Information on pre-approval access
People living with serious illnesses who have exhausted all available treatment options and cannot participate in a clinical trial may be eligible for approval by local regulators/health authorities to receive an investigational therapy before it is approved.
Such individual use of an investigational therapy is often called "expanded access" or “compassionate use,” but may go by other names.
For new medicines to be approved for use by global health authorities such as the U.S. Food and Drug Administration, companies are required to evaluate their safety and effectiveness in clinical trials and submit trial results to regulatory agencies. To participate in a trial, patients 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 patient “informed consent” is a required step in the process of enrolling. argenx has clinical trials ongoing across its antibody pipeline.
Find an argenx clinical trial here.
Pre-approval access or Compassionate Use
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 pre-approval access to an investigational drug. Requests for pre-approval access must be made by the patient’s physician.
For more information about pre-approval access, please visit the National Organization for Rare Diseases Expanded Access FAQ page here.
argenx Pre-approval Access Policy
Pre-approval access is a term used to describe access to investigational therapies that have not yet been approved by regulatory bodies. In certain cases, argenx may provide an investigational product outside of a clinical trial to a patient or group of patients through their treating physicians.
argenx is committed to delivering innovative new therapies to help improve patients' lives. To achieve this, we keep patients’ needs and aspirations top of mind and integrate their insights into all that we do, including how we design our clinical trials. This collaborative approach is aimed at addressing the complex challenges patients often face with rare diseases.
We consider making pre-approval access available to provide investigational therapies for patients diagnosed with a serious or life-threatening disease or condition, who have exhausted approved treatment options, and who are not eligible for clinical trials. Globally, there are many terms used to describe types of pre-approval access. Many countries/regions have unique names, established criteria and regulatory requirements to participate in pre-approval access (e.g. compassionate use, early access medicinal program, expanded access, named patient program, special access program, managed access programs, special access schemes).
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 pre-approval access to an investigational therapy.
How decisions are made for pre-approval access
argenx is committed to a fair and impartial evaluation of each request for access to our investigational therapies. Pre-approval requests are carefully considered to ensure patient safety and that the potential medical benefits outweigh the risks. Considerations include: whether all other approved treatment options have been exhausted; if enrollment in a clinical trial is possible; whether there is a scientific rationale to support that the requested investigational therapy may provide benefit and that the potential benefits outweigh the potential risks; and if there is sufficient amounts of the investigational therapy available to patients outside the clinical trial setting.