Compassionate Use, Expanded Access, and Patient Assistance ? Obtaining Life-Saving Medications When a Clinical Trial is Not Available (207-2)

5:30 pm – 6:00 pm Thursday, September 15
The vast majority of clinical trials conducted in oncology are not open to patients under the age of 18 years old, leaving pediatric cancer patients with a very limited selection of FDA-approved therapies. Though clinical trials are the safest and most-beneficial means of determining the effectiveness of medications, not all patients have the option to enroll in clinical trials due to barriers such as patient clinical status, geographic location, or enrollment caps. In other circumstances where FDA-approved therapies are commercially available, insurance companies simply deny payment for off-label use of these expensive medications based on disease categories instead of oncogenic targets. Compassionate use programs, also known as expanded access trials or single patient investigational new drugs, provide opportunity for these patients to obtain possibly life-saving therapies. This interactive session will help demystify the process of gaining access to medical therapies on behalf of our patients. The different terms used in the compassionate use process will be discussed, as well as the specific steps oncology teams can take to apply to the FDA for approval of experimental medications. Case studies will provide examples of how the compassionate use process works in different circumstances as well as demonstrate clinical outcomes. Payment assistance programs and other resources to help patients pay for FDA-approved medications will also be covered.


Anne Raines, MSN, RN, CPHON

CNE Hours: