REG210 FDA Guidance on Rare Disease Drug Development

The Orphan Drug Act was enacted in 1983 to help make development of drugs for rare diseases more financially viable. The Act DOES NOT create a different statutory standard for rare disease products. This module will help you understand the areas where rare disease drug development differs from traditional drug development.


Promulgate, sponsors, large molecules, homogeneity, clinical endpoint, nonclinical studies, toxicology studies, pharmacology, explant, Good Laboratory Practice, tox coverage, acute toxicology studies, sub-chronic toxiciology studies, chronic toxicology studes, clinical studies, safety signals, developmental toxicology studies, reproductive toxicology studies, carcinogenicity studies, review division, New Chemical Entities, drug disposition studies, drug interaction studies, biospecimens, exposure-response relationship, predictive biomarkers, responsive biomarkers, bias, placebo effect, Randomization, Concurrent controls, external controls, historical controls, test group, control group, performance bias, reporting bias, double blinding, prognostic factors, placebo-arm, active-arm, Bayesian designs, adaptive designs, n-of-1 studies, Randomized delayed start study designs, crossover designs, Master Protocol, prevalence, Auxiliary Cohorts, Expanded Access, Pre-Approval Access programs, misbranding, adulteration, Fast-track designation, Breakthrough Therapy, Pediatric Priority Review Vouchers, Pediatric considerations