Expanded Access Program (EAP)
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 have not yet been approved by government regulatory agencies. The information below explains options that may be available to patients in these circumstances.
1. Clinical Trials
For new medicines to be legally approved for use, companies like CTI 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 patient “informed consent” is a required step in the process of enrolling.
- Learn more about clinical trials as a patient.
- Find a CTI clinical trial on clinicaltrials.gov.
- Search for open recruiting trials by the investigational drug name or therapeutic area.
- Search for available CTI BioPharma sponsored expanded access protocols by expanded access or the investigational drug name.
- Find a trial (all sponsors, including CTI) on clinicialtrials.gov.
2. Expanded Access or Compassionate Use
In cases where a clinical trial is not 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 “expanded access” or “compassionate use” but may go by other names. CTI refers to these requests as expanded access.
On February 28th, 2022, the US Food and Drug Administration approved VONJO™ (pacritinib) for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. As a result of this approval, CTI has subsequently closed the Pacritinib Expanded Access Program for all patients in the United States.
For health care providers and patients outside of the United States: CTI is presently not accepting requests for expanded access (compassionate use) to pacritinib. CTI currently is enrolling patients on the PACIFICA trial.
Please feel free to contact us at CTI with any questions.