FDA Announces New Approval Pathway for Drugs Treating Ultra-Rare Diseases

On February 23, the FDA announced a new approval pathway designed to support the development of drugs for ultra-rare diseases. Often, treatments for ultra-rare diseases have difficulty meeting the agency’s rigorous approval requirements. The FDA has issued a guidance document for the “Plausible Mechanism Framework,” which will allow drugmakers to bypass certain clinical trial requirements when advancing drugs treating ultra-rare conditions.... By: Cozen O'Connor