[Federal Register Volume 89, Number 6 (Tuesday, January 9, 2024)]
[Notices]
[Pages 1101-1103]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-00217]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-N-4804]


Agency Information Collection Activities; Proposed Collection; 
Comment Request; Expedited Programs for Serious Conditions--Drugs and 
Biologics

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
an opportunity for public comment on the proposed collection of certain 
information by the Agency. Under the Paperwork Reduction Act of 1995 
(PRA), Federal Agencies are required to publish notice in the Federal 
Register concerning each proposed collection of information, including 
each proposed extension of an existing collection of information, and 
to allow 60 days for public comment in response to the notice. This 
notice solicits comments on information collection pertaining to 
``Expedited Programs for Serious Conditions--Drugs and Biologics.''

DATES: Submit either electronic or written comments on the collection 
of information by March 11, 2024.

ADDRESSES: You may submit comments as follows. Please note that late, 
untimely filed comments will not be considered. The https://www.regulations.gov electronic filing system will accept comments until 
11:59 p.m. Eastern Time at the end of March 11, 2024. Comments received 
by mail/hand delivery/courier (for written/paper submissions) will be 
considered timely if they are received on or before that date.

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-2023-N-4804 for ``Expedited Programs for Serious Conditions--Drugs 
and Biologics.'' 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, 240-402-7500.
     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.govinfo.gov/content/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, 240-402-7500.

FOR FURTHER INFORMATION CONTACT: Amber Sanford, Office of Operations, 
Food and Drug Administration, Three White Flint North, 10A-12M, 11601

[[Page 1102]]

Landsdown St., North Bethesda, MD 20852, 301-796-8867, 
[email protected].

SUPPLEMENTARY INFORMATION: Under the PRA (44 U.S.C. 3501-3521), Federal 
Agencies must obtain approval from the Office of Management and Budget 
(OMB) for each collection of information they conduct or sponsor. 
``Collection of information'' is defined in 44 U.S.C. 3502(3) and 5 CFR 
1320.3(c) and includes Agency requests or requirements that members of 
the public submit reports, keep records, or provide information to a 
third party. Section 3506(c)(2)(A) of the PRA (44 U.S.C. 3506(c)(2)(A)) 
requires Federal Agencies to provide a 60-day notice in the Federal 
Register concerning each proposed collection of information, including 
each proposed extension of an existing collection of information, 
before submitting the collection to OMB for approval. To comply with 
this requirement, FDA is publishing notice of the proposed collection 
of information set forth in this document.
    With respect to the following collection of information, FDA 
invites comments on these topics: (1) whether the proposed collection 
of information is necessary for the proper performance of FDA's 
functions, including whether the information will have practical 
utility; (2) the accuracy of FDA's estimate of the burden of the 
proposed collection of information, including the validity of the 
methodology and assumptions used; (3) ways to enhance the quality, 
utility, and clarity of the information to be collected; and (4) ways 
to minimize the burden of the collection of information on respondents, 
including through the use of automated collection techniques, when 
appropriate, and other forms of information technology.

Expedited Programs for Serious Conditions--Drugs and Biologics

OMB Control Number 0910-0765--Extension

    This information collection supports regulations governing FDA 
expedited programs for serious conditions. These provisions are set 
forth in 21 CFR part 312, subpart E and are intended to speed the 
availability of new therapies to patients with serious conditions, 
especially when there are no satisfactory alternative therapies, while 
preserving appropriate standards for safety and effectiveness. The 
regulations call for earlier attention to drugs that have promise in 
treating such conditions, including early consultation with FDA for 
sponsors of such products. Respondents to the information collection 
are sponsors of drug or biologic product applications submitted to FDA.
    To assist respondents with the information collection, we developed 
Agency guidance entitled ``Guidance for Industry Expedited Programs for 
Serious Conditions--Drugs and Biologics'' (May 2014). The guidance is a 
resource for information on FDA's policies and procedures related to 
the following expedited programs for serious conditions: (1) fast track 
designation, (2) breakthrough therapy designation, (3) accelerated 
approval, and (4) priority review designation, and describes threshold 
criteria generally applicable to expedited programs, including what is 
meant by serious condition, unmet medical need, and available therapy. 
The guidance addresses the applicability of expedited programs to rare 
diseases, clarification on available therapy, and additional detail on 
possible flexibility in manufacturing and product quality. It also 
clarifies the qualifying criteria for breakthrough therapy designation, 
provides examples of surrogate endpoints and intermediate clinical 
endpoints used to support accelerated approval, and priority review.
    In addition, we developed Agency guidance entitled ``Expedited 
Programs for Regenerative Medicine Therapies for Serious Conditions,'' 
(February 2019) describing the criteria for participation in the 
Regenerative Medicine Advanced Therapy (RMAT) program. The RMAT 
expedited program was approved as part of the 21st Century CURES Act, 
signed December 13, 2016. An RMAT product is intended to treat, modify, 
reverse, or cure serious or life-threatening diseases or conditions, 
and preliminary clinical evidence indicate that the drug has the 
potential to address unmet medical needs for such diseases or 
conditions. This is a Center Biologics Evaluation and Research (CBER) 
program and is included as an expedited program available for serious 
conditions.
    For a sponsor or applicant who seeks fast track, priority, 
breakthrough, RMAT or accelerated approval designation review, approval 
is required to submit a request showing that the drug product: (1) is 
intended for a serious or life-threatening condition and (2) has the 
potential to (a) address an unmet medical need, (b) demonstrate 
substantial improvement over available therapy, or (c) fill an unmet 
need to be approved based on a surrogate endpoint. We expect that most 
information to support a designation request will have been gathered 
under existing requirements for preparing an investigational new drug 
(IND), new drug application (NDA), or biologics license application 
(BLA). If such information has already been submitted to us, the 
information may be summarized in the designation request. A designation 
request should include, where applicable, additional information not 
specified elsewhere by statute or regulation. For example, additional 
information may be needed to show that a product has the potential to 
address an unmet medical need where an approved therapy exists for the 
serious or life-threatening condition to be treated. Such information 
may include clinical data, published reports, summaries of data and 
reports, and a list of references. The amount of information and 
discussion in a designation request should be sufficient to permit a 
reviewer to assess whether the criteria for fast track, priority, 
breakthrough, RMAT or accelerated approval designation have been met.
    After we make an expedited programs designation, a sponsor or 
applicant may submit a premeeting package that may include additional 
information supporting a request to participate in certain expedited 
programs. The premeeting package serves as background information for 
the meeting and should support the intended objectives of the meeting. 
As with the request for expedited programs designation, we expect that 
most sponsors or applicants will have gathered such information to meet 
existing requirements for preparing an IND, NDA, or BLA. These may 
include descriptions of clinical safety and efficacy trials not 
conducted under an IND (e.g., foreign studies) and information to 
support a request for accelerated approval. If such information has 
already been submitted to us, the information may be summarized in the 
premeeting package.
    The guidance documents are available on our website at www.fda.gov/regulatory-information/search-fda-guidance-documents and were issued 
consistent with our good guidance practice regulations in 21 CFR 
10.115, which provide for public comment at any time.
    We estimate the burden of this collection of information as 
follows:

[[Page 1103]]



                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                     Number of
            Activity                 Number of     responses per   Total annual   Average burden    Total hours
                                    respondents     respondent       responses     per response
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                                                      CDER
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Priority Review Designation                   81            1.53             124              30           3,720
 Requests (Expedited Programs
 for Serious Conditions Guidance
 (EPSC) Section VIII)...........
Breakthrough Therapy Designation              71            1.08              77              70           5,390
 Requests (EPSC Section VI).....
Fast Track Designation Requests              235            1.18             277              60          16,620
 (EPSC Section V)...............
Accelerated Approval Designation              26            1.27              33             100           3,300
 (EPSC Section VII).............
Premeeting Packages (21 CFR                  163            1.01             165             100          16,500
 312.82)........................
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    CDER Subtotal...............  ..............  ..............             676  ..............          45,530
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                                                      CBER
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Priority Review Designation                    8               1               8              30             240
 Request (EPSC Section VIII)....
Breakthrough Therapy Designation              15             1.1              17              70           1,190
 Request (EPSC Section VI)......
Fast Track Designation Requests               64             1.2              77              60           4,620
 (EPSC Section VII).............
RMAT Designation Requests                     33             1.1              36              60           2,160
 (Regenerative Medicine
 Therapies for Serious
 Conditions Guidance (RMAT)
 Section III)...................
Guidance p 6)...................
Premeeting Packages (RMAT                    146             1.9             277             100          27,700
 Section V).....................
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    CBER Subtotal...............  ..............  ..............             415  ..............          35,910
        Total...................  ..............  ..............           1,091  ..............          81,440
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

    Based on FY 2022 receipts, we estimate that for Center for Drug 
Evaluation and Research (CDER) products, 81 respondents will submit 124 
requests for priority review designation annually, and we assume 30 
hours are needed to prepare such a request. We estimate 71 respondents 
will submit 77 requests for breakthrough designation annually, and we 
assume 70 hours are needed to prepare such a request. We estimate that 
235 respondents will submit 277 requests for fast-track designation 
requests annually, and we assume 60 hours are required to prepare such 
a request. We estimate 26 respondents will submit 33 accelerated 
approval designation requests annually and we assume 100 hours are 
required to prepare such a request. Finally, CDER received 165 pre-
meeting package submissions from 163 respondents. We assume 100 hours 
are needed to prepare a pre-meeting package.
    Similarly, also based on FY 2022 receipts, we estimate that for 
CBER products, 8 applicants will submit 8 requests for priority review 
designation annually, and we assume 30 hours are required to prepare 
such a request. We estimate 15 respondents will submit 17 requests for 
breakthrough designation annually, and we assume 70 hours are needed to 
prepare such a request. We estimate that 64 respondents will submit 78 
requests for fast-track designation annually, and we assume 60 hours is 
required to prepare such a request. We also estimate 33 respondents 
will submit 35 requests for RMAT designation annually and assume that 
60 hours are needed to prepare each RMAT designation request. Finally, 
CBER received 283 pre-meeting package submissions from 146 respondents. 
We assume 100 hours are needed to prepare a pre-meeting package.
    Based on a review of the information collection since our last 
request for OMB approval, we have increased our burden estimate by 143 
responses and 10,350 hours to reflect actual submissions we have 
received. We attribute these changes to increased interest in the 
expedited programs, new expedited programs, and an increase in the 
number of submissions we received over the last few years.

    Dated: January 4, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-00217 Filed 1-8-24; 8:45 am]
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