Orphan Drug Insights Journal
Stay ahead of industry news, regulatory policy shifts, and orphan drug designation strategies.
Only Orphans Cote breaks down what matters most to rare disease drug developers to help sponsors bring new therapies to patients faster.
Understand the core requirements of IND-enabling studies that support FDA clearance for first-in-human trials and how strategic planning accelerates approval.
Learn why FDA issues deficiency letters for Orphan Drug Designation applications. Understand the most common pitfalls in medical rationale and prevalence justification.
Learn the benefits of orphan drug designation, how market exclusivity works, and when to apply for faster approval and greater funding success.
FDA’s new Rare Disease Evidence Principles (RDEP): what this means for biotech sponsors and orphan drug developers seeking faster approvals.