Rare Diseases & Orphan Drugs

Preclinical Studies for Orphan Drug Development

Preclinical studies are a critical step in the development of orphan drugs. These studies involve testing drug candidates in animal models to assess their safety, efficacy, and pharmacokinetics. Preclinical studies can provide valuable data to inform clinical trial design and drug dosing. Regulatory agencies require preclinical data to support clinical trial applications for orphan drugs. The small patient populations of rare diseases make preclinical studies even more critical as they help identify potential safety concerns early in the development process. Collaboration between researchers, patient advocacy groups, and regulatory agencies is necessary to ensure that preclinical studies are conducted ethically and efficiently and that orphan drugs can move from preclinical studies to clinical trials as quickly as possible.