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          Rare Diseases: Natural History Studies for Drug DevelopmentSearch for FDA Guidance Documents GUIDANCE DOCUMENT
	Rare Diseases: Natural History Studies for Drug Development
		 
			Draft Guidance for Industry
		
		March 2019


	Download the  Draft  Guidance Document
	    
      	      		Read the Federal Register Notice 

    Draft

	  	    Not for implementation. Contains non-binding recommendations.
	  
	  
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    PrintDocket Number:
		    FDA-2019-D-0481
	    	    		    Issued by:
		    Guidance Issuing Office
          Office of the Commissioner, Office of Clinical Policy and Programs, Office of Orphan Products Development
          Center for Drug Evaluation and Research
          Center for Biologics Evaluation and Research


The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Natural History Studies for Drug Development.” FDA is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. A natural history study collects information about the natural history of a disease in the absence of an intervention, from the disease's onset until either its resolution or the individual's death. Although knowledge of a disease's natural history can benefit drug development for many disorders and conditions, natural history information is usually not available or is incomplete for most rare diseases; therefore, natural history information is particularly needed for these diseases.


 
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						All written comments should be identified with this document's docket number: FDA-2019-D-0481.

                                                                                  Content current as of:
      04/15/2020
         
                  
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