Policy For Access To Investigational Medicines For Compassionate Use
Patients who choose to participate in clinical trials play a critical role in developing new medicines. In some cases, patients with serious or immediately life-threatening diseases may not be eligible for any clinical trials and may not have other treatment options. In these cases, investigational medicines (those that have not been approved by any regulatory agency) are sometimes made available by pharmaceutical manufacturers through a process known as compassionate use (also referred to as expanded access). CTI BioPharma makes some of its investigational medicines available on a limited basis when and where appropriate for compassionate use.
It is important to remember that investigational medicines have not yet received regulatory approval and therefore their potential risks and benefits are not yet established (safety and efficacy have not been established). Physicians and patients should consider all possible benefits and risks when seeking expanded access to an investigational medicine—so a patient should seek the advice of his or her treating physician.
Physicians who would like to receive more information regarding the availability of a particular investigational medicine through expanded access on behalf of their patients should contact CTI BioPharma at firstname.lastname@example.org. CTI BioPharma will acknowledge receipt of all requests within 2 business days of receiving them. Different countries have various regulatory procedures required for compassionate use of an investigational medicine. In all cases, compassionate use must be allowed by local law and local regulatory approval for the proposed use is required.
General Criteria For Compassionate Use
CTI BioPharma is committed to a fair and impartial evaluation of each request for expanded access to our investigational medicines. Therefore, all decisions are based solely on clinical evidence and guided by the general criteria outlined below. Access to a CTI BioPharma investigational medicine is only for a patient(s) with a serious or life-threatening condition for whom no other treatment options or clinical trials are available.
- CTI BioPharma must be studying the requested investigational medicine as part of an ongoing clinical trial. Upon commercial availability of the medicine, following local health authority approval, the investigational medicine will no longer be made available through expanded access for an approved indication.
- Granting access to a CTI BioPharma investigational medicine must not interfere in any way with the completion of clinical trials that could support health authority approval of the medicine or otherwise obtaining health authority approval. Whenever possible, patients requesting access to a CTI BioPharma investigational medicine will be referred to an ongoing clinical trial.
- The patient must not be eligible for ongoing or pending clinical trials of the investigational medicine. Geographic limitations to participation in a clinical trial would generally not impact eligibility.
- There must be sufficient clinical evidence to support the safety and potential clinical benefit of the investigational medicine in the patient with the disease or condition to be treated. The potential benefits to the patient seeking access to the investigational medicine must outweigh the collective potential risks to the patient. CTI BioPharma evaluates these benefits and risks based on case-by-case, clinical assessments in consultation with the patient’s qualified treating physician.
- Adequate clinical data must exist to support the appropriate dose amount and frequency of the investigational medicine and duration of treatment. CTI BioPharma’s clinical team will determine whether sufficient clinical data is available.
- There must be sufficient supply of the investigational medicine to support the expanded access. Supplying the medicine through expanded access must not compromise the supply of medicine available for active or soon-opening clinical trials.
- The requesting physician must agree to comply with all applicable laws and contractual conditions, including safety reporting.