82 FR 54385 - Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 82, Issue 221 (November 17, 2017)
Food and Drug Administration
Federal Register Volume 82, Issue 221 (November 17, 2017)
| Page Range | 54385-54386 | |
| FR Document | 2017-24837 |
[Federal Register Volume 82, Number 221 (Friday, November 17, 2017)] [Notices] [Pages 54385-54386] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2017-24837] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2017-D-6159] Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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of a draft document entitled ``Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry.'' The draft guidance, when finalized, will provide stakeholders engaged in the development of regenerative medicine therapies with FDA's current thinking on the expedited development and review of these products. The draft guidance describes the expedited programs available to sponsors of regenerative medicine therapies for serious or life-threatening diseases or conditions (referred to in the draft guidance as serious conditions), including those products designated as regenerative advanced therapies (which FDA refers to as ``regenerative medicine advanced therapy'' (RMAT) designation); describes how the Center for Biologics Evaluation and Research (CBER) will work with sponsors and encourage flexibility in clinical trial design to facilitate the development of data to demonstrate the safety and effectiveness of regenerative medicine therapies being developed to address unmet medical needs in patients with serious or life- threatening diseases or conditions; and describes the opportunities for sponsors of regenerative medicine therapies to interact with CBER review staff. DATES: Submit either electronic or written comments on the draft guidance by February 15, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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: http://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to http://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 http://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-2017-D-6159 for ``Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry.'' Received comments will be placed in the docket and, except for those submitted as ``Confidential Submissions,'' publicly viewable at http://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 http://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 [[Page 54386]] 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 Office of Communication, Outreach and Development, Center for Biologics Evaluation and Research (CBER), 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 the office in processing your requests. The draft guidance may also be obtained by mail by calling CBER at 1-800-835-4709 or 240-402-8010. See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. FOR FURTHER INFORMATION CONTACT: Tami Belouin, 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 document entitled ``Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry.'' The draft guidance document describes the expedited programs available to sponsors of regenerative medicine therapies for serious or life-threatening diseases or conditions (referred to in the draft guidance as serious conditions), including those products designated as RMATs; provides information about the provisions in the 21st Century Cures Act (Cures Act) (Pub. L. 114-225) regarding the use of the accelerated approval pathway for regenerative medicine therapies that have been granted designation as an RMAT; describes how CBER will encourage flexibility in clinical trial design to facilitate the development of data to demonstrate the safety and effectiveness of regenerative medicine therapies that are being developed to address unmet needs in patients with serious conditions; and describes the opportunities for sponsors of regenerative medicine therapies to interact with CBER review staff. The draft guidance document addresses regenerative medicine therapies, which are defined in section 506(g)(8) of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), as amended by the Cures Act, as including cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, except for those regulated solely under section 361 of the Public Health Service Act (PHS Act) (42 U.S.C. 264) and 21 CFR part 1271. Gene therapies, including genetically modified cells, that lead to a durable modification of cells or tissues can meet the definition of a regenerative medicine therapy. Under section 506(g) of the FD&C Act, RMAT designation is available for a drug (i.e., a human drug, including a drug that is a biological product) that meets the definition of regenerative medicine therapy; is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and with respect to which preliminary clinical evidence indicates the potential to address unmet medical needs for such disease or condition. A combination product (biologic-device, biologic-drug, or biologic-device-drug) can be eligible for RMAT designation when the biological product provides the greatest contribution to the overall intended therapeutic effects of the combination product (i.e., the primary mode of action in the combination product is conveyed by the biological product component). Elsewhere in this issue of the Federal Register, FDA is announcing the availability of a document entitled ``Evaluation of Devices Used with Regenerative Medicine Advanced Therapies; Draft Guidance for Industry.'' 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 ``Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry.'' 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 draft guidance refers to previously approved collections of information found in FDA regulations. These collections of information 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; the collections of information regarding formal meetings in ``Guidance for Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products'' have been approved under OMB control number 0910-0429; the collections of information in 21 CFR part 601 have been approved under OMB control number 0910-0338; the collections of information for expedited programs in ``Guidance for Industry: Expedited Programs for Serious Conditions--Drugs and Biologics,'' have been approved under OMB control number 0910-0765; the collections of information in 21 CFR part 314 have been approved under OMB control number 0910-0001. III. Electronic Access Persons with access to the Internet may obtain the draft guidance at either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or https://www.regulations.gov. Dated: November 13, 2017. Anna K. Abram, Deputy Commissioner for Policy, Planning, Legislation, and Analysis. [FR Doc. 2017-24837 Filed 11-16-17; 8:45 am] BILLING CODE 4164-01-P
![54386 Federal Register / Vol. 82, No. 221 / Friday, November 17, 2017 / Notices
and other applicable disclosure law. For development of data to demonstrate the II. Paperwork Reduction Act of 1995
more information about FDA’s posting safety and effectiveness of regenerative This draft guidance refers to
of comments to public dockets, see 80 medicine therapies that are being previously approved collections of
FR 56469, September 18, 2015, or access developed to address unmet needs in information found in FDA regulations.
the information at: https://www.gpo.gov/ patients with serious conditions; and These collections of information are
fdsys/pkg/FR-2015-09-18/pdf/2015- describes the opportunities for sponsors subject to review by the Office of
23389.pdf. of regenerative medicine therapies to Management and Budget (OMB) under
Docket: For access to the docket to interact with CBER review staff. the Paperwork Reduction Act of 1995
read background documents or the The draft guidance document
(44 U.S.C. 3501–3520). The collections
electronic and written/paper comments addresses regenerative medicine
of information in 21 CFR part 312 have
received, go to https:// therapies, which are defined in section
506(g)(8) of the Federal Food, Drug, and been approved under OMB control
www.regulations.gov and insert the number 0910–0014; the collections of
docket number, found in brackets in the Cosmetic Act (the FD&C Act), as
amended by the Cures Act, as including information regarding formal meetings
heading of this document, into the in ‘‘Guidance for Formal Meetings
‘‘Search’’ box and follow the prompts cell therapies, therapeutic tissue
engineering products, human cell and Between the FDA and Sponsors or
and/or go to the Dockets Management Applicants of PDUFA Products’’ have
Staff, 5630 Fishers Lane, Rm. 1061, tissue products, and combination
products using any such therapies or been approved under OMB control
Rockville, MD 20852. number 0910–0429; the collections of
You may submit comments on any products, except for those regulated
solely under section 361 of the Public information in 21 CFR part 601 have
guidance at any time (see 21 CFR been approved under OMB control
10.115(g)(5)). Health Service Act (PHS Act) (42 U.S.C.
264) and 21 CFR part 1271. Gene number 0910–0338; the collections of
Submit written requests for single information for expedited programs in
copies of the draft guidance to the Office therapies, including genetically
modified cells, that lead to a durable ‘‘Guidance for Industry: Expedited
of Communication, Outreach and Programs for Serious Conditions—Drugs
Development, Center for Biologics modification of cells or tissues can meet
the definition of a regenerative medicine and Biologics,’’ have been approved
Evaluation and Research (CBER), Food under OMB control number 0910–0765;
and Drug Administration, 10903 New therapy. Under section 506(g) of the
FD&C Act, RMAT designation is the collections of information in 21 CFR
Hampshire Ave., Bldg. 71, Rm. 3128, part 314 have been approved under
Silver Spring, MD 20993–0002. Send available for a drug (i.e., a human drug,
including a drug that is a biological OMB control number 0910–0001.
one self-addressed adhesive label to
assist the office in processing your product) that meets the definition of III. Electronic Access
requests. The draft guidance may also be regenerative medicine therapy; is
intended to treat, modify, reverse, or Persons with access to the Internet
obtained by mail by calling CBER at 1– may obtain the draft guidance at either
800–835–4709 or 240–402–8010. See cure a serious or life-threatening disease
or condition; and with respect to which https://www.fda.gov/
the SUPPLEMENTARY INFORMATION section BiologicsBloodVaccines/
for electronic access to the draft preliminary clinical evidence indicates
the potential to address unmet medical GuidanceCompliance
guidance document. RegulatoryInformation/Guidances/
needs for such disease or condition. A
FOR FURTHER INFORMATION CONTACT: default.htm or https://
combination product (biologic-device,
Tami Belouin, Center for Biologics biologic-drug, or biologic-device-drug) www.regulations.gov.
Evaluation and Research, Food and can be eligible for RMAT designation Dated: November 13, 2017.
Drug Administration, 10903 New when the biological product provides Anna K. Abram,
Hampshire Ave., Bldg. 71, Rm. 7301, the greatest contribution to the overall
Silver Spring, MD 20993–0002, 240– Deputy Commissioner for Policy, Planning,
intended therapeutic effects of the Legislation, and Analysis.
402–7911. combination product (i.e., the primary [FR Doc. 2017–24837 Filed 11–16–17; 8:45 am]
SUPPLEMENTARY INFORMATION: mode of action in the combination BILLING CODE 4164–01–P
I. Background product is conveyed by the biological
product component).
FDA is announcing the availability of Elsewhere in this issue of the Federal DEPARTMENT OF HEALTH AND
a draft document entitled ‘‘Expedited Register, FDA is announcing the HUMAN SERVICES
Programs for Regenerative Medicine availability of a document entitled
Therapies for Serious Conditions; Draft ‘‘Evaluation of Devices Used with Food and Drug Administration
Guidance for Industry.’’ The draft Regenerative Medicine Advanced
guidance document describes the Therapies; Draft Guidance for Industry.’’ [Docket No. FDA–2014–D–1167]
expedited programs available to This draft guidance is being issued
Controlled Correspondence Related to
sponsors of regenerative medicine consistent with FDA’s good guidance
Generic Drug Development; Draft
therapies for serious or life-threatening practices regulation (21 CFR 10.115).
Guidance for Industry; Availability;
diseases or conditions (referred to in the The draft guidance, when finalized, will
Correction
draft guidance as serious conditions), represent the current thinking of FDA
including those products designated as on ‘‘Expedited Programs for AGENCY: Food and Drug Administration,
RMATs; provides information about the Regenerative Medicine Therapies for HHS.
provisions in the 21st Century Cures Act Serious Conditions; Draft Guidance for
sradovich on DSK3GMQ082PROD with NOTICES
ACTION: Notice; correction.
(Cures Act) (Pub. L. 114–225) regarding Industry.’’ It does not establish any
the use of the accelerated approval rights for any person and is not binding SUMMARY: The Food and Drug
pathway for regenerative medicine on FDA or the public. You can use an Administration is correcting a notice
therapies that have been granted alternative approach if it satisfies the entitled ‘‘Controlled Correspondence
designation as an RMAT; describes how requirements of the applicable statutes Related to Generic Drug Development;
CBER will encourage flexibility in and regulations. This guidance is not Draft Guidance for Industry;
clinical trial design to facilitate the subject to Executive Order 12866. Availability’’ that appeared in the
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Document Created: 2017-11-17 05:14:47
Document Modified: 2017-11-17 05:14:47
| 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 February 15, 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 | Tami Belouin, 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 | 82 FR 54385 |
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