119-s705

[Congressional Bills 119th Congress]
[From the U.S. Government Publishing Office]
[S. 705 Introduced in Senate

    (IS) ]

<DOC>

119th CONGRESS
 1st Session
 S. 705

 To amend the Federal Food, Drug, and Cosmetic Act with respect to 
 molecularly targeted pediatric cancer investigations, and for other 
 purposes.

_______________________________________________________________________

 IN THE SENATE OF THE UNITED STATES

 February 25, 2025

 Mr. Reed (for himself and Mrs. Capito) introduced the following bill; 
 which was read twice and referred to the Committee on Health, 
 Education, Labor, and Pensions

_______________________________________________________________________

 A BILL

 To amend the Federal Food, Drug, and Cosmetic Act with respect to 
 molecularly targeted pediatric cancer investigations, and for other 
 purposes.

 Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

 This Act may be cited as the ``Innovation in Pediatric Drugs Act of 
2025''.

SEC. 2. ENSURING COMPLETION OF PEDIATRIC STUDY REQUIREMENTS.

    (a) Equal Accountability for Pediatric Study Requirements.--

Section 505B
            (d) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c
            (d) ) 
is amended--

    (1) in paragraph

    (1) , by striking ``Beginning 270'' and 
 inserting ``Noncompliance letter.--Beginning 270'';

    (2) in paragraph

    (2) --
        (A) by striking ``The drug or'' and inserting 
 ``Effect of noncompliance.--The drug or''; and
        (B) by striking ``(except that the drug or 
 biological product shall not be subject to action under 
 

section 303)'' and inserting ``(except that the drug or 
 biological product shall be subject to action under 
 

section 303 only if such person demonstrated a lack of 
 due diligence in satisfying the applicable 
 requirement)''; and

    (3) by adding at the end the following:
 ``

    (3) Limitation.--The Secretary shall not issue 
 enforcement actions under 

section 303 for failures under this 
 subsection in the case of a drug or biological product that is 
 no longer marketed.''.

    (b) Due Diligence.--

Section 505B
            (d) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355c
            (d) ), as amended by subsection

    (a) , is 
further amended by adding at the end the following:
 ``

    (4) Due diligence.--Before the Secretary may conclude 
 that a person failed to submit or otherwise meet a requirement 
 as described in the matter preceding paragraph

    (1) , the 
 Secretary shall--
 ``
        (A) issue a noncompliance letter pursuant to 
 paragraph

    (1) ;
 ``
        (B) provide such person with a 45-day period 
 beginning on the date of receipt of such noncompliance 
 letter to respond in writing as set forth in such 
 paragraph; and
 ``
            (C) after reviewing such written response, 
 determine whether the person demonstrated a lack of due 
 diligence in satisfying such requirement.''.
            (c) Conforming Amendments.--

Section 303

    (f)

    (4)
        (A) of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 333

    (f)

    (4)
        (A) ) is amended by 
striking ``or 505-1'' and inserting ``505-1, or 505B''.
            (d) Transition Rule.--The Secretary of Health and Human Services 
may take enforcement action under 

section 303 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 333) for failures described in 

section 505B
            (d) of such Act (21 U.S.C. 355c
            (d) ) only if such failures 
occur on or after the date that is 180 days after the date of enactment 
of this Act.

SEC. 3. FDA REPORT ON PREA ENFORCEMENT.

 

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

    (11) , by striking the semicolon at the end 
 and inserting ``, including an evaluation of compliance with 
 deadlines provided for in deferrals and deferral extensions;'';

    (2) in paragraph

    (15) , by striking ``and'' at the end;

    (3) in paragraph

    (16) , by striking the period at the end 
 and inserting ``; and''; and

    (4) by adding at the end the following:
 ``

    (17) a listing of penalties, settlements, or payments 
 under 

section 303 of the Federal Food, Drug, and Cosmetic Act 
 (21 U.S.C. 353) for failure to comply with requirements under 
 such 

section 505B, including, for each penalty, settlement, or 
 payment, the name of the drug, the sponsor thereof, and the 
 amount of the penalty, settlement, or payment imposed.''.

SEC. 4. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.

 

Section 409I
            (d) of the Public Health Service Act (42 U.S.C. 
284m
            (d) ) is amended to read as follows:
 ``
            (d) Funding.--Of the amount made available for pediatric research 
to each national research institute and national center under this 
title for each of fiscal years 2026 through 2030, the Director of NIH 
is authorized to make available up to one percent of such amount for 
pediatric research under this section.''.

SEC. 5. PEDIATRIC STUDIES OF ORPHAN DRUGS.

    (a) Application of Pediatric Research Requirements to Orphan 
Drugs.--

    (1) In general.--

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.--This section does not apply to a drug or 
 biological product for an indication for which orphan 
 designation has been granted under 

section 526 unless the 
 Secretary determines that pediatric assessments of such drug or 
 biological product required under this section could represent 
 a meaningful therapeutic benefit as described in subsection
            (c) .
 ``

    (2) Deferrals and waivers.--Deferrals and waivers under 
 subsections

    (a)

    (4) and

    (a)

    (5) shall apply to assessments 
 described in this subsection to the same extent and in the same 
 manner as such deferrals and waivers apply with respect to the 
 assessments under subsection

    (a)

    (1) , and waivers under 
 subsection

    (b)

    (2) shall apply to assessments described in this 
 subsection to the same extent and in the same manner as such 
 waivers apply with respect to the assessments required pursuant 
 to subsection

    (b)

    (1) .''.

    (2) Applicability.--The amendment made by paragraph

    (1) applies only to applications described in subparagraph
        (A) or
        (B) of 

section 505B

    (a)

    (1) of the Federal Food, Drug, and 
 Cosmetic Act (21 U.S.C. 355c

    (a)

    (1) ) that are submitted on or 
 after the later of--
        (A) the date that is 18 months after the date of 
 issuance of the final guidance under subsection

    (b) ; 
 and
        (B) such later date as may be specified by the 
 Secretary of Health and Human Services (referred to in 
 this section as the ``Secretary'') by regulation.

    (b) Guidance.--

    (1) Issuance.--The Secretary shall--
        (A) not later than 1 year after the date of 
 enactment of this Act, issue draft guidance describing 
 how, upon the applicability of the amendment made by 
 subsection

    (a)

    (1) , the requirements of 

section 505B of 
 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
 355c) will apply with respect to any drug or biological 
 product for an indication within a disease or condition 
 for which orphan designation has been granted under 
 

section 526 of such Act (21 U.S.C. 360bb); and
        (B) not later than 18 months after the date of the 
 public meeting required by subsection
            (c) (1) , finalize 
 such draft guidance.

    (2) Contents.--The guidance under subsection

    (b) shall 
 address the following:
        (A) Information regarding how full and partial 
 waivers under subsections

    (a)

    (5)
        (A) ,

    (a)

    (5)
        (B) , and

    (b)

    (2) of 

section 505B of the Federal Food, Drug, and 
 Cosmetic Act (21 U.S.C. 355c) for any drug or 
 biological product for an indication within a disease 
 or condition for which orphan designation has been 
 granted under 

section 526 of such Act (21 U.S.C. 360bb) 
 will be granted.
        (B) Application of the requirements of 

section 505B

    (e) of such Act (21 U.S.C. 355c

    (e) ) to drugs or 
 biological products for an indication within a disease 
 or condition for which orphan designation has been 
 granted under 

section 526 of such Act (21 U.S.C. 
 360bb), including submission and timing of planned 
 requests for full or partial waivers and responses by 
 the Food and Drug Administration to those requests.
            (C) Rare diseases and conditions (as defined in 
 

section 526

    (a)

    (2) of such Act (21 U.S.C. 360bb

    (a)

    (2) ) 
 that should be added to the lists under 

section 505B

    (a)

    (5)
        (E) and 505B

    (b)

    (2)
        (E) of such Act, as added 
 by subsection

    (f) , and a process for regularly updating 
 such lists.
            (D) Situations where the initial pediatric study 
 plan under 

section 505B

    (e) of such Act (21 U.S.C. 
 355c

    (e) ) includes a plan to fulfill the requirements of 
 

section 505B

    (a) of such Act (21 U.S.C. 355c

    (a) ) without 
 requesting waivers in any age group.
        (E) Consideration of how the Secretary will balance 
 the unique scientific challenges of rare disease drug 
 development with the need for improved pediatric 
 labeling of drugs and biological products for 
 indications within diseases or conditions for which 
 orphan designation has been granted under 

section 526 
 of such Act (21 U.S.C. 360bb).
        (F) Considerations of the strengths, weaknesses, 
 appropriateness, and limitations of different types of 
 real-world evidence specific to the fulfillment of 
 requirements under 

section 505B of such Act (21 U.S.C. 
 355c).
        (G) Consideration of input received from the public 
 meeting set forth in subsection
            (c) .
            (c) Public Meeting.--The Secretary shall--

    (1) not later than 6 months after the date of issuance of 
 the draft guidance under subsection

    (b)

    (1)
        (A) , hold a public 
 meeting to inform the final guidance to be issued under 
 subsection

    (b)

    (1)
        (B) ; and

    (2) publish prior notice of such meeting in the Federal 
 Register.
            (d) GAO Study.--Not later than 4 years after the applicability date 
described in subsection

    (a)

    (2) , the Comptroller General of the United 
States shall submit to the Committee on Energy and Commerce and the 
Committee on Ways and Means of the House of Representatives and the 
Committee on Health, Education, Labor, and Pensions of the Senate a 
report that--

    (1) addresses the impacts of this Act on--
        (A) rare disease drug development in the United 
 States; and
        (B) the availability of pediatric information on 
 drugs and biological products within diseases or 
 conditions for indications for which orphan designation 
 has been granted under 

section 526 of the Federal Food, 
 Drug, and Cosmetic Act (21 U.S.C. 360bb); and

    (2) includes--
        (A) the findings of a survey of companies of 
 varying sizes engaged in the development of orphan 
 drugs, which shall include questions regarding the 
 feasibility and other challenges of conducting 
 pediatric studies for such indications;
        (B) input from patient groups and medical provider 
 associations; and
            (C) an assessment of the impact changes to required 
 pediatric studies had on drug development for rare 
 diseases.

    (e) Rule of Construction.--Nothing in this section shall be 
construed to limit requirements for investigations, as described in 

section 505B

    (a)

    (3) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355c

    (a)

    (3) ), of molecularly targeted pediatric cancer drugs for 
which orphan designation has been granted under 

section 526 of such Act 
(21 U.S.C. 360bb).

    (f) Certainty Regarding Waivers.--

Section 505B of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355c) is amended--

    (1) in subsection

    (a)

    (5) , by adding at the end the 
 following:
 ``
        (E) Automatic full waiver list.--The Secretary 
 shall maintain a list, posted on the website of the 
 Food and Drug Administration, of adult-related diseases 
 and conditions--
 ``
            (i) with respect to which the necessary 
 studies are impossible or highly impracticable, 
 as described in subparagraph
        (A)
            (i) ; or
 ``
            (ii) for which a drug or biological 
 product for such disease or condition otherwise 
 meets the criteria described in subparagraph
        (A) .'';

    (2) in subsection

    (b)

    (2) , by adding at the end the 
 following:
 ``
        (E) Automatic full waiver list.--The Secretary 
 shall maintain a list, posted on the website of the 
 Food and Drug Administration, of adult-related diseases 
 and conditions with respect to which the necessary 
 studies would meet the criteria for a full waiver under 
 subparagraph
        (A) .''; and

    (3) in subsection

    (e)

    (4) , by adding at the end the 
 following: ``If, at the time of an applicant's submission of 
 the initial pediatric study plan, the disease or condition for 
 which the drug is intended to treat appears on the list under 
 subsection

    (a)

    (5)
        (E) , then the assessments for such disease or 
 condition shall be waived under subsection

    (a)

    (5) .''.
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