I 

117TH CONGRESS 

2D SESSION H. R. 6888 

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

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

IN THE HOUSE OF REPRESENTATIVES 

MARCH 1, 2022 

Mr. TONKO (for himself and Mr. MCKINLEY) introduced the following bill; 

which was referred to the Committee on Energy and Commerce 

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, 


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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 the end of each 

5 of the 4 years following the date of enactment of the Help6 ing Experts Accelerate Rare Treatments Act of 2022, the 

7 Secretary shall submit to the Congress a report summa8 rizing the activities of the Food and Drug Administration 

9 related to designating drugs under section 526 for a rare 

10 disease or condition and approving such drugs under sec11 tion 505 of this Act or licensing such drugs under section 

12 351 of the Public Health Service Act, including— 

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

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

15 Public Health Service Act received by the Food and 

16 Drug Administration, the number of such applica17 tions accepted and rejected for filing, and the num18 ber of such applications pending, approved, and dis19 approved by the Food and Drug Administration, 

20 arrayed by the review division assigned to the appli21 cation; and 

22 ‘‘(2) assess the extent to which the Food and 

23 Drug Administration is consulting with external ex24 perts pursuant to section 569(a)(2) on topics per25 taining to drugs for a rare disease or condition, inVerDate Sep 11 2014 06:00 Apr 07, 2022 Jkt 029200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H6888.IH H6888

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1 cluding how and when any such consultation is oc2 curring. 

3 ‘‘(b) PUBLIC AVAILABILITY.—The Secretary shall 

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

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

6 the Food and Drug Administration. 

7 ‘‘(c) DEFINITION.—In this section, the term ‘rare 

8 disease or condition’ means a disease or condition affect9 ing fewer than 200,000 persons in the United States.’’. 

10 (b) STUDY ON EUROPEAN UNION SAFETY AND EFFI11 CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON12 DITIONS.— 

13 (1) IN GENERAL.—The Comptroller General of 

14 the United States shall enter into a contract with an 

15 appropriate entity to conduct a study on the Euro16 pean Union process for evaluating the safety and ef17 ficacy of drugs for rare diseases or conditions, in18 cluding— 

19 (A) any flexibilities, authorities, or mecha20 nisms available in the European Union specific 

21 to rare diseases or conditions; and 

22 (B) consideration and use of supplemental 

23 data submitted during the review process, in24 cluding data associated with open label extenVerDate Sep 11 2014 06:00 Apr 07, 2022 Jkt 029200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H6888.IH H6888

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1 sion studies and expanded access programs spe2 cific to rare diseases or conditions. 

3 (2) CONSULTATION.—The contract under para4 graph (1) shall provide for consultation with relevant 

5 stakeholders, including— 

6 (A) rare disease or condition patients; and 

7 (B) patient groups that— 

8 (i) represent rare disease or condition 

9 patients; and 

10 (ii) have international patient out11 reach. 

12 (3) REPORT.—The contract under paragraph 

13 (1) shall provide for— 

14 (A) not later than 2 years after the date 

15 of enactment of this Act— 

16 (i) the completion of the study under 

17 paragraph (1); and 

18 (ii) the submission of a report on the 

19 results of such study to the Congress; and 

20 (B) the inclusion in the report under sub21 paragraph (A)(ii) of recommendations for 

22 changes to the processes and authorities of the 

23 Food and Drug Administration to facilitate de24 velopment of, and access to, treatments for rare 

25 diseases or conditions. 


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1 (4) PUBLIC AVAILABILITY.—The contract under 

2 paragraph (1) shall provide for the appropriate enti3 ty referred to in paragraph (1) to make the report 

4 under paragraph (3) available to the public, includ5 ing by posting the report on the website of the ap6 propriate entity. 

7 (c) PUBLIC MEETING.— 

8 (1) IN GENERAL.—Not later than December 31, 

9 2023, the Secretary of Health and Human Services, 

10 acting through the Commissioner of Food and 

11 Drugs, shall convene one or more public meetings to 

12 solicit input from stakeholders regarding the ap13 proaches described in paragraph (2). 

14 (2) APPROACHES.—The public meeting or 

15 meetings under paragraph (1) shall address ap16 proaches to increasing and improving engagement 

17 with rare disease or condition patients, groups rep18 resenting such patients, rare disease or condition ex19 perts, and experts on small population studies, in 

20 order to improve the understanding with respect to 

21 rare diseases or conditions of— 

22 (A) patient burden; 

23 (B) treatment options; and 

24 (C) side effects of treatments, including— 


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1 (i) comparing the side effects of treat2 ments; and 

3 (ii) understanding the risks of side ef4 fects relative to the health status of the pa5 tient and the progression of the disease or 

6 condition. 

7 (3) PUBLIC DOCKET.—The Secretary of Health 

8 and Human Services shall establish a public docket 

9 to receive written comments related to the ap10 proaches addressed during each public meeting 

11 under paragraph (1). Such public docket shall re12 main open for 60 days following the date of each 

13 such public meeting. 

14 (4) REPORTS.—Not later than 180 days after 

15 each public meeting under paragraph (1), the Com16 missioner of Food and Drugs shall develop and pub17 lish on the website of the Food and Drug Adminis18 tration a report on— 

19 (A) the approaches discussed at the public 

20 meeting; and 

21 (B) any related recommendations. 

22 (d) CONSULTATION ON THE SCIENCE OF SMALL

23 POPULATION STUDIES.—Section 569(a)(2) of the Federal 

24 Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb–8(b)) 

25 is amended by adding at the end the following: 


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1 ‘‘(C) SMALL POPULATION STUDIES.—The 

2 external experts on the list maintained pursuant 

3 to subparagraph (A) may include experts on the 

4 science of small population studies.’’. 

5 (e) STUDY ON SUFFICIENCY AND USE OF FDA 

6 MECHANISMS FOR INCORPORATING THE PATIENT AND

7 CLINICIAN PERSPECTIVE IN FDA PROCESSES RELATED

8 TO APPLICATIONS CONCERNING DRUGS FOR RARE DIS9 EASES OR CONDITIONS.— 

10 (1) IN GENERAL.—The Comptroller General of 

11 the United States shall conduct a study on the use 

12 of Food and Drug Administration mechanisms and 

13 tools to ensure that patient and physician perspec14 tives are considered and incorporated throughout the 

15 processes of the Food and Drug Administration— 

16 (A) for approving or licensing under sec17 tion 505 of the Federal Food, Drug, or Cos18 metic Act (21 U.S.C. 355) or section 351 of the 

19 Public Health Service Act (42 U.S.C. 262) a 

20 drug designated as a drug for a rare disease or 

21 condition under section 526 of the Federal 

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

23 360bb); and 


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1 (B) in making any determination related 

2 to such a drug’s approval, including assessment 

3 of the drug’s— 

4 (i) safety or effectiveness; or 

5 (ii) postapproval safety monitoring. 

6 (2) TOPICS.—The study under paragraph (1) 

7 shall— 

8 (A) identify and compare the processes 

9 that the Food and Drug Administration has 

10 formally put in place and utilized to gather ex11 ternal expertise (including patients, patient 

12 groups, and physicians) on specific applications 

13 for diseases or conditions affecting 20,000 or 

14 fewer patients in the United States and specific 

15 applications for diseases or conditions affecting 

16 200,000 or fewer patients in the United States; 

17 (B) examine tools or mechanisms to im18 prove efforts and initiatives of the Food and 

19 Drug Administration to collect and consider 

20 such external expertise with respect to applica21 tions for diseases or conditions affecting 20,000 

22 or fewer patients in the United States com23 pared to applications for diseases or conditions 

24 affecting 200,000 or fewer patients in the 

25 United States throughout the application review 


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1 and approval or licensure processes, including 

2 within internal benefit-risk assessments, advi3 sory committee processes, and postapproval 

4 safety monitoring; and 

5 (C) examine processes or alternatives to 

6 address or resolve conflicts of interest that im7 pede the Food and Drug Administration in 

8 gaining external expert input on rare diseases 

9 or conditions with a limited set of clinical and 

10 research experts. 

11 (3) REPORT.—Not later than 2 years after the 

12 date of enactment of this Act, the Comptroller Gen13 eral of the United States shall— 

14 (A) complete the study under paragraph 

15 (1); 

16 (B) submit a report on the results of such 

17 study to the Congress; and 

18 (C) include in such report recommenda19 tions, if appropriate, for changes to the proc20 esses and authorities of the Food and Drug Ad21 ministration to improve the collection and con22 sideration of external expert opinions of pa23 tients, patient groups, and physicians with ex24 pertise in rare diseases or conditions, including 

25 any specific recommendations for diseases or 


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1 conditions affecting 20,000 or fewer patients in 

2 the United States. 

3 (f) DEFINITION.—In this section, the term ‘‘rare dis4 ease or condition’’ means a disease or condition affecting 

5 fewer than 200,000 persons in the United States. 

Æ 


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