[Federal Register Volume 83, Number 39 (Tuesday, February 27, 2018)]
[Notices]
[Pages 8485-8487]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-03961]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-0663]


Tissue Agnostic Therapies in Oncology: Regulatory Considerations 
for Orphan Drug Designation; Public Workshop; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop; request for comments.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA or the Agency) is 
announcing the following public workshop entitled ``Tissue Agnostic 
Therapies in Oncology: Regulatory Considerations for Orphan Drug 
Designation.'' The purpose of the public workshop is to discuss factors 
FDA should consider when evaluating drugs for orphan designation that 
treat a tissue agnostic disease or condition in oncology, and 
additional factors related to orphan exclusivity FDA should consider 
when approving a product with a tissue agnostic indication.

DATES: The public workshop will be held on May 9, 2018, from 9 a.m. to 
5 p.m. The public workshop may be extended or may end early depending 
on the level of public participation. Submit either electronic or 
written comments on this public workshop by June 8, 2018. See the 
SUPPLEMENTARY INFORMATION section for registration date and 
information.

ADDRESSES: The public workshop will be held at the FDA White Oak 
Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the 
Great Room (Rm. 1503, Section A), Silver Spring, MD 20993-0002. 
Entrance for the public workshop 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 June 8, 2018. The https://www.regulations.gov 
electronic filing system will accept comments until midnight Eastern 
Time at the end of June 8, 2018. 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

[[Page 8486]]

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-0663 for ``Tissue Agnostic Therapies in Oncology: Regulatory 
Considerations for Orphan Drug Designation; Public Workshop; Request 
for Comments.'' 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/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: Nicole Wolanski, Office of Orphan 
Products Development, Food and Drug Administration, 10903 New Hampshire 
Ave., Bldg. 32, Rm. 5210, Silver Spring, MD 20933, 301-796-6570, 
[email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    The combination of government incentives, scientific advances, and 
the promise of commercial opportunity has fueled extraordinary 
investment in orphan drugs. Since the Orphan Drug Act was first passed 
in 1983, over 650 rare disease indications for drugs and biologics have 
been developed and approved for marketing. In fact, rare disease drug 
approvals have accounted for approximately 40 percent of the new 
molecular entities and therapeutic biologic products in the Center for 
Drug Evaluation and Research for the last several years.
    Not only have we seen tremendous growth in the development of 
products for rare diseases, but the very landscape of rare disease 
product development is changing, with an increase in the development of 
targeted therapies, more interest in the development of biologics 
(including gene therapies), and tremendous growth in the oncology 
space. For example, in 2017 alone, FDA granted its first tissue 
agnostic approval (pembrolizumab for patients with unresectable or 
metastatic, microsatellite instability-high (MSI-H) or mismatch repair 
deficient (dMMR) solid tumors) and first tissue agnostic orphan drug 
designations (larotrectinib and entrectinib, each for the treatment of 
solid tumors with NTRK-fusion proteins). FDA also approved the first 
cell-based gene therapy, KYMRIAH, for use in treating a rare pediatric 
cancer.
    As advancements in genomics and precision medicine continue, FDA 
has been taking these new developments into account as it considers 
what constitutes a ``disease or condition.'' For example, one question 
that has already arisen in oncology is whether a disease should be 
defined in a tissue/organ-specific or a tissue agnostic manner. Because 
the continued development of targeted therapies for molecularly defined 
groups has the potential to alter the landscape of orphan drug 
development, FDA is holding the public workshop to obtain input on the 
complex scientific and regulatory issues surrounding molecularly 
targeted drugs and biologics in oncology and the appropriate 
application of orphan drug incentives in that paradigm. This discussion 
will inform how the Agency can incorporate the latest science and drug 
development trends into the implementation of the Orphan Drug Act, all 
while continuing to reflect the goals intended by Congress.

II. Topics for Discussion at the Public Workshop

    This public workshop will consist of both presentations and 
interactive panel discussions. The presentations will provide 
information to outline the goals of the workshop and help promote 
interactive discussions. Following the presentations, there will be a 
moderated discussion where speakers and additional panelists will be 
asked to provide their individual perspectives. The presentations and 
discussions will focus on several related topics. Topics will involve 
discussion of and seek input on factors FDA should consider when 
evaluating drugs for orphan designation that treat a tissue agnostic 
disease or condition in oncology and additional factors related to 
orphan exclusivity to consider when approving a product with a tissue 
agnostic indication. A detailed agenda will be posted on the following 
website in advance of the workshop: https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm592778.htm.

III. Participating in the Public Workshop

    Registration: To register for the public workshop, please visit the 
following website by April 25, 2018: https://

[[Page 8487]]

www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm592778.htm. 
Please provide complete contact information for each attendee, 
including name, title, affiliation, address, email, and telephone.
    Registration is free and based on space availability, with priority 
given to early registrants. Persons interested in attending this public 
workshop must register by April 25, 2018, 5 p.m. Eastern Time. Early 
registration is recommended because seating is limited; therefore, FDA 
may limit the number of participants from each organization. 
Registrants will receive confirmation when their registration has been 
received. If time and space permit, onsite registration on the day of 
the public workshop will be provided beginning an hour prior to the 
start of the meeting.
    If you need special accommodations due to a disability, please 
contact Nicole Wolanski, at 301-796-6570, or 
[email protected] no later than April 25, 2018.
    An agenda for the workshop and any other background materials will 
be made available 5 days before the workshop at https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm592778.htm.
    Streaming Webcast of the Public Workshop: For those unable to 
attend in person, FDA will provide a live webcast of the workshop. To 
register for the streaming webcast of the public workshop, please visit 
the following website by May 8, 2018: https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm592778.htm.
    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 workshop 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 
internet at https://www.fda.gov/NewsEvents/MeetingsConferencesWorkshops/ucm592778.htm.

    Dated: February 22, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-03961 Filed 2-26-18; 8:45 am]
 BILLING CODE 4164-01-P


