80 FR 49246 - Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 158 (August 17, 2015)

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Rare Diseases: Common Issues in Drug Development.'' The purpose of this draft guidance is to advance and facilitate the development of drugs and biologics to treat rare diseases. Drug development for rare diseases has many challenges related to the nature of these diseases. This draft guidance is intended to assist sponsors of drug and biological products for treating rare diseases in conducting more efficient and successful development programs.

[Federal Register Volume 80, Number 158 (Monday, August 17, 2015)]
[Notices]
[Pages 49246-49247]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-20235]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-2818]


Rare Diseases: Common Issues in Drug Development; Draft Guidance 
for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Rare 
Diseases: Common Issues in Drug Development.'' The purpose of this 
draft guidance is to advance and facilitate the development of drugs 
and biologics to treat rare diseases. Drug development for rare 
diseases has many challenges related to the nature of these diseases. 
This draft guidance is intended to assist sponsors of drug and 
biological products for treating rare diseases in conducting more 
efficient and successful development programs.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by October 16, 2015.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 
20993-0002; or Office of Communication, Outreach, and Development, 
Center for Biologics Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver 
Spring, MD 20993-0002. Send one self-addressed adhesive label to assist 
that office in processing your requests. See the SUPPLEMENTARY 
INFORMATION section for electronic access to the draft guidance 
document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Jonathan Goldsmith, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6480, Silver Spring, MD 20903-0002, 240-
402-9959; or 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.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Rare Diseases: Common Issues in Drug Development.'' This 
guidance is intended to assist sponsors of drug and biological products 
for treating rare diseases in conducting more efficient and successful 
development programs through a discussion of selected issues commonly 
encountered in rare disease drug development. Although these issues are 
encountered in other drug development programs, they are frequently 
more difficult to address in the context of a rare disease than a 
common disease for which there is greater and more widespread medical 
experience. These issues are also more acute with increasing rarity of 
the disorder. A rare disease is defined by the Orphan Drug Act as a 
disorder or condition that affects less than 200,000 persons in the 
United States; however, most rare diseases affect far fewer persons.
    Most rare disorders are serious conditions with no approved 
treatments, and rare disease patients have considerable unmet medical 
needs. Collectively, rare diseases are highly diverse. FDA is committed 
to helping sponsors of drugs for rare diseases create successful 
programs that address the particular challenges posed by each disease.
    This guidance addresses the following important components of drug 
development:
     Adequate description and understanding of the disease's 
natural history
     Adequate understanding of the pathophysiology of the 
disease and the drug's proposed mechanism of action
     Nonclinical pharmacotoxicology considerations to support 
the proposed clinical investigation(s)
     Standard of evidence to establish safety and effectiveness
     Drug manufacturing considerations during drug development
    Early consideration of these issues allows sponsors to efficiently 
and adequately address them during the course of drug development, from 
drug discovery to confirmatory efficacy and safety studies, and to have 
productive meetings with FDA.
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the current thinking of FDA on common issues 
in drug development for rare diseases. 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.

II. The Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information that are subject to review by the Office of Management and 
Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-
3520). The collections of information in 21 CFR part 312 have been 
approved under OMB control number 0910-0014, and the collections of 
information in 21 CFR part 314 have been approved under OMB control 
number 0910-0001.

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the

[[Page 49247]]

docket number found in brackets in the heading of this document. 
Received comments may be seen in the Division of Dockets Management 
between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to 
the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the document at 
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or http://www.regulations.gov.

    Dated: August 12, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-20235 Filed 8-14-15; 8:45 am]
 BILLING CODE 4164-01-P