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2019-03-22
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Rare Diseases: Natural History Studies for Drug Development; Draft Guidance for Industry; Availability
Notices
D09002ee1bdb7933c
D09002ee1bdb793b5
United States
Department of Health and Human Services
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United States Government Agency or Subagency
United States
Food and Drug Administration
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United States Government Agency or Subagency
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Rare Diseases: Natural History Studies for Drug Development.'' FDA is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological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.
84 FR 11110
https://www.govinfo.gov/app/details/FR-2019-03-25/2019-05655
2019-05655
fr25mr19-74
4164-01-P
Docket No. FDA-2019-D-0481
https://www.govinfo.gov/app/details/FR-2019-03-25/2019-05655
https://www.govinfo.gov/content/pkg/FR-2019-03-25/html/2019-05655.htm
https://www.govinfo.gov/content/pkg/FR-2019-03-25/pdf/2019-05655.pdf
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84 FR 11110
Rare Diseases: Natural History Studies for Drug Development; Draft Guidance for Industry; Availability; Federal Register Vol. 84, Issue
NOTICE
2019-05655
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
2019-05-24
Docket No. FDA-2019-D-0481
4164-01-P
2019-05655
Notice of availability.
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Rare Diseases: Natural History Studies for Drug Development.'' FDA is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological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.
Submit either electronic or written comments on the draft guidance by May 24, 2019 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.
Lucas Kempf, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 6460, Silver Spring, MD 20993, 301-796- 1140; Stephen Ripley, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911; or Aaron Friedman, Office of Orphan Products Development, Office of Special Medical Programs, Office of the Commissioner, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5295, Silver Spring, MD 20993- 0002, 301-796-8660.
Guidance:
Rare Diseases: Natural History Studies for Drug Development
,
https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm
https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm
https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm
https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf
https://www.regulations.gov
Federal Register
Vol. 84, no. 57
Office of the Federal Register, National Archives and Records Administration
2019-03-25
continuing
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Table of Contents:
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https://www.govinfo.gov/app/details/FR-2019-03-25
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