FDA’s Orphan Products Grants Program awards significant funding for rare diseases

The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara.

Each year, the FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.  In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase III trial – totaling $17.2 million over the next four years. The FDA also funds natural history studies under the grants program. The Agency announced the FY2024 funding for the three natural history study grant awardees provides $4.7 million over four years.

While the various expedited programs for serious conditions (Fast Track Designation, Breakthrough Therapy Designation, Accelerated Approval, and Priority Review Designation) are in many cases well understood by companies and academic sponsors developing therapies for rare diseases, the Orphan Products Grants Program represents a lesser known but highly impactful program that importantly can serve to support advancement of novel therapies for rare disease patient populations.