Rare Diseases: Natural History Studies for Drug Development; Draft Guidance for Industry; Availability, 11110-11112 [2019-05655]

Download as PDF 11110 Federal Register / Vol. 84, No. 57 / Monday, March 25, 2019 / Notices Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. • For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in ‘‘Instructions.’’ Instructions: All submissions received must include the Docket No. FDA– 2019–N–0795 for ‘‘Antimicrobial Drugs Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments.’’ Received comments, those filed in a timely manner (see ADDRESSES), will be placed in the docket and, except for those submitted as ‘‘Confidential Submissions,’’ publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday. • Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states ‘‘THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.’’ FDA will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/ blacked out, will be available for public viewing and posted on https:// www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify the information as ‘‘confidential.’’ Any information marked as ‘‘confidential’’ will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https://www.gpo.gov/ fdsys/pkg/FR-2015-09-18/pdf/201523389.pdf. Docket: For access to the docket to read background documents or the electronic and written/paper 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 Dockets Management VerDate Sep<11>2014 16:47 Mar 22, 2019 Jkt 247001 Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. FOR FURTHER INFORMATION CONTACT: Lauren Tesh, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 31, Rm. 2417, Silver Spring, MD 20993–0002, 301–796–9001, Fax: 301–847–8533, AMDAC@fda.hhs.gov; or the FDA Advisory Committee Information Line, 1–800–741–8138 (301–443–0572 in the Washington, DC area). A notice in the Federal Register about last minute modifications that impact a previously announced advisory committee meeting cannot always be published quickly enough to provide timely notice. Therefore, you should always check FDA’s website at https://www.fda.gov/Advisory Committees/default.htm and scroll down to the appropriate advisory committee meeting link, or call the advisory committee information line to learn about possible modifications before coming to the meeting. SUPPLEMENTARY INFORMATION: Agenda: The committee will discuss the safety and effectiveness of bacitracin for intramuscular injection for the treatment of infants with pneumonia and empyema caused by staphylococci shown to be susceptible to the drug, which is the only approved indication for bacitracin for intramuscular injection. The committee will also consider whether there are other uses for bacitracin for intramuscular injection that could be studied. FDA will present background information on the regulatory history of bacitracin for intramuscular injection and information on the current use of bacitracin for intramuscular injection. FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its website prior to the meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA’s website after the meeting. Background material is available at https://www.fda.gov/ AdvisoryCommittees/Calendar/ default.htm. Scroll down to the appropriate advisory committee meeting link. Procedure: Interested persons may present data, information, or views, orally or in writing, on issues pending before the committee. All electronic and written submissions submitted to the Docket (see ADDRESSES) on or before April 11, 2019, will be provided to the committee. Oral presentations from the PO 00000 Frm 00066 Fmt 4703 Sfmt 4703 public will be scheduled between approximately 10:30 a.m. and 11:30 a.m. Those individuals interested in making formal oral presentations should notify the contact person and submit a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation on or before April 3, 2019. Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing session. The contact person will notify interested persons regarding their request to speak by April 4, 2019. Persons attending FDA’s advisory committee meetings are advised that FDA is not responsible for providing access to electrical outlets. For press inquiries, please contact the Office of Media Affairs at fdaoma@ fda.hhs.gov or 301–796–4540. FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with disabilities. If you require accommodations due to a disability, please contact Lauren Tesh (see FOR FURTHER INFORMATION CONTACT) at least 7 days in advance of the meeting. FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our website at https://www.fda.gov/Advisory Committees/AboutAdvisoryCommittees/ ucm111462.htm for procedures on public conduct during advisory committee meetings. Notice of this meeting is given under the Federal Advisory Committee Act (5 U.S.C. app. 2). Dated: March 20, 2019. Lowell J. Schiller, Acting Associate Commissioner for Policy. [FR Doc. 2019–05652 Filed 3–22–19; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2019–D–0481] Rare Diseases: Natural History Studies for Drug Development; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. E:\FR\FM\25MRN1.SGM 25MRN1 Federal Register / Vol. 84, No. 57 / Monday, March 25, 2019 / Notices ACTION: Written/Paper Submissions 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. SUMMARY: 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. DATES: You may submit comments on any guidance at any time as follows: ADDRESSES: Electronic Submissions Submit electronic comments in the following way: • Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https:// www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov. • If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see ‘‘Written/Paper Submissions’’ and ‘‘Instructions’’). VerDate Sep<11>2014 16:47 Mar 22, 2019 Jkt 247001 Submit written/paper submissions as follows: • Mail/Hand delivery/Courier (for written/paper submissions): Dockets Management Staff (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. • For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in ‘‘Instructions.’’ Instructions: All submissions received must include the Docket No. FDA– 2019–D–0481 for ‘‘Rare Diseases: Natural History Studies for Drug Development; Draft Guidance for Industry.’’ Received comments will be placed in the docket and, except for those submitted as ‘‘Confidential Submissions,’’ publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday. • Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states ‘‘THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.’’ The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on https://www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as ‘‘confidential.’’ Any information marked as ‘‘confidential’’ will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https://www.gpo.gov/ fdsys/pkg/FR-2015-09-18/pdf/201523389.pdf. Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https:// PO 00000 Frm 00067 Fmt 4703 Sfmt 4703 11111 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 Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. You may submit comments on any guidance at any time (see 21 CFR 10.115(g)(5)). 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; the 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; or the 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. 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. FOR FURTHER INFORMATION CONTACT: 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. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a draft guidance for industry entitled ‘‘Rare Diseases: Natural History Studies for Drug Development.’’ This draft guidance is intended 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. Although FDA has published guidance concerning common issues encountered in drug E:\FR\FM\25MRN1.SGM 25MRN1 11112 Federal Register / Vol. 84, No. 57 / Monday, March 25, 2019 / Notices development for rare diseases, this draft guidance expands on the topic of natural history studies specifically. There are approximately 7,000 recognized rare diseases. Individually, rare diseases affect a small number of people, but collectively rare diseases affect about 1 in 10 people in the United States. Most rare diseases have no approved therapies and thus present a significant unmet public health need. 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. This draft guidance describes the potential uses of a natural history study in all phases of drug development and in the postmarketing period, the strengths and weaknesses of various types of natural history studies that might be conducted to support drug development, data elements and research plans, and a practical framework for the conduct of a natural history study. The draft guidance also discusses patient confidentiality and data protection issues in natural history studies and the potential nature of interactions with FDA related to these studies. 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 ‘‘Rare Diseases: Natural History Studies for Drug Development.’’ 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. This guidance is not subject to Executive Order 12866. II. 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 parts 312 and 314 have been approved under OMB control numbers 0910–0014 and 0910– 0001, respectively. The collections of information in 21 CFR parts 50 and 56 (Protection of Human Subjects: Informed Consent; Institutional Review Boards) have been approved under OMB control number 0910–0755. VerDate Sep<11>2014 16:47 Mar 22, 2019 Jkt 247001 III. Electronic Access Persons with access to the internet may obtain the draft guidance at https:// www.fda.gov/Drugs/Guidance ComplianceRegulatoryInformation/ Guidances/default.htm, https:// www.fda.gov/BiologicsBloodVaccines/ GuidanceComplianceRegulatory Information/Guidances/default.htm, https://www.fda.gov/ForIndustry/ DevelopingProductsforRareDiseases Conditions/default.htm, or https:// www.regulations.gov. Dated: March 20, 2019. Lowell J. Schiller, Acting Associate Commissioner for Policy. [FR Doc. 2019–05655 Filed 3–22–19; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES [Document Identifier: OS–0937–New] Agency Information Collection Request; 30-Day Public Comment Request Office of the Secretary, HHS. Notice. AGENCY: ACTION: In compliance with the requirement of the Paperwork Reduction Act of 1995, the Office of the Secretary (OS), Department of Health and Human Services, is publishing the following summary of a proposed collection for public comment. DATES: Comments on the ICR must be received on or before April 24, 2019. ADDRESSES: Submit your comments to OIRA_submission@omb.eop.gov or via facsimile to (202) 395–5806. FOR FURTHER INFORMATION CONTACT: Sherrette Funn, Sherrette.Funn@hhs.gov or (202) 795–7714. When submitting comments or requesting information, please include the document identifier 0937-Fertility Knowledge Survey-30D and project title for reference. SUPPLEMENTARY INFORMATION: Interested persons are invited to send comments regarding this burden estimate or any other aspect of this collection of information, including any of the following subjects: (1) The necessity and utility of the proposed information collection for the proper performance of the agency’s functions; (2) the accuracy of the estimated burden; (3) ways to enhance the quality, utility, and clarity of the information to be collected; and (4) the use of automated collection techniques or other forms of information technology to minimize the information collection burden. Title of the Collection: Fertility Knowledge Survey. SUMMARY: PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 Type of Collection: New. Abstract: The Office of the Assistant Secretary for Health/Office of Population Affairs (OPA) is requesting a three-year approval by the Office of Management and Budget of a new information collection. We are seeking to collect information to increase understanding of (1) adolescent and young adult knowledge of human (female and male) fertility and (2) how this knowledge is related to behaviors and intentions involving childbearing. We propose to collect this information through a 20-minute web survey (Fertility Knowledge Survey) of 2,100 females and 1,900 males, aged 15 to 29 years, using an online panel that is based on a probability-based sample of the U.S. population. Respondents will be members of the general public, and consist of English-speaking females and males, aged 15 to 29 years, who are able to get pregnant or to biologically father a child, respectively. The survey will produce evidence and findings that are expected to be generalizable to the population of individuals in the United States with these characteristics. Possessing accurate knowledge about human fertility is important information that enables reproductive-aged women and men to make informed decisions and plans about reproduction and empowers them to seek appropriate and timely health services (e.g., family planning, related preventive healthcare, or infertility assessment) to achieve those plans. OPA requires high-quality information on the fertility knowledge and related behaviors of U.S. adolescents and young adults to inform Title X policies and strategies that aim to close knowledge gaps, enhance reproductive life planning, and increase access to appropriate and evidenceinformed care. The web survey (Fertility Knowledge Survey) will be self-administered once by each respondent using a personal computer, tablet, or smart phone. A web survey has numerous methodological advantages, including increased accuracy in measurement of key variables of interest, and reduced burden on study participants. This collection will not involve small business or small entities. The estimated annualized hour burden of responding to this information collection is 1,333 hours, or a weighted average of 20 minutes (.33 hours) per respondent. The hour-burden estimate includes the time spent by a respondent to read the email invitation, review the online consent or assent (minor), and complete the survey. Participation is voluntary and there are E:\FR\FM\25MRN1.SGM 25MRN1

Agencies

[Federal Register Volume 84, Number 57 (Monday, March 25, 2019)]
[Notices]
[Pages 11110-11112]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-05655]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-0481]


Rare Diseases: Natural History Studies for Drug Development; 
Draft Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

[[Page 11111]]


ACTION: Notice of availability.

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

SUMMARY: 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.

DATES: 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.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2019-D-0481 for ``Rare Diseases: Natural History Studies for Drug 
Development; Draft Guidance for Industry.'' Received comments will be 
placed in the docket and, except for those submitted as ``Confidential 
Submissions,'' publicly viewable at https://www.regulations.gov or at 
the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through 
Friday.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on https://www.regulations.gov. 
Submit both copies to the Dockets Management Staff. If you do not wish 
your name and contact information to be made publicly available, you 
can provide this information on the cover sheet and not in the body of 
your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR 56469, September 18, 2015, or 
access the information at: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper 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 Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    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; the 
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; or 
the 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. 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.

FOR FURTHER INFORMATION CONTACT: 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.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Rare Diseases: Natural History Studies for Drug 
Development.'' This draft guidance is intended 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. Although FDA has published guidance 
concerning common issues encountered in drug

[[Page 11112]]

development for rare diseases, this draft guidance expands on the topic 
of natural history studies specifically.
    There are approximately 7,000 recognized rare diseases. 
Individually, rare diseases affect a small number of people, but 
collectively rare diseases affect about 1 in 10 people in the United 
States. Most rare diseases have no approved therapies and thus present 
a significant unmet public health need. 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.
    This draft guidance describes the potential uses of a natural 
history study in all phases of drug development and in the 
postmarketing period, the strengths and weaknesses of various types of 
natural history studies that might be conducted to support drug 
development, data elements and research plans, and a practical 
framework for the conduct of a natural history study. The draft 
guidance also discusses patient confidentiality and data protection 
issues in natural history studies and the potential nature of 
interactions with FDA related to these studies.
    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 ``Rare 
Diseases: Natural History Studies for Drug Development.'' 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. This guidance 
is not subject to Executive Order 12866.

II. 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 parts 312 and 314 have 
been approved under OMB control numbers 0910-0014 and 0910-0001, 
respectively. The collections of information in 21 CFR parts 50 and 56 
(Protection of Human Subjects: Informed Consent; Institutional Review 
Boards) have been approved under OMB control number 0910-0755.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm, or https://www.regulations.gov.

    Dated: March 20, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-05655 Filed 3-22-19; 8:45 am]
 BILLING CODE 4164-01-P
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