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.

Examining the Role of Social Support for Adolescents from Low Socioeconomic Backgrounds in a College Access Program

Prior research has shown that college access programs help to increase college-going for youth from lower-income backgrounds. In addition to increasing college access, these programs also provide social support to youth and impact their academic and non-academic outcomes. Guided by risk and resilience frameworks, the present study examined social support from the college access program as a promotive factor for adolescents from lower socioeconomic backgrounds. The results indicated that social support was positively associated with personal resources, future college-going, and confidence in academic abilities. Overall, the current findings affirm the importance of investigating social support for adolescents from lower socioeconomic backgrounds. These findings also have implications for future research related to social support from college access programs.