About FDA

Guidances: Annotated Listing for Rare Diseases


There are many guidances on FDA’s website pertaining to a wide variety of topics.

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 Agency guidances means that something is suggested or recommended, but not required.

Selected Guidance Documents

Guidances that may be especially helpful for rare disease drug development include:

Rare Diseases: Common Issues in Drug Development
This guidance assists sponsors of drug and biological products intended to treat or prevent rare diseases in conducting more efficient and successful development programs through a discussion of selected issues commonly encountered in rare disease drug development.

Rare Pediatric Disease Priority Review Vouchers
This guidance provides information on the implementation of section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA), which added section 529 to the Federal Food, Drug, and Cosmetic Act (the FD&C Act). Under section 529, FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section.

Expedited Programs for Serious Conditions - Drugs and Biologics
The following four FDA programs are intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of a serious or life-threatening condition: fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation.

Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products
This guidance provides recommendations to industry on formal meetings between the Food and Drug Administration (FDA) and sponsors or applicants relating to the development and review of drug or biological drug products.

Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products
This guidance is intended to provide guidance to sponsors planning to file NDAs, BLAs or supplemental applications to demonstrate effectiveness.

Qualification Process for Drug Development Tools
This guidance describes the qualification process for drug development tools (DDTs) intended for potential use, over time, in multiple drug development programs.

Best Practices for Communication Between IND Sponsors and FDA Drug Development
This guidance describes best practices and procedures for timely, transparent, and effective communications between investigational new drug application (IND) sponsors and FDA at critical junctures in drug development, which may facilitate earlier availability of safe and effective drugs to the American public.

Page Last Updated: 08/09/2016
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