II 

118TH CONGRESS 

2D SESSION S. 4426 

To amend the Federal Food, Drug, and Cosmetic Act to establish a timelimited conditional approval pathway, subject to specific obligations, for 

certain drugs and biological products, and for other purposes. 

IN THE SENATE OF THE UNITED STATES 

MAY 23, 2024 

Mr. BRAUN (for himself, Mrs. GILLIBRAND, Mr. CRAMER, Mr. MANCHIN, Mr. 

SCHMITT, Mr. PADILLA, Mr. VANCE, Mr. BOOKER, Mr. HAWLEY, Mr. 

WELCH, and Ms. MURKOWSKI) 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 biological products, and for other purposes. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Promising Pathway 

5 Act 2.0’’. 


ssavage on LAPJG3WLY3PROD with BILLS
2 

•S 4426 IS

1 SEC. 2. CONDITIONAL APPROVAL OF NEW HUMAN DRUGS 

2 FOR INDIVIDUALS WITH RARE, PROGRES3 SIVE, AND SERIOUS DISEASES. 

4 (a) IN GENERAL.—Subchapter A of chapter V of the 

5 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351 

6 et seq.) is amended by adding at the end of the following: 

7 ‘‘SEC. 524C. CONDITIONAL APPROVAL OF HUMAN DRUGS 

8 FOR INDIVIDUALS WITH RARE, PROGRES9 SIVE, AND SERIOUS DISEASES. 

10 ‘‘(a) CONDITIONAL APPROVAL; PRIORITY REVIEW; 

11 OTHER DESIGNATIONS.— 

12 ‘‘(1) IN GENERAL.—The sponsor of a drug may 

13 file with the Secretary an application for conditional 

14 approval of an eligible drug described in subsection 

15 (b). The Secretary shall approve or deny such appli16 cation in accordance with subsection (c). 

17 ‘‘(2) PRIORITY REVIEW.—The Secretary shall 

18 give priority review to an application for conditional 

19 approval of an eligible drug described in subsection 

20 (b). 

21 ‘‘(3) OTHER DESIGNATIONS.—If a drug that is 

22 granted conditional approval under this section is el23 igible for a special designation by the Secretary 

24 under this Act, including as a drug for a rare dis25 ease or condition under section 526, all applicable 

26 benefits of such other designation shall be available 


ssavage on LAPJG3WLY3PROD with BILLS
3 

•S 4426 IS

1 for use under such conditional approval, including 

2 any tax credits and waiving of fees under chapter 

3 VII. 

4 ‘‘(4) OTHER PROGRAMS.—A sponsor of a drug 

5 seeking conditional approval of such drug under this 

6 section may also seek designation, exclusivity, or ap7 proval, as applicable, of such drug under other appli8 cable provisions of this Act or the Public Health 

9 Service Act, subject to the requirements of such pro10 visions. 

11 ‘‘(b) ELIGIBILITY.— 

12 ‘‘(1) IN GENERAL.—A drug may be eligible for 

13 conditional approval under this section if such drug 

14 is intended to treat a disease or condition that is— 

15 ‘‘(A) rapidly progressive, terminal, and has 

16 substantial unmet medical need, as determined 

17 by the Secretary; or 

18 ‘‘(B) a rare disease or condition (as de19 fined in section 526(a)(2)) that results in a 

20 substantially shortened lifespan, substantial re21 duction in quality of life, or other substantial 

22 adverse health effects, as determined by the 

23 Secretary. 

24 ‘‘(2) EXCLUSION FROM ELIGIBILITY.—A drug 

25 that is intended to treat or respond to a material 


ssavage on LAPJG3WLY3PROD with BILLS
4 

•S 4426 IS

1 threat identified by the Secretary of Homeland Secu2 rity under section 319F–2(c)(2)(A)(ii) shall not be 

3 eligible for conditional approval under this section. 

4 ‘‘(c) STANDARD OF REVIEW FOR CONDITIONAL AP5 PROVAL.— 

6 ‘‘(1) REQUIREMENTS.—The Secretary shall 

7 only approve an application for conditional approval 

8 of a drug under this section if— 

9 ‘‘(A) the Secretary determines that— 

10 ‘‘(i)(I) evidence of safety for the drug 

11 has been established by— 

12 ‘‘(aa) the completion of a phase 1 

13 clinical investigation of the drug (as 

14 described in section 312.21 of title 21, 

15 Code of Federal Regulations (or suc16 cessor regulations)); or 

17 ‘‘(bb) another demonstration of 

18 safety, as determined appropriate by 

19 the Secretary; and 

20 ‘‘(II) evidence of effectiveness in 

21 treating a given indication (which indica22 tion is congruent with the eligibility re23 quirements of subsection (b)), as estab24 lished by an ongoing or completed phase 2 

25 clinical investigation of the drug (as deVerDate Sep 11 2014 20:07 Jul 16, 2024 Jkt 049200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S4426.IS S4426

ssavage on LAPJG3WLY3PROD with BILLS
5 

•S 4426 IS

1 scribed in section 312.21 of title 21, Code 

2 of Federal Regulations (or successor regu3 lations)); or 

4 ‘‘(ii) in the case of a drug that is in5 tended to treat a terminal pediatric rare 

6 disease or condition (as defined in section 

7 526(a)(2)) that does not predominately af8 fect adults— 

9 ‘‘(I) evidence of safety for the 

10 drug has been established in accord11 ance with clause (i)(I); and 

12 ‘‘(II) the drug shows preliminary 

13 evidence of clinical effectiveness based 

14 upon studies in animal models; and 

15 ‘‘(B) the sponsor has provided a written 

16 affirmation of the sponsor’s intent to pursue 

17 under section 505 of this Act or section 351 of 

18 the Public Health Service Act approval of the 

19 drug, which affirmation shall include a justifica20 tion and a plan for pursuing such approval. 

21 ‘‘(2) ROLLING, REAL-TIME REVIEW.— 

22 ‘‘(A) IN GENERAL.—If the Secretary deter23 mines, after preliminary evaluation of data sub24 mitted by the sponsor, that a drug may meet 

25 the standard for conditional approval, the sponVerDate Sep 11 2014 20:07 Jul 16, 2024 Jkt 049200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S4426.IS S4426

ssavage on LAPJG3WLY3PROD with BILLS
6 

•S 4426 IS

1 sor may submit portions of an application for 

2 conditional approval of a drug under this sec3 tion for evaluation by the Secretary before the 

4 sponsor submits a complete application, which 

5 submission shall include— 

6 ‘‘(i) a schedule for submission of in7 formation necessary to make the applica8 tion complete; and 

9 ‘‘(ii) a payment of any fee that may 

10 be required under section 736. 

11 ‘‘(B) REVIEW.—The Secretary— 

12 ‘‘(i) shall evaluate each application 

13 submitted under subparagraph (A) to as14 sess whether such application is complete 

15 or ready to be filed; and 

16 ‘‘(ii) may commence review of portions 

17 of such application for approval. 

18 ‘‘(3) USE OF REAL-WORLD EVIDENCE.— 

19 ‘‘(A) IN GENERAL.—The Secretary shall 

20 allow the use of real world evidence (as defined 

21 in section 505F(b)), including real world data 

22 used to generate real world evidence, and of ex23 ternal sources of data, including prospective or 

24 retrospective natural history data, to support an 


ssavage on LAPJG3WLY3PROD with BILLS
7 

•S 4426 IS

1 application for conditional approval under this 

2 section. 

3 ‘‘(B) DATA INTEGRITY REQUIREMENTS.— 

4 In using evidence described in subparagraph 

5 (A) to support an application for conditional 

6 approval under this section, the sponsor shall 

7 consider the guidance of the Food and Drug 

8 Administration entitled ‘Data Standards for 

9 Drug and Biological Product Submissions Con10 taining Real-World Data’ and dated December 

11 2023 (or successor guidance). 

12 ‘‘(d) FDA AUTHORITY TO WITHDRAW CONDITIONAL

13 APPROVAL.— 

14 ‘‘(1) IN GENERAL.—The Secretary may with15 draw the conditional approval of a drug under this 

16 section if— 

17 ‘‘(A) after adequate review of appropriate 

18 safety data, including data from an observa19 tional registry established under subsection (g), 

20 the Secretary determines that such data no 

21 longer supports conditional approval; 

22 ‘‘(B) the Secretary determines that the ap23 plication for conditional approval submitted 

24 under subsection (a)(1) contained an untrue 

25 statement of material fact; or 


ssavage on LAPJG3WLY3PROD with BILLS
8 

•S 4426 IS

1 ‘‘(C) the Secretary determines that the 

2 drug is no longer eligible under subsection (b). 

3 ‘‘(2) FDA EXAMINATION AUTHORITY.— 

4 ‘‘(A) IN GENERAL.—For purposes deter5 mining whether to withdraw the conditional ap6 proval of a drug under paragraph (1), the Sec7 retary may— 

8 ‘‘(i) review any available clinical data 

9 made available through clinical trials or an 

10 observational registry under subsection (g), 

11 applicable to such drug; and 

12 ‘‘(ii) determine whether the sponsor of 

13 such drug is in violation of a requirement 

14 established under paragraph (3) or (4) of 

15 section 505(o) or section 505–1 with re16 spect to the drug. 

17 ‘‘(B) TRANSPARENCY.— 

18 ‘‘(i) IN GENERAL.—The Secretary 

19 may require drug sponsors and observa20 tional registries under subsection (g) to 

21 submit the data described in subparagraph 

22 (A) for the purposes of the review under 

23 that subparagraph. 

24 ‘‘(ii) FINES.—The Secretary may levy 

25 fines on sponsors and observational regVerDate Sep 11 2014 20:07 Jul 16, 2024 Jkt 049200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\S4426.IS S4426

ssavage on LAPJG3WLY3PROD with BILLS
9 

•S 4426 IS

1 istries that do not comply with a request 

2 for data under clause (i) within such rea3 sonable timeframe as is established by the 

4 Secretary. 

5 ‘‘(3) EFFECT OF WITHDRAWAL.— 

6 ‘‘(A) AVAILABILITY TO NEW PATIENTS.— 

7 ‘‘(i) IN GENERAL.—If a conditional 

8 approval is withdrawn under this sub9 section, the sponsor may not make the 

10 drug available to any new patients, but 

11 may continue to make such drug available 

12 to patients who started taking the drug 

13 prior to the date of withdrawal. 

14 ‘‘(ii) EFFECT.—Nothing in this sub15 paragraph shall be construed to require— 

16 ‘‘(I) a patient to continue taking 

17 a conditionally approved drug if such 

18 patient decides to stop taking such 

19 drug; or 

20 ‘‘(II) the sponsor to ensure such 

21 drug continues to be manufactured 

22 after the date of withdrawal. 

23 ‘‘(B) CIVIL MONETARY PENALTY.—Any 

24 sponsor who makes available to new patients a 

25 drug for which conditional approval has been 


ssavage on LAPJG3WLY3PROD with BILLS
10 

•S 4426 IS

1 withdrawn under this subsection shall be sub2 ject to such civil monetary penalty as is deter3 mined by the Secretary. 

4 ‘‘(4) WITHDRAWAL NOTICE.—Upon deter5 mining to withdraw the conditional approval of a 

6 drug under paragraph (1), the Secretary shall sub7 mit written notice to the sponsor of such drug and 

8 such withdrawal shall be effective on the date that 

9 is 14 days after the date of such submission of no10 tice. 

11 ‘‘(5) APPEALS.—Not later than 180 days after 

12 the date of enactment of the Promising Pathway Act 

13 2.0, the Secretary, by rule, shall establish a process 

14 by which a sponsor of a drug for which conditional 

15 approval was withdrawn under paragraph (1) may 

16 appeal such withdrawal. 

17 ‘‘(6) AUTOMATIC WITHDRAWAL.— 

18 ‘‘(A) IN GENERAL.—If the sponsor of a 

19 drug that receives conditional approval under 

20 this section does not submit an application for 

21 renewal of such conditional approval under sub22 section (f)(2) by the deadline under that sub23 section, such conditional approval shall auto24 matically be withdrawn in accordance with 


ssavage on LAPJG3WLY3PROD with BILLS
11 

•S 4426 IS

1 paragraph (3) on the date on which such condi2 tional approval expires. 

3 ‘‘(B) MARKETING REQUIREMENT.—If any 

4 drug that receives conditional approval under 

5 this section is not brought to market within 1 

6 year of the date on which the conditional ap7 proval is granted, such conditional approval, 

8 along with any benefits described in subsection 

9 (a)(3), shall automatically be withdrawn in ac10 cordance with paragraph (3) on such date. 

11 ‘‘(C) NO RIGHT TO APPEAL; EFFECT OF

12 AUTOMATIC WITHDRAWAL.— 

13 ‘‘(i) IN GENERAL.—A sponsor shall 

14 not have the right to appeal an automatic 

15 withdrawal under this paragraph. 

16 ‘‘(ii) EFFECT.—The Secretary shall 

17 have no means or power to prevent an 

18 automatic withdrawal under this para19 graph from occurring. 

20 ‘‘(e) LABELING; REVIEW OF MATERIALS.— 

21 ‘‘(1) IN GENERAL.—Sponsors may not make 

22 available to patients a drug conditionally approved 

23 under this section, unless— 

24 ‘‘(A) all labeling and advertising of such 

25 drug contains the statement ‘conditionally apVerDate Sep 11 2014 20:07 Jul 16, 2024 Jkt 049200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\S4426.IS S4426

ssavage on LAPJG3WLY3PROD with BILLS
12 

•S 4426 IS

1 proved for a limited population’ in a prominent 

2 manner and adjacent to, and not more promi3 nent than— 

4 ‘‘(i) the proprietary name of such 

5 drug, if any; or 

6 ‘‘(ii) if there is no proprietary name, 

7 the established name of such drug, if any, 

8 as defined in section 502(e)(3), or, in the 

9 case of a drug that is a biological product, 

10 the proper name, as defined by regulation; 

11 and 

12 ‘‘(B) the prescribing information for the 

13 drug required by section 201.57 of title 21, 

14 Code of Federal Regulations (or any successor 

15 regulation) includes the following statement: 

16 ‘This drug is conditionally approved for use in 

17 a limited and specific population. This drug has 

18 not received full approval by the Food and 

19 Drug Administration. Conditional approval of 

20 this drug may be withdrawn at short notice.’. 

21 ‘‘(2) SUBMISSION.—Not later than 45 days be22 fore such materials are distributed, all promotional, 

23 educational, and marketing materials for such drug 

24 shall be submitted to the Secretary for review. 


ssavage on LAPJG3WLY3PROD with BILLS
13 

•S 4426 IS

1 ‘‘(3) PUBLIC LIST.—The Secretary shall main2 tain a list of all drugs conditionally approved under 

3 this section on a publicly accessible website. Such 

4 website shall briefly describe what each conditionally 

5 approved drugs is and list the 1 or more diseases or 

6 conditions for which the drug is indicated. 

7 ‘‘(f) RENEWAL OF CONDITIONAL APPROVAL; RE8 QUIREMENT TO BRING DRUG TO MARKET.— 

9 ‘‘(1) DURATION; RENEWALS.—The conditional 

10 approval for a drug under this section is effective for 

11 a 2-year period. The sponsor may request renewal of 

12 such conditional approval for up to 3 subsequent 2- 

13 year periods. Conditional approval with respect to a 

14 drug shall not exceed a total of 8 years from the ini15 tial date the drug was granted conditional approval. 

16 ‘‘(2) APPLICATIONS FOR RENEWAL OF CONDI17 TIONAL APPROVAL.— 

18 ‘‘(A) IN GENERAL.—Except as provided in 

19 subparagraph (C), the sponsor of a drug seek20 ing a renewal of conditional approval for such 

21 drug under this subsection shall submit to the 

22 Secretary, not later than 180 days before the 

23 date on which such conditional approval expires, 

24 an application that contains the applicable in25 formation described in paragraph (3) in a 


ssavage on LAPJG3WLY3PROD with BILLS
14 

•S 4426 IS

1 standardized format determined by the Sec2 retary. 

3 ‘‘(B) PROCESS FOR GRANTING RENEW4 ALS.—Not later than 180 days after the date of 

5 enactment of the Promising Pathway Act 2.0, 

6 the Secretary, by rule, shall establish the proc7 ess for granting a renewal under this sub8 section. 

9 ‘‘(C) EXEMPTION FOR SMALL POPULATION

10 DISEASES.— 

11 ‘‘(i) IN GENERAL.—The Secretary 

12 shall exempt from the requirements of sub13 paragraph (A) and paragraph (3) an appli14 cation for a renewal of conditional approval 

15 for a drug under this subsection if the Sec16 retary determines that the population af17 fected by the disease or condition that the 

18 drug is intended to treat does not support 

19 additional preliminary evidence of effective20 ness (as defined in paragraph (3)(D)). 

21 ‘‘(ii) APPLICATION FOR EXEMP22 TION.—Sponsors may submit an applica23 tion for exemption under this subpara24 graph not later than 180 days before the 


ssavage on LAPJG3WLY3PROD with BILLS
15 

•S 4426 IS

1 date on which the conditional approval ex2 pires. 

3 ‘‘(iii) APPLICATION PROCESS.—Not 

4 later than 180 days after the date of en5 actment of the Promising Pathway Act 

6 2.0, the Secretary shall establish a stand7 ardized application process for purposes of 

8 this subparagraph. 

9 ‘‘(iv) DEADLINE.—The Secretary shall 

10 approve or deny an application under this 

11 subparagraph before the date on which the 

12 conditional approval expires. 

13 ‘‘(v) APPEALS.—Not later than 180 

14 days after the date of enactment of the 

15 Promising Pathway Act 2.0, the Secretary 

16 shall establish a process under which a 

17 sponsor my appeal a denial of an applica18 tion under this subparagraph. 

19 ‘‘(3) ADDITIONAL PRELIMINARY EVIDENCE OF

20 EFFECTIVENESS.—The information described in this 

21 paragraph is the following: 

22 ‘‘(A) FOR THE FIRST APPROVAL RE23 NEWAL.—With respect to an application under 

24 paragraph (2) for the first renewal of condi25 tional approval for a drug under this subVerDate Sep 11 2014 20:07 Jul 16, 2024 Jkt 049200 PO 00000 Frm 00015 Fmt 6652 Sfmt 6201 E:\BILLS\S4426.IS S4426

ssavage on LAPJG3WLY3PROD with BILLS
16 

•S 4426 IS

1 section, additional preliminary evidence of effec2 tiveness of the drug, as compared to the evi3 dence provided in the initial application for con4 ditional approval for the drug under subsection 

5 (c). 

6 ‘‘(B) FOR THE SECOND APPROVAL RE7 NEWAL.—With respect to an application under 

8 paragraph (2) for the second renewal of condi9 tional approval for a drug under this sub10 section, additional preliminary evidence of effec11 tiveness of the drug, as compared to the evi12 dence provided in the renewal application de13 scribed in subparagraph (A). 

14 ‘‘(C) FOR THE FINAL APPROVAL RE15 NEWAL.—With respect to an application under 

16 paragraph (2) for the third renewal of condi17 tional approval for a drug under this sub18 section, a written affirmation from the head of 

19 the drug’s review division of the Office of New 

20 Drugs or the Office of Therapeutic Products 

21 asserting that a third renewal is necessary— 

22 ‘‘(i) for patients who have benefitted 

23 from such drug to retain access to such 

24 drug; and 


ssavage on LAPJG3WLY3PROD with BILLS
17 

•S 4426 IS

1 ‘‘(ii) to generate additional prelimi2 nary evidence of effectiveness for the pur3 poses of attaining approval under section 

4 505 of this Act or section 351 of the Pub5 lic Health Service Act. 

6 ‘‘(D) DEFINITION.—In this paragraph, the 

7 term ‘preliminary evidence of effectiveness’ 

8 means— 

9 ‘‘(i) clinical evidence generated by an 

10 ongoing or completed clinical trial con11 ducted in accordance with section 11.22 of 

12 title 42, Code of Federal Regulations (or 

13 successor regulations); 

14 ‘‘(ii) real-world evidence (as defined in 

15 section 505F(b)); or 

16 ‘‘(iii) evidence from an observational 

17 registry under subsection (g). 

18 ‘‘(4) DENIAL OF RENEWAL ON THE BASIS OF

19 DATA FRAUD.—The Secretary may deny the applica20 tion for renewal of conditional approval for a drug 

21 under this subsection if the Secretary, in conducting 

22 a review under subsection (d)(2), finds that the evi23 dence provided in such application under subpara24 graph (A) or (B) of paragraph (3) was fraudulently 

25 manipulated by the applicable observational registry 


ssavage on LAPJG3WLY3PROD with BILLS
18 

•S 4426 IS

1 and that such application substantially relies on 

2 such data. 

3 ‘‘(g) OBSERVATIONAL REGISTRIES.— 

4 ‘‘(1) ESTABLISHMENT.— 

5 ‘‘(A) IN GENERAL.—Subject to subpara6 graph (C), the sponsor of a drug conditionally 

7 approved under this section shall establish an 

8 observational registry, for patients who are or 

9 will be treated with such drug, that pertains to 

10 the disease or condition that the drug is in11 tended to treat. 

12 ‘‘(B) REGISTRIES.—In establishing an ob13 servational registry for a drug under subpara14 graph (A), the sponsor may— 

15 ‘‘(i) establish a new observational reg16 istry; 

17 ‘‘(ii) use an existing observational reg18 istry that pertains to the disease or condi19 tion such drug is intended to treat; 

20 ‘‘(iii) combine 1 or more existing ob21 servational registries that pertain to the 

22 disease or condition such drug is intended 

23 to treat with a new observational registry; 

24 or 


ssavage on LAPJG3WLY3PROD with BILLS
19 

•S 4426 IS

1 ‘‘(iv) combine 2 or more existing ob2 servational registries that pertain to the 

3 disease or condition such drug is intended 

4 to treat. 

5 ‘‘(C) APPROVAL OF REGISTRY AND RIGHT

6 TO APPEAL.—Not later than 180 days after the 

7 date of enactment of the Promising Pathway 

8 Act 2.0, the Secretary shall establish— 

9 ‘‘(i) a process to approve or deny the 

10 establishment of an observational registry 

11 under subparagraph (A); and 

12 ‘‘(ii) a process for sponsors that re13 ceived such a denial to appeal the denial. 

14 ‘‘(2) REQUIREMENT FOR PATIENTS TO ENROLL

15 IN OBSERVATIONAL REGISTRY.— 

16 ‘‘(A) IN GENERAL.—A drug conditionally 

17 approved under this section shall not be made 

18 available to a patient unless such patient is en19 rolled in the applicable observational registry 

20 described in paragraph (1). 

21 ‘‘(B) INFORMED CONSENT.— 

22 ‘‘(i) IN GENERAL.—Prior to enrolling 

23 in an observational registry under subpara24 graph (A), a patient shall provide informed 

25 consent in accordance with clause (ii). 


ssavage on LAPJG3WLY3PROD with BILLS
20 

•S 4426 IS

1 ‘‘(ii) APPLICATION OF CERTAIN RE2 QUIREMENTS.—The requirements for in3 formed consent under part 50 of sub4 chapter A of chapter I of title 21, Code of 

5 Federal Regulations (or successor regula6 tions), shall apply to enrollment an obser7 vational registry under this paragraph. 

8 ‘‘(3) SUBMISSION OF PATIENT DATA.— 

9 ‘‘(A) IN GENERAL.—The sponsor of a drug 

10 conditionally approved under this section shall 

11 be responsible for obtaining and submitting pa12 tient data to the applicable observational reg13 istry described in paragraph (1). 

14 ‘‘(B) SUBMISSION STANDARDS.—Not later 

15 than 180 days after date of enactment of the 

16 Promising Pathway Act 2.0, the Secretary shall 

17 establish data submission standards for spon18 sors to comply with for purposes of subpara19 graph (A) to ensure that registry data is con20 sistent and clinically informed. 

21 ‘‘(4) REQUIREMENTS FOR REGISTRIES.—An ob22 servational registry described in paragraph (1) for a 

23 drug conditionally approved under this section may 

24 be operated by the sponsor of such drug or, at the 


ssavage on LAPJG3WLY3PROD with BILLS
21 

•S 4426 IS

1 sponsor’s discretion, a third party, for-profit organi2 zation, or nonprofit organization. 

3 ‘‘(5) RISK AND BENEFIT DATA.— 

4 ‘‘(A) IN GENERAL.—The sponsor of a drug 

5 conditionally approved under this section shall 

6 submit relevant risk and benefit data to the ap7 plicable observational registry described in 

8 paragraph (1). 

9 ‘‘(B) ONLINE PORTAL.—The Secretary 

10 shall operate an online portal on an existing 

11 website of the Secretary for sponsors to submit 

12 data described in subparagraph (A). 

13 ‘‘(6) ACCESSIBILITY.— 

14 ‘‘(A) IN GENERAL.—An observational reg15 istry described in paragraph (1) shall— 

16 ‘‘(i) not later than 30 days after re17 ceipt of a request, provide patients (or 

18 their designated representatives) with ac19 cess to such patient’s personal registry in20 formation; and 

21 ‘‘(ii) provide approved researchers and 

22 medical professionals access to de-identi23 fied and aggregated data from the registry 

24 for the purposes of indication- and disease25 specific and translational research into 


ssavage on LAPJG3WLY3PROD with BILLS
22 

•S 4426 IS

1 conditions and diseases relating to the dis2 ease or condition that the drug tracked by 

3 the observational registry is intended to 

4 treat. 

5 ‘‘(B) APPROVED RESEARCHERS AND MED6 ICAL PROFESSIONALS.—Not later than 180 

7 days after the date of enactment of the Prom8 ising Pathway Act 2.0, the Secretary, by rule, 

9 shall establish a process for approving research10 ers and medical professionals for purposes of 

11 subparagraph (A)(ii). 

12 ‘‘(7) EFFECT.—Nothing in this section shall be 

13 construed to modify or limit the Secretary’s author14 ity to require for a drug conditionally approved 

15 under this section any type of postapproval study 

16 under any other provision of law, including sections 

17 505(o)(3), 505B, and 506. 

18 ‘‘(h) PURSUIT OF A DIFFERENT INDICATION.— 

19 ‘‘(1) IN GENERAL.—In the case of a drug con20 ditionally approved under this section for which such 

21 approval was withdrawn under subsection (d), ex22 pired under subsection (f)(1), or was denied for re23 newal under subsection (f)(4), not later than 2 years 

24 after the date of withdrawal, expiration, or denial, as 

25 applicable, the sponsor of such drug shall have the 


ssavage on LAPJG3WLY3PROD with BILLS
23 

•S 4426 IS

1 opportunity to petition the Secretary to receive con2 ditional approval of such drug, in accordance with 

3 this section, for a different indication. 

4 ‘‘(2) PROCESS.—Not later than 180 days after 

5 the date of enactment of the Promising Pathway Act 

6 2.0, the Secretary shall establish a process for peti7 tions under paragraph (1). 

8 ‘‘(i) TRANSITION TO OTHER FORMS OF APPROVAL.— 

9 ‘‘(1) IN GENERAL.—A drug that receives condi10 tional approval under this section may be granted 

11 approval under section 505 of this Act or section 

12 351 of the Public Health Service Act during the pe13 riod in which such conditional approval is in effect. 

14 Effective on the date on which approval for such 

15 drug is granted under section 505 of this Act or sec16 tion 351 of the Public Health Service Act, such con17 ditional approval shall be automatically withdrawn in 

18 accordance with subsection (d)(3). 

19 ‘‘(2) CONSIDERATION OF CERTAIN EVI20 DENCE.—In determining whether to approve under 

21 section 505 of this Act or section 351 of the Public 

22 Health Service Act a drug that has received condi23 tional approval under this section, the Secretary may 

24 consider evidence from the observational registry for 

25 the drug under subsection (g). 


ssavage on LAPJG3WLY3PROD with BILLS
24 

•S 4426 IS

1 ‘‘(j) INFORMED CONSENT.— 

2 ‘‘(1) IN GENERAL.—Prior to being prescribed a 

3 drug conditionally approved under this section, a pa4 tient shall provide informed consent in accordance 

5 with paragraph (2). 

6 ‘‘(2) APPLICATION OF CERTAIN REQUIRE7 MENTS.—The requirements for informed consent 

8 under part 50 of subchapter A of chapter I of title 

9 21, Code of Federal Regulations (or successor regu10 lations), shall apply to drugs conditionally approved 

11 under this section. 

12 ‘‘(3) OBSERVATIONAL REGISTRIES.—An obser13 vational registry established for a drug in accord14 ance with subsection (g) may obtain, and maintain 

15 records of, informed consent of a patient on behalf 

16 of the drug sponsor, in accordance with paragraph 

17 (2). 

18 ‘‘(4) COMMON RULE.—Drugs conditionally ap19 proved under this section shall comply with subpart 

20 A of part 46 of title 45, Code of Federal Regulations 

21 (commonly known as the ‘Common Rule’) (or suc22 cessor regulations), if applicable. 

23 ‘‘(k) LIMITATION ON LIABILITY.—With respect to 

24 any claim under State law relating to a drug made avail25 able pursuant to a grant of conditional approval under this 


ssavage on LAPJG3WLY3PROD with BILLS
25 

•S 4426 IS

1 section, no liability shall lie against a sponsor or manufac2 turer of the drug, or any health care provider who pre3 scribes or administers the drug, absent intentional wrong4 doing. 

5 ‘‘(l) REPORT TO CONGRESS.— 

6 ‘‘(1) IN GENERAL.—Not later than 2 years 

7 after the date of enactment of the Promising Path8 way Act 2.0, and once every 2 years thereafter, the 

9 Secretary, in collaboration with drug sponsors, shall 

10 submit a report to Congress on all drugs granted 

11 conditional approval under this section. Such report 

12 shall include— 

13 ‘‘(A) an estimated number of patients 

14 treated with each such drug, and the number of 

15 patients tracked in an observational registry 

16 under subsection (g) with respect to each such 

17 drug, if applicable; 

18 ‘‘(B) a discussion, at an aggregate level , 

19 of the types and amounts of data obtained 

20 through observational registries under sub21 section (g), such as patient treatments and 

22 uses, length of use, side effects encountered, 

23 relevant biomarkers, scan results, cause of 

24 death and how long the patient lived, and ad25 verse drug effects; 


ssavage on LAPJG3WLY3PROD with BILLS
26 

•S 4426 IS

1 ‘‘(C) a list of all such drugs for which an 

2 application for approval under this section, or 

3 an application for an extension of conditional 

4 approval under this section, has been sub5 mitted; and 

6 ‘‘(D) the number of all applications grant7 ed and denied conditional approval under this 

8 section. 

9 ‘‘(2) SPONSOR PARTICIPATION.—Not later than 

10 180 days before the date on which the Secretary 

11 submits a report under paragraph (1), the sponsor 

12 of a drug conditionally approved under this section 

13 shall provide to the Secretary the information de14 scribed in subparagraphs (A) and (B) of paragraph 

15 (1), as applicable. 

16 ‘‘(3) NOTICE AUTHORITY.—The Secretary may 

17 notify sponsors of drugs conditionally approved 

18 under this section and observational registries under 

19 subsection (g) as necessary to complete a report 

20 under paragraph (1).’’. 

21 (b) CONFORMING AMENDMENT.—Section 505(a) of 

22 the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

23 355(a)) is amended by inserting ‘‘, or there is in effect 

24 a conditional approval under section 524C with respect to 

25 such drug’’ before the period. 


ssavage on LAPJG3WLY3PROD with BILLS
27 

•S 4426 IS

1 (c) REIMBURSEMENT.— 

2 (1) PRIVATE HEALTH INSURERS.—Section 

3 2719A of the Public Health Service Act (42 U.S.C. 

4 300gg–19a) is amended by adding at the end the 

5 following: 

6 ‘‘(f) COVERAGE OF CERTAIN DRUGS.—A group 

7 health plan or health insurance issuer offering group or 

8 individual health insurance coverage shall provide coverage 

9 for, and shall not impose any cost sharing requirements 

10 for, drugs conditionally approved under section 524C of 

11 the Federal Food, Drug, and Cosmetic Act for patients 

12 who have the disease or condition the drug is intended 

13 to treat.’’. 

14 (2) FEDERAL HEALTH CARE PROGRAMS.—The 

15 requirement under subsection (f) of section 2719A 

16 of the Public Health Service Act (as added by para17 graph (1)) shall apply with respect to coverage de18 terminations under a Federal health care program 

19 (as defined in section 1128B(f) of the Social Secu20 rity Act (42 U.S.C. 1320a–7b(f))) in the same man21 ner such requirement applies under such subsection 

22 (f). 

23 (3) CONFORMING AMENDMENT.—Section 

24 1927(k)(2)(A)(i) of the Social Security Act (42 

25 U.S.C. 1396r–8(k)(2)(A)(i)) is amended— 


ssavage on LAPJG3WLY3PROD with BILLS
28 

•S 4426 IS

1 (A) by striking ‘‘or which’’ and inserting ‘‘, 

2 which’’; and 

3 (B) by inserting ‘‘, or which is condi4 tionally approved under section 524C of such 

5 Act’’ before the semicolon. 

Æ 


ssavage on LAPJG3WLY3PROD with BILLS