Innovative Methods for Rare Disease Drug Development (Chapman & Hall/CRC Biostatistics Series)

"In the United States, a rare disease is defined by the Orphan Drug Act as a disorder or condition that affects less than 200,000 persons. For approval of "orphan" drug products for rare diseases, the traditional approach of power analysis for sample size calculation is not feasible because there are only limited number of subjects available for clinical trials. In this case, innovative approaches are needed for providing substantial evidence meeting the same standard as drugs in common conditions with certain statistical assurance. Innovative Methods for Rare Diseases Drug Development focuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives"--