II 

117TH CONGRESS 

2D SESSION S. 4071 

To amend the Federal Food, Drug, and Cosmetic Act to improve the 

treatment of rare diseases and conditions, and for other purposes. 

IN THE SENATE OF THE UNITED STATES 

APRIL 7, 2022 

Mr. CASEY (for himself and Mr. SCOTT of South Carolina) 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 

improve the treatment of rare diseases and conditions, 

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 ‘‘Helping Experts Accel5 erate Rare Treatments Act of 2022’’. 

6 SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES 

7 AND CONDITIONS. 

8 (a) ANNUAL REPORT ON ORPHAN DRUG PRO9 GRAM.—Subchapter B of chapter V of the Federal Food, 


kjohnson on DSK79L0C42PROD with BILLS
2 

•S 4071 IS

1 Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.) is 

2 amended by adding at the end the following new section: 

3 ‘‘SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM. 

4 ‘‘(a) IN GENERAL.—Not later than 1 year after the 

5 date of enactment of the Helping Experts Accelerate Rare 

6 Treatments Act of 2022, and not less frequently than an7 nually thereafter, the Secretary shall submit to Congress 

8 a report summarizing the activities of the Food and Drug 

9 Administration related to designating drugs under section 

10 526 for a rare disease or condition and approving such 

11 drugs under section 505 of this Act or licensing such 

12 drugs under section 351 of the Public Health Service Act, 

13 including— 

14 ‘‘(1) the number of applications for such drugs 

15 under section 505 of this Act and section 351 of the 

16 Public Health Service Act received by the Food and 

17 Drug Administration, the number of such applica18 tions accepted for filing, the number of such applica19 tions rejected for filing, and the numbers of such ap20 plications pending, approved, and disapproved by the 

21 Food and Drug Administration, arrayed by the re22 view division assigned to the application; 

23 ‘‘(2) the size of the affected population in the 

24 United States of each disease or condition addressed 

25 by an application described in paragraph (1), asVerDate Sep 11 2014 02:56 Apr 12, 2022 Jkt 029200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\S4071.IS S4071

kjohnson on DSK79L0C42PROD with BILLS
3 

•S 4071 IS

1 sessed taking into consideration the documentation 

2 required by section 316.20 of title 21, Code of Fed3 eral Regulations (or any successor regulation) and 

4 other relevant information available to the Secretary; 

5 and 

6 ‘‘(3) the extent of consultation with stake7 holders and external experts pursuant to section 

8 569. 

9 ‘‘(b) MANNER OF SUBMISSION.—The information re10 quired to be reported under this section may be submitted 

11 as part of another report to Congress related to the regu12 lation of drugs. 

13 ‘‘(c) PUBLIC AVAILABILITY.—The Secretary shall 

14 make each report under subsection (a) available to the 

15 public, including by posting the report on the website of 

16 the Food and Drug Administration.’’. 

17 (b) STUDY ON EUROPEAN UNION SAFETY AND EFFI18 CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON19 DITIONS.— 

20 (1) IN GENERAL.—Not later than 1 year after 

21 the date of enactment of this Act, the Secretary of 

22 Health and Human Services shall seek to enter into 

23 an agreement with the National Academies of 

24 Sciences, Engineering, and Medicine (referred to in 

25 this section as the ‘‘National Academies’’) to examVerDate Sep 11 2014 02:56 Apr 12, 2022 Jkt 029200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S4071.IS S4071

kjohnson on DSK79L0C42PROD with BILLS
4 

•S 4071 IS

1 ine and report on European Union safety and effi2 cacy reviews of drugs for rare diseases and condi3 tions, the use and sufficiency of existing mechanisms 

4 and tools of the Food and Drug Administration in 

5 ensuring that patient and physician perspectives are 

6 considered throughout such reviews, and opportuni7 ties to improve such reviews in the United States. 

8 (2) CONTENT.—The report developed under 

9 paragraph (1) shall— 

10 (A) assess and evaluate, with respect to 

11 drugs for rare diseases and conditions— 

12 (i) any flexibilities, authorities, or 

13 mechanisms available in the European 

14 Union; 

15 (ii) consideration and use by the Eu16 ropean Medicines Agency of supplemental 

17 data submitted during the orphan drug ap18 plication review process, including data as19 sociated with open label extension studies 

20 and expanded access programs; and 

21 (iii) each formal or informal process 

22 that the Food and Drug Administration 

23 has utilized to gather external expertise on 

24 orphan drug applications, separately con25 sidering orphan drugs for diseases or conVerDate Sep 11 2014 02:56 Apr 12, 2022 Jkt 029200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\S4071.IS S4071

kjohnson on DSK79L0C42PROD with BILLS
5 

•S 4071 IS

1 ditions that affect fewer than 20,000 indi2 viduals in the United States, compared to 

3 orphan drugs for other rare diseases or 

4 conditions; and 

5 (B) provide recommendations for changes 

6 to the processes and authorities of the Food 

7 and Drug Administration to facilitate develop8 ment of, and access to, orphan drugs, which 

9 may include— 

10 (i) new tools or mechanisms to im11 prove efforts and initiatives of the Food 

12 and Drug Administration to collect and 

13 consider external expertise on orphan drug 

14 applications, separately considering treat15 ments for diseases or conditions that affect 

16 fewer than 20,000 individuals in the 

17 United States, including with respect to 

18 processes related to application review, in19 cluding structured benefit-risk assess20 ments, advisory committee deliberations, 

21 and postapproval safety monitoring; and 

22 (ii) in the case of a need for input 

23 from external experts where there are lim24 ited clinical and research experts available 

25 with respect to a rare disease or condition, 


kjohnson on DSK79L0C42PROD with BILLS
6 

•S 4071 IS

1 setting forth alternative processes to ad2 dress or resolve any conflicts of interest 

3 that would otherwise impede inclusion of 

4 input from such external experts. 

5 (3) INPUT.—In conducting the study and devel6 oping the report under paragraph (2), the National 

7 Academies shall— 

8 (A) consider input from the Department of 

9 Health and Human Services, and any other 

10 Federal agencies, as appropriate; and 

11 (B) consult with relevant stakeholders, 

12 which may include conducting public meetings 

13 and other forms of engagement, as appropriate, 

14 with patient groups, health care providers, med15 ical professional societies, public health experts, 

16 medical product manufacturers, and other enti17 ties with relevant experience. 

18 (4) DEFINITIONS.—In this subsection— 

19 (A) the term ‘‘orphan drug’’ means a drug 

20 designated as a drug for a rare disease or con21 dition under section 526 of the Federal Food, 

22 Drug, and Cosmetic Act (21 U.S.C. 360bb); 

23 and 

24 (B) the term ‘‘rare disease or condition’’ 

25 has the meaning given such term in section 526 


kjohnson on DSK79L0C42PROD with BILLS
7 

•S 4071 IS

1 of the Federal Food, Drug, and Cosmetic Act 

2 (21 U.S.C. 360bb). 

3 (c) REVIEW PROCESS.— 

4 (1) CONSULTATION WITH STAKEHOLDERS.— 

5 Section 569(a)(1) of the Federal Food, Drug, and 

6 Cosmetic Act (21 U.S.C. 360bbb–8(a)(1)) is amend7 ed— 

8 (A) by striking ‘‘at a time’’ and inserting 

9 ‘‘at any time’’; 

10 (B) by striking ‘‘Consistent with sections’’ 

11 and inserting the following: 

12 ‘‘(A) IN GENERAL.—Consistent with sec13 tions’’; and 

14 (C) by adding at the end the following: 

15 ‘‘(B) CONSULTATION WITH PATIENTS AND

16 PATIENT GROUPS.— 

17 ‘‘(i) IN GENERAL.—The Secretary 

18 may, as appropriate, consult with patients 

19 and relevant patient groups impacted by 

20 the rare disease or condition, together with 

21 at least one expert included on the list 

22 under paragraph (2)(A) and selected by 

23 such groups— 

24 ‘‘(I) during the review process of 

25 an application for a new drug or bioVerDate Sep 11 2014 02:56 Apr 12, 2022 Jkt 029200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\S4071.IS S4071

kjohnson on DSK79L0C42PROD with BILLS
8 

•S 4071 IS

1 logical product for a rare disease or 

2 condition or a drug or biological prod3 uct that is genetically targeted; and 

4 ‘‘(II) as applicable, during meet5 ings between the Food and Drug Ad6 ministration and sponsors prior to the 

7 submission of an application for a 

8 drug described in subclause (I). 

9 ‘‘(ii) CONFLICTS OF INTEREST.—For 

10 purposes of clause (i), to be eligible for 

11 consultation pursuant to clause (i), pa12 tients and relevant patient groups may not 

13 have any financial interest in the applica14 ble drug or biological product, and external 

15 experts shall be in compliance with applica16 ble law, including section 208 of title 18, 

17 United States Code. 

18 ‘‘(C) CONSULTATION WITH DISPROPOR19 TIONATELY AFFECTED COMMUNITIES.—To the 

20 extent an application for a new drug or biologi21 cal product relates to a rare disease or condi22 tion that disproportionately affects communities 

23 of color or other historically underrepresented 

24 and vulnerable populations, the Secretary is en25 couraged to consult with patients of that subVerDate Sep 11 2014 02:56 Apr 12, 2022 Jkt 029200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\S4071.IS S4071

kjohnson on DSK79L0C42PROD with BILLS
9 

•S 4071 IS

1 population, or one or more patient groups that 

2 represent that subpopulation.’’. 

3 (2) REQUIRING APPROPRIATE EXPERT CON4 SULTATION.—Section 569(a)(2) of the Federal 

5 Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb– 

6 8(a)(2)) is amended— 

7 (A) in subparagraph (A), by striking the 

8 second sentence; and 

9 (B) by striking subparagraph (B) and in10 serting the following: 

11 ‘‘(B) CONSULTATION.—With respect to 

12 any application under section 505 of this Act or 

13 section 351 of the Public Health Service Act for 

14 a drug designated under section 526 for a rare 

15 disease or condition or a drug or biological 

16 product that is genetically targeted, the Sec17 retary may, as appropriate, consult— 

18 ‘‘(i) with an expert with respect to the 

19 disease or condition referenced in the ap20 plication who appears on the list described 

21 in subparagraph (A); or 

22 ‘‘(ii) if no such expert is available, in23 cluding because of conflicts of interest, 

24 with an expert on the list described in subVerDate Sep 11 2014 02:56 Apr 12, 2022 Jkt 029200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\S4071.IS S4071

kjohnson on DSK79L0C42PROD with BILLS
10 

•S 4071 IS

1 paragraph (A) in the science of small pop2 ulation studies. 

3 ‘‘(C) AVAILABILITY AT MEETINGS.—In 

4 connection with each drug product advisory 

5 committee meeting concerning a drug or bio6 logical product for a rare disease or condition, 

7 the Secretary may, as appropriate— 

8 ‘‘(i) include— 

9 ‘‘(I) an expert in the rare disease 

10 or condition; or 

11 ‘‘(II) if no such expert is avail12 able, including because of conflicts of 

13 interest, an expert in the science of 

14 small population studies; and 

15 ‘‘(ii) invite at least one disease or con16 dition expert identified by the relevant pa17 tient groups to participate as a nonvoting 

18 member of the advisory committee.’’. 

19 (3) ADDITIONAL TOPIC FOR CONSULTATION.— 

20 Section 569(b) of the Federal Food, Drug, and Cos21 metic Act (21 U.S.C. 360bbb–8(b)) is amended— 

22 (A) in paragraph (6), by striking ‘‘; and’’ 

23 and inserting ‘‘;’’; 

24 (B) in paragraph (7), by striking the pe25 riod and inserting ‘‘; and’’; and 


kjohnson on DSK79L0C42PROD with BILLS
11 

•S 4071 IS

1 (C) by adding at the end the following: 

2 ‘‘(8) the science of small population studies.’’. 

Æ 


kjohnson on DSK79L0C42PROD with BILLS