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  4. Tissue Agnostic Therapies: Regulatory Considerations for Orphan Drug Designation; Public Workshop - 05/09/2018
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Workshop | In Person

Event Title
Tissue Agnostic Therapies: Regulatory Considerations for Orphan Drug Designation; Public Workshop
May 9, 2018

May 9, 2018

The Food and Drug Administration (FDA) is holding a public workshop 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.

As advances in genomics and precision medicine continue to be made, FDA has been taking 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 based on molecular markers 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 tissue agnostic therapies in oncology and the appropriate application of orphan drug incentives in that paradigm.

Issue Summary

Workshop Date and Time
Wednesday, May 9, 2018
9:30 a.m. to 2:30 p.m.

Workshop Location
FDA White Oak Campus
10903 New Hampshire Avenue
Building 31 Conference Center
the Great Room (Rm. 1503, Section A)
Silver Spring, MD 20993-0002

The entrance for public hearing participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For parking and security information, please refer to Public Meetings at the FDA White Oak Campus.

Meeting Materials

Federal Register Notice


If you wish to attend the public workshop in person, please register by Wednesday, April 25, 2018 at 5 p.m. Eastern Time.

FDA will provide a free-of-charge live webcast for viewing the public workshop. We will post a link to the archived webcast on this page approximately one week after the hearing. A written transcript of the hearing will be made available in the public docket.

If you need special accommodations due to a disability, please contact Nicole Wolanski at 301-796-6570, or email OOPDorphanevents@fda.hhs.gov or theresa.wells@fda.hhs.gov no later than Wednesday, April 25, 2018.


Submitting Comments

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 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: Regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to 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 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."


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, will be placed in the docket and, except for those submitted as "Confidential Submissions," publicly viewable at 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 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.


For access to the docket to read background documents or the electronic and written/paper comments received, go to docket number FDA-2018-N-0663 on regulations.gov and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.


If you have questions about registering for the public workshop, contact Nicole Wolanski, Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5209, Silver Spring, MD, 20933, phone 301-796-6570, e-mail OOPDOrphanEvents@fda.hhs.gov or theresa.wells@fda.hhs.gov.

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