Compassionate use, also known as expanded access, refers to a program or regulatory pathway that allows seriously ill patients who have exhausted all other available treatment options to access investigational or unapproved medical therapies. These patients may not be eligible for clinical trials or may not have access to the standard treatments for their condition, and their situations are often life-threatening.
Compassionate use is a regulatory pathway that allows seriously ill patients to access investigational or unapproved medical therapies.
In other word compassionate use is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial.
They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.
Expanded Access Treatment Mechanisms:
- Treatment INDs or Individual Patient Access to Investigational Drugs/Devices for Serious Diseases: These mechanisms are primarily intended to give seriously ill patients access to experimental drugs or devices where no comparable or satisfactory alternative treatment is available. Although the test article sponsor is expected to continue conventional clinical trials and pursue marketing approvals with due diligence, expanded access studies involve systematic use of experimental treatments, and, with very rare exceptions, require the same review and approval as research, including both IRB approval and FDA approval in the form of an IDE (medical device) or an IND (drug/biologic).
- Open Protocols (Parallel Track, Open Label Protocol, Open Label IND) or Continued Access IDEs: These uncontrolled studies are typically used when controlled trials have ended and treatment is continued so the subjects may continue to receive the benefits of the test article until marketing approval is obtained. Informed consent and prior IRB approval are required.
Ethical considerations of compassionate use
The use of compassionate use programs can raise ethical, legal, and logistical considerations, as it involves providing unapproved treatments to patients without the rigorous testing and safety data that approved therapies undergo. However, it is seen as a way to balance the need to protect patient safety with the need to provide potentially life-saving options to those with no other alternatives.
Keynote on compassionate use
Compassionate use is typically a way for these patients to receive experimental drugs, devices, or therapies that are still undergoing clinical trials or awaiting approval by regulatory agencies such as the U.S. Food and Drug Administration (FDA). The main purpose of compassionate use is to provide these individuals with a last-resort option in the hope of improving their health or extending their lives.