[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]
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