FDA Introduces New Pathways for Rare Disease Therapies Amid Unanswered Questions
The FDA has introduced new pathways, including the plausible mechanism pathway and rare disease evidence principles (RDEP), to accelerate the development of therapies for rare diseases. These initiatives aim to provide faster routes to market for investigational therapies, particularly those targeting rare conditions with small patient populations. However, the specifics of these pathways remain unclear, leaving industry experts with questions about their implementation and impact. The plausible mechanism pathway, for instance, is designed to allow approval based on evidence of biological target engagement and clinical improvement, but its commercial implications and regulatory exclusivities are still uncertain. 
