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. The information below explains options that may be available to patients in these circumstances.
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, a patient 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.
Compassionate Use Programs
In cases where a clinical trial isn’t an option, and the patient has exhausted all available treatment options, regulators/health authorities may grant permission for us to provide a treating physician with an investigational drug pre-approval. Such individual use of an investigational drug pre-approval is often called “compassionate use” but may go by other names.