[Federal Register Volume 83, Number 201 (Wednesday, October 17, 2018)]
[Notices]
[Pages 52477-52478]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-22565]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-3633]


Oncology Center of Excellence: Pediatric Oncology Program; 
Establishment of a Public Docket; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice; establishment of a public docket; request for comments.

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SUMMARY: The Oncology Center of Excellence (OCE) Pediatric Oncology 
Program of the Food and Drug Administration (FDA or the Agency) 
announces the creation of a list of molecular targets that have been 
determined to be substantially relevant to the growth or progression of 
a pediatric cancer (Candidate Pediatric Molecular Target List) and a 
list of molecular targets of new cancer drugs and biological products 
in development for which requirements for studies in pediatric cancers 
would be automatically waived. The former list includes molecular 
targets for which prevailing evidence and/or a scientific rationale 
exists to determine their potential relevance to the growth or 
progression of one or more pediatric cancers. The latter list details 
those targets that are unlikely to be associated with the growth or 
progression of pediatric cancers such that statutory requirements for 
early pediatric evaluation would be waived. These lists fulfill one of 
FDA's obligations under the FDA Reauthorization Act of 2017 (FDARA) and 
provide information to industry in planning for initial pediatric study 
plan submissions for certain oncology drugs or biological products in 
accordance with the amended provisions of the Federal Food, Drug, and 
Cosmetic Act (FD&C Act). FDA is establishing this docket for public 
comment on possible additions to or deletions from the list on the 
lists described above.
    The lists can be found on the Oncology Center of Excellence: 
Pediatric Oncology website at the following link: https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/OCE/ucm544641.htm.

DATES: Submit either electronic or written comments. This docket will 
remain open indefinitely.

ADDRESSES: You may submit comments as follows:

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 below (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-3633 for ``Oncology Center of Excellence: Pediatric Oncology 
Program; Establishment of a Public Docket; Request for Comments.'' 
Received comments 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.'' FDA 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 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 the 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://

[[Page 52478]]

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: Christine Lincoln, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave. Bldg. 22, Rm. 2118, Silver Spring, MD 20993-0002, email: 
[email protected].

SUPPLEMENTARY INFORMATION: The use of tumor genetic profiling in cancer 
treatment decision making has transformed therapeutic strategies in 
many adult cancers. Extension of this approach to treatment decision 
making for children with cancer, however, has been greatly diminished 
due to delays in evaluation of potentially active drugs. Until the 
passage of section 504 of FDARA, section 505B of the FD&C Act (21 
U.S.C. 355c) has not typically been a useful mechanism to require the 
development of drugs for pediatric cancers, since most of the oncology 
drugs approved for adults are used to treat cancers that very rarely or 
never occur in children (e.g. cancers of the lung, prostate and 
breast). Therefore, historically, drug sponsors have requested and 
obtained waivers for conducting the required assessments of these drugs 
in pediatric patients. Additionally, drugs developed for rare cancer 
indications that received orphan designation are exempted from the pre-
FDARA requirement to conduct pediatric assessments--even if the cancers 
those products are intended to treat occur in both adult and pediatric 
patients--due to the fact that the orphan designation exempts them from 
such studies (see section 505B(k) of the FD&C Act). However, FDARA 
amended section 505B so that the requirement for pediatric 
investigations of drugs directed at molecular targets determined to be 
substantially relevant to the growth and progression of a pediatric 
cancer apply even when the adult indication has received an orphan 
designation, or when the adult indication does not occur, in the 
pediatric population (e.g., prostate cancer).
    Although requirements to study investigational therapies in 
pediatric oncology were exceedingly rare, other incentives have been 
put into place to promote the development of oncology products for 
pediatric cancer. Section 505A of the FD&C Act (21 U.S.C. 355a) 
provides incentives, in the form of 6 months of additional marketing 
exclusivity, to encourage sponsors of investigational therapies to 
conduct pediatric studies of medicines with the potential for use in 
children. To date, section 505A has been one of the few mechanisms 
available to incentivize evaluation of new oncology products in 
children and adolescents. Nevertheless, further development of more 
novel products that address the substantial unmet needs of the 
pediatric population is needed.
    Section 504 of FDARA requires FDA, with input from the National 
Cancer Institute (NCI) and others, to develop and regularly update: (1) 
A list of molecular targets that are determined to be substantially 
relevant to the growth and progression of a pediatric cancer, and that 
may trigger the requirement for pediatric investigations under section 
505B of the FD&C Act, and (2) a list of molecular targets of new cancer 
drugs and biological products in development for which the requirement 
for pediatric investigations under section 505B of the FD&C Act would 
be automatically waived.
    To date, a total of 205 candidate molecular targets were identified 
from peer-reviewed literature searches, review of publicly available 
genomic databases, such as NCI Genomic Data Commons, TARGET 
(Therapeutically Applicable Research to Generate Effective Targets), 
St. Jude PeCan Data Portal, Ped PanCan, and INFORM (Individualized 
Therapy for Relapsed Malignancies in Childhood), and input from 
international subject matter experts. Of these, 62 (30.3 percent) 
target a gene abnormality, 40 (19.5 percent) target a cell lineage 
determinant, 21 (10.2 percent) target the tumor microenvironment or the 
immune system, and 77 (37.6 percent) are classified as ``Others.'' Five 
(2.4 percent) are candidates for automatic waivers.

    Dated: October 11, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-22565 Filed 10-16-18; 8:45 am]
 BILLING CODE 4164-01-P


