Union Calendar No. 594
118th CONGRESS 2d Session |
[Report No. 118–700]
To amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes.
May 17, 2023
Mr. McCaul (for himself, Ms. Eshoo, Mr. Kelly of Pennsylvania, Mrs. Kim of California, Mr. Smith of New Jersey, Ms. Schrier, Mr. Bacon, Mr. Moylan, Mr. Buchanan, Mr. Fitzpatrick, Mr. Huizenga, Mr. Grothman, Mr. Johnson of Ohio, and Mr. Phillips) introduced the following bill; which was referred to the Committee on Energy and Commerce
September 20, 2024
Additional sponsors: Ms. Kuster, Mr. Wilson of South Carolina, Ms. Salazar, Mr. Neguse, Mr. LaLota, Mr. Valadao, Mr. Mullin, Mr. Waltz, Mr. Allred, Mr. Lawler, Ms. Castor of Florida, Ms. Ross, Ms. Lofgren, Mr. Cárdenas, Mr. Calvert, Mr. Kilmer, Mr. Rouzer, Mr. Davis of North Carolina, Ms. Mace, Ms. Pettersen, Ms. Leger Fernandez, Ms. Perez, Ms. Caraveo, Mr. Pappas, Mr. Jackson of North Carolina, Mr. Rutherford, Mr. Owens, Mr. Baird, Mr. Nehls, Mr. Schneider, Mr. Van Drew, Mr. Smith of Nebraska, Mr. Bean of Florida, Mr. Westerman, Mr. Ferguson, Mr. Yakym, Ms. Sewell, Mr. Kean of New Jersey, Mr. Casten, Ms. De La Cruz, Mr. Steube, Mr. Lamborn, Mr. Cohen, Ms. Titus, Ms. Brown, Mr. Veasey, Ms. Slotkin, Mr. Garbarino, Mr. Luetkemeyer, Mr. Peters, Mr. McGarvey, Mr. Lieu, Mr. Zinke, Mr. Harder of California, Mr. Gooden of Texas, Mr. Grijalva, Mr. Norman, Ms. Blunt Rochester, Mr. McGovern, Mr. Moulton, Mr. Quigley, Mr. Crow, Mr. LaHood, Mr. Thanedar, Mr. Gottheimer, Mr. Moskowitz, Mr. Swalwell, Mr. Austin Scott of Georgia, Mr. Bera, Mr. Diaz-Balart, Ms. Budzinski, Mr. Aderholt, Ms. Tokuda, Ms. Granger, Mr. Cline, Mr. Finstad, Ms. Velázquez, Mr. Nunn of Iowa, Ms. Wasserman Schultz, Mr. Trone, Mr. Tonko, Mrs. Trahan, Mr. Carl, Ms. Matsui, Mr. Keating, Ms. Houlahan, Mrs. Miller of West Virginia, Mrs. Foushee, Mrs. Watson Coleman, Mr. Thompson of Pennsylvania, Mr. Carey, Mr. Nickel, Mr. Fleischmann, Mr. Donalds, Ms. Waters, Mr. Ivey, Mr. Sablan, Mrs. Cherfilus-McCormick, Mrs. Napolitano, Mr. Lynch, Mr. Correa, Mr. Goldman of New York, Mr. Deluzio, Mr. Raskin, Mr. Mike Garcia of California, Mr. Larson of Connecticut, Mr. Panetta, Ms. DelBene, Mrs. Fletcher, Mr. Arrington, Ms. Brownley, Mr. Sessions, Mr. LaMalfa, Mr. Amodei, Mr. D'Esposito, Mr. Scott Franklin of Florida, Ms. Jacobs, Mr. Costa, Mr. Graves of Missouri, Ms. Meng, Ms. Wild, Ms. Norton, Mr. Kim of New Jersey, Mr. DeSaulnier, Ms. Pingree, Mr. Takano, Ms. Clarke of New York, Mr. Khanna, Ms. Lee of Pennsylvania, Mr. Kildee, Mrs. Dingell, Ms. Malliotakis, Mr. Mills, Ms. Spanberger, Ms. Stansbury, Mr. Burchett, Mr. Kelly of Mississippi, Mr. Jackson of Illinois, Mr. Soto, Mr. Castro of Texas, Mrs. Bice, Mr. Mooney, Mr. Mann, Ms. Scanlon, Ms. Porter, Mr. Bucshon, Ms. Craig, Mr. Ruiz, Mr. Cuellar, Mr. Carter of Louisiana, Mr. Flood, Mrs. Hayes, Mr. Clyde, Ms. Jayapal, Mr. LaTurner, Ms. Letlow, Ms. Kamlager-Dove, Mr. Pocan, Ms. Wexton, Ms. Sherrill, Ms. Strickland, Mrs. Kiggans of Virginia, Ms. Barragán, Mr. Higgins of Louisiana, Mr. Sorensen, Ms. Crockett, Mr. Ciscomani, Mr. Alford, Mr. Kiley, Mr. Smucker, Mr. McHenry, Mr. James, Ms. Stefanik, Mr. McCormick, Mrs. Miller of Illinois, Mr. Meuser, Mrs. Harshbarger, Ms. Davids of Kansas, Mr. Williams of New York, Mr. Krishnamoorthi, Mr. Morelle, Ms. Tenney, Mr. Posey, Mr. Weber of Texas, Mr. Schweikert, Mr. Gimenez, Mr. Vicente Gonzalez of Texas, Ms. Bush, Mrs. Hinson, Mr. DesJarlais, Mr. Miller of Ohio, Mr. Jackson of Texas, Mr. Boyle of Pennsylvania, Ms. Williams of Georgia, Mr. Robert Garcia of California, Ms. DeGette, Mrs. Luna, Mr. Carbajal, Mr. Thompson of Mississippi, Mr. Suozzi, Mr. Garamendi, Ms. Bonamici, Ms. Kaptur, Ms. McCollum, Ms. Jackson Lee, Mr. Cleaver, Mr. Espaillat, Ms. Moore of Wisconsin, Mr. Nadler, Mr. Sarbanes, Ms. Salinas, Mrs. Beatty, Mr. García of Illinois, Mr. Norcross, Ms. McClellan, Ms. Lee of California, Mr. Bilirakis, Ms. Schakowsky, Mr. Evans, Ms. Scholten, Mr. Levin, and Mr. Wittman
September 20, 2024
Reported with an amendment, committed to the Committee of the Whole House on the State of the Union, and ordered to be printed
[Strike out all after the enacting clause and insert the part printed in italic]
[For text of introduced bill, see copy of bill as introduced on May 17, 2023]
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,
(b) Table of contents.—The table of contents for this Act is as follows:
Sec. 1. Short title; table of contents.
Sec. 101. Research into pediatric uses of drugs; additional authorities of Food and Drug Administration regarding molecularly targeted cancer drugs.
Sec. 102. Ensuring completion of pediatric study requirements.
Sec. 103. FDA report on PREA enforcement.
Sec. 104. Extension of authority to issue priority review vouchers to encourage treatments for rare pediatric diseases.
Sec. 105. Limitations on exclusive approval or licensure of orphan drugs.
Sec. 106. Program for pediatric studies of drugs.
Sec. 201. Establishment of Abraham Accords Office within Food and Drug Administration.
Sec. 301. Registration fees.
(a) In general.—
(1) ADDITIONAL ACTIVE INGREDIENT FOR APPLICATION DRUG; LIMITATION REGARDING NOVEL-COMBINATION APPLICATION DRUG.—Section 505B(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(3)) is amended—
(B) by striking subparagraph (A) and inserting the following:
“(A) IN GENERAL.—For purposes of paragraph (1)(B), the investigation described in this paragraph is (as determined by the Secretary) a molecularly targeted pediatric cancer investigation of—
“(i) the drug or biological product for which the application referred to in such paragraph is submitted; or
“(ii) such drug or biological product in combination with—
“(I) an active ingredient of a drug or biological product—
“(II) an active ingredient of a drug or biological product—
“(B) ADDITIONAL REQUIREMENTS.—
“(i) DESIGN OF INVESTIGATION.—A molecularly targeted pediatric cancer investigation referred to in subparagraph (A) shall be designed to yield clinically meaningful pediatric study data that is 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.
“(ii) LIMITATION.—An investigation described in subparagraph (A)(ii) may be required only if the drug or biological product for which the application referred to in paragraph (1)(B) contains either—
(2) DETERMINATION OF APPLICABLE REQUIREMENTS.—Section 505B(e)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(e)(1)) is amended by adding at the end the following: “The Secretary shall determine whether subparagraph (A) or (B) of subsection (a)(1) shall apply with respect to an application before the date on which the applicant is required to submit the initial pediatric study plan under paragraph (2)(A).”.
(3) CLARIFYING APPLICABILITY.—Section 505B(a)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(1)) is amended by adding at the end the following:
“(C) RULE OF CONSTRUCTION.—No application that is subject to the requirements of subparagraph (B) shall be subject to the requirements of subparagraph (A), and no application (or supplement to an application) that is subject to the requirements of subparagraph (A) shall be subject to the requirements of subparagraph (B).”.
(4) CONFORMING AMENDMENTS.—Section 505B(a) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)) is amended—
(b) Guidance.—The Secretary of Health and Human Services, acting through the Commissioner of Food and Drugs, shall—
(c) Applicability.—The amendments made by this section apply with respect to any application under section 505(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(b)) and any application under section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)), that is submitted on or after the date that is 3 years after the date of enactment of this Act.
(d) Reports to Congress.—
(1) SECRETARY OF HEALTH AND HUMAN SERVICES.—Not later than 2 years after the date of enactment of this Act, the Secretary of Health and Human Services shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report on the Secretary’s efforts, in coordination with industry, to ensure implementation of the amendments made by subsection (a).
(2) GAO STUDY AND REPORT.—
(A) STUDY.—Not later than 3 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 subsection (a), in the development of drugs and biological products for pediatric cancer indications.
(B) FINDINGS.—Not later than 7 years after the date of enactment of this Act, the Comptroller General shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report containing the findings of the study conducted under subparagraph (A).
(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)—
(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
(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—
(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) only for failures described in section 505B(d) of such Act (21 U.S.C. 355c(d)) that occur on or after the date that is 180 days after the date of enactment of this Act.
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;”;
(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; and”.
(a) Extension.—Paragraph (5) of section 529(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(b)) is amended by striking “September 30, 2024, unless” and all that follows through the period at the end and inserting “September 30, 2029.”.
(b) GAO report on effectiveness of rare pediatric disease priority voucher awards in incentivizing rare pediatric disease drug development.—
(1) GAO STUDY.—
(A) STUDY.—The Comptroller General of the United States shall conduct a study of the effectiveness of awarding rare pediatric disease priority vouchers under section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff), as amended by subsection (a), in the development of human drug products that treat or prevent rare pediatric diseases (as defined in such section 529).
(B) CONTENTS OF STUDY.—In conducting the study under subparagraph (A), the Comptroller General shall examine the following:
(i) The indications for each drug or biological product that—
(I) is the subject of a rare pediatric disease product application (as defined in section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff)) for which a priority review voucher was awarded; and
(II) was approved under section 505 of the Federal Food, Drug, and Cosmetic Act (42 U.S.C. 355) or licensed under section 351 of the Public Health Service Act (42 U.S.C. 262).
(ii) Whether, and to what extent, an unmet need related to the treatment or prevention of a rare pediatric disease was met through the approval or licensure of such a drug or biological product.
(iii) The size of the company to which a priority review voucher was awarded under section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff) for such a drug or biological product.
(v) Identification of each drug for which a priority review voucher awarded under such section 529 was used.
(vii) The length of the period of time between the date on which a priority review voucher was awarded under such section 529 and the date on which it was used.
(viii) Whether, and to what extent, an unmet need related to the treatment or prevention of a rare pediatric disease was met through the approval under section 505 of the Federal Food, Drug, and Cosmetic Act (42 U.S.C. 355) or licensure under section 351 of the Public Health Service Act (42 U.S.C. 262) of a drug for which a priority review voucher was used.
(ix) Whether, and to what extent, companies were motivated by the availability of priority review vouchers under section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff) to attempt to develop a drug for a rare pediatric disease.
(x) Whether, and to what extent, pediatric review vouchers awarded under such section were successful in stimulating development and expedited patient access to drug products for treatment or prevention of a rare pediatric disease that wouldn’t otherwise take place without the incentive provided by such vouchers.
(2) REPORT ON FINDINGS.—Not later than 5 years after the date of the enactment of this Act, the Comptroller General of the United States shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report containing the findings of the study conducted under paragraph (1).
(a) In general.—Section 527 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360cc) is amended—
(1) in subsection (a), in the matter following paragraph (2), by striking “same disease or condition” and inserting “same approved use or indication within such rare disease or condition”;
(2) in subsection (b)—
(3) in subsection (c)(1), by striking “same rare disease or condition as the already approved drug” and inserting “same use or indication for which the already approved or licensed drug was approved or licensed”; and
(4) by adding at the end the following:
“(f) Approved use or indication defined.—In this section, the term ‘approved use or indication’ means the use or indication approved under section 505 of this Act or licensed under section 351 of the Public Health Service Act for a drug designated under section 526 for a rare disease or condition.”.
(b) Application of amendments.—The amendments made by subsection (a) shall apply with respect to any drug designated under section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb), regardless of the date on which the drug was so designated, and regardless of the date on which the drug was approved under section 505 of such Act (21 U.S.C. 355) or licensed under section 351 of the Public Health Service Act (42 U.S.C. 262).
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 2025, 2026, and 2027, the Director of NIH is authorized to make available up to one percent of such amount for pediatric research under this section.”.
(a) In general.—Chapter X of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 391 et seq.) is amended by adding at the end the following:
“SEC. 1015. Abraham Accords Office.
“(a) In general.—The Secretary, acting through the Commissioner of Food and Drugs, shall establish within the Food and Drug Administration an office, to be known as the Abraham Accords Office, to be headed by a director.
“(b) Office.—Not later than two years after the date of enactment of this section, the Secretary shall—
“(c) Duties.—The Secretary, acting through the Director of the Abraham Accords Office, shall—
“(1) after the Abraham Accords Office is established—
“(A) as part of the Food and Drug Administration’s work to strengthen the international oversight of regulated commodities, provide technical assistance to regulatory partners in Abraham Accords countries on strengthening regulatory oversight and converging regulatory requirements for the oversight of regulated products, including good manufacturing practices and other issues relevant to manufacturing medical products that are regulated by the Food and Drug Administration;
“(B) facilitate interactions between the Food and Drug Administration and interested parties in Abraham Accords countries, including by sharing relevant information regarding United States regulatory pathways with such parties; and
“(C) facilitate feedback between the Food and Drug Administration and such parties located within Abraham Accords countries prior to submission of an application under section 505(b), 505(j), or 515 of this Act or section 351(a) or 351(k) of the Public Health Service Act, or a notification under section 510(k) of this Act, such as feedback on research, development, and manufacturing of drugs, biologics, and medical devices; and
(b) Report to Congress.—
(1) IN GENERAL.—Not later than 3 years after the date of enactment of this Act, the Secretary of Health and Human Services shall submit to the Congress a report on the Abraham Accords Office, including—
(A) an evaluation of how the Office has advanced progress toward conformance with Food and Drug Administration regulatory requirements by manufacturers in the Abraham Accords countries;
(B) a numerical count of parties that the Office has helped facilitate interactions or feedback pursuant to subparagraphs (B) and (C) of section 1015(c)(1) of the Federal Food, Drug, and Cosmetic Act (as added by subsection (a));
Section 372 of the Public Health Service Act (42 U.S.C. 274) is amended by adding at the end the following:
“(d) Registration fees.—
“(1) IN GENERAL.—The Secretary may collect registration fees from any member of the Organ Procurement and Transplantation Network for each transplant candidate such member places on the list described in subsection (b)(2)(A)(i). Such registration fees shall only be collected and distributed to support the operation of the Organ Procurement and Transplantation Network. Such registration fees are authorized to remain available until expended.
“(2) COLLECTION.—The Secretary may collect the registration fees under paragraph (1) directly or through awards made under subsection (b)(1)(A).
“(3) DISTRIBUTION.—The Secretary may distribute such fees among the awardees described in subsection (b)(1)(A).
“(4) TRANSPARENCY.—The Secretary shall—
Union Calendar No. 594 | |||||
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[Report No. 118–700] | |||||
A BILL | |||||
To amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes. | |||||
September 20, 2024 | |||||
Reported with an amendment, committed to the Committee of the Whole House on the State of the Union, and ordered to be printed |