Breakthrough Therapies Act
Bill: S.5123 | Sponsor: Sen. Booker, Cory A. (D-NJ)
Policy area: Crime and Law Enforcement

Summary:
Breakthrough Therapies Act
This bill provides for the classification of drugs or other substances that are part of approved breakthrough therapies as schedule II controlled substances under the Controlled Substances Act. It also provides for expedited controlled substance registrations through the Drug Enforcement Administration for researchers whose work involves investigational drugs.

To amend the Controlled Substances Act to modify the registration 
       requirements relating to research, 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 ``Breakthrough Therapies Act''.

SEC. 2. REGISTRATION REQUIREMENTS RELATED TO RESEARCH.

    (a) Alternative Registration Process for Schedule I Research.--
Section 303 of the Controlled Substances Act (21 U.S.C. 823) is amended 
by adding at the end the following new subsection:
    ``(l) Special Provisions for Those Conducting Certain Research With 
Schedule I Controlled Substances.--
            ``(1) In general.--Notwithstanding subsection (f), a 
        practitioner may conduct research that is described by 
        paragraph (2) and that is with 1 or more schedule I substances 
        if one of the following conditions is satisfied:
                    ``(A) Researcher with a current schedule i or ii 
                research registration.--If the practitioner is 
                registered to conduct research with a controlled 
                substance in schedule I or II, the practitioner may 
                conduct research under this paragraph 30 days after the 
                practitioner has sent a notice to the Attorney General 
                containing the following information, with respect to 
                each substance with which the research will be 
                conducted:
                            ``(i) The chemical name of the substance.
                            ``(ii) The quantity of the substance to be 
                        used in such research.
                            ``(iii) Demonstration that the research is 
                        in the category described by paragraph (2), 
                        which demonstration can be satisfied--
                                    ``(I) in the case of a grant, 
                                contract, cooperative agreement, or 
                                other transaction, or intramural 
                                research project, by identifying the 
                                sponsoring agency and supplying 
                                information related to the grant, 
                                contract, cooperative agreement, other 
                                transaction, or project; or
                                    ``(II) in the case of an 
                                application under section 505(i) of the 
                                Federal Food, Drug, and Cosmetic Act, 
                                by supplying the application number and 
                                the sponsor of record on such 
                                application.
                            ``(iv) Demonstration that the researcher is 
                        authorized to conduct research with respect to 
                        the substance under the laws of the State in 
                        which the research will take place.
                    ``(B) Researcher without a current schedule i or 
                ii.--
                            ``(i) Research registration.--If the 
                        practitioner is not currently registered to 
                        conduct research with a controlled substance in 
                        schedule I or II, the practitioner may send a 
                        notice to the Attorney General containing the 
                        information listed in subparagraph (A), with 
                        respect to each substance with which the 
                        research will be conducted, and the Attorney 
                        General will treat such notice as a sufficient 
                        application for a research registration. Not 
                        later than 45 days of receiving such a notice 
                        that contains all information required by 
                        subparagraph (A), the Attorney General shall 
                        register the applicant, or serve an order to 
                        show cause upon the applicant in accordance 
                        with section 824(c) of this title.
                    ``(C) Verification of information.--On request from 
                the Attorney General, the Secretary of Health and Human 
                Services or the Secretary of Veterans Affairs, as 
                appropriate, shall verify information submitted by an 
                applicant under subparagraph (A)(iii).
            ``(2) Research subject to expedited procedure.--Research is 
        described by this paragraph if--
                    ``(A) the research is the subject of an application 
                under section 505(i) of the Federal Food, Drug, and 
                Cosmetic Act for the investigation of a drug which is 
                in effect in accordance with section 312.40 of title 
                21, Code of Federal Regulations; or
                    ``(B) the research is conducted by the Department 
                of Health and Human Services or the Department of 
                Veterans Affairs or is funded partly or entirely by a 
                grant, contract, cooperative agreement, or other 
                transaction from the Department of Health and Human 
                Services, Department of Veterans Affairs, or a State 
                health department.
            ``(3) Electronic submissions.--The Attorney General shall 
        provide a means to permit practitioners to submit notifications 
        under paragraph (1) electronically.
            ``(4) Limitation on amounts.--A practitioner conducting 
        research with a schedule I substance pursuant to this 
        subsection shall only be permitted to possess the amounts of 
        schedule I substance identified in--
                    ``(A) the notification to the Attorney General 
                under paragraph (1); or
                    ``(B) a supplemental notification that the 
                practitioner may send if the practitioner needs 
                additional amounts for the research, which supplemental 
                notification shall include the registrant's name, the 
                additional quantity needed of the substance, and an 
                attestation that the research to be conducted with the 
                substance is consistent with the scope of the research 
                that was the subject of the notification under 
                paragraph (1).
            ``(5) Importation and exportation requirements not 
        affected.--Nothing in this section alters the requirements of 
        part A of title III, regarding the importation and exportation 
        of controlled substances.''.
    (b) Separate Registrations Not Required for Additional Researcher 
in Same Institution.--Section 302 of the Controlled Substances Act (21 
U.S.C. 822) is amended in subsection (c), by adding the following 
paragraph:
            ``(4) An agent or employee of a research institution that 
        is conducting research with a controlled substance if--
                    ``(A) such agent or employee is acting within the 
                scope of his or her professional practice;
                    ``(B) another agent or employee of such institution 
                is registered to conduct research with a controlled 
                substance in the same schedule;
                    ``(C) the researcher who is so registered--
                            ``(i) informs the Attorney General of the 
                        name, position title, and employing institution 
                        of the agent or employee who is not separately 
                        registered;
                            ``(ii) authorizes such agent or employee to 
                        perform research under the registered 
                        researcher's registration; and
                            ``(iii) affirms that all acts taken by such 
                        agent or employee involving controlled 
                        substances shall be attributable to the 
                        registered researcher, as if the researcher had 
                        directly committed such acts, for purposes of 
                        any proceeding under section 304(a) (21 U.S.C. 
                        824(a)) to suspend or revoke the registration 
                        of the registered researcher; and
                    ``(D) the Attorney General does not, within 30 days 
                of receiving the information, authorization, and 
                affirmation described in subparagraph (C), refuse, for 
                a reason listed in section 304(a) (21 U.S.C. 824(a)), 
                to allow such agent or employee to possess such 
                substance without a separate registration.''.
    (c) Single Registration for Related Research Sites.--Such section 
302 is further amended in subsection (e) by adding at the end the 
following new paragraph:
            ``(3)(A) Notwithstanding paragraph (1), a person registered 
        to conduct research with a controlled substance under section 
        303(f) may conduct such research under a single registration 
        if--
                    ``(i) such research occurs exclusively on sites all 
                of which are within the same city or county and are 
                under the control of the same institution, 
                organization, or agency; and
                    ``(ii) the researcher notifies the Attorney General 
                of all sites where the research will be conducted or 
                where the controlled substance will be stored or 
                administered prior to commencing such research.
            ``(B) A site described by subparagraph (A) shall be 
        included in such registration only if the researcher has 
        notified the Attorney General of such site--
                    ``(i) in the application for such registration; or
                    ``(ii) before the research is conducted, or before 
                the controlled substance is stored or administered, at 
                such site.
            ``(C) The Attorney General may, in consultation with the 
        Secretary of Health and Human Services, issue regulations 
        addressing--
                    ``(i) the manner in which controlled substances may 
                be delivered to the research sites described in 
                subparagraph (A);
                    ``(ii) the storage and security of controlled 
                substances at such research sites;
                    ``(iii) the maintenance of records for such 
                research sites; and
                    ``(iv) any other matters necessary to ensure 
                effective controls against diversion at such research 
                sites.''.
    (d) New Inspection Not Required in Certain Situations.--Such 
section 302 is further amended in subsection (f)--
            (1) by striking ``(f) The'' and inserting ``(f)(1) The''; 
        and
            (2) by adding a new paragraph, as follows:
            ``(2)(A) If a person is registered to conduct research with 
        a controlled substance and applies for a registration, or for a 
        modification of a registration, to conduct research with a 
        second controlled substance that is in the same schedule as the 
        first controlled substance, or is in a schedule with a higher 
        numerical designation than the schedule of the first controlled 
        substance, a new inspection by the Attorney General of the 
        registered location is not required.
            ``(B) Nothing in this paragraph shall prohibit the Attorney 
        General from conducting any inspection if the Attorney General 
        deems it necessary to ensure that the registrant maintains 
        effective controls against diversion.''.
    (e) Continuation of Research on Substances Newly Added to Schedule 
I.--Such section 302 is further amended by adding at the end the 
following new subsection:
    ``(h) Continuation of Research on Substances Newly Added to 
Schedule I.--If a person is conducting research on a substance at the 
time the substance is added to schedule I, and such person is already 
registered to conduct research with a controlled substance in schedule 
I, then--
            ``(1) the person shall, not later than 90 days of the 
        scheduling of the newly scheduled substance, submit a completed 
        application for registration or modification of existing 
        registration, to conduct research on such substance, in 
        accordance with the regulations issued by the Attorney General;
            ``(2) the person may, notwithstanding subsections (a) and 
        (b), continue to conduct the research on such substance until 
        the person withdraws such application or until the Attorney 
        General serves on the person an order to show cause proposing 
        the denial of the application pursuant to section 304(c);
            ``(3) if the Attorney General serves such an order to show 
        cause and the person requests a hearing, such hearing shall be 
        held on an expedited basis and not later than 45 days after the 
        request is made, except that the hearing may be held at a later 
        time if so requested by the person; and
            ``(4) if the person sends a copy of the application 
        referred to in that paragraph to a manufacturer or distributor 
        of such substance, receipt of such copy by such manufacturer or 
        distributor shall constitute sufficient evidence that the 
        person is authorized to receive such substance.''.
    (f) Treatment of Certain Manufacturing Activities as Coincident to 
Research.--Such section 302 (21 U.S.C. 822) is further amended by 
adding at the end the following new subsection:
    ``(j) Treatment of Certain Manufacturing Activities as Coincident 
to Research.--
            ``(1) In general.--Except as specified in paragraph (3), a 
        person who is registered to perform research on a controlled 
        substance may perform manufacturing activities with small 
        quantities of that substance, including activities listed in 
        paragraph (2), without being required to obtain a manufacturing 
        registration, if such activities are performed for the purpose 
        of the research and if the activities and the quantities of the 
        substance involved in those activities are stated in--
                    ``(A) a notification submitted to the Attorney 
                General under section 303(l);
                    ``(B) a protocol filed with an application for 
                registration approval, under section 303(f); or
                    ``(C) a notification to the Attorney General that 
                includes the registrant's name and an attestation that 
                the research to be conducted with the small quantities 
                of manufactured substance is consistent with the scope 
                of the research that is the basis for the registration.
            ``(2) Activities included.--Activities permitted under 
        paragraph (1) include--
                    ``(A) processing the substance to create extracts, 
                tinctures, oils, solutions, derivatives, or other forms 
                of the substance consistent the information provided as 
                part of a notification submitted to the Attorney 
                General under section 303(l) (21 U.S.C. 823(l)) or a 
                research protocol filed with the application for 
                registration approval; and
                    ``(B) dosage form development studies performed for 
                the purpose of satisfying FDA regulatory requirements 
                for submitting an investigational new drug application.
            ``(3) Exception regarding marihuana.--The authority under 
        paragraph (1) to manufacture substances does not include 
        authority to grow marihuana.''.
    (g) Transparency Regarding Special Procedures.--Section 303 of such 
Act (21 U.S.C. 823) is further amended by adding at the end the 
following new subsection:
    ``(m) Transparency Regarding Special Procedures.--
            ``(1) In general.--If the Attorney General determines, with 
        respect to a controlled substance, that an application by a 
        practitioner to conduct research with such substance should be 
        considered under a process, or subject to criteria, different 
        from the process or criteria applicable to applications to 
        conduct research with other controlled substances in the same 
        schedule, the Attorney General shall make public, including by 
        posting on the website of the Drug Enforcement Administration--
                    ``(A) the identities of all substances for which 
                such determinations have been made;
                    ``(B) the process and criteria that shall be 
                applied to applications to conduct research with such 
                substances; and
                    ``(C) how such process and criteria differ from 
                those applicable to applications to conduct research 
                with other controlled substances in the same schedule.
            ``(2) Timing of posting.--The Attorney General shall make 
        such information public upon making such determination, 
        regardless of whether a practitioner has submitted such an 
        application at that time.''.

SEC. 3. CURRENTLY ACCEPTED MEDICAL USE WITH SEVERE RESTRICTIONS.

    (a) Definitions.--Section 102 of the Controlled Substances Act (21 
U.S.C. 802) is amended by inserting after paragraph (7) the following:
            ``(7)(A) Subject to subparagraph (B), the term `currently 
        accepted medical use with severe restrictions', with respect to 
        a drug or other substance, includes a drug or other substance 
        that is an active moiety or active ingredient (whether in 
        natural or synthetic form) of an investigational new drug for 
        which a waiver is in effect under section 505(i) of the Federal 
        Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or section 
        351(a)(3) of the Public Health Service Act (42 U.S.C. 
        262(a)(3)) and that the Secretary--
                    ``(i) designates as a breakthrough therapy under 
                section 506(a) of the Federal Food, Drug, and Cosmetic 
                Act (21 U.S.C. 356(a)); or
                    ``(ii) authorizes for expanded access under 
                subsection (b) or (c) of section 561 of the Federal 
                Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb), either 
                alone or as part of a therapeutic protocol, to treat 
                patients with serious or life-threatening diseases for 
                which no comparable or satisfactory therapies are 
                available.
            ``(B) A drug or other substance shall not meet the criteria 
        under subparagraph (A) for having a currently accepted medical 
        use with severe restrictions if--
                    ``(i) in the case of a drug or other substance 
                described in subparagraph (A)(ii)--
                            ``(I) the Secretary places the expanded 
                        access or protocol for such drug on clinical 
                        hold as described in section 312.42 of title 
                        21, Code of Federal Regulations (or any 
                        successor regulations);
                            ``(II) there is no other investigational 
                        new drug containing the drug or other substance 
                        for which expanded access has been authorized 
                        under section 561(a) of the Federal Food, Drug, 
                        and Cosmetic Act (21 U.S.C. 360bbb(a)); and
                            ``(III) the drug or other substance does 
                        not meet the requirements of subparagraph 
                        (A)(i); or
                    ``(ii) the drug or other substance is an active 
                moiety or active ingredient (whether natural or 
                synthetic) of an application approved under section 505 
                of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
                355) or section 351 of the Public Health Service Act 
                (42 U.S.C. 262).''.
    (b) Authority and Criteria for Classification of Substances.--
Section 201(j) of the Controlled Substances Act (21 U.S.C. 811(j)) is 
amended--
            (1) in paragraph (1), by inserting ``a drug designated as a 
        breakthrough therapy under section 506(a) of the Federal Food, 
        Drug, and Cosmetic Act (21 U.S.C. 356(a)), or a drug authorized 
        for expanded access under subsection (b) or (c) of section 561 
        of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
        360bbb)'' after ``subsection (f),''; and
            (2) in paragraph (2)--
                    (A) in subparagraph (A), by striking ``; or'' and 
                inserting a semicolon;
                    (B) in subparagraph (B), by striking the period at 
                the end and inserting a semicolon; and
                    (C) by adding at the end the following:
                    ``(C) the date on which the Attorney General 
                receives notification from the Secretary of Health and 
                Human Services that the Secretary has designated a drug 
                as a breakthrough therapy under section 506(a) of the 
                Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356(a)) 
                or authorized a drug for expanded access under 
                subsection (b) or (c) of section 561 of the Federal 
                Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb); or
                    ``(D) the date on which the Attorney General 
                receives any written notification demonstrating that 
                the Secretary, before the date of enactment of this 
                subparagraph, designated a drug as a breakthrough 
                therapy under section 506(a) of the Federal Food, Drug, 
                and Cosmetic Act (21 U.S.C. 356(a)) or authorized a 
                drug for expanded access under subsection (b) or (c) of 
                section 561 of the Federal Food, Drug, and Cosmetic Act 
                (21 U.S.C. 360bbb).''.
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