I 
118TH CONGRESS 
1ST SESSION H. R. 6094 
To amend titles XVIII and XIX of the Social Security Act and title XXVII 
of the Public Health Service Act to refine the set of information sources 
for determining coverage of certain drugs and biologicals used in the 
treatment or management of a rare disease or condition, and for other 
purposes. 
IN THE HOUSE OF REPRESENTATIVES 
OCTOBER 26, 2023 
Ms. MATSUI (for herself, Mr. DUNN of Florida, Mr. THOMPSON of California, 
and Mr. KELLY of Pennsylvania) introduced the following bill; which was 
referred to the Committee on Energy and Commerce, and in addition to 
the Committee on Ways and Means, for a period to be subsequently de-
termined by the Speaker, in each case for consideration of such provisions 
as fall within the jurisdiction of the committee concerned 
A BILL 
To amend titles XVIII and XIX of the Social Security Act 
and title XXVII of the Public Health Service Act to 
refine the set of information sources for determining 
coverage of certain drugs and biologicals used in the 
treatment or management of a rare disease or condition, 
and for other purposes. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
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SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Providing Realistic Op-
portunity To Equal and Comparable Treatment for Rare 
Act’’ or the ‘‘PROTECT Rare Act’’. 
SEC. 2. COVERAGE OF CERTAIN DRUGS USED IN TREAT-
MENT OR MANAGEMENT OF RARE DISEASE 
OR CONDITION. 
(a) MEDICARE.— 
(1) IN
GENERAL.—Section 1861(t)(2) of the 
Social Security Act (42 U.S.C. 1395x(t)(2)) is 
amended— 
(A) in subparagraph (A), by inserting after 
‘‘regimen’’ the following: ‘‘, or in the treatment 
or management of a disease or condition affect-
ing 200,000 or fewer individuals in the United 
States,’’; and 
(B) in subparagraph (B)(ii)— 
(i) in subclause (I), by striking ‘‘, or’’ 
at the end and inserting a semicolon; 
(ii) in subclause (II), by striking the 
period at the end and inserting ‘‘; or’’; and 
(iii) by adding at the end the fol-
lowing new subclause: 
‘‘(III) in the case of a drug that 
is used in the treatment or manage-
ment of a disease or condition affect-
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ing 200,000 or fewer individuals in 
the United States, such use is sup-
ported by peer-reviewed medical lit-
erature, including clinical guidelines, 
and is not reviewed unfavorably in the 
compendia 
described 
in 
section 
1927(g)(1)(B)(i), or listed as a con-
traindication in the FDA-approved la-
beling.’’. 
(2) MEDICALLY ACCEPTED USES OF COVERED 
PART D DRUGS IN TREATING RARE CONDITIONS.— 
Section 1860D–2(e)(4)(A) of the Social Security Act 
(42 U.S.C. 1395w–102(e)(4)(A)) is amended— 
(A) in clause (i)(II), by striking ‘‘and’’; 
(B) by redesignating clause (ii) as clause 
(iii); and 
(C) by inserting after clause (i)(II) the fol-
lowing new clause: 
‘‘(ii) in the case of a covered part D 
drug used in the treatment or management 
of a disease or condition affecting 200,000 
or fewer individuals in the United States, 
in section 1861(t)(2)(B); and’’. 
(3) EFFECTIVE DATE.—The amendments made 
by this subsection apply with respect to items and 
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services furnished on or after the date that is 30 
days after the date of the enactment of this Act. 
(b) MEDICAID.— 
(1) IN GENERAL.—Section 1927(k)(6) of the 
Social Security Act (42 U.S.C. 1396r–8(k)(6)) is 
amended to read as follows: 
‘‘(6) MEDICALLY ACCEPTED INDICATION.—The 
term ‘medically accepted indication’ means any use 
for a covered drug— 
‘‘(A) which is approved under the Federal 
Food, Drug, and Cosmetic Act; 
‘‘(B) which is supported by one or more ci-
tations included or approved for inclusion in 
any of the compendia described in subsection 
(g)(1)(B)(i); or 
‘‘(C) in the case of a drug used to treat or 
manage 
a 
disease 
or 
condition 
affecting 
200,000 or fewer individuals in the United 
States— 
‘‘(i) the use of such drug is supported 
by peer-reviewed medical literature, includ-
ing clinical guidelines; and 
‘‘(ii) is not reviewed unfavorably in 
the compendia described in subsection 
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(g)(1)(B)(i), or listed as a contraindication 
in the FDA-approved labeling.’’. 
(2) 
CONFORMING
AMENDMENT.—Section 
1927(d)(4)(C) of the Social Security Act (42 U.S.C. 
1396r–8(d)(4)(C)) is amended by striking ‘‘com-
pendia’’ and inserting ‘‘sources’’. 
(3) EFFECTIVE DATE.—The amendments made 
by this subsection apply with respect to covered out-
patient drugs furnished on or after the date that is 
30 days after the date of the enactment of this Act. 
(c) PRIVATE HEALTH INSURANCE.— 
(1) IN GENERAL.—Subpart II of part A of title 
XXVII of the Public Health Service Act (42 U.S.C. 
300gg–19) is amended by adding at the end the fol-
lowing new section: 
‘‘SEC. 2730. EXPEDITED PROCESSES FOR REVIEW ASSOCI-
ATED WITH CERTAIN DRUGS USED IN TREAT-
MENT OR MANAGEMENT OF A RARE DISEASE 
OR CONDITION. 
‘‘A group health plan or a health insurance issuer of-
fering group or individual health insurance coverage shall 
provide a mechanism for expedited formulary exception, 
reconsideration, and appeal of any denial of coverage for 
a drug or biological— 
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