| 1 | 1 | | II |
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| 2 | 2 | | 119THCONGRESS |
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| 3 | 3 | | 1 |
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| 4 | 4 | | STSESSION S. 705 |
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| 5 | 5 | | To amend the Federal Food, Drug, and Cosmetic Act with respect to |
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| 6 | 6 | | molecularly targeted pediatric cancer investigations, and for other purposes. |
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| 7 | 7 | | IN THE SENATE OF THE UNITED STATES |
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| 8 | 8 | | FEBRUARY25, 2025 |
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| 9 | 9 | | Mr. R |
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| 10 | 10 | | EED(for himself and Mrs. CAPITO) introduced the following bill; which |
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| 11 | 11 | | was read twice and referred to the Committee on Health, Education, |
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| 12 | 12 | | Labor, and Pensions |
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| 13 | 13 | | A BILL |
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| 14 | 14 | | To amend the Federal Food, Drug, and Cosmetic Act with |
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| 15 | 15 | | respect to molecularly targeted pediatric cancer investiga- |
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| 16 | 16 | | tions, and for other purposes. |
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| 17 | 17 | | Be it enacted by the Senate and House of Representa-1 |
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| 18 | 18 | | tives of the United States of America in Congress assembled, 2 |
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| 19 | 19 | | SECTION 1. SHORT TITLE. 3 |
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| 20 | 20 | | This Act may be cited as the ‘‘Innovation in Pediatric 4 |
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| 21 | 21 | | Drugs Act of 2025’’. 5 |
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| 22 | 22 | | SEC. 2. ENSURING COMPLETION OF PEDIATRIC STUDY RE-6 |
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| 23 | 23 | | QUIREMENTS. 7 |
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| 24 | 24 | | (a) E |
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| 25 | 25 | | QUALACCOUNTABILITY FOR PEDIATRICSTUDY 8 |
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| 26 | 26 | | R |
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| 27 | 27 | | EQUIREMENTS.—Section 505B(d) of the Federal Food, 9 |
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| 30 | 30 | | •S 705 IS |
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| 31 | 31 | | Drug, and Cosmetic Act (21 U.S.C. 355c(d)) is amend-1 |
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| 32 | 32 | | ed— 2 |
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| 33 | 33 | | (1) in paragraph (1), by striking ‘‘Beginning 3 |
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| 34 | 34 | | 270’’ and inserting ‘‘N |
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| 35 | 35 | | ONCOMPLIANCE LETTER .— 4 |
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| 36 | 36 | | Beginning 270’’; 5 |
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| 37 | 37 | | (2) in paragraph (2)— 6 |
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| 38 | 38 | | (A) by striking ‘‘The drug or’’ and insert-7 |
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| 39 | 39 | | ing ‘‘E |
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| 40 | 40 | | FFECT OF NONCOMPLIANCE .—The drug 8 |
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| 41 | 41 | | or’’; and 9 |
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| 42 | 42 | | (B) by striking ‘‘(except that the drug or 10 |
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| 43 | 43 | | biological product shall not be subject to action 11 |
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| 44 | 44 | | under section 303)’’ and inserting ‘‘(except that 12 |
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| 45 | 45 | | the drug or biological product shall be subject 13 |
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| 46 | 46 | | to action under section 303 only if such person 14 |
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| 47 | 47 | | demonstrated a lack of due diligence in satis-15 |
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| 48 | 48 | | fying the applicable requirement)’’; and 16 |
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| 49 | 49 | | (3) by adding at the end the following: 17 |
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| 50 | 50 | | ‘‘(3) L |
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| 51 | 51 | | IMITATION.—The Secretary shall not 18 |
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| 52 | 52 | | issue enforcement actions under section 303 for fail-19 |
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| 53 | 53 | | ures under this subsection in the case of a drug or 20 |
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| 54 | 54 | | biological product that is no longer marketed.’’. 21 |
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| 55 | 55 | | (b) D |
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| 56 | 56 | | UEDILIGENCE.—Section 505B(d) of the Fed-22 |
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| 57 | 57 | | eral Food, Drug, and Cosmetic Act (21 U.S.C. 355c(d)), 23 |
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| 58 | 58 | | as amended by subsection (a), is further amended by add-24 |
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| 59 | 59 | | ing at the end the following: 25 |
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| 62 | 62 | | •S 705 IS |
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| 63 | 63 | | ‘‘(4) DUE DILIGENCE.—Before the Secretary 1 |
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| 64 | 64 | | may conclude that a person failed to submit or oth-2 |
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| 65 | 65 | | erwise meet a requirement as described in the mat-3 |
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| 66 | 66 | | ter preceding paragraph (1), the Secretary shall— 4 |
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| 67 | 67 | | ‘‘(A) issue a noncompliance letter pursuant 5 |
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| 68 | 68 | | to paragraph (1); 6 |
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| 69 | 69 | | ‘‘(B) provide such person with a 45-day 7 |
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| 70 | 70 | | period beginning on the date of receipt of such 8 |
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| 71 | 71 | | noncompliance letter to respond in writing as 9 |
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| 72 | 72 | | set forth in such paragraph; and 10 |
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| 73 | 73 | | ‘‘(C) after reviewing such written response, 11 |
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| 74 | 74 | | determine whether the person demonstrated a 12 |
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| 75 | 75 | | lack of due diligence in satisfying such require-13 |
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| 76 | 76 | | ment.’’. 14 |
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| 77 | 77 | | (c) C |
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| 78 | 78 | | ONFORMING AMENDMENTS.—Section 15 |
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| 79 | 79 | | 303(f)(4)(A) of the Federal Food, Drug, and Cosmetic Act 16 |
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| 80 | 80 | | (21 U.S.C. 333(f)(4)(A)) is amended by striking ‘‘or 505– 17 |
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| 81 | 81 | | 1’’ and inserting ‘‘505–1, or 505B’’. 18 |
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| 82 | 82 | | (d) T |
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| 83 | 83 | | RANSITIONRULE.—The Secretary of Health 19 |
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| 84 | 84 | | and Human Services may take enforcement action under 20 |
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| 85 | 85 | | section 303 of the Federal Food, Drug, and Cosmetic Act 21 |
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| 86 | 86 | | (21 U.S.C. 333) for failures described in section 505B(d) 22 |
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| 87 | 87 | | of such Act (21 U.S.C. 355c(d)) only if such failures occur 23 |
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| 88 | 88 | | on or after the date that is 180 days after the date of 24 |
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| 89 | 89 | | enactment of this Act. 25 |
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| 92 | 92 | | •S 705 IS |
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| 93 | 93 | | SEC. 3. FDA REPORT ON PREA ENFORCEMENT. 1 |
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| 94 | 94 | | Section 508(b) of the Food and Drug Administration 2 |
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| 95 | 95 | | Safety and Innovation Act (21 U.S.C. 355c–1(b)) is 3 |
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| 96 | 96 | | amended— 4 |
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| 97 | 97 | | (1) in paragraph (11), by striking the semicolon 5 |
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| 98 | 98 | | at the end and inserting ‘‘, including an evaluation 6 |
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| 99 | 99 | | of compliance with deadlines provided for in defer-7 |
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| 100 | 100 | | rals and deferral extensions;’’; 8 |
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| 101 | 101 | | (2) in paragraph (15), by striking ‘‘and’’ at the 9 |
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| 102 | 102 | | end; 10 |
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| 103 | 103 | | (3) in paragraph (16), by striking the period at 11 |
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| 104 | 104 | | the end and inserting ‘‘; and’’; and 12 |
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| 105 | 105 | | (4) by adding at the end the following: 13 |
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| 106 | 106 | | ‘‘(17) a listing of penalties, settlements, or pay-14 |
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| 107 | 107 | | ments under section 303 of the Federal Food, Drug, 15 |
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| 108 | 108 | | and Cosmetic Act (21 U.S.C. 353) for failure to 16 |
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| 109 | 109 | | comply with requirements under such section 505B, 17 |
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| 110 | 110 | | including, for each penalty, settlement, or payment, 18 |
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| 111 | 111 | | the name of the drug, the sponsor thereof, and the 19 |
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| 112 | 112 | | amount of the penalty, settlement, or payment im-20 |
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| 113 | 113 | | posed.’’. 21 |
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| 114 | 114 | | SEC. 4. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS. 22 |
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| 115 | 115 | | Section 409I(d) of the Public Health Service Act (42 23 |
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| 116 | 116 | | U.S.C. 284m(d)) is amended to read as follows: 24 |
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| 117 | 117 | | ‘‘(d) F |
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| 118 | 118 | | UNDING.—Of the amount made available for 25 |
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| 119 | 119 | | pediatric research to each national research institute and 26 |
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| 122 | 122 | | •S 705 IS |
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| 123 | 123 | | national center under this title for each of fiscal years 1 |
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| 124 | 124 | | 2026 through 2030, the Director of NIH is authorized to 2 |
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| 125 | 125 | | make available up to one percent of such amount for pedi-3 |
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| 126 | 126 | | atric research under this section.’’. 4 |
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| 127 | 127 | | SEC. 5. PEDIATRIC STUDIES OF ORPHAN DRUGS. 5 |
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| 128 | 128 | | (a) A |
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| 129 | 129 | | PPLICATION OF PEDIATRICRESEARCHRE-6 |
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| 130 | 130 | | QUIREMENTS TOORPHANDRUGS.— 7 |
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| 131 | 131 | | (1) I |
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| 132 | 132 | | N GENERAL.—Section 505B(k) of the Fed-8 |
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| 133 | 133 | | eral Food, Drug, and Cosmetic Act (21 U.S.C. 9 |
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| 134 | 134 | | 355c(k)) is amended to read as follows: 10 |
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| 135 | 135 | | ‘‘(k) R |
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| 136 | 136 | | ELATION TOORPHANDRUGS.— 11 |
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| 137 | 137 | | ‘‘(1) I |
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| 138 | 138 | | N GENERAL.—This section does not apply 12 |
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| 139 | 139 | | to a drug or biological product for an indication for 13 |
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| 140 | 140 | | which orphan designation has been granted under 14 |
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| 141 | 141 | | section 526 unless the Secretary determines that pe-15 |
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| 142 | 142 | | diatric assessments of such drug or biological prod-16 |
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| 143 | 143 | | uct required under this section could represent a 17 |
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| 144 | 144 | | meaningful therapeutic benefit as described in sub-18 |
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| 145 | 145 | | section (c). 19 |
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| 146 | 146 | | ‘‘(2) D |
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| 147 | 147 | | EFERRALS AND WAIVERS .—Deferrals 20 |
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| 148 | 148 | | and waivers under subsections (a)(4) and (a)(5) 21 |
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| 149 | 149 | | shall apply to assessments described in this sub-22 |
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| 150 | 150 | | section to the same extent and in the same manner 23 |
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| 151 | 151 | | as such deferrals and waivers apply with respect to 24 |
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| 152 | 152 | | the assessments under subsection (a)(1), and waiv-25 |
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| 155 | 155 | | •S 705 IS |
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| 156 | 156 | | ers under subsection (b)(2) shall apply to assess-1 |
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| 157 | 157 | | ments described in this subsection to the same ex-2 |
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| 158 | 158 | | tent and in the same manner as such waivers apply 3 |
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| 159 | 159 | | with respect to the assessments required pursuant to 4 |
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| 160 | 160 | | subsection (b)(1).’’. 5 |
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| 161 | 161 | | (2) A |
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| 162 | 162 | | PPLICABILITY.—The amendment made by 6 |
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| 163 | 163 | | paragraph (1) applies only to applications described 7 |
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| 164 | 164 | | in subparagraph (A) or (B) of section 505B(a)(1) of 8 |
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| 165 | 165 | | the Federal Food, Drug, and Cosmetic Act (21 9 |
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| 166 | 166 | | U.S.C. 355c(a)(1)) that are submitted on or after 10 |
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| 167 | 167 | | the later of— 11 |
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| 168 | 168 | | (A) the date that is 18 months after the 12 |
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| 169 | 169 | | date of issuance of the final guidance under 13 |
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| 170 | 170 | | subsection (b); and 14 |
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| 171 | 171 | | (B) such later date as may be specified by 15 |
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| 172 | 172 | | the Secretary of Health and Human Services 16 |
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| 173 | 173 | | (referred to in this section as the ‘‘Secretary’’) 17 |
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| 174 | 174 | | by regulation. 18 |
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| 175 | 175 | | (b) G |
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| 176 | 176 | | UIDANCE.— 19 |
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| 177 | 177 | | (1) I |
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| 178 | 178 | | SSUANCE.—The Secretary shall— 20 |
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| 179 | 179 | | (A) not later than 1 year after the date of 21 |
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| 180 | 180 | | enactment of this Act, issue draft guidance de-22 |
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| 181 | 181 | | scribing how, upon the applicability of the 23 |
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| 182 | 182 | | amendment made by subsection (a)(1), the re-24 |
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| 183 | 183 | | quirements of section 505B of the Federal 25 |
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| 186 | 186 | | •S 705 IS |
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| 187 | 187 | | Food, Drug, and Cosmetic Act (21 U.S.C. 1 |
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| 188 | 188 | | 355c) will apply with respect to any drug or bi-2 |
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| 189 | 189 | | ological product for an indication within a dis-3 |
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| 190 | 190 | | ease or condition for which orphan designation 4 |
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| 191 | 191 | | has been granted under section 526 of such Act 5 |
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| 192 | 192 | | (21 U.S.C. 360bb); and 6 |
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| 193 | 193 | | (B) not later than 18 months after the 7 |
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| 194 | 194 | | date of the public meeting required by sub-8 |
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| 195 | 195 | | section (c)(1), finalize such draft guidance. 9 |
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| 196 | 196 | | (2) C |
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| 197 | 197 | | ONTENTS.—The guidance under sub-10 |
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| 198 | 198 | | section (b) shall address the following: 11 |
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| 199 | 199 | | (A) Information regarding how full and 12 |
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| 200 | 200 | | partial waivers under subsections (a)(5)(A), 13 |
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| 201 | 201 | | (a)(5)(B), and (b)(2) of section 505B of the 14 |
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| 202 | 202 | | Federal Food, Drug, and Cosmetic Act (21 15 |
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| 203 | 203 | | U.S.C. 355c) for any drug or biological product 16 |
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| 204 | 204 | | for an indication within a disease or condition 17 |
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| 205 | 205 | | for which orphan designation has been granted 18 |
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| 206 | 206 | | under section 526 of such Act (21 U.S.C. 19 |
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| 207 | 207 | | 360bb) will be granted. 20 |
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| 208 | 208 | | (B) Application of the requirements of sec-21 |
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| 209 | 209 | | tion 505B(e) of such Act (21 U.S.C. 355c(e)) 22 |
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| 210 | 210 | | to drugs or biological products for an indication 23 |
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| 211 | 211 | | within a disease or condition for which orphan 24 |
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| 212 | 212 | | designation has been granted under section 526 25 |
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| 215 | 215 | | •S 705 IS |
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| 216 | 216 | | of such Act (21 U.S.C. 360bb), including sub-1 |
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| 217 | 217 | | mission and timing of planned requests for full 2 |
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| 218 | 218 | | or partial waivers and responses by the Food 3 |
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| 219 | 219 | | and Drug Administration to those requests. 4 |
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| 220 | 220 | | (C) Rare diseases and conditions (as de-5 |
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| 221 | 221 | | fined in section 526(a)(2) of such Act (21 6 |
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| 222 | 222 | | U.S.C. 360bb(a)(2)) that should be added to 7 |
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| 223 | 223 | | the lists under section 505B(a)(5)(E) and 8 |
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| 224 | 224 | | 505B(b)(2)(E) of such Act, as added by sub-9 |
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| 225 | 225 | | section (f), and a process for regularly updating 10 |
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| 226 | 226 | | such lists. 11 |
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| 227 | 227 | | (D) Situations where the initial pediatric 12 |
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| 228 | 228 | | study plan under section 505B(e) of such Act 13 |
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| 229 | 229 | | (21 U.S.C. 355c(e)) includes a plan to fulfill 14 |
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| 230 | 230 | | the requirements of section 505B(a) of such 15 |
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| 231 | 231 | | Act (21 U.S.C. 355c(a)) without requesting 16 |
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| 232 | 232 | | waivers in any age group. 17 |
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| 233 | 233 | | (E) Consideration of how the Secretary 18 |
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| 234 | 234 | | will balance the unique scientific challenges of 19 |
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| 235 | 235 | | rare disease drug development with the need for 20 |
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| 236 | 236 | | improved pediatric labeling of drugs and bio-21 |
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| 237 | 237 | | logical products for indications within diseases 22 |
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| 238 | 238 | | or conditions for which orphan designation has 23 |
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| 239 | 239 | | been granted under section 526 of such Act (21 24 |
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| 240 | 240 | | U.S.C. 360bb). 25 |
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| 243 | 243 | | •S 705 IS |
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| 244 | 244 | | (F) Considerations of the strengths, weak-1 |
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| 245 | 245 | | nesses, appropriateness, and limitations of dif-2 |
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| 246 | 246 | | ferent types of real-world evidence specific to 3 |
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| 247 | 247 | | the fulfillment of requirements under section 4 |
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| 248 | 248 | | 505B of such Act (21 U.S.C. 355c). 5 |
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| 249 | 249 | | (G) Consideration of input received from 6 |
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| 250 | 250 | | the public meeting set forth in subsection (c). 7 |
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| 251 | 251 | | (c) P |
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| 252 | 252 | | UBLICMEETING.—The Secretary shall— 8 |
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| 253 | 253 | | (1) not later than 6 months after the date of 9 |
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| 254 | 254 | | issuance of the draft guidance under subsection 10 |
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| 255 | 255 | | (b)(1)(A), hold a public meeting to inform the final 11 |
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| 256 | 256 | | guidance to be issued under subsection (b)(1)(B); 12 |
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| 257 | 257 | | and 13 |
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| 258 | 258 | | (2) publish prior notice of such meeting in the 14 |
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| 259 | 259 | | Federal Register. 15 |
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| 260 | 260 | | (d) GAO S |
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| 261 | 261 | | TUDY.—Not later than 4 years after the 16 |
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| 262 | 262 | | applicability date described in subsection (a)(2), the 17 |
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| 263 | 263 | | Comptroller General of the United States shall submit to 18 |
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| 264 | 264 | | the Committee on Energy and Commerce and the Com-19 |
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| 265 | 265 | | mittee on Ways and Means of the House of Representa-20 |
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| 266 | 266 | | tives and the Committee on Health, Education, Labor, 21 |
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| 267 | 267 | | and Pensions of the Senate a report that— 22 |
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| 268 | 268 | | (1) addresses the impacts of this Act on— 23 |
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| 269 | 269 | | (A) rare disease drug development in the 24 |
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| 270 | 270 | | United States; and 25 |
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| 274 | 274 | | (B) the availability of pediatric information 1 |
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| 275 | 275 | | on drugs and biological products within diseases 2 |
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| 276 | 276 | | or conditions for indications for which orphan 3 |
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| 277 | 277 | | designation has been granted under section 526 4 |
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| 278 | 278 | | of the Federal Food, Drug, and Cosmetic Act 5 |
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| 279 | 279 | | (21 U.S.C. 360bb); and 6 |
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| 280 | 280 | | (2) includes— 7 |
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| 281 | 281 | | (A) the findings of a survey of companies 8 |
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| 282 | 282 | | of varying sizes engaged in the development of 9 |
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| 283 | 283 | | orphan drugs, which shall include questions re-10 |
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| 284 | 284 | | garding the feasibility and other challenges of 11 |
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| 285 | 285 | | conducting pediatric studies for such indica-12 |
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| 286 | 286 | | tions; 13 |
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| 287 | 287 | | (B) input from patient groups and medical 14 |
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| 288 | 288 | | provider associations; and 15 |
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| 289 | 289 | | (C) an assessment of the impact changes 16 |
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| 290 | 290 | | to required pediatric studies had on drug devel-17 |
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| 291 | 291 | | opment for rare diseases. 18 |
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| 292 | 292 | | (e) R |
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| 293 | 293 | | ULE OFCONSTRUCTION.—Nothing in this sec-19 |
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| 294 | 294 | | tion shall be construed to limit requirements for investiga-20 |
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| 295 | 295 | | tions, as described in section 505B(a)(3) of the Federal 21 |
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| 296 | 296 | | Food, Drug, and Cosmetic Act (21 U.S.C. 355c(a)(3)), of 22 |
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| 297 | 297 | | molecularly targeted pediatric cancer drugs for which or-23 |
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| 298 | 298 | | phan designation has been granted under section 526 of 24 |
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| 299 | 299 | | such Act (21 U.S.C. 360bb). 25 |
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| 302 | 302 | | •S 705 IS |
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| 303 | 303 | | (f) CERTAINTYREGARDING WAIVERS.—Section 1 |
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| 304 | 304 | | 505B of the Federal Food, Drug, and Cosmetic Act (21 2 |
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| 305 | 305 | | U.S.C. 355c) is amended— 3 |
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| 306 | 306 | | (1) in subsection (a)(5), by adding at the end 4 |
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| 307 | 307 | | the following: 5 |
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| 308 | 308 | | ‘‘(E) A |
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| 309 | 309 | | UTOMATIC FULL WAIVER LIST .— 6 |
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| 310 | 310 | | The Secretary shall maintain a list, posted on 7 |
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| 311 | 311 | | the website of the Food and Drug Administra-8 |
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| 312 | 312 | | tion, of adult-related diseases and conditions— 9 |
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| 313 | 313 | | ‘‘(i) with respect to which the nec-10 |
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| 314 | 314 | | essary studies are impossible or highly im-11 |
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| 315 | 315 | | practicable, as described in subparagraph 12 |
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| 316 | 316 | | (A)(i); or 13 |
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| 317 | 317 | | ‘‘(ii) for which a drug or biological 14 |
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| 318 | 318 | | product for such disease or condition oth-15 |
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| 319 | 319 | | erwise meets the criteria described in sub-16 |
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| 320 | 320 | | paragraph (A).’’; 17 |
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| 321 | 321 | | (2) in subsection (b)(2), by adding at the end 18 |
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| 322 | 322 | | the following: 19 |
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| 323 | 323 | | ‘‘(E) A |
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| 324 | 324 | | UTOMATIC FULL WAIVER LIST .— 20 |
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| 325 | 325 | | The Secretary shall maintain a list, posted on 21 |
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| 326 | 326 | | the website of the Food and Drug Administra-22 |
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| 327 | 327 | | tion, of adult-related diseases and conditions 23 |
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| 328 | 328 | | with respect to which the necessary studies 24 |
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| 329 | 329 | | VerDate Sep 11 2014 20:53 Mar 13, 2025 Jkt 059200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\S705.IS S705 |
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| 330 | 330 | | ssavage on LAPJG3WLY3PROD with BILLS 12 |
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| 331 | 331 | | •S 705 IS |
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| 332 | 332 | | would meet the criteria for a full waiver under 1 |
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| 333 | 333 | | subparagraph (A).’’; and 2 |
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| 334 | 334 | | (3) in subsection (e)(4), by adding at the end 3 |
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| 335 | 335 | | the following: ‘‘If, at the time of an applicant’s sub-4 |
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| 336 | 336 | | mission of the initial pediatric study plan, the dis-5 |
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| 337 | 337 | | ease or condition for which the drug is intended to 6 |
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| 338 | 338 | | treat appears on the list under subsection (a)(5)(E), 7 |
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| 339 | 339 | | then the assessments for such disease or condition 8 |
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| 340 | 340 | | shall be waived under subsection (a)(5).’’. 9 |
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| 341 | 341 | | Æ |
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| 342 | 342 | | VerDate Sep 11 2014 20:53 Mar 13, 2025 Jkt 059200 PO 00000 Frm 00012 Fmt 6652 Sfmt 6301 E:\BILLS\S705.IS S705 |
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| 343 | 343 | | ssavage on LAPJG3WLY3PROD with BILLS |
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