CATO SMS has significant experience and expertise in the development of drugs for orphan indications. Rare disease trials differ from common disease trials not only by the small number of patients available for participation in clinical trials across multiple geographies, but also by the complex interactions and collaborations with several stakeholders, including regulators, investigators, patient advocacy groups, and patient assistance organizations. The successful execution of rare disease trials requires a special commitment by the trial’s management team and solid experience in extracting maximal information from small-population clinical trials.

CATO SMS has authored and submitted more than 40 successful orphan drug applications (ODAs), amendments, and grants to the FDA, and designed and conducted clinical trials for more than 20 different types of orphan indications, typically with a high percentage of pediatric subjects. Our experience with orphan indications covers a broad range of diseases, including:

  • Rare CNS disorders
  • Metabolic storage diseases
  • Multiple cancers
  • Infectious diseases
  • Cardiovascular diseases
  • Immunologic disorders