Drug Development and Rare Pediatric Diseases: Issues, Strategic Plan, and Voucher Program Assessment


Frederick Johnston (Editor)

Series: Pharmacology – Research, Safety Testing and Regulation
BISAC: MED071000, MED069000

Over the past few decades, a number of legislative actions have been taken to encourage the development of therapies to treat people affected by rare diseases and to encourage therapies for pediatric patients. During this same time period, the Food and Drug Administration (FDA) has invested substantial effort in support of these same goals. As described in this book, recent legislative actions have prompted new initiatives. Continuing the response to the laws’ specific requirements and FDA’s commitment under PDUFA, this book includes input from the public and FDA and discusses the many complex issues involved in medical product development for people with rare diseases. Following a brief overview of both legislative and FDA efforts over the years to foster development of therapies for these patients, the book summarizes the three days of the public meeting and presents FDA’s strategic plan for accelerating the development of the urgently needed therapies for pediatric rare diseases. (Imprint: Nova Biomedical)

Table of Contents

Table of Contents


Chapter 1. Report: Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases Including Strategic Plan: Accelerating the Development of Therapies for Pediatric Rare Diseases
Food and Drug Administration

Chapter 2. Rare Diseases: Too Early to Gauge Effectiveness of FDA’s Pediatric Voucher Program
United States Government Accountability Office


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