RARE Act of 2019

I 116TH CONGRESS 1ST SESSION H. R. 4228 To expand and improve the programs and activities of the Department of Health and Human Services for awareness, education, research, surveil- lance, diagnosis, and treatment concerning rare diseases and conditions. IN THE HOUSE OF REPRESENTATIVES SEPTEMBER 6, 2019 Mr. CARSON of Indiana (for himself, Mr. HUDSON, Ms. BROWNLEY of Cali- fornia, Mr. BUCSHON, Mr. DEFAZIO, Mr. DEUTCH, Mr. FITZPATRICK, Ms. JACKSON LEE, Mr. LUJA´N, Mrs. CAROLYN B. MALONEY of New York, Ms. MOORE, Mrs. MURPHY, Ms. NORTON, Mr. RASKIN, Mr. ROUDA, Mr. SUOZZI, and Mrs. WALORSKI) introduced the following bill; which was referred to the Committee on Energy and Commerce A BILL To expand and improve the programs and activities of the Department of Health and Human Services for aware- ness, education, research, surveillance, diagnosis, and treatment concerning rare diseases and conditions. 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 ‘‘Rare disease Advance- 4 ment, surveillance Research, and Education Act of 2019’’ 5 or the ‘‘RARE Act of 2019’’. 6 VerDate Sep 11 2014 20:32 Sep 09, 2019 Jkt 089200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 2 •HR 4228 IH SEC. 2. NIH RARE DISEASE REGIONAL CENTERS OF EXCEL- 1 LENCE. 2 Paragraph (1) of section 402A(a) of the Public 3 Health Service Act (42 U.S.C. 282a(a)) is amended by 4 adding at the end the following: ‘‘In addition to the 5 amounts authorized to be appropriated by the preceding 6 sentence, there are authorized to be appropriated such 7 sums as may be necessary for each of fiscal years 2020 8 through 2024 for carrying out section 481A (relating to 9 rare disease regional centers of excellence).’’. 10 SEC. 3. CDC SURVEILLANCE OF RARE DISEASES AND CON- 11 DITIONS. 12 Title III of the Public Health Service Act is amended 13 by inserting after section 317T of such Act (42 U.S.C. 14 247b–22) the following: 15 ‘‘SEC. 317U. CDC SURVEILLANCE OF RARE DISEASES AND 16 CONDITIONS. 17 ‘‘(a) IN GENERAL.—The Secretary may, as appro- 18 priate— 19 ‘‘(1) enhance and expand infrastructure and ac- 20 tivities to track the epidemiology of up to 4 rare dis- 21 eases and conditions selected under subsection 22 (c)(1); and 23 ‘‘(2) incorporate information obtained through 24 such activities into an integrated surveillance system 25 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 3 •HR 4228 IH to be known as the National Rare Disease or Condi- 1 tion Surveillance System. 2 ‘‘(b) RESEARCH.—The Secretary shall ensure that 3 the National Rare Disease or Condition Surveillance Sys- 4 tem is designed in a manner that provides information 5 that facilitates further research on rare diseases and con- 6 ditions. 7 ‘‘(c) CONTENT.—In carrying out subsection (a), the 8 Secretary— 9 ‘‘(1) shall select up to 4 rare diseases and con- 10 ditions that are determined by the Secretary to 11 have— 12 ‘‘(A) a high rate of mortality or morbidity; 13 or 14 ‘‘(B) potential for meaningful research and 15 treatment; 16 ‘‘(2) shall provide for the collection and storage 17 of information on the incidence and prevalence of 18 such rare diseases and conditions in the United 19 States; 20 ‘‘(3) to the extent practicable, shall provide for 21 the collection and storage of other available informa- 22 tion on such rare diseases and conditions, including 23 information related to persons living with such a 24 rare disease or condition who choose to participate 25 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 4 •HR 4228 IH in surveillance activities, and including information 1 on— 2 ‘‘(A) demographics, such as age, race, eth- 3 nicity, sex, geographic location, family history, 4 and other information, as appropriate; 5 ‘‘(B) risk factors that may be associated 6 with the rare disease or condition, such as ge- 7 netic and environmental risk factors and other 8 information, as appropriate; and 9 ‘‘(C) diagnosis and progression markers; 10 ‘‘(4) to the extent practicable, shall provide for 11 the collection and storage of information relevant to 12 analysis on such rare diseases and conditions, such 13 as information concerning— 14 ‘‘(A) the natural history of the diseases 15 and conditions; 16 ‘‘(B) the prevention of the disease or con- 17 dition, including secondary diseases and condi- 18 tions; 19 ‘‘(C) the detection, management, and 20 treatment approaches for the diseases and con- 21 ditions; and 22 ‘‘(D) the development of outcomes meas- 23 ures; and 24 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 5 •HR 4228 IH ‘‘(5) may address issues identified during the 1 consultation process under subsection (d). 2 ‘‘(d) CONSULTATION.—In carrying out this section, 3 the Secretary shall consult with individuals with appro- 4 priate expertise, which may include— 5 ‘‘(1) epidemiologists with experience in disease 6 surveillance; 7 ‘‘(2) representatives of national voluntary 8 health associations that— 9 ‘‘(A) focus on rare diseases or conditions; 10 and 11 ‘‘(B) have demonstrated experience in re- 12 search, care, or patient services; 13 ‘‘(3) health information technology experts or 14 other information management specialists; 15 ‘‘(4) clinicians with expertise in rare diseases or 16 conditions; 17 ‘‘(5) research scientists with expertise in rare 18 diseases or conditions, or experience conducting 19 translational research or utilizing surveillance sys- 20 tems for scientific research purposes; and 21 ‘‘(6) patients and caregivers of patients with 22 rare diseases or conditions. 23 ‘‘(e) GRANTS.—The Secretary may award grants to, 24 or enter into contracts or cooperative agreements with, 25 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 6 •HR 4228 IH public or private nonprofit entities to carry out activities 1 under this section. 2 ‘‘(f) COORDINATION WITH OTHER FEDERAL, STATE, 3 AND LOCAL AGENCIES.—Subject to subsection (h), the 4 Secretary shall— 5 ‘‘(1) make information and analysis in the Na- 6 tional Rare Disease or Condition Surveillance Sys- 7 tem available, as appropriate— 8 ‘‘(A) to Federal departments and agencies, 9 such as the National Institutes of Health and 10 the Department of Veterans Affairs; and 11 ‘‘(B) to State and local agencies; and 12 ‘‘(2) identify, build upon, leverage, and coordi- 13 nate among existing data and surveillance systems, 14 surveys, registries, and other Federal public health 15 infrastructure, wherever practicable. 16 ‘‘(g) PUBLIC ACCESS.—Subject to subsection (h), the 17 Secretary shall ensure that information and analysis in the 18 National Rare Disease or Conditions Surveillance System 19 are available, as appropriate, to the public, including re- 20 searchers. 21 ‘‘(h) PRIVACY.—The Secretary shall ensure that in- 22 formation and analysis in the National Rare Disease or 23 Condition Surveillance System are made available only to 24 the extent permitted by applicable Federal and State law, 25 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 7 •HR 4228 IH and in a manner that protects personal privacy, to the ex- 1 tent required by applicable Federal and State privacy law, 2 at a minimum. 3 ‘‘(i) SUPPLEMENT NOT SUPPLANT.—The activities 4 under this section may supplement, but shall not supplant, 5 any activities with respect to spina bifida, muscular dys- 6 trophy, or fragile X syndrome that are ongoing as of the 7 date of enactment of this section. 8 ‘‘(j) REPORTS.— 9 ‘‘(1) REPORT ON INFORMATION AND ANAL- 10 YSES.—Not later than 2 years after the date on 11 which any system is established under this section, 12 the Secretary shall submit an interim report to the 13 Committee on Health, Education, Labor, and Pen- 14 sions of the Senate and the Committee on Energy 15 and Commerce of the House of Representatives re- 16 garding aggregate information collected pursuant to 17 this section and epidemiological analyses, as appro- 18 priate. Such report shall be posted on the Internet 19 website of the Department of Health and Human 20 Services and shall be updated biennially. 21 ‘‘(2) IMPLEMENTATION REPORT.—Not later 22 than 4 years after the date of the enactment of this 23 section, the Secretary shall submit a report to the 24 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 8 •HR 4228 IH Congress concerning the implementation of this sec- 1 tion. Such report shall include information on— 2 ‘‘(A) the development and maintenance of 3 the National Rare Disease or Condition Surveil- 4 lance System; 5 ‘‘(B) the type of information collected and 6 stored in the surveillance system; 7 ‘‘(C) the use and availability of such infor- 8 mation, including guidelines for such use; and 9 ‘‘(D) the use and coordination of databases 10 that collect or maintain information on rare dis- 11 eases or conditions. 12 ‘‘(k) DEFINITIONS.—In this section: 13 ‘‘(1) NATIONAL VOLUNTARY HEALTH ASSOCIA- 14 TION.—The term ‘national voluntary health associa- 15 tion’ means a national nonprofit organization with 16 chapters, other affiliated organizations, or networks 17 in States throughout the United States with experi- 18 ence serving the population of individuals with a 19 rare disease or condition and have demonstrated ex- 20 perience in rare disease or condition research, care, 21 and patient services. 22 ‘‘(2) RARE.—The term ‘rare’, with respect to a 23 disease or condition, means having a prevalence of 24 fewer than 200,000 individuals in the United States. 25 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 9 •HR 4228 IH ‘‘(l) AUTHORIZATION OF APPROPRIATIONS.—To 1 carry out this section, there are authorized to be appro- 2 priated $10,000,000 for each of fiscal years 2020 through 3 2025.’’. 4 SEC. 4. INCREASING HEALTH PROFESSIONALS’ AWARENESS 5 OF RARE DISEASES. 6 (a) IN GENERAL.—The Director of the Agency for 7 Healthcare Research and Quality shall expand and inten- 8 sify the activities of the Agency to increase the awareness 9 and knowledge of health care providers about rare diseases 10 and conditions. 11 (b) DEFINITION.—In this section, the term ‘‘rare dis- 12 eases and conditions’’, with respect to a disease or condi- 13 tion, means having a prevalence of fewer than 200,000 14 individuals in the United States. 15 SEC. 5. REPORT. 16 (a) IN GENERAL.—The Secretary of Health and 17 Human Services shall seek to enter into an arrangement 18 with the National Academies (or another appropriate enti- 19 ty if the National Academies decline) to update and repub- 20 lish, by not later than 3 years after the date of enactment 21 of this Act, the 2010 report of the National Academies 22 entitled ‘‘Rare Diseases and Orphan Products: Accel- 23 erating Research and Development’’. 24 VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS 10 •HR 4228 IH (b) AUTHORIZATION OF APPROPRIATIONS.—To carry 1 out this section, there is authorized to be appropriated 2 $1,000,000. 3 Æ VerDate Sep 11 2014 00:20 Sep 07, 2019 Jkt 089200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6301 E:\BILLS\H4228.IH H4228 jspears on DSK3GMQ082PROD with BILLS