What is the goal of the Orphan Drug Act?

The goal of the Orphan Drug Act is to encourage the development of treatments for rare diseases. This legislation was specifically designed to address the challenges faced by pharmaceutical companies in developing drugs for conditions that affect a small percentage of the population. These conditions are often not financially viable for companies to pursue without incentives, as the limited market size can lead to insufficient return on investment.

The Orphan Drug Act provides several incentives for companies, such as tax credits for clinical research, assistance in the drug development process, and seven years of market exclusivity for the approved drug. This framework is intended to foster innovation and ensure that patients with rare diseases have access to effective treatments that might otherwise be overlooked. By creating a supportive environment for the development of orphan drugs, the Act aims to meet the healthcare needs of these patients and improve their quality of life.