[Federal Register Volume 84, Number 155 (Monday, August 12, 2019)]
[Notices]
[Pages 39856-39858]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-17209]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2019-N-3453]
Promoting Effective Drug Development Programs: Opportunities and
Priorities for the Food and Drug Administration's Office of New Drugs;
Public Meeting; Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public meeting; request for comments.
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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is
holding a public meeting on November 7, 2019 entitled ``Promoting
Effective Drug Development Programs: Opportunities and Priorities for
FDA's Office of New Drugs.'' The purpose of the public meeting is to
solicit specific, actionable policy suggestions that could be
implemented in the near-term by the review staff of the Center for Drug
Evaluation and Research's (CDER's) Office of New Drugs to promote
effective drug development programs without compromising our regulatory
standards for the assessment of safety and effectiveness.
DATES: The public meeting will be held on November 7, 2019, from 9 a.m.
to 5 p.m. The public meeting may be extended or may end early depending
on the level of public participation. Persons can attend the event in-
person or via webcast. In-person attendees can also request to give a
formal presentation as part of the registration process. See the
SUPPLEMENTARY INFORMATION section for registration date and
information. Electronic or written comments will be accepted after the
public hearing until January 7, 2020.
ADDRESSES: The public meeting will be held at the FDA White Oak Campus,
10903 New Hampshire Ave., Building 31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993-0002. Entrance for public meeting
participants (non-FDA employees) is through Building 1, where routine
security check procedures will be performed. For parking and security
information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
You may submit comments as follows. Please note that late, untimely
filed comments will not be considered. Electronic comments must be
submitted on or before January 7, 2020. The https://www.regulations.gov
electronic filing system will accept comments until 11:59 p.m. Eastern
Time at the end of January 7, 2020. Comments received by mail/hand
delivery/courier (for written/paper submissions) will be considered
timely if they are postmarked or the delivery service acceptance
receipt is 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-2019-N-3453 for ``Promoting Effective Drug Development Programs:
Opportunities and Priorities for FDA's Office of New Drugs.'' 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.
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/
[[Page 39857]]
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: Eithu Lwin, Center for Drug Evaluation
and Research, Food and Drug Administration, 10903 New Hampshire Ave.,
Bldg. 22, Rm. 6236, Silver Spring, MD 20993, 301-796-0728,
[email protected].
SUPPLEMENTARY INFORMATION:
I. Background
FDA regulates drugs, including those that are licensed as
biological products, under the Federal Food, Drug, and Cosmetic Act
(FD&C Act), the Public Health Service Act (PHS Act), and relevant
implementing regulations to promote and protect the public health.
The central mission of CDER's Office of New Drugs (OND) is the
proper and, where appropriate, flexible implementation of review
authorities to ensure that safe and effective drugs and biologics are
available to the American people. Rapid scientific and technological
advances have enhanced our understanding of disease pathologies and, in
many cases, have identified potential actionable targets that translate
into drug development programs with an increased emphasis on precision
medicine, rare diseases, and defined subsets of common diseases. FDA
can help to ensure these innovations reach patients through the
development and dissemination of clear policies regarding our
expectations and standards.
FDA is engaged in multiple, high-priority policy initiatives to
facilitate effective drug development. Effective drug development
programs leverage the best available scientific knowledge to
characterize the benefits and risks of a potential product and generate
the data necessary to support product approval. Current FDA endeavors
in this area include evaluating the potential use of real-world
evidence in regulatory decision-making and the incorporation of the
patient perspective into drug development and review. FDA has
previously released comprehensive plans or frameworks for the
advancement of these efforts; therefore, these topics are not the focus
of this meeting. Instead, stakeholders are encouraged to provide input
on these initiatives at topic-specific public meetings or in response
to draft guidance.
Consistent with FDA's broader initiatives and modernization
efforts, the clinical and scientific leaders of OND are seeking
suggestions on where we can provide regulatory clarity to promote
innovative and effective drug development across multiple therapeutic
areas. FDA will hold a public meeting on November 7, 2019, from 9 a.m.
to 5 p.m., to solicit input from stakeholders regarding where the
Office of New Drugs can focus policy priorities while those broader
Agency- and Center-wide efforts, described above, continue. In
particular, OND welcomes specific policy suggestions that can be
implemented in the near-term.
The format of the meeting involves presentations from the public to
a panel of leaders from OND review divisions. The Agency will not be
inviting specific presenters; rather, with this document, FDA is
soliciting presentations from interested stakeholders. FDA also invites
interested stakeholders to submit written comments to the docket on the
topics described below.
II. Topics for Discussion at the Public Meeting
FDA is soliciting specific, actionable policy suggestions that
could be implemented in the near-term by the review staff of CDER's
Office of New Drugs to promote effective drug development programs
without compromising our regulatory standards for the assessment of
safety and effectiveness.
The Agency welcomes any relevant information that stakeholders wish
to share at the meeting or in a submission to the docket, but we
emphasize that the focus of this meeting is to seek input that is
distinct from parallel, topic-specific initiatives related to real-
world evidence and patient-focused drug development. Furthermore, to
best inform policy priorities, we anticipate that the most informative
suggestions would not be specific to a therapeutic area or disease but
rather apply across multiple therapeutic areas or diseases. We are
particularly interested in the topics that follow.
1. We are interested in input from stakeholders about where OND can
provide additional guidance or prioritize additional scientific
discussion in the near-term to improve clarity and encourage effective
drug development. Given that OND's portfolio includes a diverse
spectrum of drugs and diseases, such input should focus on specific
policy needs for various clinical areas linked by a shared therapeutic
context (e.g., drugs intended to treat serious, life-threatening rare
diseases; non-serious, self-limited conditions; etc.), rather than
focusing on any specific disease or condition.
2. Over the past decade, advances in scientific knowledge have led
to unprecedented targeting of drugs to the underlying genetic or
molecular pathophysiology of a disease. For many diseases, however, the
current state of knowledge does not provide opportunities for such
precise targeting, but patients living with these diseases require
therapeutic innovation as well. Recognizing that each disease has
unique considerations, we are interested in specific suggestions for
guidance or policy development that OND could undertake to facilitate
drug development for diseases not currently amenable to targeted
therapies.
3. Some therapeutic areas, particularly those that include serious
and life-threatening diseases, have begun to implement novel trial
designs, such as the use of master protocols to study multiple
therapies and/or multiple diseases under a common infrastructure. We
are interested in stakeholders' views regarding the advantages and
disadvantages of extending these approaches to additional therapeutic
areas, and what guidance development would be most useful.
4. FDA has published many guidances intended to explain the
Agency's current thinking regarding drug development topics that are
not specific to a particular disease or indication. If stakeholders
believe that OND review divisions are implementing these guidances in
different ways, which are not explained by case-specific features, this
may reflect a need for guidance revision or additional policy
development. We are interested in hearing specific recommendations for
topics where further clarity of the Agency's current thinking may be
warranted.
5. Innovative approaches can bring additional uncertainty to drug
development, since the advantages and disadvantages of the approaches
may not yet be fully understood by either the Agency or sponsors
because of their novelty. Sometimes, a well-understood development
pathway may be chosen solely because of existing precedents in the
therapeutic area. We would like to hear how OND can promote effective
drug development programs when this tension exists.
[[Page 39858]]
III. Participating in the Public Meeting
Registration: Persons interested in attending this public meeting
must register online at https://promotingeffectivedrugdevelopmentprograms.eventbrite.com by midnight on
October 10, 2019. Please provide complete contact information for each
attendee, including name, title, affiliation, address, email, and
telephone. Please also indicate whether attendance will be by webcast
or in person. Early registration is recommended because seating is
limited; therefore, FDA may limit the number of participants from each
organization. If registration reaches maximum capacity, FDA will post a
notice closing registration at https://www.fda.gov.
If you need special accommodations due to a disability, please
contact Eithu Lwin (see FOR FURTHER INFORMATION CONTACT) no later than
October 30, 2019.
Requests for Oral Presentations: During online registration you may
indicate if you wish to present. To facilitate agenda development,
registrants requesting to present will be contacted to provide
information regarding which topics they intend to address and the title
of their presentation. We will do our best to accommodate requests to
present. Individuals and organizations with common interests are urged
to consolidate or coordinate their presentations, and request time for
a joint presentation, or submit requests for designated representatives
to participate. All requests to make oral presentations must be
received by the close of registration on October 10, 2019.
Following the close of registration, we will determine the amount
of time allotted to each presenter and the approximate time each oral
presentation is to begin, and we will select and notify participants by
October 24, 2019. If selected for presentation, registered presenters
planning to use an electronic slide deck should submit an electronic
copy of their presentation (PowerPoint or PDF), to
[email protected] with the subject line ``Promoting
Effective Drug Development Programs: Opportunities and Priorities for
FDA's Office of New Drugs'' on or before October 31, 2019. If
presenters choose not to use a slide deck, they are requested to submit
a single slide with their name, affiliation, title of presentation, and
contact information. Persons registered to present are encouraged to
arrive at the meeting room early and check in at the onsite
registration table to confirm their designated presentation time. No
commercial or promotional material will be permitted to be presented or
distributed at the public meeting.
Streaming Webcast of the Public Meeting: This public meeting will
also be webcast. To join the meeting via the Webcast, visit https://collaboration.fda.gov/ond110719/.
If you have never attended a Connect Pro event before, test your
connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a quick overview of the Connect Pro program,
visit https://www.adobe.com/go/connectpro_overview. FDA has verified
the website addresses in this document, as of the date this document
publishes in the Federal Register, but websites are subject to change
over time.
Transcripts: Please be advised that as soon as a transcript of the
public meeting is available, it will be accessible at https://www.regulations.gov. It may be viewed at the Dockets Management Staff
(see ADDRESSES). A link to the transcript will also be available on the
Agency's website at https://www.fda.gov.
Dated: August 7, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-17209 Filed 8-9-19; 8:45 am]
BILLING CODE 4164-01-P