Rare diseases: considerations for the development of drugs and biological products : guidance for industry

Rare diseases: considerations for the development of drugs and biological products : guidance for industry

Collection:
Health Policy and Services Research
Uniform Title(s):
Rare diseases (U.S. Department of Health and Human Services, Food and Drug Administration)
Contributor(s):
Center for Drug Evaluation and Research (U.S.), issuing body.
Center for Biologics Evaluation and Research (U.S.), issuing body.
Publication:
Silver Spring, MD : Center for Drug Evaluation and Research, December 2023
Language(s):
English
Format:
Text
Subject(s):
Biological Products
Drug Development
Drug Industry
Government Regulation
Rare Diseases -- drug therapy
United States
Genre(s):
Guideline
Technical Report
Abstract:
The purpose of this guidance is to assist sponsors of drugs for the treatment of rare diseases in conducting efficient and successful drug development programs. The statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs. These issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, poorly understood natural history data, sample size constraints, and lack of drug development experience. This guidance does not contain discussion of the general issues of statistical analysis. Those topics are addressed in other documents, including ICH guidances for industry E9 Statistical Principles for Clinical Trials (September 1998) and E10 Choice of Control Group and Related Issues in Clinical Trials (May 2001), respectively. Additional FDA guidances cover other specific topics that may be of specialized interest.
Copyright:
The National Library of Medicine believes this item to be in the public domain. (More information)
Extent:
1 online resource (1 PDF file (26 pages))
NLM Unique ID:
9918751183706676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918751183706676