Tissue Agnostic Therapies in Oncology: Regulatory Considerations for Orphan Drug Designation; Public Workshop; Request for Comments, 8485-8487 [2018-03961]

Download as PDF 8485 Federal Register / Vol. 83, No. 39 / Tuesday, February 27, 2018 / Notices ANNUAL BURDEN ESTIMATES Instrument Number of respondents Number of responses per respondent Average burden hours per response Total burden hours Mandatory Grant Financial Report .................................................................. 900 4 5 18,000 Estimated Total Annual Burden Hours: Additional Information Copies of the proposed collection may be obtained by writing to the Administration for Children and Families, Office of Planning, Research and Evaluation, 330 C Street SW, Washington, DC 20201. Attention Reports Clearance Officer. All requests should be identified by the title of the information collection. Email address: infocollection@acf.hhs.gov. OMB Comment OMB is required to make a decision concerning the collection of information between 30 and 60 days after publication of this document in the Federal Register. Therefore, a comment is best assured of having its full effect if OMB receives it within 30 days of publication. Written comments and recommendations for the proposed information collection should be sent directly to the following: Office of Management and Budget, Paperwork Reduction Project, Email: OIRA_ SUBMISSION@OMB.EOP.GOV. Attn: Desk Officer for the Administration for Children and Families. abbreviated new drug applications from multiple applicants, withdrawn as of July 21, 2017. The document indicated that FDA was withdrawing approval of NDA 204508, Clinolipid 20% (olive oil and soybean oil) USP, 16%/4%, after receiving a request from the NDA holder, Baxter Healthcare Corp. (Baxter), 32650 N Wilson Rd., Round Lake, IL 60073. Before the approval of NDA 204508 was withdrawn, Baxter informed FDA that it did not want the approval of this NDA withdrawn. Because Baxter timely requested that approval of this NDA not be withdrawn, the approval of NDA 204508 is still in effect. FOR FURTHER INFORMATION CONTACT: Florine Purdie, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6366, Silver Spring, MD 20993–0002, 301– 796–3601. SUPPLEMENTARY INFORMATION: In the Federal Register of Wednesday, June 21, 2017, appearing on page 28322 in FR Doc. 2017–12908, the following correction is made: On page 28329, in table 1, the entry for NDA 204508 is removed. Dated: February 21, 2018. Leslie Kux, Associate Commissioner for Policy. Robert Sargis, Reports Clearance Officer. [FR Doc. 2018–03976 Filed 2–26–18; 8:45 am] [FR Doc. 2018–03925 Filed 2–26–18; 8:45 am] BILLING CODE P BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration Food and Drug Administration [Docket No. FDA–2017–N–3203] [Docket No. FDA–2018–N–0663] Wyeth Pharmaceuticals Inc. et al.; Withdrawal of Approval of 121 New Drug Applications and 161 Abbreviated New Drug Applications; Correction AGENCY: Food and Drug Administration, HHS. daltland on DSKBBV9HB2PROD with NOTICES ACTION: The Food and Drug Administration (FDA) is correcting a notice that appeared in the Federal Register of June 21, 2017 (82 FR 28322). The document announced the withdrawal of approval of 121 new drug applications (NDAs) and 161 SUMMARY: 19:49 Feb 26, 2018 AGENCY: Food and Drug Administration, HHS. Notice; correction. VerDate Sep<11>2014 Tissue Agnostic Therapies in Oncology: Regulatory Considerations for Orphan Drug Designation; Public Workshop; Request for Comments Jkt 244001 Notice of public workshop; request for comments. ACTION: The Food and Drug Administration (FDA or the Agency) is announcing the following public workshop entitled ‘‘Tissue Agnostic Therapies in Oncology: Regulatory SUMMARY: PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 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/WhiteOak CampusInformation/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 E:\FR\FM\27FEN1.SGM 27FEN1 8486 Federal Register / Vol. 83, No. 39 / Tuesday, February 27, 2018 / Notices daltland on DSKBBV9HB2PROD with NOTICES 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 VerDate Sep<11>2014 19:49 Feb 26, 2018 Jkt 244001 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/201523389.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, OOPDOrphanEvents@fda.hhs.gov. 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) PO 00000 Frm 00069 Fmt 4703 Sfmt 4703 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/organspecific 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/Meetings ConferencesWorkshops/ 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:// E:\FR\FM\27FEN1.SGM 27FEN1 daltland on DSKBBV9HB2PROD with NOTICES Federal Register / Vol. 83, No. 39 / Tuesday, February 27, 2018 / Notices www.fda.gov/NewsEvents/Meetings ConferencesWorkshops/ 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 OOPDOrphanEvents@fda.hhs.gov 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/News Events/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/Meetings ConferencesWorkshops/ 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. VerDate Sep<11>2014 19:49 Feb 26, 2018 Jkt 244001 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 DEPARTMENT OF HEALTH AND HUMAN SERVICES Health Resources and Service Administration Women’s Preventive Services Guidelines Health Resources and Services Administration (HRSA), Department of Health and Human Services (HHS). ACTION: Notice. AGENCY: 8487 information about the December 20, 2016, updates, please see https:// www.federalregister.gov/documents/ 2016/12/27/2016-31129/updating-thehrsa-supported-womens-preventiveservices-guidelines. In addition, the December 20, 2016, updates, including information related to coverage of contraceptive services and exemption for objecting organizations from requirements related to the provision of contraceptive services, can be found at https://www.hrsa.gov/womensguidelines-2016/. Information regarding the two new services that were accepted by the HRSA Administrator on December 29, 2017, is set out below: 1. Screening for Diabetes Mellitus After Pregnancy SUMMARY: Applicable as of December 29, The Women’s Preventive Services 2017, HRSA updated the HRSAsupported Women’s Preventive Services Initiative recommends women with a history of gestational diabetes mellitus Guidelines for purposes of health (GDM) who are not currently pregnant insurance coverage for preventive and who have not previously been services that address health needs diagnosed with type 2 diabetes mellitus specific to women based on clinical should be screened for diabetes recommendations from the Women’s Preventive Services Initiative. This 2017 mellitus. Initial testing should ideally occur within the first year postpartum update adds two additional services— and can be conducted as early as 4–6 Screening for Diabetes Mellitus after weeks postpartum. Pregnancy and Screening for Urinary Women with a negative initial Incontinence—to the nine preventive postpartum screening test result should services included in the 2016 update to be rescreened at least every 3 years for the HRSA-supported Women’s a minimum of 10 years after pregnancy. Preventive Services Guidelines. The For women with a positive postpartum nine services included in the 2016 screening test result, testing to confirm update are as follows: Breast Cancer the diagnosis of diabetes is indicated Screening for Average Risk Women, regardless of the initial test (e.g., oral Breastfeeding Services and Supplies, glucose tolerance test, fasting plasma Screening for Cervical Cancer, Contraception, Screening for Gestational glucose, or hemoglobin A1c). Repeat Diabetes Mellitus, Screening for Human testing is indicated in women who were screened with hemoglobin A1c in the Immunodeficiency Virus Infection, first six months postpartum regardless Screening for Interpersonal and of the result (see Implementation Domestic Violence, Counseling for Considerations below). Sexually Transmitted Infections, and Well-Woman Preventive Visits. This 2. Screening for Urinary Incontinence notice serves as an announcement of the The Women’s Preventive Services decision to update the guidelines as Initiative recommends screening women listed below. Please see https:// for urinary incontinence annually. www.hrsa.gov/womens-guidelines/ Screening should ideally assess whether index.html for additional information. women experience urinary incontinence FOR FURTHER INFORMATION CONTACT: and whether it impacts their activities Kimberly C. Sherman, Maternal and and quality of life. The Women’s Child Health Bureau, HRSA at phone: Preventive Services Initiative (301) 443–0543; email: wellwomancare@ recommends referring women for hrsa.gov. further evaluation and treatment if SUPPLEMENTARY INFORMATION: The indicated. complete set of updated 2017 HRSAsupported Women’s Preventive Services HRSA-Supported Women’s Preventive Services Guidelines Guidelines includes those that were The HRSA-supported Women’s accepted by the Acting HRSA Administrator on December 20, 2016, as Preventive Services Guidelines were originally established in 2011 based on well as two new services, Screening for recommendations from an HHS Diabetes Mellitus After Pregnancy and Screening for Urinary Incontinence. For commissioned study by the Institute of Medicine, now known as the National a complete listing and detailed PO 00000 Frm 00070 Fmt 4703 Sfmt 4703 E:\FR\FM\27FEN1.SGM 27FEN1

Agencies

[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

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.