Preparing Now For FDA's START Program For Rare Disease Drug Developers

By Michael Fusakio, Ph.D.

The U.S. Food and Drug Administration (FDA) has launched a pilot program, Support for Clinical Trials Advancing Rare Disease Therapeutics (START), to expedite the development of innovative drugs and biological products for rare diseases. The initiative will enable a select group of sponsors to engage in more frequent communication with the FDA to tackle clinical development challenges. The START program aims to enhance the efficiency of creating potentially life-saving treatments for rare diseases and assist sponsors in producing high-quality data for future marketing applications.

The pilot program will be accessible to three sponsors each from the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). Applications for the inaugural pilot program are due by March 1, 2024. This move by the FDA marks a significant step towards addressing the urgent need for effective treatments for rare diseases.

Access a full overview of the START pilot program, expectations to consider if you are applying to the program, and early preparation considerations by reading the article below.