[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[S. 4071 Introduced in Senate (IS)]

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117th CONGRESS
  2d Session
                                S. 4071

   To amend the Federal Food, Drug, and Cosmetic Act to improve the 
   treatment of rare diseases and conditions, and for other purposes.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                             April 7, 2022

Mr. Casey (for himself and Mr. Scott of South Carolina) introduced the 
 following bill; which was read twice and referred to the Committee on 
                 Health, Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL


 
   To amend the Federal Food, Drug, and Cosmetic Act to improve the 
   treatment of rare diseases and conditions, 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 ``Helping Experts Accelerate Rare 
Treatments Act of 2022''.

SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES AND CONDITIONS.

    (a) Annual Report on Orphan Drug Program.--Subchapter B of chapter 
V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.) 
is amended by adding at the end the following new section:

``SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM.

    ``(a) In General.--Not later than 1 year after the date of 
enactment of the Helping Experts Accelerate Rare Treatments Act of 
2022, and not less frequently than annually thereafter, the Secretary 
shall submit to Congress a report summarizing the activities of the 
Food and Drug Administration related to designating drugs under section 
526 for a rare disease or condition and approving such drugs under 
section 505 of this Act or licensing such drugs under section 351 of 
the Public Health Service Act, including--
            ``(1) the number of applications for such drugs under 
        section 505 of this Act and section 351 of the Public Health 
        Service Act received by the Food and Drug Administration, the 
        number of such applications accepted for filing, the number of 
        such applications rejected for filing, and the numbers of such 
        applications pending, approved, and disapproved by the Food and 
        Drug Administration, arrayed by the review division assigned to 
        the application;
            ``(2) the size of the affected population in the United 
        States of each disease or condition addressed by an application 
        described in paragraph (1), assessed taking into consideration 
        the documentation required by section 316.20 of title 21, Code 
        of Federal Regulations (or any successor regulation) and other 
        relevant information available to the Secretary; and
            ``(3) the extent of consultation with stakeholders and 
        external experts pursuant to section 569.
    ``(b) Manner of Submission.--The information required to be 
reported under this section may be submitted as part of another report 
to Congress related to the regulation of drugs.
    ``(c) Public Availability.--The Secretary shall make each report 
under subsection (a) available to the public, including by posting the 
report on the website of the Food and Drug Administration.''.
    (b) Study on European Union Safety and Efficacy Reviews of Drugs 
for Rare Diseases and Conditions.--
            (1) In general.--Not later than 1 year after the date of 
        enactment of this Act, the Secretary of Health and Human 
        Services shall seek to enter into an agreement with the 
        National Academies of Sciences, Engineering, and Medicine 
        (referred to in this section as the ``National Academies'') to 
        examine and report on European Union safety and efficacy 
        reviews of drugs for rare diseases and conditions, the use and 
        sufficiency of existing mechanisms and tools of the Food and 
        Drug Administration in ensuring that patient and physician 
        perspectives are considered throughout such reviews, and 
        opportunities to improve such reviews in the United States.
            (2) Content.--The report developed under paragraph (1) 
        shall--
                    (A) assess and evaluate, with respect to drugs for 
                rare diseases and conditions--
                            (i) any flexibilities, authorities, or 
                        mechanisms available in the European Union;
                            (ii) consideration and use by the European 
                        Medicines Agency of supplemental data submitted 
                        during the orphan drug application review 
                        process, including data associated with open 
                        label extension studies and expanded access 
                        programs; and
                            (iii) each formal or informal process that 
                        the Food and Drug Administration has utilized 
                        to gather external expertise on orphan drug 
                        applications, separately considering orphan 
                        drugs for diseases or conditions that affect 
                        fewer than 20,000 individuals in the United 
                        States, compared to orphan drugs for other rare 
                        diseases or conditions; and
                    (B) provide recommendations for changes to the 
                processes and authorities of the Food and Drug 
                Administration to facilitate development of, and access 
                to, orphan drugs, which may include--
                            (i) new tools or mechanisms to improve 
                        efforts and initiatives of the Food and Drug 
                        Administration to collect and consider external 
                        expertise on orphan drug applications, 
                        separately considering treatments for diseases 
                        or conditions that affect fewer than 20,000 
                        individuals in the United States, including 
                        with respect to processes related to 
                        application review, including structured 
                        benefit-risk assessments, advisory committee 
                        deliberations, and postapproval safety 
                        monitoring; and
                            (ii) in the case of a need for input from 
                        external experts where there are limited 
                        clinical and research experts available with 
                        respect to a rare disease or condition, setting 
                        forth alternative processes to address or 
                        resolve any conflicts of interest that would 
                        otherwise impede inclusion of input from such 
                        external experts.
            (3) Input.--In conducting the study and developing the 
        report under paragraph (2), the National Academies shall--
                    (A) consider input from the Department of Health 
                and Human Services, and any other Federal agencies, as 
                appropriate; and
                    (B) consult with relevant stakeholders, which may 
                include conducting public meetings and other forms of 
                engagement, as appropriate, with patient groups, health 
                care providers, medical professional societies, public 
                health experts, medical product manufacturers, and 
                other entities with relevant experience.
            (4) Definitions.--In this subsection--
                    (A) the term ``orphan drug'' means a drug 
                designated as a drug for a rare disease or condition 
                under section 526 of the Federal Food, Drug, and 
                Cosmetic Act (21 U.S.C. 360bb); and
                    (B) the term ``rare disease or condition'' has the 
                meaning given such term in section 526 of the Federal 
                Food, Drug, and Cosmetic Act (21 U.S.C. 360bb).
    (c) Review Process.--
            (1) Consultation with stakeholders.--Section 569(a)(1) of 
        the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb-
        8(a)(1)) is amended--
                    (A) by striking ``at a time'' and inserting ``at 
                any time'';
                    (B) by striking ``Consistent with sections'' and 
                inserting the following:
                    ``(A) In general.--Consistent with sections''; and
                    (C) by adding at the end the following:
                    ``(B) Consultation with patients and patient 
                groups.--
                            ``(i) In general.--The Secretary may, as 
                        appropriate, consult with patients and relevant 
                        patient groups impacted by the rare disease or 
                        condition, together with at least one expert 
                        included on the list under paragraph (2)(A) and 
                        selected by such groups--
                                    ``(I) during the review process of 
                                an application for a new drug or 
                                biological product for a rare disease 
                                or condition or a drug or biological 
                                product that is genetically targeted; 
                                and
                                    ``(II) as applicable, during 
                                meetings between the Food and Drug 
                                Administration and sponsors prior to 
                                the submission of an application for a 
                                drug described in subclause (I).
                            ``(ii) Conflicts of interest.--For purposes 
                        of clause (i), to be eligible for consultation 
                        pursuant to clause (i), patients and relevant 
                        patient groups may not have any financial 
                        interest in the applicable drug or biological 
                        product, and external experts shall be in 
                        compliance with applicable law, including 
                        section 208 of title 18, United States Code.
                    ``(C) Consultation with disproportionately affected 
                communities.--To the extent an application for a new 
                drug or biological product relates to a rare disease or 
                condition that disproportionately affects communities 
                of color or other historically underrepresented and 
                vulnerable populations, the Secretary is encouraged to 
                consult with patients of that subpopulation, or one or 
                more patient groups that represent that 
                subpopulation.''.
            (2) Requiring appropriate expert consultation.--Section 
        569(a)(2) of the Federal Food, Drug, and Cosmetic Act (21 
        U.S.C. 360bbb-8(a)(2)) is amended--
                    (A) in subparagraph (A), by striking the second 
                sentence; and
                    (B) by striking subparagraph (B) and inserting the 
                following:
                    ``(B) Consultation.--With respect to any 
                application under section 505 of this Act or section 
                351 of the Public Health Service Act for a drug 
                designated under section 526 for a rare disease or 
                condition or a drug or biological product that is 
                genetically targeted, the Secretary may, as 
                appropriate, consult--
                            ``(i) with an expert with respect to the 
                        disease or condition referenced in the 
                        application who appears on the list described 
                        in subparagraph (A); or
                            ``(ii) if no such expert is available, 
                        including because of conflicts of interest, 
                        with an expert on the list described in 
                        subparagraph (A) in the science of small 
                        population studies.
                    ``(C) Availability at meetings.--In connection with 
                each drug product advisory committee meeting concerning 
                a drug or biological product for a rare disease or 
                condition, the Secretary may, as appropriate--
                            ``(i) include--
                                    ``(I) an expert in the rare disease 
                                or condition; or
                                    ``(II) if no such expert is 
                                available, including because of 
                                conflicts of interest, an expert in the 
                                science of small population studies; 
                                and
                            ``(ii) invite at least one disease or 
                        condition expert identified by the relevant 
                        patient groups to participate as a nonvoting 
                        member of the advisory committee.''.
            (3) Additional topic for consultation.--Section 569(b) of 
        the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb-
        8(b)) is amended--
                    (A) in paragraph (6), by striking ``; and'' and 
                inserting ``;'';
                    (B) in paragraph (7), by striking the period and 
                inserting ``; and''; and
                    (C) by adding at the end the following:
            ``(8) the science of small population studies.''.
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