{"product_id":"innovative-methods-for-rare-disease-drug-development-9780367502904","title":"Innovative Methods for Rare Disease Drug Development","description":"\u003cp\u003e • Author(s): Shein-Chung Chow\u003cbr\u003e • Publisher: Taylor \u0026amp; Francis\u003cbr\u003e • Publisher Imprint: CRC Press\u003cbr\u003e • BISAC: Probability \u0026amp; Statistics - General\u003c\/p\u003e\u003cp\u003e\u003c\/p\u003e\u003cp\u003eIn the United States, a rare disease is defined by the Orphan Drug Act as a disorder or condition that affects fewer than 200,000 persons. For the 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 standards for statistical assurance as drugs used to treat common conditions. \u003cb\u003e\u003ci\u003eInnovative Methods for Rare Disease Drug Development\u003c\/i\u003e\u003c\/b\u003e\u003ci\u003e \u003c\/i\u003efocuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives. \u003c\/p\u003e\u003cb\u003e \u003c\/b\u003e\u003cp\u003eKey Features: \u003c\/p\u003e \u003cul\u003e\n\u003cb\u003e\u003c\/b\u003e \u003cp\u003e \u003c\/p\u003e\n\u003cli\u003eReviews critical issues (e.g., endpoint\/margin selection, sample size requirements, and complex innovative design).\u003c\/li\u003e \u003cp\u003e\u003c\/p\u003e \u003cp\u003e \u003c\/p\u003e\n\u003cli\u003eProvides better understanding of statistical concepts and methods which may be used in regulatory review and approval.\u003c\/li\u003e \u003cp\u003e\u003c\/p\u003e \u003cp\u003e \u003c\/p\u003e\n\u003cli\u003eClarifies controversial statistical issues in regulatory review and approval accurately and reliably.\u003c\/li\u003e \u003cp\u003e\u003c\/p\u003e \u003cp\u003e \u003c\/p\u003e\n\u003cli\u003eMakes recommendations to evaluate rare diseases regulatory submissions.\u003c\/li\u003e \u003cp\u003e\u003c\/p\u003e \u003cp\u003e \u003c\/p\u003e\n\u003cli\u003eProposes innovative study designs and statistical methods for rare diseases drug development, including \u003ci\u003en\u003c\/i\u003e-of-1 trial design, adaptive trial design, and master protocols like platform trials.\u003c\/li\u003e \u003cp\u003e\u003c\/p\u003e \u003cp\u003e \u003c\/p\u003e\n\u003cli\u003eProvides insight regarding current regulatory guidance on rare diseases drug development like gene therapy.\u003c\/li\u003e \u003cp\u003e\u003c\/p\u003e\n\u003c\/ul\u003e","brand":"Taylor \u0026 Francis","offers":[{"title":"Paperback","offer_id":45241805078679,"sku":"9780367502904","price":4326.0,"currency_code":"INR","in_stock":true}],"thumbnail_url":"\/\/cdn.shopify.com\/s\/files\/1\/0666\/3471\/1191\/files\/9780367502904.webp?v=1769229897","url":"https:\/\/atlanticbooks.com\/products\/innovative-methods-for-rare-disease-drug-development-9780367502904","provider":"Atlantic Books","version":"1.0","type":"link"}