Accessing Investigational Products Outside of a Trial: Considerations for Neuromuscular Providers

Purpose of Review: People with fatal neuromuscular diseases such as ALS want to access investigational products. Trials are our preferred pathway for this, but most people with these diseases will not be able to participate due to restrictive inclusion criteria, travel burdens, or design features they will not accept. This leaves FDA Expanded Access Programs (EAPs), the Right To Try (RTT) pathway, and self-purchase of alternative and off-label treatments (AOTs). Recent Findings: A recent survey highlighted physician barriers to the above pathways, including lack of knowledge and concerns about time burdens and risks. Emerging resources are highlighted that can mitigate some of these concerns. Summary: With the information in this chapter, we hope that neuromuscular clinicians will feel more knowledgeable and confident in supporting patient request for investigational products.

Duke Scholars

Author Richard Stanley Bedlack Neurology, Neuromuscular Disease