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FDA Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases

蒋千琴 12/2/2021 浏览 254 FDA United States

标准简介

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases[附网盘链接]是于不久前发布的FDA标准，适用于United States。

标准截图

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases[附网盘链接]
Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases[附网盘链接](截图)

 

标准文档说明

标准文档类型为Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases[附网盘链接]高清PDF版本（文字版），标准文档内可进行搜索，可以复制原文，可粘贴。

标准部分原文

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases Guidance for Industry This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, contact the FDA office responsible for this guidance as listed on the title page. I. INTRODUCTION 1

This guidance is intended for sponsors of drugs and biological products (hereafter drugs ) who are considering submitting requests for orphan-drug designation for their drugs under section 526 of the Federal Food, Drug, and Cosmetic Act (FD&C Act). The Food and Drug Administration (FDA) does not expect to grant any additional orphan-drug designation to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of 200,000 or greater). Pediatric-subpopulation designations that have already been granted will not be affected by this change. In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in FDA’s guidances means that something is suggested or recommended, but not required. II. BACKGROUND Congress enacted the Orphan Drug Act (ODA) to give sponsors incentives to develop drugs to prevent, diagnose, or treat rare diseases and conditions, including in pediatric patients with such 2

diseases. Orphan-drug designation may cover diseases in pediatric patients in several ways, including the following: 1. If a disease is rare (i.e., the prevalence of the disease is less than 200,000), a drug may be eligible for designation for the entire disease. The pediatric population, as part of the population being affected by that disease, would be covered under that orphan designation. 1

For the purposes of this guidance, the term drugs refers to both human drug and biological products regulated by the Center for Drug Evaluation and Research or the Center for Biologics Evaluation and Research. 2

Pub. L. 97-414 (1983), codified as amended at 21 U.S.C. §§ 360aa - 360ee. The ODA generally defines a rare disease or condition as any disease or condition that affects fewer than 200,000 persons in the United States. 1

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