Expanded Access to Investigational Therapies

When a patient requests expanded access to an investigational therapy, physicians should: 

1. Assess the patient’s individual clinical situation to determine whether an investigational therapy would be appropriate, including: 
   1. whether there is a satisfactory alternative therapy available to diagnose, monitor, or treat the patient’s disease or condition; 
   2. the nature of potential risks of the investigational therapy and whether those risks are not unreasonable in the context of the patient’s disease or condition;  
   3. whether the potential benefit to the patient justifies the risks of the investigational therapy; 
   4. whether the patient meets inclusion criteria for an existing clinical trial of the investigational therapy. 
2. As part of the informed consent process, advise the patient (or parent/guardian if the patient is a minor) that the investigational therapy has not yet been demonstrated to be effective in treating the patient’s condition and may pose as yet unknown risks. Physicians should explain the importance of clinical trials, encourage patients who meet inclusion criteria to participate in an existing trial rather than seek access to investigational therapy through the FDA expanded access program, and direct patients who wish to participate in research to appropriate resources. 
3. Decline to support an application for expanded access to an investigational therapy when: 
   1. the physician judges the treatment with the investigational therapy not to be in the patient’s best interest, and explain why; or  
   2. the physician does not have appropriate resources and ability to safely supervise the patient’s care under expanded access.  

   In such cases, physicians should refer the patient to another physician with whom to discuss possible application for expanded access. 

4. Discuss the implications of expanded access for the patient and family and help them form realistic expectations about what it will mean to be treated with the investigational therapy outside a clinical trial. Physicians should alert patients:  
   1. to the possibility of financial or other responsibilities associated with receiving an investigational therapy through expanded access; 
   2. to the lack of infrastructure to systematically monitor and evaluate the effects of the investigational therapy outside a clinical trial; 
   3. that they need information about how to contact the manufacturer for guidance if they seek emergency care from a health care professional who is not affiliated with a clinical trial of the investigational therapy; 
   4. that the physician has a responsibility to collect and share clinical information about the patient’s course of treatment with the investigational therapy, as well as to report any adverse events that may occur over the course of treatment; 
   5. to the conditions under which the physician would recommend stopping treatment with the investigational therapy.