US Codex
Pub. L.
Notes

Title V — Pediatric Drugs and Devices

115th Congress · Approved Aug 18, 2017 · 131 Stat. 1005

TITLE V Pediatric Drugs and Devices

SEC. 501. Best Pharmaceuticals for Children.

Section 409I of the Public Health Service Act (42 U.S.C. 284m) is amended—
(1)
in subsection (a)(2)(A)(ii), by inserting “ and identification of biomarkers for such diseases, disorders, or conditions,” after “ biologics,”;
(2)
in subsection (c)—
(A)
in paragraph (6)—
(i)
by amending subparagraph (B) to read as follows:

“(B) Availability of reports.—

“(i) In general.—Each report submitted under subparagraph (A) shall be considered to be in the public domain (subject to section 505A(d)(4) of the Federal Food, Drug, and Cosmetic Act) and not later than 90 days after submission of such report, shall be—

“(I) posted on the internet website of the National Institutes of Health in a manner that is accessible and consistent with all applicable Federal laws and regulations, including such laws and regulations for the protection of—

“(aa) human research participants, including with respect to privacy, security, informed consent, and protected health information; and

“(bb) proprietary interests, confidential commercial information, and intellectual property rights; and

“(II) assigned a docket number by the Commissioner of Food and Drugs and made available for the submission of public comments.

“(ii) Submission of comments.—An interested person may submit written comments concerning such pediatric studies to the Commissioner of Food and Drugs, and the submitted comments shall become part of the docket file with respect to each of the drugs.”

; and

(ii)
in subparagraph (C), by striking “ appropriate action” and all that follows through the period and inserting “ action in a timely and appropriate manner in response to the reports submitted under subparagraph (A), and shall begin such action upon receipt of the report under subparagraph (A), in accordance with paragraph (7).”; and
(B)
in paragraph (7)—
(i)
in the matter preceding subparagraph (A), by striking “ During” and inserting “ Within”;
(ii)
in subparagraph (C)(i), by striking “ place” and all that follows through “ and of” and inserting “ include in the public docket file a reference to the location of the report on the internet website of the National Institutes of Health and a copy of”; and
(iii)
in clause (ii), by striking “ in the Federal Register and”;
(3)
by striking subsection (d);
(4)
by redesignating subsection (e) as subsection (d); and
(5)
in paragraph (1) of subsection (d), as so redesignated, by striking “ 2013 through 2017” and inserting “ 2018 through 2022”.

SEC. 502. Pediatric Devices.

(a)
Pediatric Use of Devices.— Section 515A(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360e–1(a)(3)) is amended—
(1)
by redesignating subparagraphs (B) through (D) as subparagraphs (D) through (F), respectively;
(2)
by inserting after subparagraph (A) the following:

“(B) any information, based on a review of data available to the Secretary, regarding devices used in pediatric patients but not labeled for such use for which the Secretary determines that approved pediatric labeling could confer a benefit to pediatric patients;

“(C) the number of pediatric devices that receive a humanitarian use exemption under section 520(m);”

(3)
in subparagraph (E), as so redesignated, by striking “ ; and” and inserting “ ;”;
(4)
in subparagraph (F) (as so redesignated), by striking “ (B), and (C).” and inserting “ (C), (D), and (E);”; and
(5)
by adding at the end the following:

“(G) the number of devices for which the Secretary relied on data with respect to adults to support a determination of a reasonable assurance of safety and effectiveness in pediatric patients; and

“(H) the number of devices for which the Secretary relied on data from one pediatric subpopulation to support a determination of a reasonable assurance of safety and effectiveness in another pediatric subpopulation.

(b)
Humanitarian Device Exemption.— Section 520(m) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360j(m)) is amended—
(1)
in paragraph (4)—
(A)
in subparagraph (B), by inserting “ or an appropriate local committee” after “ review committee” each place such term appears; and
(B)
in the matter following subparagraph (B), by inserting “ or an appropriate local committee” after “ review committee” each place such term appears; and
(2)
in paragraph (6)(A)(iv), by striking “ 2017” and inserting “ 2022”.
(c)
Demonstration Grants for Improving Pediatric Availability.— Section 305 of the Pediatric Medical Device Safety and Improvement Act of 2007 (Public Law 110–85; 42 U.S.C. 282 note)) is amended—
(1)
in subsection (c)—
(A)
in paragraph (4), by striking “ and” at the end;
(B)
in paragraph (5), by striking the period and inserting “ ; and”; and
(C)
by adding at the end the following:

“(6) providing regulatory consultation to device sponsors in support of the submission of an application for a pediatric device, where appropriate.”

; and

(2)
in subsection (e), by striking “ 2013 through 2017” and inserting “ 2018 through 2022”.
(d)
Meeting on Pediatric Device Development.—
(1)
In general.— Not later than 1 year after the date of enactment of this Act, the Secretary of Health and Human Services shall convene a public meeting on the development, approval or clearance, and labeling of pediatric medical devices. The Secretary shall invite to such meeting representatives from the medical device industry, academia, recipients of funding under section 305 of the Pediatric Medical Device Safety and Improvement Act of 2007 (Public Law 110–85; 42 U.S.C. 282 note), medical provider organizations, and organizations representing patients and consumers.
(2)
Topics.— The meeting described in paragraph (1) shall include consideration of ways to—
(A)
improve research infrastructure and research networks to facilitate the conduct of clinical studies of devices for pediatric populations that would result in the approval or clearance, and labeling, of medical devices for such populations;
(B)
appropriately use extrapolation under section 515A(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360e–1(b));
(C)
enhance the appropriate use of postmarket registries and data to increase pediatric medical device labeling;
(D)
increase Food and Drug Administration assistance to medical device manufacturers in developing devices for pediatric populations that are approved or cleared, and labeled, for their use; and
(E)
identify current barriers to pediatric device development and incentives to address such barriers.
(3)
Report.— The report submitted under section 515A(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360e–1(a)(3)) with respect to the calendar year in which the meeting described in paragraph (1) is held shall include a summary of, and responses to, recommendations raised in such meeting.

SEC. 503. Early Meeting on Pediatric Study Plan.

(a)
In General.— Clause (i) of section 505B(e)(2)(C) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)(2)(C)) is amended to read as follows:

“(i) shall meet with the applicant—

“(I) if requested by the applicant with respect to a drug or biological product that is intended to treat a serious or life-threatening disease or condition, to discuss preparation of the initial pediatric study plan, not later than the end-of-Phase 1 meeting (as such term is used in section 312.82(b) of title 21, Code of Federal Regulations, or successor regulations) or within 30 calendar days of receipt of such request, whichever is later;

“(II) to discuss the initial pediatric study plan as soon as practicable, but not later than 90 calendar days after the receipt of such plan under subparagraph (A); and

“(III) to discuss the bases for the deferral under subsection (a)(4) or a full or partial waiver under subsection (a)(5);”

(b)
Conforming Changes.— Section 505B(e) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)) is amended—
(1)
in the heading of paragraph (2), by striking “ meeting” and inserting “ meetings”;
(2)
in the heading of paragraph (2)(C), by striking “ Meeting” and inserting “ Meetings”;
(3)
in clauses (ii) and (iii) of paragraph (2)(C), by striking “ no meeting” each place it appears and inserting “ no meeting under clause (i)(II)”; and
(4)
in paragraph (3) by striking “ meeting under paragraph (2)(C)(i)” and inserting “ meeting under paragraph (2)(C)(i)(II)”.

SEC. 504. Development of Drugs and Biological Products for Pediatric Cancers.

(a)
Molecular Targets Regarding Cancer Drugs and Biological Products.— Section 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c) is amended—
(1)
in subsection (a)—
(A)
in paragraph (1)—
(i)
by redesignating subparagraphs (A) and (B) as clauses (i) and (ii), respectively, and adjusting the margins accordingly;
(ii)
by striking “ A person” and inserting the following:

“(A) General requirements.—Except with respect to an application for which subparagraph (B) applies, a person”

(iii)
in clause (i), as so redesignated, by striking “ , or” at the end and inserting “ ; or”; and
(iv)
by adding after subparagraph (A), as so designated by clause (ii), the following:

“(B) Certain molecularly targeted cancer indications.—A person that submits, on or after the date that is 3 years after the date of enactment of the FDA Reauthorization Act of 2017, an original application for a new active ingredient under section 505 of this Act or section 351 of the Public Health Service Act, shall submit with the application reports on the investigation described in paragraph (3) if the drug or biological product that is the subject of the application is—

“(i) intended for the treatment of an adult cancer; and

“(ii) directed at a molecular target that the Secretary determines to be substantially relevant to the growth or progression of a pediatric cancer.”

(B)
in paragraph (2)(A), by striking “ paragraph (1)” and inserting “ paragraph (1)(A)”;
(C)
by redesignating paragraphs (3) and (4) as paragraphs (4) and (5), respectively;
(D)
by inserting after paragraph (2) the following:

“(3) Molecularly targeted pediatric cancer investigation.—

“(A) In general.—With respect to a drug or biological product described in paragraph (1)(B), the investigation described in this paragraph is a molecularly targeted pediatric cancer investigation, which shall be designed to yield clinically meaningful pediatric study data, gathered using appropriate formulations for each age group for which the study is required, regarding dosing, safety, and preliminary efficacy to inform potential pediatric labeling.

“(B) Extrapolation of data.—Paragraph (2)(B) shall apply to investigations described in this paragraph to the same extent and in the same manner as paragraph (2)(B) applies with respect to the assessments required under paragraph (1)(A).

“(C) Deferrals and waivers.—Deferrals and waivers under paragraphs (4) and (5) shall apply to investigations described in this paragraph to the same extent and in the same manner as such deferrals and waivers apply with respect to the assessments under paragraph (2)(B).”

(E)
in paragraph (4), as so redesignated—
(i)
by striking “ assessments required under paragraph (1)” each place it appears and inserting “ assessments required under paragraph (1)(A) or reports on the investigation required under paragraph (1)(B)”;
(ii)
in subparagraph (A)(ii)(I), by inserting “ or reports on the investigation” after “ assessments”;
(iii)
in subparagraph (B)(ii), by striking “ assessment under paragraph (1)” and inserting “ assessment under paragraph (1)(A) or reports on the investigation under paragraph (1)(B)”; and
(iv)
in subparagraph (C)(ii)(II), by inserting “ or investigation” after “ assessment”; and
(F)
in paragraph (5), as so redesignated, by inserting “ or reports on the investigation” after “ assessments” each place it appears;
(2)
in subsection (d)—
(A)
by striking “ subsection (a)(3)” each place it appears and inserting “ subsection (a)(4)”;
(B)
by inserting “ and Reports on the Investigation” after “ Submission of Assessments” in the heading; and
(C)
by inserting “ or the investigation described in subsection (a)(3)” after “ assessment described in subsection (a)(2)” each place it appears;
(3)
in subsection (e)—
(A)
in paragraph (1), by inserting “ or the investigation described in subsection (a)(3)” after “ under subsection (a)(2)”; and
(B)
in paragraph (2)(A)(i), by inserting “ or the investigation described in subsection (a)(3)” after “ under subsection (a)(2)”; and
(4)
by adding at the end the following:

“(m) List of Primary Molecular Targets.—

“(1) In general.—Within one year of the date of enactment of the FDA Reauthorization Act of 2017, the Secretary shall establish and update regularly, and shall publish on the internet website of the Food and Drug Administration—

“(A) a list of molecular targets considered, on the basis of data the Secretary determines to be adequate, to be substantially relevant to the growth and progression of a pediatric cancer, and that may trigger the requirements under this section; and

“(B) a list of molecular targets of new cancer drugs and biological products in development for which pediatric cancer study requirements under this section will be automatically waived.

“(2) Consultation.—In establishing the lists described in paragraph (1), the Secretary shall consult the National Cancer Institute, members of the internal committee under section 505C, and the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee, and shall take into account comments from the meeting under subsection (c).

“(3) Rule of construction.—Nothing in paragraph (1) shall be construed—

“(A) to require the inclusion of a molecular target on the list published under such paragraph as a condition for triggering the requirements under subsection (a)(1)(B) with respect to a drug or biological product directed at such molecular target; or

“(B) to authorize the disclosure of confidential commercial information, as prohibited under section 301(j) of this Act or section 1905 of title 18, United States Code.”

(b)
Orphan Drugs.— Section 505B(k) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(k)) is amended to read as follows:

“(k) Relation to Orphan Drugs.—

“(1) In general; exemption for orphan indications.—Unless the Secretary requires otherwise by regulation and except as provided in paragraph (2), this section does not apply to any drug or biological product for an indication for which orphan designation has been granted under section 526.

“(2) Applicability despite orphan designation of certain indications.—This section shall apply with respect to a drug or biological product for which an indication has been granted orphan designation under 526 if the investigation described in subsection (a)(3) applies to the drug or biological product as described in subsection (a)(1)(B).”

(c)
Meeting, Consultation, and Guidance.—
(1)
Meeting.— The Secretary of Health and Human Services (referred to in this subsection as the “Secretary”), acting through the Commissioner of Food and Drugs and in collaboration with the Director of the National Cancer Institute, shall convene a public meeting not later than 1 year after the date of enactment of this Act to solicit feedback from physicians and researchers (including pediatric oncologists and rare disease specialists), patients, and other stakeholders to provide input on development of the guidance under paragraph (2) and the list under subsection (m) of section 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c), as added by subsection (a). The Secretary shall seek input at such meeting on—
(A)
the data necessary to determine that there is scientific evidence that a drug or biological product is directed at a molecular target that is considered to be substantially relevant to the growth or progression of a pediatric cancer;
(B)
the data necessary to determine that there is scientific evidence that a molecular target is considered to be substantially relevant to the growth or progression of a pediatric cancer;
(C)
the data needed to meet the requirement of conducting an investigation described in section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act, as amended by subsection (a);
(D)
considerations when developing the list under section 505B(m) of the Federal Food, Drug, and Cosmetic Act that contains molecular targets shared between different tumor types;
(E)
the process the Secretary shall utilize to update regularly a list of molecular targets that may trigger a pediatric study under section 505B of the Federal Food, Drug, and Cosmetic Act, as so amended, and how often such updates shall occur;
(F)
how to overcome the challenges related to pediatric cancer drug and biological product development, including issues related to the ethical, practical, and other barriers to conducting clinical trials in pediatric cancer with small patient populations;
(G)
scientific or operational challenges associated with performing an investigation described in section 505B(a)(1)(B) of the Federal Food, Drug, and Cosmetic Act, including the effect on pediatric studies currently underway in a pediatric patient population, treatment of a pediatric patient population, and the ability to complete adult clinical trials;
(H)
the advantages and disadvantages of innovative clinical trial designs in addressing the development of cancer drugs or biological products directed at molecular targets in pediatric cancer patients;
(I)
the ways in which the Secretary can improve the current process outlined under sections 505A and 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a, 355c) to encourage additional research and development of pediatric cancer treatments;
(J)
the ways in which the Secretary might streamline and improve the written request process, including when studies contained in a request under such section 505A are not feasible due to the ethical, practical, or other barriers to conducting clinical trials in pediatric cancer populations;
(K)
how the Secretary will facilitate collaboration among pediatric networks, academic centers and experts in pediatric cancer to conduct an investigation described in such section 505B(a)(3);
(L)
how the Secretary may facilitate collaboration among sponsors of same-in-class drugs and biological products that would be subject to the requirements for an investigation under such section 505B based on shared molecular targets; and
(M)
the ways in which the Secretary will help to mitigate the risks, if any, of discouraging the research and development of orphan drugs when implementing such section 505B as amended.
(2)
Guidance.— Not later than 2 years after the date of enactment of this Act, the Secretary, acting through the Commissioner of Food and Drugs, shall issue final guidance on implementation of the amendments to section 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c) regarding molecularly targeted cancer drugs made by this section, including—
(A)
the scientific criteria, types of data, and regulatory considerations for determining whether a molecular target is substantially relevant to the growth or progression of a pediatric cancer and would trigger an investigation under section 505B of the Federal Food, Drug, and Cosmetic Act, as amended;
(B)
the process by which the Secretary will engage with sponsors to discuss determinations, investigation requirements, deferrals, waivers, and any other issues that need to be resolved to ensure that any required investigation based on a molecular target can be reasonably conducted;
(C)
the scientific or operational challenges for which the Secretary may issue deferrals or waivers for an investigation described in subsection (a)(3) of such section 505B, including adverse impacts on current pediatric studies underway in a pediatric patient population, studies involving drugs designated as orphan drugs, treatment of a pediatric patient population, or the ability to complete adult clinical trials;
(D)
how the Secretary and sponsors will facilitate collaboration among pediatric networks, academic centers, and experts in pediatric cancer to conduct an investigation described in subsection (a)(3) of such section 505B;
(E)
scientific and regulatory considerations for study designs, including the applicability of innovative clinical trial designs for pediatric cancer drug and biological product developments under sections 505A and 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a, 355c);
(F)
approaches to streamline and improve the amendment process, including when studies contained in a request under such section 505A are not feasible due to the ethical, practical, or other barriers to conducting clinical trials in pediatric cancer populations;
(G)
the process for submission of an initial pediatric study plan for the investigation described in section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(3)), including the process for a sponsor to meet and reach agreement with the Secretary on the initial pediatric study plan; and
(H)
considerations for implementation of such section 505B, as so amended, and waivers of the requirements of such section 505B with regard to molecular targets for which several drugs or biological products may be under investigation.
(d)
Report to Congress.— Section 508(b) of the Food and Drug Administration Safety and Innovation Act (21 U.S.C. 355c–1(b)) is amended—
(1)
in paragraph (10), by striking “ ; and” and inserting “ ;”; and
(2)
by striking paragraph (11) and inserting the following:

“(11) an assessment of the impact of the amendments to such section 505B made by the FDA Reauthorization Act of 2017 on pediatric research and labeling of drugs and biological products and pediatric labeling of molecularly targeted drugs and biological products for the treatment of cancer;

“(12) an assessment of the efforts of the Secretary to implement the plan developed under section 505C–1 of the Federal Food, Drug, and Cosmetic Act, regarding earlier submission of pediatric studies under sections 505A and 505B of such Act and section 351(m) of the Public Health Service Act, including—

“(A) the average length of time after the approval of an application under section 505(b)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(b)(1)) or section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)) before studies conducted pursuant to such section 505A, 505B, or section 351(m) are completed, submitted, and incorporated into labeling;

“(B) the average length of time after the receipt of a proposed pediatric study request before the Secretary responds to such request;

“(C) the average length of time after the submission of a proposed pediatric study request before the Secretary issues a written request for such studies;

“(D) the number of written requests issued for each investigational new drug or biological product prior to the submission of an application under section 505(b)(1) of the Federal Food, Drug, and Cosmetic Act or section 351(a) of the Public Health Service Act; and

“(E) the average number, and range of numbers, of amendments to written requests issued, and the time the Secretary requires to review and act on proposed amendments to written requests;

“(13) a list of sponsors of applications or holders of approved applications who received exclusivity under such section 505A or such section 351(m) after receiving a letter issued under such section 505B(d)(1) for any drug or biological product before the studies referred to in such letter were completed and submitted;

“(14) a list of assessments and investigations required under such section 505B;

“(15) how many requests under such section 505A for molecular targeted cancer drugs, as defined by subsection (a)(1)(B) of such section 505B, approved prior to 3 years after the date of enactment of the FDA Reauthorization Act of 2017, have been issued by the Food and Drug Administration, and how many such requests have been completed; and

“(16) the Secretary’s assessment of the overall impact of the amendments made by section 504 of the FDA Reauthorization Act of 2017 on the conduct and effectiveness of pediatric cancer research and the orphan drug program, as well any subsequent recommendations.”

(e)
Rule of Construction.— Nothing in this section, including the amendments made by this section, shall limit the authority of the Secretary of Health and Human Services to issue written requests under section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) or section 351(m) of the Public Health Service Act (42 U.S.C. 262(m)), or to negotiate or implement amendments to such requests proposed by the an applicant.
(f)
GAO Report.—
(1)
In general.— Beginning on the date that is 5 years after the date of enactment of this Act, the Comptroller General of the United States shall conduct a study of the effectiveness of requiring assessments and investigations described in section 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c), as amended by this section, in the development of drugs and biological products for pediatric cancer indications. The Comptroller General shall examine—
(A)
the indications and associated molecular targets studied in assessments and investigations required for drugs or biological products intended for the treatment of an adult cancer;
(B)
the indication for which the study was requested as compared to the indication requested under the new drug application filed by the sponsor;
(C)
the number of pediatric cancer indications for which assessments and investigations have been required under such section 505B;
(D)
the number of requests for deferral and waiver of pediatric assessments and investigations required under such section and the number of such deferral and waiver requests granted and denied;
(E)
the number of orphan-designated indications for drugs and biological products for which assessments and investigations were required under such section;
(F)
the number of drugs and biological products approved for the treatment of cancer in the pediatric population for which the supportive studies were required to be conducted under such section;
(G)
the number of written requests made under section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) relating to investigations required under subsection (a)(1)(B) of such section 505B; and
(H)
any additional considerations by the Secretary regarding the effectiveness of requiring pediatric assessments described in such section 505B in the development of drugs and biological products for pediatric cancer indications.
(2)
Review.— The study under paragraph (1) shall include a review of the Food and Drug Administration’s use of the authority under section 505B of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c), as amended by this section, including the amendments to the deferral and waiver criteria under such section and how such criteria have been applied.
(3)
Consultation.— In conducting the study under paragraph (1), the Comptroller General of the United States shall consult with appropriate stakeholders that may be required to conduct the trials under section 505B of the Federal Food, Drug, and Cosmetic Act, and the ability of such stakeholders to adhere to the requests issued by the Food and Drug Administration.
(4)
Report.— Not later than the date that is 6 years after the date of enactment of this Act, the Comptroller General of the United States shall submit a report containing the results of the study under paragraph (1) to the Secretary of Health and Human Services, the Committee on Health, Education, Labor, and Pensions of the Senate, and the Committee on Energy and Commerce of the House of Representatives.

SEC. 505. Additional Provisions on Development of Drugs and Biological Products for Pediatric Use.

(a)
Informing Internal Review Committee.— Section 505A(f) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a(f)) is amended by adding at the end the following:

“(7) Informing internal review committee.—The Secretary shall provide to the committee referred to in paragraph (1) any response issued to an applicant or holder with respect to a proposed pediatric study request.”

(b)
Action on Submissions.—
(1)
In general.— Section 505A(d) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a(d)) is amended—
(A)
by redesignating paragraphs (3) through (5) as paragraphs (4) through (6), respectively; and
(B)
by inserting after paragraph (2) the following:

“(3) Action on submissions.—The Secretary shall review and act upon a submission by a sponsor or holder of a proposed pediatric study request or a proposed amendment to a written request for pediatric studies within 120 calendar days of the submission.”

(2)
Conforming amendments.—
(A)
Federal food, drug, and cosmetic act.— Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a), as amended by paragraph (1), is further amended by striking subsection “ (d)(3)” each place it appears and inserting “ (d)(4)”.
(B)
Public health service act.— Paragraphs (2), (3), and (4) of section 351(m) of the Public Health Service Act (42 U.S.C. 262(m)) are amended by striking “ section 505A(d)(3)” each place it appears and inserting “ section 505A(d)(4)”.
(c)
Plan.— The Secretary of Health and Human Services, acting through the internal review committee established under section 505C of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355d) shall, not later than one year after the date of enactment of this Act, develop and implement a plan to achieve, when appropriate, earlier submission of pediatric studies under section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) or section 351(m) of the Public Health Service Act (42 U.S.C. 262(m)). Such plan shall include recommendations to achieve—
(1)
earlier discussion of proposed pediatric study requests and written requests with sponsors, and if appropriate, discussion of such requests at the meeting required under section 505B(e)(2)(C) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)(2)(C)), as amended by section 503(a);
(2)
earlier issuance of written requests for a pediatric study under such section 505A, including for investigational new drugs prior to the submission of an application under section 505(b)(1) of such Act (21 U.S.C. 355(b)(1)); and
(3)
shorter timelines, when appropriate, for the completion of studies pursuant to a written request under such section 505A or such section 351(m).
(d)
Neonatology Expertise.—
(1)
In general.— Section 6(d) of the Best Pharmaceuticals for Children Act (21 U.S.C. 393a(d)) is amended by striking “ For the 5-year period beginning on the date of enactment of this subsection, at” and inserting “ At”.
(2)
Draft guidance.— Not later than 2 years after the date of enactment of this Act, the Secretary shall issue draft guidance on clinical pharmacology considerations for neonatal studies for drugs and biological products.
(e)
Submission of Assessments.— Section 505B(d)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)(1)) is amended by adding at the end the following: “ The Secretary shall inform the Pediatric Advisory Committee of letters issued under this paragraph and responses to such letters.”.
(f)
Internal Committee.— Section 505C of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355d) is amended by inserting “ or pediatric rare diseases” after “ psychiatry”.
(g)
Report on Labeling of Orphan Drugs.—
(1)
In general.— Not later than 2 years after the date of the enactment of this Act, the Secretary of Health and Human Services shall submit to the Committee on Health, Education, Labor and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives, and make publicly available, including through posting on the internet website of the Food and Drug Administration, a report on the lack of information in the labeling of drugs for indications that have received an orphan designation under section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) with respect to the use of such drugs pediatric populations.
(2)
Contents.— The report described in paragraph (1) shall include—
(A)
a list of drugs for which—
(i)
an indication was granted an orphan designation under section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb);
(ii)
an application described under section 505B(a)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(1)) for such indication was submitted to the Secretary of Health and Human Services on or after April 1, 1999; and
(iii)
the labeling for such indication lacks important pediatric information, including information related to safety, dosing, and effectiveness;
(B)
a description of the lack of information referred to in subparagraph (A)(iii) for each drug for an indication on such list; and
(C)
Federal policy recommendations to improve the labeling of drugs for indications that have received an orphan designation under such section 526 with respect to the use of such drugs pediatric populations.”