83 FR 16868 - Pilot Meetings Program for Model-Informed Drug Development Approaches

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 74 (April 17, 2018)

Page Range		16868-16870
FR Document		2018-08010

The sixth iteration of the Prescription Drug User Fee Act (PDUFA VI), incorporated as part of the FDA Reauthorization Act of 2017 (FDARA), highlights the goal of advancing model-informed drug development (MIDD). The Food and Drug Administration (FDA or Agency) is announcing a pilot program that affords sponsors or applicants who are selected for participation the opportunity to meet with Agency staff to discuss MIDD approaches in medical product development. Meetings under the pilot program will be conducted by FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) during fiscal years 2018 to 2022. This pilot program is being conducted to fulfill FDA's performance commitment under PDUFA VI. For this pilot program, MIDD is defined as the application of exposure- based, biological, and/or statistical models derived from preclinical and clinical data sources to address drug development and/or regulatory issues (see Supplementary Information, I. Background, and II. Eligibility and Selection for Participation of this notice). For each approved proposal, the pilot program consists of two meetings between sponsors or applicants and the relevant center and will provide an opportunity for drug developers and FDA to discuss the application of MIDD approaches to the development and regulatory evaluation of medical products in development.

Federal Register, Volume 83 Issue 74 (Tuesday, April 17, 2018)

[Federal Register Volume 83, Number 74 (Tuesday, April 17, 2018)]
[Notices]
[Pages 16868-16870]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-08010]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-1203]


Pilot Meetings Program for Model-Informed Drug Development 
Approaches

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The sixth iteration of the Prescription Drug User Fee Act 
(PDUFA VI), incorporated as part of the FDA Reauthorization Act of 2017 
(FDARA), highlights the goal of advancing model-informed drug 
development (MIDD). The Food and Drug Administration (FDA or Agency) is 
announcing a pilot program that affords sponsors or applicants who are 
selected for participation the opportunity to meet with Agency staff to 
discuss MIDD approaches in medical product development. Meetings under 
the pilot program will be conducted by FDA's Center for Drug Evaluation 
and Research (CDER) and Center for Biologics Evaluation and Research 
(CBER) during fiscal years 2018 to 2022. This pilot program is being 
conducted to fulfill FDA's performance commitment under PDUFA VI. For 
this pilot program, MIDD is defined as the application of exposure-
based, biological, and/or statistical models derived from preclinical 
and clinical data sources to address drug development and/or regulatory 
issues (see Supplementary Information, I. Background, and II. 
Eligibility and Selection for Participation of this notice). For each 
approved proposal, the pilot program consists of two meetings between 
sponsors or applicants and the relevant center and will provide an 
opportunity for drug developers and FDA to discuss the application of 
MIDD approaches to the development and regulatory evaluation of medical 
products in development.

DATES: FDA will accept requests to participate in the program on a 
continuous basis beginning on April 17, 2018 through June 15, 2022. See 
section III of this notice for instructions about how to request 
participation in the pilot program. Meeting-granted and -denied 
decisions will be made the last 2 weeks of each quarter of the fiscal 
year based on submissions received to date. Requesters will receive a 
meeting-granted or -denied notification the first week of the new 
quarter.
    The pilot program meetings will begin in Q4 of FY 2018 (July 1-
September 30, 2018), and run through Q4 of FY 2022 (September 30, 
2022). Proposals not selected for a given quarter will be so notified 
by the Agency. Sponsors who are not chosen to participate in the pilot 
program may seek Agency interaction through existing channels (e.g., 
Type C meeting requests, critical path innovation meetings).

ADDRESSES: Comments about this pilot program can be submitted until May 
17, 2018. You may submit comments about the MIDD pilot meetings program 
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-N-1203 for ``Pilot Meetings Program for Model-Informed Drug 
Development Approaches.'' 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

[[Page 16869]]

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.

FOR FURTHER INFORMATION CONTACT: 
    CDER: Yvonne Knight, Center for Drug Evaluation and Research, Food 
and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 2142, 
Silver Spring, MD 20993-0002, 301-796-2133, [email protected], 
with the subject line ``MIDD Pilot Meetings Program for CDER.''
    CBER: Jason Claeys, Center for Biologics Evaluation and Research, 
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 
1223, Silver Spring, MD 20993-0002, 240-402-8589, 
[email protected], with the subject line ``MIDD Pilot Meetings 
Program for CBER.''

SUPPLEMENTARY INFORMATION: 

I. Background

    Under FDARA FDA agreed, in accordance with the ``PDUFA 
Reauthorization Performance Goals and Procedures Fiscal Years 2018 
Through 2022: I. Ensuring the Effectiveness of the Human Drug Review 
Program, Part J. Enhancing Regulatory Decision Tools to Support Drug 
Development and Review'' to provide information on how a sponsor can 
apply to participate in a pilot meetings program with FDA to discuss 
MIDD approaches (https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf).
    FDA is announcing this pilot meetings program to satisfy the above-
mentioned commitment and to facilitate MIDD approaches. This excludes 
statistical designs involving complex adaptations, Bayesian methods, or 
other features requiring computer simulations to determine the 
operating characteristics of a confirmatory clinical trial. MIDD 
approaches use a variety of quantitative methods to help balance the 
risks and benefits of drug products in development. When successfully 
applied, MIDD approaches can improve clinical trial efficiency, 
increase the probability of regulatory success, and optimize drug 
dosing/therapeutic individualization in the absence of dedicated 
trials.
    The goal of the early meeting discussions granted under this pilot 
program is to provide advice on how specific, proposed MIDD approaches 
can be used in a specific drug development program. FDA has committed 
to accepting two to four meeting requests quarterly each fiscal year. 
The meetings granted will include an initial and followup meeting on 
the same drug development issues within the span of approximately 120 
days.
    The listed eligibility factors and procedures outlined in this 
Federal Register notice reflect the current thinking at the time of 
publication. Processes may be revised and will be communicated as this 
pilot program evolves. The most current pilot program eligibility 
factors and procedures may be found on the MIDD Pilot Program website: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm600311.htm.

II. Eligibility and Selection for Participation in the MIDD Pilot 
Program

    The requester should be a drug/biologics development company 
(interested consortia or software/device developer should come in 
partnership with a drug development company) and have an 
investigational new drug application (IND) or pre-IND (PIND) number for 
the relevant program. Recognizing that FDA will learn both from the 
number and types of submissions received for consideration into the 
pilot program, FDA welcomes submissions related to any relevant MIDD 
topics. However, given that the Agency expects to grant two to four 
meeting requests per quarter as part of the pilot program, the Agency 
will initially prioritize selecting requests that focus on:
     Dose selection or estimation (e.g., for dose/dosing 
regimen selection or refinement).
     Clinical trial simulation (e.g., based on drug-trial-
disease models to inform the duration of a trial, select appropriate 
response measures, predict outcomes).
     Predictive or mechanistic safety evaluation (e.g., use of 
systems pharmacology/mechanistic models for predicting safety or 
identifying critical biomarkers of interest).

III. Procedures and Submission Information

A. General Information

    The MIDD pilot program will be jointly administered by CDER's 
Office of Clinical Pharmacology, in the Office of Translational 
Sciences, which is the point of contact for all communications for CDER 
products, and CBER's Office of Biostatistics and Epidemiology, which is 
the point of contact for all communications for CBER products.

B. How To Submit a Meeting Request and Meeting Package

    Meeting requests should be submitted electronically to the relevant 
application (i.e., PIND, IND) with ``MIDD Pilot Program Meeting Request 
for CDER'' (CDER applications) or ``MIDD Pilot Program Meeting Request 
for CBER'' (CBER applications) in the subject line. Information about 
providing regulatory submissions in electronic format is available at: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/FormsSubmissionRequirements/%20ElectronicSubmissions/ucm153574.htm.

C. Content and Format of the Meeting Request

    Include the following information in the meeting request (no more 
than three to four pages):
    1. Product name.
    2. Application number.
    3. Chemical name and structure.
    4. Proposed indication(s) or context of product development.
    5. Brief statement of the purpose and objectives of the meeting. 
The statement should include a brief background of the MIDD issues 
underlying the agenda.
    6. MIDD approach(es) considered for the product under development 
and how MIDD can assess uncertainties about issues (e.g., dosing, 
duration, patient selection) in a way that can inform regulatory 
decision-making.
    7. List of issues for discussion with the Agency about the specific 
MIDD proposed approach for the applicable drug development program.

D. Content and Format of the Meeting Information Package

    Sponsors or applicants whose meeting requests are granted as part 
of the pilot program should submit a meeting information package 
electronically with ``MIDD Pilot Program Meeting Package for CDER'' 
(CDER applications) or ``MIDD Pilot Program Meeting Package for CBER'' 
(CBER applications) in the subject line no later than 30 days before 
each (initial and followup) meeting. This meeting package should 
include the following information:

[[Page 16870]]

    1. Product name.
    2. Application number.
    3. Chemical name and structure.
    4. Proposed indication(s) or context of product development.
    5. Background section that includes a brief history of the 
development program and the events leading up to the meeting, and the 
status of product development.
    6. Proposed agenda, including estimated times needed for discussion 
of each agenda item.
    7. List of questions for discussion with a brief summary for each 
question to explain the need or context for the question.
    8. Drug development issue (e.g., dosing, clinical trial design, 
safety prediction), including the proposed MIDD approach to the 
solution, information to support discussion (e.g., a description of the 
data used for developing the models, model development, simulation 
plan, results), and how the Agency can help guide any next steps 
relative to the regulatory decision making process, which should be 
summarized and clearly articulated with any supporting data imperative 
to the discussion.

E. Meeting Summaries

    A meeting summary will be sent to the requester within 60 days of 
each meeting.

IV. Paperwork Reduction Act of 1995

    This notice refers to 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 collection 
of information resulting from formal meetings between sponsors or 
applicants and FDA has been approved under OMB control number 0910-
0429. The collection of information in 21 CFR part 312 (INDs) has been 
approved under OMB control number 0910-0014.

    Dated: April 12, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-08010 Filed 4-16-18; 8:45 am]
 BILLING CODE 4164-01-P

16870 Federal Register / Vol. 83, No. 74 / Tuesday, April 17, 2018 / Notices

1. Product name. DEPARTMENT OF HEALTH AND Service Act (PHS Act) (42 U.S.C.
2. Application number. HUMAN SERVICES 262(a)). Section 351(a) requires that
manufacturers of biological products,
3. Chemical name and structure. Food and Drug Administration which include blood and blood
4. Proposed indication(s) or context of [Docket No. FDA–2017–N–6931] components intended for further
product development. manufacturing into products, have a
5. Background section that includes a Agency Information Collection license, issued upon a demonstration
brief history of the development Activities; Submission for Office of that the product is safe, pure, and potent
Management and Budget Review; and that the manufacturing
program and the events leading up to
Comment Request; Current Good establishment meets all applicable
the meeting, and the status of product
Manufacturing Practices and Related standards, including those prescribed in
development. the FDA regulations designed to ensure
Regulations for Blood and Blood
6. Proposed agenda, including Components; and Requirements for the continued safety, purity, and
estimated times needed for discussion Donation Testing, Donor Notification, potency of the product. In addition,
of each agenda item. and ‘‘Lookback’’ under section 361 of the PHS Act (42
7. List of questions for discussion U.S.C. 264), by delegation from the
AGENCY: Food and Drug Administration, Secretary of Health and Human
with a brief summary for each question
HHS. Services, FDA may make and enforce
to explain the need or context for the
ACTION: Notice. regulations necessary to prevent the
question.
introduction, transmission, or spread of
8. Drug development issue (e.g., SUMMARY: The Food and Drug communicable diseases from foreign
dosing, clinical trial design, safety Administration (FDA, we, or Agency) is countries into the States or possessions,
prediction), including the proposed announcing that a proposed collection or from one State or possession into any
MIDD approach to the solution, of information has been submitted to the other State or possession.
information to support discussion (e.g., Office of Management and Budget Section 351(j) of the PHS Act states
a description of the data used for (OMB) for review and clearance under that the Federal Food, Drug, and
developing the models, model the Paperwork Reduction Act of 1995. Cosmetic Act (FD&C Act) also applies to
development, simulation plan, results), DATES: Fax written comments on the biological products. Blood and blood
and how the Agency can help guide any collection of information by May 17, components for transfusion or for
next steps relative to the regulatory 2018. further manufacturing into products are
decision making process, which should drugs, as that term is defined in section
ADDRESSES: To ensure that comments on
201(g)(1) of the FD&C Act (21 U.S.C.
be summarized and clearly articulated the information collection are received,
321(g)(1)). Because blood and blood
with any supporting data imperative to OMB recommends that written
components are drugs under the FD&C
the discussion. comments be faxed to the Office of
Act, blood and plasma establishments
Information and Regulatory Affairs,
E. Meeting Summaries must comply with the provisions and
OMB, Attn: FDA Desk Officer, Fax: 202–
related regulatory scheme of the FD&C
A meeting summary will be sent to 395–7285, or emailed to oira_
Act. For example, under section 501 of
the requester within 60 days of each submission@omb.eop.gov. All
the FD&C Act (21 U.S.C. 351), drugs are
comments should be identified with the deemed ‘‘adulterated’’ if the methods
meeting.
OMB control number 0910–0116. Also used in their manufacturing, processing,
IV. Paperwork Reduction Act of 1995 include the FDA docket number found packing, or holding do not conform to
in brackets in the heading of this current good manufacturing practice
This notice refers to collections of document.
information that are subject to review by (CGMP) and related regulations.
FOR FURTHER INFORMATION CONTACT: Ila The CGMP regulations (part 606) (21
the Office of Management and Budget
S. Mizrachi, Office of Operations, Food CFR part 606) and related regulations
(OMB) under the Paperwork Reduction implement FDA’s statutory authority to
and Drug Administration, Three White
Act of 1995 (44 U.S.C. 3501–3520). The Flint North, 10A–12M, 11601 ensure the safety, purity, and potency of
collection of information resulting from Landsdown St., North Bethesda, MD blood and blood components. The
formal meetings between sponsors or 20852, 301–796–7726, PRAStaff@ public health objective in testing human
applicants and FDA has been approved fda.hhs.gov. blood donations for evidence of relevant
under OMB control number 0910–0429. transfusion-transmitted infections and
The collection of information in 21 CFR SUPPLEMENTARY INFORMATION: In
in notifying donors is to prevent the
part 312 (INDs) has been approved compliance with 44 U.S.C. 3507, FDA
transmission of relevant transfusion-
has submitted the following proposed
under OMB control number 0910–0014. transmitted infections. For example, the
collection of information to OMB for
Dated: April 12, 2018. ‘‘lookback’’ requirements are intended
review and clearance.
to help ensure the continued safety of
Leslie Kux,
Current Good Manufacturing Practices the blood supply by providing necessary
Associate Commissioner for Policy. and Related Regulations for Blood and information to consignees of blood and
[FR Doc. 2018–08010 Filed 4–16–18; 8:45 am] Blood Components; and Requirements blood components and appropriate
BILLING CODE 4164–01–P for Donation Testing, Donor notification of recipients of blood
Notification, and ‘‘Lookback’’ components that are at increased risk for
daltland on DSKBBV9HB2PROD with NOTICES

transmitting human immunodeficiency
OMB Control Number 0910–0116— virus (HIV) or hepatitis C virus (HCV)
Extension infection.
All blood and blood components The information collection
introduced or delivered for introduction requirements in the CGMP, donation
into interstate commerce are subject to testing, donor notification, and
section 351(a) of the Public Health ‘‘lookback’’ regulations provide FDA

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Document Created: 2018-04-17 02:52:59
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Category		Regulatory Information
Collection		Federal Register
sudoc Class		AE 2.7: GS 4.107: AE 2.106:
Publisher		Office of the Federal Register, National Archives and Records Administration
Section		Notices
Action		Notice.
Dates		FDA will accept requests to participate in the program on a continuous basis beginning on April 17, 2018 through June 15, 2022. See section III of this notice for instructions about how to request participation in the pilot program. Meeting-granted and -denied decisions will be made the last 2 weeks of each quarter of the fiscal year based on submissions received to date. Requesters will receive a meeting-granted or -denied notification the first week of the new quarter.
Contact		CDER: Yvonne Knight, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 2142, Silver Spring, MD 20993-0002, 301-796-2133, [email protected], with the subject line ``MIDD Pilot Meetings Program for CDER.''
FR Citation		83 FR 16868

83 FR 16868 - Pilot Meetings Program for Model-Informed Drug Development Approaches

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 83, Issue 74 (April 17, 2018)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration