to participate in a clinical trial for the investigational drug.More items...•
Is compassionate use the same as right to try?
What is the Food & Drug Administration's (FDA) Expanded Access Program? Also known as “compassionate use,” this program allows people with immediately life-threatening conditions or serious diseases or conditions to gain access to investigational medical products.
When did compassionate use start?
In the US, compassionate use started with the provision of investigational medicine to certain patients in the late 1970s, and a formal program was established in 1987 in response to HIV/AIDS patients requesting access to drugs in development.
What compassionate use means for gene therapies?
Compassionate use is pre-approval access to an investigational medical product outside a clinical trial. Trial participation is the preferred mechanism for pre-approval access to investigational drugs because the systematic collection of comparative data is necessary to support applications for marketing authorization.
Is compassionate use research?
IRB agreement that a particular case meets FDA criteria for emergency use applies to the treatment of one patient only and is not the same as IRB approval to conduct a research study. Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product.
What is compassionate supply?
In some circumstances and at their own discretion, a sponsor may provide a therapeutic good to a patient on a compassionate basis (referred to as compassionate supply) at reduced or no cost.
What does compassionate use of Remdesivir mean?
We provided remdesivir on a compassionate-use basis to patients hospitalized with Covid-19, the illness caused by infection with SARS-CoV-2. Patients were those with confirmed SARS-CoV-2 infection who had an oxygen saturation of 94% or less while they were breathing ambient air or who were receiving oxygen support.
What Is Compassionate Drug use?
Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no ot...
Is Compassionate Drug Use legal?
Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. For people who aren’t in clinical trials, a dru...
Who Might Benefit from Using Unapproved Drugs?
According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions: 1. Have u...
Is It Difficult to Get Drugs For Compassionate use?
Perhaps the biggest problem with compassionate drug use is that it’s often hard to get the drug. The simplest way to get an unapproved drug is thro...
What Should I Ask My Doctor About Compassionate Drug use?
Here are some questions you may want to ask if your doctor is thinking about compassionate drug use: 1. Are there any approved treatments that I ha...
Where Do I Start If I Want to Apply For Compassionate Drug use?
Your doctor or one of the office staff will work with you on this process. The US Food and Drug Administration, Center for Drug Evaluation and Rese...
What is the phone number for the FDA?
All questions about and requests for expanded access for emergency use for drugs, biologics and medical devices should be directed to the FDA Emergency Call Center, telephone: 866-300-4374.
What is an informed consent statement?
Informed Consent Statement that states that informed consent and approval of an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment.
What is the phone number for expanded access for investigational drugs?
For general questions about expanded access for emergency use for investigational drugs, contact CDER’s Division of Drug Information (DDI) at phone: 301-796-3400 or 855-543-3784; fax: 301-431-6353; or e-mail: [email protected].
Is Form 1571 required for expanded access?
It can also be used for certain submissions to FDA after the initial application is filed ( if you have trouble opening this form you may need to open it in Internet Explorer or right click on the form and save it as a PDF ). FDA Form 1571 and 1572 are no longer required for individual patient expanded access to investigational drugs and biologics.
What time does the FDA open for emergency calls?
2. After 4:30 p.m. ET weekdays and all day on weekends. For emergency requests for all medical products (drugs, biologics, and medical devices) contact FDA's Emergency Call Center at 866-300-4374. Report from the 2018 external assessment of the Expanded Access Program.
What is the phone number for FDA?
Investigational medical devices: 301-796-7100 or [email protected] [CDRH's Division of Industry and Consumer Education] Investigational biologics: 240-402-8020 or 800-835-4709 or [email protected] [CBER's Office of Communication, Outreach and Development]
When is expanded access appropriate?
Expanded access may be appropriate when all the following apply: Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition. There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
What is FDA regulation for investigational drugs?
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for: individual patients (single-patient IND applications for treatment in emergency settings and non-emergency settings) larger populations for use of the drug under a treatment protocol or Treatment IND Application.
What is a patient benefit?
Patient (s) have a serious or immediately life-threatening disease or condition , and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; The potential patient benefit justifies the potential risks of the treatment and the potential risks are not unreasonable in the context ...
Can expanded use of investigational drugs interfere with clinical investigations?
The expanded use of the investigational drug for the requested treatment will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the product.
What happens before FDA requests?
Several things must happen before the request is sent to FDA: You and your licensed physician agree that expanded access is a good option for you, and your licensed physician agrees to manage the process, monitor your treatment, and keep records and reports.*. The company agrees to provide their investigational medical product for treatment ...
What does the FDA determine about expanded access?
The FDA must determine the following: The patient (or patients) to be treated has a serious or life-threatening disease or condition. There is no comparable or satisfactory therapy to diagnose, monitor, or treat the disease or condition.
What does a licensed physician have to determine about an investigational medical product?
Your licensed physician must determine that the possible risks from the investigational medical product are not greater than the possible risks from the disease. It is also possible that your licensed physician is not willing or able to oversee your treatment of an investigational medical product.
What happens if investigational medical product treatment is not allowed to proceed?
If the investigational medical product treatment is not allowed to proceed, FDA will inform the licensed physician or the company ( whoever submitted the request) of this decision. FDA will communicate what issues need to be resolved in order for treatment to move forward.
How many decisions are required for expanded access to the investigational medical product?
Treatment can begin once everyone agrees to expanded access to the medical product. Expanded access to the investigational medical product generally requires five key decisions: Your licensed physician agrees to oversee your treatment and submit records and reports.
Why do you need to participate in a clinical trial?
Participation in a clinical trial can help with the development process of a medical product so it can be used in other people who have the same disease or condition.
Is there a law that requires a company to provide access to investigational medical products outside of the clinical trial
There is no law or policy that requires a company to provide access to investigational medical products outside of the clinical trial. They may not have enough of the investigational medical product for all patients requesting expanded access.
What is the 21st Century Cures Act?
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.
What is expanded access treatment?
For example, FDA reviewers recognize that: expanded access treatment generally occurs outside a controlled clinical trial setting; 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;
What happens to patients who receive expanded access?
patients who receive a drug through expanded access may suffer from one or more comorbidities.
Does FDA review expanded access?
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.
Can an investigational medicine be used in a clinical trial?
Whenever possible, an investigational medical product should be used as part of a clinical trial.
Is early identification of adverse events beneficial?
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 ...
Is investigational medicine FDA approved?
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. ...
When a physician wants to submit a single patient expanded access request to obtain an unapproved investigational drug for an
When a physician wants to submit a Single Patient Expanded Access request to obtain an unapproved investigational drug for an individual patient, he or she must first ensure that the manufacturer is willing to provide the investigational drug for expanded access use. If the manufacturer agrees to provide the drug, the physician should follow the steps below to submit an Investigational New Drug Application (IND) to the FDA.
Is Form 1571 required for expanded access?
Form FDA 1571 and 1572 are still required for other expanded access submissions involving more than one patient (e.g., intermediate access or treatment INDs) and for IND submissions by commercial sponsors or drug manufacturers. Upon receipt of the single patient expanded access application, FDA will review to make a decision.
Can the FDA put an IND on hold?
The FDA will either allow the treatment to proceed or place the IND on hold. FDA allows over 99% of single patient expanded access requests to proceed. FDA may contact the physician to request more information or clarification in order to avoid placing the IND on hold.
What is compassionate use?
Compassionate use can be for devices that are being studied in a clinical trial under an IDE for patients who do not meet the requirements for inclusion in the clinical investigation but for whom the treating physician believes the device may provide a benefit in treating or diagnosing their disease or condition.
What does the FDA expect from a physician?
The FDA expects the physician to make the determination that the patient's circumstances meet the above criteria, to assess the potential for benefit from the use of the unapproved device, and to have substantial reason to believe that benefits will exist.
What is a potential patient benefit?
Potential patient benefit justifies the potential risks of the investigational device.
When does emergency use apply?
Emergency use may apply if the device is being studied in clinical trials under an investigational device exemption (IDE) such as when a physician who is not part of the IDE clinical study wishes to use the device to treat a patient in an immediately life-threatening situation.
When to use an investigational device?
Use of an investigational device when an individual patient is in a life-threatening situation and needs immediate treatment (there are no alternative options and no time to use existing procedures to get FDA approval for the use) No.
What are the criteria for emergency use?
Criteria for Emergency Use. The patient has a life-threatening condition that needs immediate treatment; No generally acceptable alternative treatment for the condition exists; and. Because of the immediate need to use the device, there is no time to use existing procedures to obtain FDA approval for the use.
Is compassionate use a medical device?
Yes. Compassionate use. Use of an investigational device to treat or diagnose an individual patient or a small group of patients with a serious disease or condition when there are no available alternative options. Yes.
Understanding Unapproved Use of Approved Drugs "Off-Label"
Has your healthcare provider ever talked to you about using a drug to treat your disease or medical condition that has not been studied on patients with your disease? It maybe helpful for you to understand what "Off-Label" use means and the questions you may need to ask your healthcare provider.
Understanding Investigational Drugs
Has your healthcare provider ever talked to you about using an investigational drug to treat your disease or medical condition. It maybe helpful for you to understand what an investigational drug is before deciding if one might be right for you. Learn the basics of what they are and how you can get access.
Understanding Expanded Access (Compassionate Use)
Information about FDA's current expanded access policies, requirements for enrolling in expanded access programs, and steps you can take to get more information.
Understanding Right to Try
The Right to Try Act, or the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, was signed into law May 30, 2018.