[Federal Register Volume 83, Number 235 (Friday, December 7, 2018)]
[Notices]
[Pages 63178-63179]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-26546]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-4000]


Framework for a Real-World Evidence Program; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is 
establishing a public docket to collect comments on a framework created 
by the Center for Drug Evaluation and Research and the Center for 
Biologic Evaluation and Research for implementing a program to evaluate 
the potential use of real-world evidence (RWE) in regulatory decision 
making. This framework is entitled ``Framework for the Real-World 
Evidence Program.'' The 21st Century Cures Act (Cures Act) was enacted 
on December 13, 2016, and requires that FDA establish a framework for 
implementing a program to evaluate the potential use of RWE to help 
support the approval of a new indication for a drug approved under the 
Federal Food, Drug, and Cosmetic Act (FD&C Act) and to help support or 
satisfy postapproval study requirements. FDA has created this framework 
to satisfy the Cures Act mandate and is establishing a docket to 
receive public comments.

DATES: Submit either electronic or written comments on the draft 
document by February 5, 2019 to ensure that the Agency considers your 
comment before it begins work to implement the program.

ADDRESSES: You may submit comments 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:

[[Page 63179]]

     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-4000 for ``Framework for a Real-World Evidence Program; 
Availability.'' 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.

FOR FURTHER INFORMATION CONTACT: Dianne Paraoan, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 3326, Silver Spring, MD 20993-0002, 301-
796-2500, [email protected]; 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, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is establishing a public docket to collect comments on its 
``Framework for a Real-World Evidence Program.'' Section 3022 of the 
Cures Act amended the FD&C Act to add section 505F, Utilizing real 
world evidence (21 U.S.C. 355g). This section requires the 
establishment of a program to evaluate the potential use of RWE to help 
support the approval of a new indication for a drug approved under 
section 505(c) of the FD&C Act (21 U.S.C. 355(c)) and to help to 
support or satisfy postapproval study requirements. This section also 
requires FDA publish a framework for that program. In addition to drug 
and biological products approved under section 505(c) of the FD&C Act, 
FDA is also applying this framework to biological products licensed 
under section 351 of the Public Health Service Act (42 U.S.C. 262).
    The statute directs that the framework for the RWE program include 
information describing sources of RWE, gaps in data collection 
activities, standards and methodologies for collecting and analyzing 
RWE, and priority areas, remaining challenges, and potential pilot 
opportunities to address the overarching Cures Act requirements. To 
help meet a requirement in the Cures Act for consultation in developing 
the program framework, on September 13, 2017, through its cooperative 
agreement with the Duke Margolis Center for Health Policy, FDA convened 
a public meeting that explored the use of RWE for regulatory decisions. 
Representatives from industry, academia, and patient advocacy groups 
discussed, among other things, opportunities and challenges associated 
with applying real-world data and RWE, the evidence derived from that 
data, to demonstrate product effectiveness, including data acquisition, 
study design, and analytic methods necessary to establish causal 
inference. The workshop helped to inform FDA's RWE framework. FDA will 
continue to consult stakeholders through public-private partnerships, 
public workshops, and demonstration projects as it implements its RWE 
program.

II. Electronic Access

    Persons with access to the internet may obtain the ``Framework for 
the Real-World Evidence Program'' at https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/21stCenturyCuresAct/ucm562475.htm.

    Dated: November 30, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-26546 Filed 12-6-18; 8:45 am]
 BILLING CODE 4164-01-P