FDA opens doors for more treatments for rare diseases

The US Food and Drug Administration (FDA) said on Thursday that it is at a turning point in understanding the science that is opening doors to rare disease treatments that were unthinkable only a few years ago, such as gene therapies and drugs that work on gene expression.

Despite this progress, there are still many rare diseases that lack treatment options, and we need to do more to alleviate the suffering of patients and their families. For this reason, the FDA says it is taking steps to help accelerate the development of novel drugs and biologics through the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.

The FDA’s Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) initiated the START Pilot Program with hopes that the insight gained through this pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies for rare diseases and help sponsors generate high-quality, actionable data to support future new drug or biologics license applications.