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.
Comprehensive guide to FDA tropical disease priority review vouchers (PRVs) worth $160M+. Learn eligibility requirements, qualifying diseases, and how to structure your development program for PRV success.
Learn which cancers qualify for FDA orphan designation based on U.S. prevalence, how globally common cancers may still be eligible, and what benefits sponsors unlock for rare cancer drug development.
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.