[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