What is Expanded Access?
Expanded access, also known as compassionate use, is a pathway for patients to gain access to investigational drugs, biologics, or medical devices outside of clinical trials. This is particularly crucial for patients with serious or life-threatening conditions who have exhausted all other treatment options.
Who is Eligible for Expanded Access?
Eligibility criteria for expanded access typically include having a serious or immediately life-threatening condition, no comparable or satisfactory alternative therapy options, and being unable to enroll in a clinical trial. The patient's physician must also determine that the potential benefits justify the potential risks of the investigational treatment.
The treating physician identifies a potential investigational treatment.
The physician submits a request to the manufacturer of the investigational product.
If the manufacturer agrees, an application is submitted to the
FDA or relevant regulatory body.
Institutional Review Board (IRB) approval may also be required.
Regulatory hurdles and extensive paperwork.
Limited availability of the investigational product.
Potential costs, which might not be covered by insurance.
Ethical considerations, including
informed consent and balancing risks and benefits.
Success Stories and Case Studies
There have been notable success stories where patients benefitted significantly from expanded access. For instance, some women with treatment-resistant ovarian cancer have gained additional years of life through investigational therapies, underscoring the potential life-saving impact of this pathway.Conclusion
Expanded access serves as a vital option for patients dealing with severe gynecological conditions who have no other viable treatment options. While the process can be complex and challenging, the potential benefits often outweigh the risks, offering hope to those in dire need.
