US WorldMeds is committed to serving patients by providing safe, fair, and sustainable patient access to its medicines. USWM may be able to provide patients access to its investigational drug, DFMO (also called eflornithine or difluoromethylornithine), outside of a clinical trial if certain conditions are met and if permitted by local law and regulation. The use of an investigational medical product (i.e., one that has not been approved by the Food and Drug Administration [FDA]) outside of a clinical trial is known as Expanded Access (sometimes referred to as “compassionate use”). Generally, there are two types of Expanded Access—Group Expanded Access and Individual Patient Expanded Access. For more information on the different types of Expanded Access, visit the following FDA website: https://www.fda.gov/news-events/public-health-focus/expanded-access.
USWM collaborates with Beat Childhood Cancer Research Consortium (BCC) and Sponsor-Investigator, Dr. Giselle Sholler and makes investigational product DFMO 192 mg tablets available for pediatric oncology indications through multiple clinical trials and a Group Expanded Access Program (also called Intermediate Population Expanded Access).
USWM does not currently provide Individual Patient Expanded Access; however, detailed written requests for unique circumstances will be reviewed on a fair and equitable basis.
Expanded Access may not always be available. Information about the Group Expanded Access Program, including a full list of eligibility criteria and site contact information can be found here: https://clinicaltrials.gov/ct2/show/NCT03581240. In addition to meeting the eligibility criteria outlined at the link above, the following criteria must be met for entry into the Group Expanded Access Program:
- USWM must have sufficient supply of DFMO to reasonably accommodate the anticipated duration of treatment.
- The patient’s licensed physician must be willing to collaborate with the enrolling center.
- The patient’s physician and enrolling physician determine there is no comparable or satisfactory therapy available to treat the patient’s disease or condition and they agree that the patient is clinically stable and able to receive this medication.
- The patient’s parent/guardian is willing to travel with the patient to BCC at study entry and every three months while on treatment.
Licensed physicians may contact USWM with general requests or questions regarding Expanded Access at regulatoryaffairs@usworldmeds.com. USWM will endeavor to acknowledge general requests and questions within 5 business days. In line with the 21st Century Cures Act, USWM cannot guarantee access to investigational product to all patients. USWM may revise this policy at any time; at such time, the revised policy will be made publicly available.