Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability, 49246-49247 [2015-20235]

Download as PDF 49246 Federal Register / Vol. 80, No. 158 / Monday, August 17, 2015 / Notices III. Comments Interested persons may submit either electronic comments regarding this document to https://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 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 https:// www.regulations.gov. IV. Electronic Access Persons with access to the Internet may obtain the document at either https://www.fda.gov/Drugs/ GuidanceCompliance RegulatoryInformation/Guidances/ default.htm or https:// www.regulations.gov. Dated: August 12, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015–20228 Filed 8–14–15; 8:45 am] BILLING CODE 4164–01–P 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SERVICES Food and Drug Administration [Docket No. FDA–2015–D–2818] Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ‘‘Rare Diseases: Common Issues in Drug Development.’’ The purpose of this draft guidance is to advance and facilitate the development of drugs and biologics to treat rare diseases.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 This draft guidance is intended to assist sponsors of drug and biological products for treating rare diseases in conducting more efficient and successful development programs.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comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments mstockstill on DSK4VPTVN1PROD with NOTICES SUMMARY: VerDate Sep<11>2014 16:57 Aug 14, 2015 Jkt 235001 on the draft guidance by October 16, 2015. Submit written requests for single copies of the draft guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th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 https:// 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: ADDRESSES: 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. PO 00000 Frm 00049 Fmt 4703 Sfmt 4703 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 https://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 E:\FR\FM\17AUN1.SGM 17AUN1 Federal Register / Vol. 80, No. 158 / Monday, August 17, 2015 / Notices 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 https:// www.regulations.gov. IV. Electronic Access Persons with access to the Internet may obtain the document at https:// www.fda.gov/Drugs/ GuidanceCompliance RegulatoryInformation/Guidances/ default.htm, https://www.fda.gov/ BiologicsBloodVaccines/ GuidanceCompliance RegulatoryInformation/default.htm, or https://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 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2015–D–1176] Compounding Animal Drugs From Bulk Drug Substances; Draft Guidance for Industry; Extension of Comment Period AGENCY: Food and Drug Administration, HHS. ACTION: period. The Food and Drug Administration (FDA) is extending the comment period for the document that appeared in the Federal Register of May 19, 2015. In the document, FDA requested comments on draft guidance for industry (GFI) #230 entitled ‘‘Compounding Animal Drugs from Bulk Drug Substances.’’ FDA is taking this action in response to a request for an extension to allow interested persons additional time to submit comments. DATES: FDA is extending the comment period on the document published May 19, 2015 (80 FR 28624). Submit either electronic or written comments on the draft guidance by November 16, 2015. ADDRESSES: You may submit comments by any of the following methods. mstockstill on DSK4VPTVN1PROD with NOTICES SUMMARY: Electronic Submissions Submit electronic comments in the following way: • Federal eRulemaking Portal: https:// www.regulations.gov. Follow the instructions for submitting comments. 16:57 Aug 14, 2015 Jkt 235001 Submit written comments in the following ways: • Mail/Hand delivery/Courier (for paper submissions): Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Instructions: All submissions received must include the Docket No. FDA– 2015–D–1176. All comments received may be posted without change to https://www.regulations.gov, including any personal information provided. For additional information on submitting comments, see the ‘‘Request for Comments’’ heading of the SUPPLEMENTARY INFORMATION section of this document. Docket: For access to the docket to read background documents or comments received, go to https:// www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the ‘‘Search’’ box and follow the prompts and/or go to the Division of Dockets Management, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. FOR FURTHER INFORMATION CONTACT: Division of Compliance, Center for Veterinary Medicine, Food and Drug Administration (HFV–230), 7519 Standish Pl., Rockville, MD 20855, 240– 402–7001, CVMCompliance@ fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background Notice; extension of comment VerDate Sep<11>2014 Written Submissions In the Federal Register of May 19, 2015, FDA published a document with a 90-day comment period for draft GFI #230 entitled ‘‘Compounding Animal Drugs from Bulk Drug Substances.’’ The draft guidance describes FDA’s policies with regard to compounding animal drugs from bulk drug substances. When final, the guidance will reflect FDA’s current thinking on the issues addressed by the guidance. FDA has received a request for a 90day extension of the comment period. The request conveyed concern that the current 90-day comment period does not allow sufficient time to respond. FDA has considered the request and is extending the comment period for 90 days, until November 16, 2015. FDA believes that a 90-day extension allows adequate time for interested persons to submit comments without significantly delaying further FDA action on this guidance document. II. Specific Topics for Comment In addition to comments on the draft guidance as written, we are specifically PO 00000 Frm 00050 Fmt 4703 Sfmt 4703 49247 requesting comments on the following issues: • Should the final guidance address the issue of FDA-approved animal and human drugs that are in shortage or are otherwise unavailable (e.g., disruptions in the manufacture or supply chain; business decisions to stop marketing the drug; drug is subject to Agency action based on safety, effectiveness, or manufacturing concerns)? If so: Æ How should these situations be addressed in the final guidance? Æ How should the final guidance define the terms ‘‘shortage’’ and ‘‘unavailable’’? Æ What criteria should FDA use to determine if an approved animal or human drug is in shortage or otherwise unavailable? • Do United States Pharmacopeia and National Formulary (USP–NF) 1 chapters 795 and 797 provide suitable standards for animal drugs compounded by veterinarians, and if not, what standards of safety, purity, and quality should apply to animal drugs compounded by veterinarians? • Should licensed veterinarians be able to sell or transfer an animal drug compounded from bulk drug substances by a State-licensed pharmacy or an outsourcing facility to owners or caretakers of animals under the veterinarian’s care? • Should the final guidance include a condition on the amount or percentage of compounded animal drugs that a pharmacy or outsourcing facility can ship in interstate commerce? If so, what would a reasonable amount be? • Is additional guidance needed to address the repackaging of drugs for animal use? Æ How widespread is the practice of repackaging drugs for animal use? Æ What types of drugs are repackaged for animal use, and why are they repackaged? Æ Have problems been identified with repackaged drugs for animal use? • Is additional guidance needed to address the compounding of animal drugs from approved animal or human drugs under section 512(a)(4) or (a)(5) of the Federal Food, Drug, and Cosmetic Act (the FD&C Act) (21 U.S.C. 360b(a)(4) and (a)(5)) and 21 CFR part 530? • Is additional guidance needed to address the compounding of animal drugs from bulk drug substances for food-producing animals? 1 Chapters <795> ‘‘Pharmaceutical Compounding—Nonsterile Preparations’’ and <797> ‘‘Pharmaceutical Compounding—Sterile Preparations’’ can be found in both the USP Compounding Compendium and the combined United States Pharmacopeia and National Formulary (USP–NF). These compendia are available at https://www.usp.org/. E:\FR\FM\17AUN1.SGM 17AUN1

Agencies

[Federal Register Volume 80, Number 158 (Monday, August 17, 2015)]
[Notices]
[Pages 49246-49247]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-20235]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 https://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 https://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 https://www.regulations.gov.

IV. Electronic Access

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