83 FR 52477 - Oncology Center of Excellence: Pediatric Oncology Program; Establishment of a Public Docket; Request for Comments
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 201 (October 17, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 201 (October 17, 2018)
| Page Range | 52477-52478 | |
| FR Document | 2018-22565 |
[Federal Register Volume 83, Number 201 (Wednesday, October 17, 2018)] [Notices] [Pages 52477-52478] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-22565] ----------------------------------------------------------------------- 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. ----------------------------------------------------------------------- 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.thefederalregister.org/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
![Federal Register / Vol. 83, No. 201 / Wednesday, October 17, 2018 / Notices 52477
A randomized study. Journal of Health potential relevance to the growth or Written/Paper Submissions
Communication, 21, 1198–1207. progression of one or more pediatric
5. Centers for Disease Control and Submit written/paper submissions as
cancers. The latter list details those follows:
Prevention. (2018a, May 18). 2016
National Health Interview Survey (NHIS)
targets that are unlikely to be associated • Mail/Hand delivery/Courier (for
data. Retrieved from https:// with the growth or progression of written/paper submissions): Dockets
www.cdc.gov/asthma/nhis/2016/table2- pediatric cancers such that statutory Management Staff (HFA–305), Food and
1.htm. requirements for early pediatric Drug Administration, 5630 Fishers
6. Centers for Disease Control and evaluation would be waived. These lists Lane, Rm. 1061, Rockville, MD 20852.
Prevention. (2018b, May 15). Most recent fulfill one of FDA’s obligations under
asthma data. Retrieved from https://
• For written/paper comments
the FDA Reauthorization Act of 2017 submitted to the Dockets Management
www.cdc.gov/asthma/most_recent_
(FDARA) and provide information to Staff, FDA will post your comment, as
data.htm.
7. Petty, R. E., & Cacioppo, J. T. (1979). Issue industry in planning for initial pediatric well as any attachments, except for
involvement can increase or decrease study plan submissions for certain information submitted, marked and
persuasion by enhancing message- oncology drugs or biological products in identified, as confidential, if submitted
relevant cognitive responses. Journal of accordance with the amended as detailed in ‘‘Instructions.’’
Personality and Social Psychology, 37, provisions of the Federal Food, Drug, Instructions: All submissions received
1915–1926. doi: 10.1037/0022- and Cosmetic Act (FD&C Act). FDA is
3514.37.10.1915. must include the Docket No. FDA–
establishing this docket for public 2018–N–3633 for ‘‘Oncology Center of
8. Petty, R. E., & Cacioppo, J. T. (1986). The
elaboration likelihood model of
comment on possible additions to or Excellence: Pediatric Oncology Program;
persuasion. Advances in Experimental deletions from the list on the lists Establishment of a Public Docket;
Social Psychology, 19, 123–205. doi: described above. Request for Comments.’’ Received
10.1016/S0065-2601(08)60214-2. The lists can be found on the comments will be placed in the docket
9. Petty, R. E., Cacioppo, J. T., & Goldman, Oncology Center of Excellence: and, except for those submitted as
R. (1981). Personal involvement as a ‘‘Confidential Submissions,’’ publicly
determinant of argument-based
Pediatric Oncology website at the
persuasion. Journal of Personality and following link: https://www.fda.gov/ viewable at https://www.regulations.gov
Social Psychology, 41, 847–855. doi: AboutFDA/CentersOffices/ or at the Dockets Management Staff
10.1037/0022-3514.41.5.847. OfficeofMedicalProductsandTobacco/ between 9 a.m. and 4 p.m., Monday
Dated: October 11, 2018. OCE/ucm544641.htm. through Friday.
• Confidential Submissions—To
Leslie Kux, DATES: Submit either electronic or submit a comment with confidential
Associate Commissioner for Policy. written comments. This docket will information that you do not wish to be
[FR Doc. 2018–22567 Filed 10–16–18; 8:45 am] remain open indefinitely. made publicly available, submit your
BILLING CODE 4164–01–P ADDRESSES: You may submit comments comments only as a written/paper
as follows: submission. You should submit two
copies total. One copy will include the
DEPARTMENT OF HEALTH AND Electronic Submissions information you claim to be confidential
HUMAN SERVICES with a heading or cover note that states
Submit electronic comments in the
following way: ‘‘THIS DOCUMENT CONTAINS
Food and Drug Administration
CONFIDENTIAL INFORMATION.’’ FDA
[Docket No. FDA–2018–N–3633] • Federal eRulemaking Portal: will review this copy, including the
https://www.regulations.gov. Follow the claimed confidential information, in its
Oncology Center of Excellence: instructions for submitting comments. consideration of comments. The second
Pediatric Oncology Program; Comments submitted electronically, copy, which will have the claimed
Establishment of a Public Docket; including attachments, to https:// confidential information redacted/
Request for Comments www.regulations.gov will be posted to blacked out, will be available for public
the docket unchanged. Because your viewing and posted on https://
AGENCY: Food and Drug Administration, comment will be made public, you are
HHS. www.regulations.gov. Submit both
solely responsible for ensuring that your copies to the Dockets Management Staff.
ACTION: Notice; establishment of a comment does not include any
public docket; request for comments. If you do not wish your name and
confidential information that you or a contact information be made publicly
SUMMARY: The Oncology Center of third party may not wish to be posted, available, you can provide this
Excellence (OCE) Pediatric Oncology such as medical information, your or information on the cover sheet and not
Program of the Food and Drug anyone else’s Social Security number, or in the body of your comments and you
Administration (FDA or the Agency) confidential business information, such must identify the information as
announces the creation of a list of as a manufacturing process. Please note ‘‘confidential.’’ Any information marked
molecular targets that have been that if you include your name, contact as ‘‘confidential’’ will not be disclosed
determined to be substantially relevant information, or other information that except in accordance with 21 CFR 10.20
to the growth or progression of a identifies you in the body of your and other applicable disclosure law. For
pediatric cancer (Candidate Pediatric comments, that information will be more information about FDA’s posting
Molecular Target List) and a list of posted on https://www.regulations.gov. of comments to public dockets, see 80
molecular targets of new cancer drugs • If you want to submit a comment FR 56469, September 18, 2015, or access
daltland on DSKBBV9HB2PROD with NOTICES
and biological products in development with confidential information that you the information at: https://www.gpo.gov/
for which requirements for studies in do not wish to be made available to the fdsys/pkg/FR-2015-09-18/pdf/2015-
pediatric cancers would be public, submit the comment as a 23389.pdf.
automatically waived. The former list written/paper submission and in the Docket: For access to the docket to
includes molecular targets for which manner detailed below (see ‘‘Written/ read background documents or the
prevailing evidence and/or a scientific Paper Submissions’’ and electronic and written/paper comments
rationale exists to determine their ‘‘Instructions’’). received, go to https://
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![52478 Federal Register / Vol. 83, No. 201 / Wednesday, October 17, 2018 / Notices
www.regulations.gov and insert the exclusivity, to encourage sponsors of DEPARTMENT OF HEALTH AND
docket number, found in brackets in the investigational therapies to conduct HUMAN SERVICES
heading of this document, into the pediatric studies of medicines with the
‘‘Search’’ box and follow the prompts potential for use in children. To date, Food and Drug Administration
and/or go to the Dockets Management section 505A has been one of the few [Docket No. FDA–2018–N–3138]
Staff, 5630 Fishers Lane, Rm. 1061, mechanisms available to incentivize
Rockville, MD 20852. evaluation of new oncology products in Agency Information Collection
FOR FURTHER INFORMATION CONTACT: children and adolescents. Nevertheless, Activities; Proposed Collection;
Christine Lincoln, Center for Drug further development of more novel Comment Request; Experimental
Evaluation and Research, Food and products that address the substantial Study of an Accelerated Approval
Drug Administration, 10903 New unmet needs of the pediatric population Disclosure
Hampshire Ave. Bldg. 22, Rm. 2118, is needed.
Silver Spring, MD 20993–0002, email: AGENCY: Food and Drug Administration,
Christine.Lincoln@fda.hhs.gov. Section 504 of FDARA requires FDA, HHS.
with input from the National Cancer ACTION: Notice.
SUPPLEMENTARY INFORMATION: The use of
tumor genetic profiling in cancer Institute (NCI) and others, to develop
and regularly update: (1) A list of SUMMARY: The Food and Drug
treatment decision making has Administration (FDA, Agency, or we) is
transformed therapeutic strategies in molecular targets that are determined to
be substantially relevant to the growth announcing an opportunity for public
many adult cancers. Extension of this comment on the proposed collection of
approach to treatment decision making and progression of a pediatric cancer,
and that may trigger the requirement for certain information by the Agency.
for children with cancer, however, has Under the Paperwork Reduction Act of
been greatly diminished due to delays pediatric investigations under section
505B of the FD&C Act, and (2) a list of 1995 (PRA), Federal Agencies are
in evaluation of potentially active drugs. required to publish notice in the
Until the passage of section 504 of molecular targets of new cancer drugs
Federal Register concerning each
FDARA, section 505B of the FD&C Act and biological products in development
proposed collection of information and
(21 U.S.C. 355c) has not typically been for which the requirement for pediatric
to allow 60 days for public comment in
a useful mechanism to require the investigations under section 505B of the response to the notice. This notice
development of drugs for pediatric FD&C Act would be automatically solicits comments on research entitled
cancers, since most of the oncology waived. ‘‘Experimental Study of an Accelerated
drugs approved for adults are used to To date, a total of 205 candidate Approval Disclosure.’’ This study will
treat cancers that very rarely or never examine the presence, wording, and
molecular targets were identified from
occur in children (e.g. cancers of the prominence of a disclosure
peer-reviewed literature searches,
lung, prostate and breast). Therefore, communicating information related to
review of publicly available genomic
historically, drug sponsors have the drug’s accelerated approval in
requested and obtained waivers for databases, such as NCI Genomic Data
Commons, TARGET (Therapeutically direct-to-consumer (DTC) promotional
conducting the required assessments of materials.
these drugs in pediatric patients. Applicable Research to Generate
Additionally, drugs developed for rare Effective Targets), St. Jude PeCan Data DATES: Submit either electronic or
cancer indications that received orphan Portal, Ped PanCan, and INFORM written comments on the collection of
designation are exempted from the pre- (Individualized Therapy for Relapsed information by December 17, 2018.
FDARA requirement to conduct Malignancies in Childhood), and input ADDRESSES: You may submit comments
pediatric assessments—even if the from international subject matter as follows. Please note that late,
cancers those products are intended to experts. Of these, 62 (30.3 percent) untimely filed comments will not be
treat occur in both adult and pediatric target a gene abnormality, 40 (19.5 considered. Electronic comments must
patients—due to the fact that the orphan percent) target a cell lineage be submitted on or before December 17,
designation exempts them from such determinant, 21 (10.2 percent) target the 2018. The https://www.regulations.gov
studies (see section 505B(k) of the FD&C tumor microenvironment or the immune electronic filing system will accept
Act). However, FDARA amended system, and 77 (37.6 percent) are comments until midnight Eastern Time
section 505B so that the requirement for classified as ‘‘Others.’’ Five (2.4 percent) at the end of December 17, 2018.
pediatric investigations of drugs are candidates for automatic waivers. Comments received by mail/hand
directed at molecular targets determined delivery/courier (for written/paper
Dated: October 11, 2018.
to be substantially relevant to the submissions) will be considered timely
growth and progression of a pediatric Leslie Kux, if they are postmarked or the delivery
cancer apply even when the adult Associate Commissioner for Policy. service acceptance receipt is on or
indication has received an orphan [FR Doc. 2018–22565 Filed 10–16–18; 8:45 am] before that date.
designation, or when the adult BILLING CODE 4164–01–P
Electronic Submissions
indication does not occur, in the
pediatric population (e.g., prostate Submit electronic comments in the
cancer). following way:
Although requirements to study • Federal eRulemaking Portal:
investigational therapies in pediatric https://www.regulations.gov. Follow the
daltland on DSKBBV9HB2PROD with NOTICES
oncology were exceedingly rare, other instructions for submitting comments.
incentives have been put into place to Comments submitted electronically,
promote the development of oncology including attachments, to https://
products for pediatric cancer. Section www.regulations.gov will be posted to
505A of the FD&C Act (21 U.S.C. 355a) the docket unchanged. Because your
provides incentives, in the form of 6 comment will be made public, you are
months of additional marketing solely responsible for ensuring that your
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Document Created: 2018-10-17 01:47:43
Document Modified: 2018-10-17 01:47:43
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice; establishment of a public docket; request for comments. | |
| Dates | Submit either electronic or written comments. This docket will remain open indefinitely. | |
| 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] | |
| FR Citation | 83 FR 52477 |
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