Conditional Approval Act

II 116TH CONGRESS 1ST SESSION S. 3133 To amend the Federal Food, Drug, and Cosmetic Act to establish a time- limited conditional approval pathway, subject to specific obligations, for certain drugs, and for other purposes. IN THE SENATE OF THE UNITED STATES DECEMBER 19, 2019 Mr. BRAUN 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 to establish a time-limited conditional approval pathway, subject to specific obligations, for certain drugs, and for other purposes. Be it enacted by the Senate and House of Representa- 1 tives of the United States of America in Congress assembled, 2 SECTION 1. SHORT TITLE. 3 This Act may be cited as the ‘‘Conditional Approval 4 Act’’. 5 VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS 2 •S 3133 IS SEC. 2. CONDITIONAL APPROVAL OF NEW HUMAN DRUGS. 1 (a) IN GENERAL.—Subchapter A of chapter V of the 2 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 3 et seq.) is amended by adding at the end of the following: 4 ‘‘SEC. 524B. CONDITIONAL AND TIME LIMITED APPROVAL 5 PATHWAY FOR NEW DRUGS. 6 ‘‘(a) PATHWAY REQUIREMENTS.—The Secretary 7 shall, at the request of the sponsor of a new drug, grant 8 provisional and time-limited approval of such drug under 9 this section, if the Secretary determines that— 10 ‘‘(1) it is likely that the sponsor will be able to 11 provide comprehensive clinical data after such drug 12 is conditionally approved; 13 ‘‘(2) such drug is intended for the treatment, 14 prevention, or medical diagnosis of a seriously debili- 15 tating disease, a life-threatening disease, or a chron- 16 ic condition; 17 ‘‘(3) the expected benefits of the drug outweigh 18 the potential risks to patients, taking into account 19 the fact that additional data are still required to as- 20 sess the drug and the severity of the underlying dis- 21 ease or condition the drug is intended to treat; 22 ‘‘(4) there are no existing meaningful treat- 23 ments for the disease or condition that the drug is 24 intended to treat; 25 VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS 3 •S 3133 IS ‘‘(5) confirmatory clinical trials are difficult or 1 costly to conduct; and 2 ‘‘(6) such drug is intended to treat a disease or 3 condition for which no more than 2 meaningful 4 treatments currently exist. 5 ‘‘(b) APPROVAL REQUIREMENTS.— 6 ‘‘(1) IN GENERAL.—Not later than 180 days 7 after the date on which the Secretary receives a re- 8 quest for conditional approval under subsection (a) 9 with respect to a new drug, the Secretary shall re- 10 quire the sponsor of such drug to— 11 ‘‘(A) complete in a timely manner clinical 12 investigations to provide full demonstration of 13 safety and effectiveness as described in section 14 505 of the Federal Food, Drug, and Cosmetic 15 Act or section 351 of the Public Health Service 16 Act, as applicable; 17 ‘‘(B) conduct clinical trials other than con- 18 firmatory trials, to demonstrate a certain de- 19 gree of safety and efficacy of the drug; and 20 ‘‘(C) demonstrate that necessary post-mar- 21 ket surveillance and risk-management tools are 22 in place with respect to the drug. 23 ‘‘(2) PERIOD OF CONDITIONAL APPROVAL.— 24 The period of conditional approval for a drug under 25 VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS 4 •S 3133 IS this subsection is effective for 1 year, and is renew- 1 able by the Secretary for up to 4 additional 1-year 2 terms. A conditional approval shall be in effect for 3 not more than 5 years from the date on which condi- 4 tional approval is first granted. 5 ‘‘(3) TIME LIMITATION.—If any conditionally 6 drug approved under this section is not brought to 7 market within 3 years of the conditional approval, 8 any conditional approval granted under this section 9 with respect to such drug shall be deemed invalid. 10 ‘‘(4) REQUIREMENTS.—As a condition on re- 11 ceipt of conditional approval under this section, the 12 Secretary shall require the sponsor of the drug to 13 agree to the following: 14 ‘‘(A) Complete in a timely manner such 15 clinical investigations to provide a full dem- 16 onstration of effectiveness as the Secretary de- 17 termines to be necessary for approval of the 18 drug under section 505 of this Act or section 19 351 of the Public Health Service Act, as appli- 20 cable. 21 ‘‘(B) Submit to the Secretary an annual 22 report on the progress of the sponsor in con- 23 ducting the clinical investigations required 24 under this section. 25 VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS 5 •S 3133 IS ‘‘(C) Ensure that all labeling and pro- 1 motional materials for the drug bear the state- 2 ment ‘conditionally approved by the FDA pend- 3 ing a full demonstration of effectiveness under 4 applicable lll’ (specifying the application 5 number assigned by the Secretary in place of 6 the blank). 7 ‘‘(5) APPLYING FOR FULL APPROVAL.—The 8 sponsor of a drug granted conditional approval pur- 9 suant to this subsection may, at any point, submit 10 an application for full approval as described under 11 section 505 of the Federal Food, Drug, and Cos- 12 metic Act or section 351 of the Public Health Serv- 13 ice Act, as applicable. 14 ‘‘(6) UTILIZATION OF REAL WORLD EVIDENCE 15 TO SUPPORT FULL APPROVAL.—The Secretary shall 16 allow the use of real world evidence, as defined in 17 section 505F(b), and collected by the sponsor of a 18 drug during the duration of conditional approval 19 granted approval to this subsection, to supplement 20 an application for full approval, in addition to other 21 post-approval studies. 22 ‘‘(c) LIMITATION ON LIABILITY.— 23 ‘‘(1) IN GENERAL.—With respect to any claim 24 under State law alleging that a drug sold or other- 25 VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS 6 •S 3133 IS wise made available pursuant to a grant of condi- 1 tional approval under this subsection is unsafe or in- 2 effective, no liability in a cause of action shall lie 3 against a sponsor or manufacturer, unless the rel- 4 evant conduct constitutes reckless or willful mis- 5 conduct, gross negligence, or an intention tort under 6 any applicable State law. 7 ‘‘(2) RULE OF CONSTRUCTION.—Except as set 8 forth in subparagraph (A), nothing in this sub- 9 section shall be construed to modify or otherwise af- 10 fect the right of any person to bring private action 11 under any Federal or State product liability, tort, 12 consumer protection, or warranty law. 13 ‘‘(d) DEFINITIONS.—In this section: 14 ‘‘(1) SERIOUSLY DEBILITATING DISEASE.—The 15 term ‘severely debilitating disease’ means a disease 16 or condition that causes major irreversible mor- 17 bidity. 18 ‘‘(2) LIFE-THREATENING DISEASE.—The term 19 ‘life-threatening disease’ means— 20 ‘‘(A) a disease or condition where the like- 21 lihood of death is high unless the course of the 22 disease is interrupted; or 23 VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS 7 •S 3133 IS ‘‘(B) a disease or condition with potentially 1 fatal outcomes, where the end point of clinical 2 trial analysis is survival. 3 ‘‘(3) CHRONIC CONDITION.—The term ‘chronic 4 condition’ means a disease or condition that— 5 ‘‘(A) usually lasts for 3 months or longer; 6 and 7 ‘‘(B)(i) requires ongoing medical attention; 8 or 9 ‘‘(ii) limits activities of daily living.’’. 10 (b) REGULATIONS AND GUIDANCE.—Not later than 11 1 year after the date of the enactment of this Act, the 12 Secretary of Health and Human Services shall issue final 13 regulations and guidance for carrying out section 524B 14 of the Federal Food, Drug, and Cosmetic Act, as added 15 by subsection (a). 16 (c) CONFORMING AMENDMENT.—Section 505(a) of 17 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 18 355(a)) is amended by inserting ‘‘, or there is in effect 19 a conditional approval under section 524B with respect 20 to such drug’’ before the period. 21 Æ VerDate Sep 11 2014 15:58 Dec 28, 2019 Jkt 099200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6301 E:\BILLS\S3133.IS S3133 pamtmann on DSKBC07HB2PROD with BILLS