83 FR 52223 - Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 200 (October 16, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 200 (October 16, 2018)
| Page Range | 52223-52224 | |
| FR Document | 2018-22435 |
[Federal Register Volume 83, Number 200 (Tuesday, October 16, 2018)] [Notices] [Pages 52223-52224] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-22435] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2018-D-3268] Rare Diseases: Early Drug Development and the Role of Pre- Investigational New Drug Application Meetings; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. 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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings.'' The purpose of this draft guidance is to assist sponsors of drug and biological products for the treatment of rare diseases in planning and conducting more efficient and productive pre-investigational new drug application (pre-IND) meetings. Drug development for rare diseases has many challenges related to the nature of these diseases. This draft guidance is intended to advance and facilitate the development of drugs and biological products for the treatment of rare diseases. DATES: Submit either electronic or written comments on the draft guidance by December 17, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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-2018-D-3268 for ``Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings; 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 [[Page 52224]] 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.thefederalregister.org/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, 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. 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 20993-0002, 301- 796-1140; 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: Early Drug Development and the Role of Pre- Investigational New Drug Application Meetings.'' This guidance is intended to assist sponsors of drug and biological products for the treatment of rare diseases in planning and conducting more efficient and productive pre-IND meetings through a discussion of selected issues commonly encountered in the early phases of rare disease drug development. Although these issues are encountered in other drug development programs, the issues are frequently more difficult to address in the context of a rare disease than in the context of a common disease, of which there is often greater and more widespread medical experience. A rare disease is defined in section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb(a)(2)) as a disease or condition that affects fewer than 200,000 people in the United States or affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug. Most rare diseases affect far fewer than 200,000 people. Most rare disorders are serious conditions with no approved treatments, and rare disease patients often have considerable unmet medical needs. Collectively, rare diseases are highly diverse. FDA is committed to helping sponsors of drugs for rare diseases have successful pre-IND meetings that address the particular challenges posed by each drug. This guidance addresses the following important topics related to pre-IND meetings: Regulatory considerations across various FDA disciplines including chemistry, manufacturing, and controls; nonclinical; clinical pharmacology; and clinical. Additional considerations, including expedited programs for serious conditions, companion diagnostics, orphan drug incentives, pediatric studies, and data standards. Early consideration of these issues allows sponsors to efficiently and adequately plan for a productive pre-IND meeting 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 ``Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings.'' 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. 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 parts 312 and 314 have been approved under OMB control numbers 0910-0014 and 0910-0001, respectively. The collection of information resulting from the draft guidance for industry ``Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products'' (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm590547.pdf) has been approved under OMB control number 0910-0429. The collection of information resulting from the guidance for industry ``Expedited Programs for Serious Conditions--Drugs and Biologics'' (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm358301.pdf) has been approved under OMB control number 0910-0765. III. Electronic Access Persons with access to the internet may obtain the draft guidance at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or https://www.regulations.gov. Dated: October 10, 2018. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2018-22435 Filed 10-15-18; 8:45 am] BILLING CODE 4164-01-P
![Federal Register / Vol. 83, No. 200 / Tuesday, October 16, 2018 / Notices 52223
Low-Frequency Molecular Subsets of a applicable statutes and regulations. This • Federal eRulemaking Portal:
Disease.’’ This guidance is intended to guidance is not subject to Executive https://www.regulations.gov. Follow the
assist sponsors in designing drug Order 12866. instructions for submitting comments.
development programs to generate the Comments submitted electronically,
II. Electronic Access
evidence needed to demonstrate efficacy including attachments, to https://
of a targeted therapy across molecular Persons with access to the internet www.regulations.gov will be posted to
subsets within a disease where some may obtain the guidance at either the docket unchanged. Because your
molecular alterations may occur at low https://www.fda.gov/Drugs/Guidance comment will be made public, you are
frequencies. ComplianceRegulatoryInformation/ solely responsible for ensuring that your
In recent years, advances in our Guidances/default.htm, https:// comment does not include any
understanding of the molecular www.fda.gov/BiologicsBloodVaccines/ confidential information that you or a
pathology of many diseases have led to GuidanceComplianceRegulatory third party may not wish to be posted,
the development of targeted therapies. Information/Guidances/default.htm, or such as medical information, your or
Although variability in drug response https://www.regulations.gov. anyone else’s Social Security number, or
has long been recognized in drug Dated: October 10, 2018. confidential business information, such
development, targeted therapies present Leslie Kux, as a manufacturing process. Please note
new challenges in addressing the Associate Commissioner for Policy. that if you include your name, contact
heterogeneity in drug response because [FR Doc. 2018–22437 Filed 10–15–18; 8:45 am]
information, or other information that
the pharmacological effect of a targeted identifies you in the body of your
BILLING CODE 4164–01–P
therapy is often related to a particular comments, that information will be
molecular alteration (e.g., a mutation, posted on https://www.regulations.gov.
gene fusion, epigenetic change, etc.). DEPARTMENT OF HEALTH AND • If you want to submit a comment
Many clinically defined diseases are HUMAN SERVICES with confidential information that you
influenced or caused by a range of do not wish to be made available to the
different molecular alterations, some of Food and Drug Administration public, submit the comment as a
which may be rare, that impact a written/paper submission and in the
[Docket No. FDA–2018–D–3268] manner detailed (see ‘‘Written/Paper
common target protein or pathway
involved in the disease pathogenesis. Rare Diseases: Early Drug Submissions’’ and ‘‘Instructions’’).
This heterogeneity in the molecular Development and the Role of Pre- Written/Paper Submissions
etiology of a given disease can result in Investigational New Drug Application
differential effects of a targeted therapy Submit written/paper submissions as
Meetings; Draft Guidance for Industry; follows:
among patients with the same disease Availability • Mail/Hand delivery/Courier (for
but who have different molecular
AGENCY: Food and Drug Administration, written/paper submissions): Dockets
alterations. Therefore, the type and
HHS. Management Staff (HFA–305), Food and
quantity of evidence that is needed to
ACTION: Notice of availability. Drug Administration, 5630 Fishers
demonstrate efficacy across molecular
Lane, Rm. 1061, Rockville, MD 20852.
subsets within a disease needs to be SUMMARY: The Food and Drug • For written/paper comments
clearly specified. Administration (FDA or Agency) is submitted to the Dockets Management
This guidance addresses the following announcing the availability of a draft Staff, FDA will post your comment, as
important topics in evaluating the guidance for industry entitled ‘‘Rare well as any attachments, except for
benefits and risks of targeted Diseases: Early Drug Development and information submitted, marked and
therapeutics within a disease where the Role of Pre-Investigational New identified, as confidential, if submitted
some molecular alterations may occur at Drug Application Meetings.’’ The as detailed in ‘‘Instructions.’’
low frequencies: purpose of this draft guidance is to Instructions: All submissions received
• Identification of patients for inclusion assist sponsors of drug and biological must include the Docket No. FDA–
in clinical trials products for the treatment of rare 2018–D–3268 for ‘‘Rare Diseases: Early
• Interpretation of study results and diseases in planning and conducting Drug Development and the Role of Pre-
generalizability of findings to the more efficient and productive pre- Investigational New Drug Application
study population investigational new drug application Meetings; Draft Guidance for Industry.’’
• Benefit-risk determination and (pre-IND) meetings. Drug development Received comments will be placed in
therapeutic product labeling for rare diseases has many challenges the docket and, except for those
• Refining the indicated population related to the nature of these diseases. submitted as ‘‘Confidential
after the initial approval This draft guidance is intended to Submissions,’’ publicly viewable at
This final guidance incorporates advance and facilitate the development https://www.regulations.gov or at the
public comments to the draft guidance of drugs and biological products for the Dockets Management Staff between 9
published in December of 2017 and treatment of rare diseases. a.m. and 4 p.m., Monday through
includes minimal revisions for clarity. DATES: Submit either electronic or Friday.
This guidance is being issued written comments on the draft guidance • Confidential Submissions—To
consistent with FDA’s good guidance by December 17, 2018 to ensure that the submit a comment with confidential
practices regulation (21 CFR 10.115). Agency considers your comment on this information that you do not wish to be
The guidance represents the current draft guidance before it begins work on made publicly available, submit your
amozie on DSK3GDR082PROD with NOTICES1
thinking of FDA on ‘‘Developing the final version of the guidance. comments only as a written/paper
Targeted Therapies in Low-Frequency ADDRESSES: You may submit comments submission. You should submit two
Molecular Subsets of a Disease.’’ It does on any guidance at any time as follows: copies total. One copy will include the
not establish any rights for any person information you claim to be confidential
and is not binding on FDA or the public. Electronic Submissions with a heading or cover note that states
You can use an alternative approach if Submit electronic comments in the ‘‘THIS DOCUMENT CONTAINS
it satisfies the requirements of the following way: CONFIDENTIAL INFORMATION.’’ The
VerDate Sep<11>2014 18:44 Oct 15, 2018 Jkt 247001 PO 00000 Frm 00036 Fmt 4703 Sfmt 4703 E:\FR\FM\16OCN1.SGM 16OCN1](https://thefederalregister.org/doc/83_FR_52423/page/1.svg)
![52224 Federal Register / Vol. 83, No. 200 / Tuesday, October 16, 2018 / Notices
Agency will review this copy, including 7301, Silver Spring, MD 20993–0002, The draft guidance, when finalized, will
the claimed confidential information, in 240–402–7911. represent the current thinking of FDA
its consideration of comments. The SUPPLEMENTARY INFORMATION: on ‘‘Rare Diseases: Early Drug
second copy, which will have the Development and the Role of Pre-
claimed confidential information I. Background
Investigational New Drug Application
redacted/blacked out, will be available FDA is announcing the availability of Meetings.’’ It does not establish any
for public viewing and posted on a draft guidance for industry entitled rights for any person and is not binding
https://www.regulations.gov. Submit ‘‘Rare Diseases: Early Drug Development on FDA or the public. You can use an
both copies to the Dockets Management and the Role of Pre-Investigational New alternative approach if it satisfies the
Staff. If you do not wish your name and Drug Application Meetings.’’ This
requirements of the applicable statutes
contact information to be made publicly guidance is intended to assist sponsors
and regulations. This guidance is not
available, you can provide this of drug and biological products for the
treatment of rare diseases in planning subject to Executive Order 12866.
information on the cover sheet and not
in the body of your comments and you and conducting more efficient and II. The Paperwork Reduction Act of
must identify this information as productive pre-IND meetings through a 1995
‘‘confidential.’’ Any information marked discussion of selected issues commonly
as ‘‘confidential’’ will not be disclosed encountered in the early phases of rare This guidance refers to previously
except in accordance with 21 CFR 10.20 disease drug development. Although approved collections of information that
and other applicable disclosure law. For these issues are encountered in other are subject to review by the Office of
more information about FDA’s posting drug development programs, the issues Management and Budget (OMB) under
of comments to public dockets, see 80 are frequently more difficult to address the Paperwork Reduction Act of 1995
FR 56469, September 18, 2015, or access in the context of a rare disease than in (44 U.S.C. 3501–3520). The collections
the information at: https://www.gpo.gov/ the context of a common disease, of of information in 21 CFR parts 312 and
fdsys/pkg/FR-2015-09-18/pdf/2015- which there is often greater and more 314 have been approved under OMB
23389.pdf. widespread medical experience. A rare control numbers 0910–0014 and 0910–
disease is defined in section 526(a)(2) of 0001, respectively. The collection of
Docket: For access to the docket to
the Federal Food, Drug, and Cosmetic
read background documents or the information resulting from the draft
Act (21 U.S.C. 360bb(a)(2)) as a disease
electronic and written/paper comments guidance for industry ‘‘Formal Meetings
or condition that affects fewer than
received, go to https:// 200,000 people in the United States or Between the FDA and Sponsors or
www.regulations.gov and insert the affects more than 200,000 in the United Applicants of PDUFA Products’’
docket number, found in brackets in the States and for which there is no (available at https://www.fda.gov/ucm/
heading of this document, into the reasonable expectation that the cost of groups/fdagov-public/@fdagov-drugs-
‘‘Search’’ box and follow the prompts developing and making available in the gen/documents/document/
and/or go to the Dockets Management United States a drug for such disease or ucm590547.pdf) has been approved
Staff, 5630 Fishers Lane, Rm. 1061, condition will be recovered from sales under OMB control number 0910–0429.
Rockville, MD 20852. in the United States of such drug. Most The collection of information resulting
You may submit comments on any rare diseases affect far fewer than from the guidance for industry
guidance at any time (see 21 CFR 200,000 people. ‘‘Expedited Programs for Serious
10.115(g)(5)). Most rare disorders are serious Conditions—Drugs and Biologics’’
Submit written requests for single conditions with no approved (available at https://www.fda.gov/ucm/
copies of the draft guidance to the treatments, and rare disease patients groups/fdagov-public/@fdagov-drugs-
Division of Drug Information, Center for often have considerable unmet medical gen/documents/document/
Drug Evaluation and Research, Food needs. Collectively, rare diseases are ucm358301.pdf) has been approved
and Drug Administration, 10001 New highly diverse. FDA is committed to under OMB control number 0910–0765.
Hampshire Ave., Hillandale Building, helping sponsors of drugs for rare
4th Floor, Silver Spring, MD 20993– diseases have successful pre-IND III. Electronic Access
0002, or Office of Communication, meetings that address the particular
challenges posed by each drug. Persons with access to the internet
Outreach, and Development, Center for
This guidance addresses the following may obtain the draft guidance at either
Biologics Evaluation and Research,
important topics related to pre-IND https://www.fda.gov/Drugs/Guidance
Food and Drug Administration, 10903
meetings: ComplianceRegulatoryInformation/
New Hampshire Ave., Bldg. 71, Rm.
3128, Silver Spring, MD 20993–0002. • Regulatory considerations across Guidances/default.htm, https://
Send one self-addressed adhesive label various FDA disciplines including www.fda.gov/BiologicsBloodVaccines/
to assist that office in processing your chemistry, manufacturing, and controls; GuidanceCompliance
requests. See the SUPPLEMENTARY nonclinical; clinical pharmacology; and RegulatoryInformation/default.htm, or
INFORMATION section for electronic clinical. https://www.regulations.gov.
access to the draft guidance document. • Additional considerations,
Dated: October 10, 2018.
including expedited programs for
FOR FURTHER INFORMATION CONTACT: serious conditions, companion Leslie Kux,
Lucas Kempf, Center for Drug diagnostics, orphan drug incentives, Associate Commissioner for Policy.
Evaluation and Research, Food and pediatric studies, and data standards. [FR Doc. 2018–22435 Filed 10–15–18; 8:45 am]
amozie on DSK3GDR082PROD with NOTICES1
Drug Administration, 10903 New Early consideration of these issues BILLING CODE 4164–01–P
Hampshire Ave., Bldg. 22, Rm. 6460, allows sponsors to efficiently and
Silver Spring, MD 20993–0002, 301– adequately plan for a productive pre-
796–1140; or Stephen Ripley, Center for IND meeting with FDA.
Biologics Evaluation and Research, This draft guidance is being issued
Food and Drug Administration, 10903 consistent with FDA’s good guidance
New Hampshire Ave., Bldg. 71, Rm. practices regulation (21 CFR 10.115).
VerDate Sep<11>2014 18:44 Oct 15, 2018 Jkt 247001 PO 00000 Frm 00037 Fmt 4703 Sfmt 9990 E:\FR\FM\16OCN1.SGM 16OCN1](https://thefederalregister.org/doc/83_FR_52423/page/2.svg)
Document Created: 2018-10-15 23:42:01
Document Modified: 2018-10-15 23:42:01
| 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 of availability. | |
| Dates | Submit either electronic or written comments on the draft guidance by December 17, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. | |
| Contact | Lucas Kempf, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 6460, Silver Spring, MD 20993-0002, 301- 796-1140; 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 | 83 FR 52223 |
2024 Federal Register | Disclaimer | Privacy Policy
USC | CFR | eCFR