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Compassionate Use and Expanded Access
Patients facing serious illnesses who have exhausted all available treatment options often want to know if and how they can receive early access to investigational drugs that haven’t yet been approved by government regulatory agencies. This website explains options that may be available to patients in these circumstances.
1. Clinical Trials
For new medicines and vaccines to be legally approved for use, companies like Pfizer are required to evaluate their safety and effectiveness in clinical trials and submit trial results to regulatory agencies. To participate in a trial, you must meet certain criteria. For those who meet the criteria to join a clinical trial, participation offers the chance to contribute to medical research that may benefit many others. Participation in a clinical trial comes with certain risks; that is why “informed consent” is a required step in the process of enrolling.
- Learn more about clinical trials
- Find a Pfizer clinical trial
- Find a trial on clinicaltrials.gov (all sponsors, including Pfizer Inc.)
2. Compassionate use, or expanded access (“compassionate access”)
In cases where a clinical trial isn’t an option and the patient has exhausted all available treatment options, regulators may grant permission for us to provide a treating physician with an unapproved drug. Such individual use of an unlicensed investigational drug is often called “compassionate use” or “expanded access” but may go by other names. Pfizer refers to these uses collectively as “compassionate access.”
It’s important to remember that investigational drugs have not yet received regulatory approval; therefore, their potential risks and benefits are not yet established. Doctors and patients should consider all possible benefits and risks when seeking compassionate access to an unapproved product.
Please feel free to contact us with any questions; some of the ones we receive most often are addressed below.
Compassionate Access Frequently Asked Questions
Who is a candidate for compassionate use of an investigational drug?
To be eligible for access to an investigational or unlicensed product, patients must meet the following criteria:
- Suffer from a serious or immediately life-threatening disease or condition
- Have undergone appropriate standard treatments without success, and comparable or satisfactory alternative treatments to diagnose, monitor or treat the disease or condition are not available
- Are ineligible for participation in any ongoing clinical study of the investigational drug, including lack of access due to geographic limitations
- Meet any other pertinent medical criteria for access to the investigational or unlicensed drug, as established by the Pfizer medical professionals working on the drug development program
How do I submit a request for compassionate use of an investigational drug?
Treating physicians may submit compassionate use or expanded access requests on behalf of patients or their caregivers. Physicians may submit requests to Pfizer at PfizerCAReS.com. Patients or non-healthcare professionals may not submit requests but may submit questions to PfizerCAReS.com.
Who decides who may access investigational drugs for compassionate use, and what criteria are used to make the decision?
Medical professionals at Pfizer who are familiar with the data collected on the investigational drug evaluate the request and respond based on the scientific evidence available to the company at the time of the request. The regulatory agency in the country of the request must also approve the proposed use of the investigational drug.
How long does it take to receive a response for a compassionate access request?
Pfizer commits to respond to compassionate access requests within no more than five business days of receipt of the request and required medical documentation. The country regulatory authority must grant final approval. The U.S. Food and Drug Administration strives to respond quickly (within a business day or two). In other countries regulatory response times may vary.
When a compassionate access request has been approved, how long does it take to receive the drug?
Pfizer works to ship drugs for compassionate use as quickly as possible. Although the delivery time may vary with international shipping requirements and restrictions, we do whatever it is within our control to help expedite.
How many preapproval access requests does Pfizer receive, and how many does it approve?
Pfizer receives thousands of preapproval access requests every year. In 2017, the PfizerCAReS portal received 4,818 requests from 59 countries, of which 98% were approved.