Access to Rare Indications Act of 2021
This bill provides for coverage under the Medicare prescription drug benefit, Medicaid, and private health insurance of certain drugs that are used to treat rare diseases or conditions (i.e., diseases or conditions that affect 200,000 or fewer individuals). Such drugs must be supported by peer-reviewed research or medical experts and must not have been reviewed unfavorably in specified compendia or used in a way that conflicts with approved labels.
To amend titles XVIII and XIX of the Social Security Act and title 
   XXVII of the Public Health Service Act to provide for coverage of 
certain drugs 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 Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Access to Rare Indications Act of 
2021''.

SEC. 2. COVERAGE OF CERTAIN DRUGS USED IN TREATMENT 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 affecting 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 following 
                        new subclause:
                                    ``(III) in the case of a drug that 
                                is used in the treatment or management 
                                of a disease or condition affecting 
                                200,000 or fewer individuals in the 
                                United States, such use is supported by 
                                peer-reviewed medical literature, 
                                clinical guidelines, or an expert in 
                                such disease or condition as identified 
                                by a medical society involved in the 
                                treatment or management of such disease 
                                or condition, and is not reviewed 
                                unfavorably in the compendia described 
                                in section 1927(g)(1)(B)(i), or listed 
                                as a contraindication in the FDA-
                                approved labeling.''.
            (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-104(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 following 
                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 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 citations 
                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, clinical 
                        guidelines, or an expert in such disease or 
                        condition as identified by a medical society 
                        involved in the treatment or management of such 
                        disease or condition; and
                            ``(ii) is not reviewed unfavorably in the 
                        compendia described in subsection (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 ``compendia'' and inserting ``sources''.
            (3) Effective date.--The amendments made by this subsection 
        apply with respect to covered outpatient 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-11 et seq.) is 
        amended by adding at the end the following new section:

``SEC. 2730. IN GENERAL COVERAGE OF CERTAIN DRUGS USED IN TREATMENT OR 
              MANAGEMENT OF A RARE DISEASE OR CONDITION.

    ``A group health plan or a health insurance issuer offering 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--
            ``(1) approved by the Food and Drug Administration;
            ``(2) for which the use is related to treatment or 
        management of a disease or condition affecting 200,000 or fewer 
        individuals in the United States; and
            ``(3) the use of which is supported by the FDA-approved 
        label, peer-reviewed literature, clinical guidelines, or an 
        expert in such disease or condition as identified by a medical 
        society involved in the treatment or management of such disease 
        or condition, and that is not reviewed unfavorably in the 
        compendia described in section 1927(g)(1)(B)(i) of the Social 
        Security Act or listed as a contraindication in the FDA-
        approved labeling.''.
            (2) Effective date.--The amendment made by this subsection 
        applies with respect to plan years beginning on or after the 
        date that is 30 days after the date of the enactment of this 
        Act.
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Access to Rare Indications Act of 2021
To amend titles XVIII and XIX of the Social Security Act and title XXVII of the Public Health Service Act to provide for coverage of certain drugs used in the treatment or management of a rare disease or condition, and for other purposes.
Access to Rare Indications Act of 2021
Rep. Matsui, Doris O.