8th Annual rare disease scientific workshop: Evaluating early access models for patients: Flashpoints, frameworks & case studies for advancement: A summary

Abstract

The 8th annual Rare Disease Scientific Workshop featured presentations on the topic of "Evaluating Models to Provide Early Access for Rare Disease Patients". The workshop included presentations from and participation by experts in Industry, the Food and Drug Administration, and academia focused on case study examples of successful mechanisms for those seeking to provide early access to experimental therapies for patients. Current regulatory frameworks were discussed and evaluated, with an emphasis on rare and serious diseases with the goal of identifying best practices and useful ideas for a set of standard approaches to early access.