80 FR 49246 - Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability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)
Food and Drug Administration
Federal Register Volume 80, Issue 158 (August 17, 2015)
| Page Range | 49246-49247 | |
| FR Document | 2015-20235 |
[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] ----------------------------------------------------------------------- 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SERVICES Food and Drug Administration [Docket No. FDA-2015-D-2818] Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Rare Diseases: Common Issues in Drug Development.'' The purpose of this draft guidance is to advance and facilitate the development of drugs and biologics to treat rare diseases.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 This draft guidance is intended to assist sponsors of drug and biological products for treating rare diseases in conducting more efficient and successful development programs.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comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments on the draft guidance by October 16, 2015. ADDRESSES: Submit written requests for single copies of the draft guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th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 See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document.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 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
![49246 Federal Register / Vol. 80, No. 158 / Monday, August 17, 2015 / Notices
III. Comments on the draft guidance by October 16, Most rare disorders are serious
Interested persons may submit either 2015. conditions with no approved
electronic comments regarding this treatments, and rare disease patients
ADDRESSES: Submit written requests for
document to http://www.regulations.gov have considerable unmet medical needs.
single copies of the draft guidance to the
or written comments to the Division of Collectively, rare diseases are highly
Division of Drug Information, Center for
Dockets Management (see ADDRESSES). It diverse. FDA is committed to helping
Drug Evaluation and Research, Food
is only necessary to send one set of sponsors of drugs for rare diseases
and Drug Administration, 10001 New
comments. Identify comments with the create successful programs that address
Hampshire Ave., Hillandale Building,
docket number found in brackets in the the particular challenges posed by each
4th Floor, Silver Spring, MD 20993–
heading of this document. Received disease.
0002; or Office of Communication, This guidance addresses the following
comments may be seen in the Division Outreach, and Development, Center for
of Dockets Management between 9 a.m. important components of drug
Biologics Evaluation and Research, development:
and 4 p.m., Monday through Friday, and Food and Drug Administration, 10903 • Adequate description and
will be posted to the docket at http:// New Hampshire Ave., Bldg. 71, Rm. understanding of the disease’s natural
www.regulations.gov. 3128, Silver Spring, MD 20993–0002. history
IV. Electronic Access Send one self-addressed adhesive label • Adequate understanding of the
to assist that office in processing your pathophysiology of the disease and the
Persons with access to the Internet requests. See the SUPPLEMENTARY
may obtain the document at either drug’s proposed mechanism of action
INFORMATION section for electronic • Nonclinical pharmacotoxicology
http://www.fda.gov/Drugs/ access to the draft guidance document.
GuidanceCompliance considerations to support the proposed
Submit electronic comments on the clinical investigation(s)
RegulatoryInformation/Guidances/ draft guidance to http:// • Standard of evidence to establish
default.htm or http:// www.regulations.gov. Submit written safety and effectiveness
www.regulations.gov. comments to the Division of Dockets • Drug manufacturing considerations
Dated: August 12, 2015. Management (HFA–305), Food and Drug during drug development
Leslie Kux, Administration, 5630 Fishers Lane, Rm. Early consideration of these issues
Associate Commissioner for Policy. 1061, Rockville, MD 20852. allows sponsors to efficiently and
[FR Doc. 2015–20228 Filed 8–14–15; 8:45 am] FOR FURTHER INFORMATION CONTACT: adequately address them during the
BILLING CODE 4164–01–P Jonathan Goldsmith, Center for Drug course of drug development, from drug
Evaluation and Research, Food and discovery to confirmatory efficacy and
Drug Administration, 10903 New safety studies, and to have productive
DEPARTMENT OF HEALTH AND Hampshire Ave., Bldg. 22, Rm. 6480, meetings with FDA.
HUMAN SERVICES Silver Spring, MD 20903–0002, 240– This draft guidance is being issued
402–9959; or Stephen Ripley, Center for consistent with FDA’s good guidance
Food and Drug Administration Biologics Evaluation and Research, practices regulation (21 CFR 10.115).
[Docket No. FDA–2015–D–2818] Food and Drug Administration, 10903 The draft guidance, when finalized, will
New Hampshire Ave., Bldg. 71, Rm. represent the current thinking of FDA
Rare Diseases: Common Issues in 7301, Silver Spring, MD 20993–0002, on common issues in drug development
Drug Development; Draft Guidance for 240–402–7911. for rare diseases. It does not establish
Industry; Availability any rights for any person and is not
SUPPLEMENTARY INFORMATION: binding on FDA or the public. You can
AGENCY: Food and Drug Administration, I. Background use an alternative approach if it satisfies
HHS. the requirements of the applicable
ACTION: Notice. FDA is announcing the availability of statutes and regulations.
a draft guidance for industry entitled
SUMMARY: The Food and Drug ‘‘Rare Diseases: Common Issues in Drug II. The Paperwork Reduction Act of
Administration (FDA or Agency) is Development.’’ This guidance is 1995
announcing the availability of a draft intended to assist sponsors of drug and This guidance refers to previously
guidance for industry entitled ‘‘Rare biological products for treating rare approved collections of information that
Diseases: Common Issues in Drug diseases in conducting more efficient are subject to review by the Office of
Development.’’ The purpose of this draft and successful development programs Management and Budget (OMB) under
guidance is to advance and facilitate the through a discussion of selected issues the Paperwork Reduction Act of 1995
development of drugs and biologics to commonly encountered in rare disease (44 U.S.C. 3501–3520). The collections
treat rare diseases. Drug development drug development. Although these of information in 21 CFR part 312 have
for rare diseases has many challenges issues are encountered in other drug been approved under OMB control
related to the nature of these diseases. development programs, they are number 0910–0014, and the collections
This draft guidance is intended to assist frequently more difficult to address in of information in 21 CFR part 314 have
sponsors of drug and biological the context of a rare disease than a been approved under OMB control
products for treating rare diseases in common disease for which there is number 0910–0001.
conducting more efficient and greater and more widespread medical
successful development programs. III. Comments
mstockstill on DSK4VPTVN1PROD with NOTICES
experience. These issues are also more
DATES: Although you can comment on acute with increasing rarity of the Interested persons may submit either
any guidance at any time (see 21 CFR disorder. A rare disease is defined by electronic comments regarding this
10.115(g)(5)), to ensure that the Agency the Orphan Drug Act as a disorder or document to http://www.regulations.gov
considers your comment on this draft condition that affects less than 200,000 or written comments to the Division of
guidance before it begins work on the persons in the United States; however, Dockets Management (see ADDRESSES). It
final version of the guidance, submit most rare diseases affect far fewer is only necessary to send one set of
either electronic or written comments persons. comments. Identify comments with the
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![Federal Register / Vol. 80, No. 158 / Monday, August 17, 2015 / Notices 49247
docket number found in brackets in the Written Submissions requesting comments on the following
heading of this document. Received issues:
Submit written comments in the
comments may be seen in the Division • Should the final guidance address
following ways: the issue of FDA-approved animal and
of Dockets Management between 9 a.m.
• Mail/Hand delivery/Courier (for human drugs that are in shortage or are
and 4 p.m., Monday through Friday, and
paper submissions): Division of Dockets otherwise unavailable (e.g., disruptions
will be posted to the docket at http://
Management (HFA–305), Food and Drug in the manufacture or supply chain;
www.regulations.gov.
Administration, 5630 Fishers Lane, Rm. business decisions to stop marketing the
IV. Electronic Access 1061, Rockville, MD 20852. drug; drug is subject to Agency action
Persons with access to the Internet Instructions: All submissions received based on safety, effectiveness, or
may obtain the document at http:// must include the Docket No. FDA– manufacturing concerns)? If so:
www.fda.gov/Drugs/ 2015–D–1176. All comments received Æ How should these situations be
GuidanceCompliance may be posted without change to addressed in the final guidance?
RegulatoryInformation/Guidances/ http://www.regulations.gov, including Æ How should the final guidance
default.htm, http://www.fda.gov/ any personal information provided. For define the terms ‘‘shortage’’ and
BiologicsBloodVaccines/ additional information on submitting ‘‘unavailable’’?
GuidanceCompliance comments, see the ‘‘Request for Æ What criteria should FDA use to
RegulatoryInformation/default.htm, or Comments’’ heading of the determine if an approved animal or
http://www.regulations.gov. SUPPLEMENTARY INFORMATION section of human drug is in shortage or otherwise
this document. unavailable?
Dated: August 12, 2015.
Docket: For access to the docket to • Do United States Pharmacopeia and
Leslie Kux, read background documents or National Formulary (USP–NF) 1
Associate Commissioner for Policy. comments received, go to http:// chapters 795 and 797 provide suitable
[FR Doc. 2015–20235 Filed 8–14–15; 8:45 am] www.regulations.gov and insert the standards for animal drugs compounded
BILLING CODE 4164–01–P docket number, found in brackets in the by veterinarians, and if not, what
heading of this document, into the standards of safety, purity, and quality
‘‘Search’’ box and follow the prompts should apply to animal drugs
DEPARTMENT OF HEALTH AND and/or go to the Division of Dockets compounded by veterinarians?
HUMAN SERVICES Management, 5630 Fishers Lane, Rm. • Should licensed veterinarians be
1061, Rockville, MD 20852. able to sell or transfer an animal drug
Food and Drug Administration compounded from bulk drug substances
FOR FURTHER INFORMATION CONTACT:
[Docket No. FDA–2015–D–1176] by a State-licensed pharmacy or an
Division of Compliance, Center for outsourcing facility to owners or
Veterinary Medicine, Food and Drug caretakers of animals under the
Compounding Animal Drugs From Administration (HFV–230), 7519
Bulk Drug Substances; Draft Guidance veterinarian’s care?
Standish Pl., Rockville, MD 20855, 240– • Should the final guidance include a
for Industry; Extension of Comment 402–7001, CVMCompliance@
Period condition on the amount or percentage
fda.hhs.gov. of compounded animal drugs that a
AGENCY: Food and Drug Administration, SUPPLEMENTARY INFORMATION: pharmacy or outsourcing facility can
HHS. ship in interstate commerce? If so, what
ACTION: Notice; extension of comment I. Background would a reasonable amount be?
period. In the Federal Register of May 19, • Is additional guidance needed to
2015, FDA published a document with address the repackaging of drugs for
SUMMARY: The Food and Drug a 90-day comment period for draft GFI animal use?
Administration (FDA) is extending the #230 entitled ‘‘Compounding Animal Æ How widespread is the practice of
comment period for the document that Drugs from Bulk Drug Substances.’’ The repackaging drugs for animal use?
appeared in the Federal Register of May draft guidance describes FDA’s policies Æ What types of drugs are repackaged
19, 2015. In the document, FDA with regard to compounding animal for animal use, and why are they
requested comments on draft guidance drugs from bulk drug substances. When repackaged?
for industry (GFI) #230 entitled final, the guidance will reflect FDA’s Æ Have problems been identified with
‘‘Compounding Animal Drugs from Bulk current thinking on the issues addressed repackaged drugs for animal use?
Drug Substances.’’ FDA is taking this • Is additional guidance needed to
by the guidance.
action in response to a request for an address the compounding of animal
FDA has received a request for a 90-
extension to allow interested persons drugs from approved animal or human
day extension of the comment period.
additional time to submit comments. drugs under section 512(a)(4) or (a)(5) of
The request conveyed concern that the
DATES: FDA is extending the comment
the Federal Food, Drug, and Cosmetic
current 90-day comment period does
period on the document published May Act (the FD&C Act) (21 U.S.C. 360b(a)(4)
not allow sufficient time to respond.
19, 2015 (80 FR 28624). Submit either and (a)(5)) and 21 CFR part 530?
FDA has considered the request and is • Is additional guidance needed to
electronic or written comments on the extending the comment period for 90 address the compounding of animal
draft guidance by November 16, 2015. days, until November 16, 2015. FDA drugs from bulk drug substances for
ADDRESSES: You may submit comments believes that a 90-day extension allows food-producing animals?
mstockstill on DSK4VPTVN1PROD with NOTICES
by any of the following methods. adequate time for interested persons to
submit comments without significantly 1 Chapters <795> ‘‘Pharmaceutical
Electronic Submissions delaying further FDA action on this Compounding—Nonsterile Preparations’’ and
Submit electronic comments in the guidance document. <797> ‘‘Pharmaceutical Compounding—Sterile
following way: Preparations’’ can be found in both the USP
• Federal eRulemaking Portal: http:// II. Specific Topics for Comment Compounding Compendium and the combined
United States Pharmacopeia and National
www.regulations.gov. Follow the In addition to comments on the draft Formulary (USP–NF). These compendia are
instructions for submitting comments. guidance as written, we are specifically available at http://www.usp.org/.
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Document Created: 2015-12-15 11:07:13
Document Modified: 2015-12-15 11:07:13
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| 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. | |
| 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. | |
| FR Citation | 80 FR 49246 |
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