Helping Experts Accelerate Rare Treatments Act of 2022
This bill requires studies on processes for approving drugs that treat rare diseases and conditions.
The Government Accountability Office (GAO) must contract with an appropriate entity to study the European Union's process for evaluating the safety and efficacy of such drugs, including any flexibilities or mechanisms that are specific to rare diseases or conditions. The GAO must also conduct a study about the Food and Drug Administration's mechanisms for ensuring that patient and physician perspectives are considered when evaluating and approving drugs for rare diseases or conditions.
The GAO must report to Congress the results of these studies.
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 the end of each of the 4 years 
following the date of enactment of the Helping Experts Accelerate Rare 
Treatments Act of 2022, the Secretary shall submit to the 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 and rejected for filing, 
        and the number of such applications pending, approved, and 
        disapproved by the Food and Drug Administration, arrayed by the 
        review division assigned to the application; and
            ``(2) assess the extent to which the Food and Drug 
        Administration is consulting with external experts pursuant to 
        section 569(a)(2) on topics pertaining to drugs for a rare 
        disease or condition, including how and when any such 
        consultation is occurring.
    ``(b) 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.
    ``(c) Definition.--In this section, the term `rare disease or 
condition' means a disease or condition affecting fewer than 200,000 
persons in the United States.''.
    (b) Study on European Union Safety and Efficacy Reviews of Drugs 
for Rare Diseases and Conditions.--
            (1) In general.--The Comptroller General of the United 
        States shall enter into a contract with an appropriate entity 
        to conduct a study on the European Union process for evaluating 
        the safety and efficacy of drugs for rare diseases or 
        conditions, including--
                    (A) any flexibilities, authorities, or mechanisms 
                available in the European Union specific to rare 
                diseases or conditions; and
                    (B) consideration and use of supplemental data 
                submitted during the review process, including data 
                associated with open label extension studies and 
                expanded access programs specific to rare diseases or 
                conditions.
            (2) Consultation.--The contract under paragraph (1) shall 
        provide for consultation with relevant stakeholders, 
        including--
                    (A) rare disease or condition patients; and
                    (B) patient groups that--
                            (i) represent rare disease or condition 
                        patients; and
                            (ii) have international patient outreach.
            (3) Report.--The contract under paragraph (1) shall provide 
        for--
                    (A) not later than 2 years after the date of 
                enactment of this Act--
                            (i) the completion of the study under 
                        paragraph (1); and
                            (ii) the submission of a report on the 
                        results of such study to the Congress; and
                    (B) the inclusion in the report under subparagraph 
                (A)(ii) of recommendations for changes to the processes 
                and authorities of the Food and Drug Administration to 
                facilitate development of, and access to, treatments 
                for rare diseases or conditions.
            (4) Public availability.--The contract under paragraph (1) 
        shall provide for the appropriate entity referred to in 
        paragraph (1) to make the report under paragraph (3) available 
        to the public, including by posting the report on the website 
        of the appropriate entity.
    (c) Public Meeting.--
            (1) In general.--Not later than December 31, 2023, the 
        Secretary of Health and Human Services, acting through the 
        Commissioner of Food and Drugs, shall convene one or more 
        public meetings to solicit input from stakeholders regarding 
        the approaches described in paragraph (2).
            (2) Approaches.--The public meeting or meetings under 
        paragraph (1) shall address approaches to increasing and 
        improving engagement with rare disease or condition patients, 
        groups representing such patients, rare disease or condition 
        experts, and experts on small population studies, in order to 
        improve the understanding with respect to rare diseases or 
        conditions of--
                    (A) patient burden;
                    (B) treatment options; and
                    (C) side effects of treatments, including--
                            (i) comparing the side effects of 
                        treatments; and
                            (ii) understanding the risks of side 
                        effects relative to the health status of the 
                        patient and the progression of the disease or 
                        condition.
            (3) Public docket.--The Secretary of Health and Human 
        Services shall establish a public docket to receive written 
        comments related to the approaches addressed during each public 
        meeting under paragraph (1). Such public docket shall remain 
        open for 60 days following the date of each such public 
        meeting.
            (4) Reports.--Not later than 180 days after each public 
        meeting under paragraph (1), the Commissioner of Food and Drugs 
        shall develop and publish on the website of the Food and Drug 
        Administration a report on--
                    (A) the approaches discussed at the public meeting; 
                and
                    (B) any related recommendations.
    (d) Consultation on the Science of Small Population Studies.--
Section 569(a)(2) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 360bbb-8(b)) is amended by adding at the end the following:
                    ``(C) Small population studies.--The external 
                experts on the list maintained pursuant to subparagraph 
                (A) may include experts on the science of small 
                population studies.''.
    (e) Study on Sufficiency and Use of FDA Mechanisms for 
Incorporating the Patient and Clinician Perspective in FDA Processes 
Related to Applications Concerning Drugs for Rare Diseases or 
Conditions.--
            (1) In general.--The Comptroller General of the United 
        States shall conduct a study on the use of Food and Drug 
        Administration mechanisms and tools to ensure that patient and 
        physician perspectives are considered and incorporated 
        throughout the processes of the Food and Drug Administration--
                    (A) for approving or licensing under section 505 of 
                the Federal Food, Drug, or Cosmetic Act (21 U.S.C. 355) 
                or section 351 of the Public Health Service Act (42 
                U.S.C. 262) 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) in making any determination related to such a 
                drug's approval, including assessment of the drug's--
                            (i) safety or effectiveness; or
                            (ii) postapproval safety monitoring.
            (2) Topics.--The study under paragraph (1) shall--
                    (A) identify and compare the processes that the 
                Food and Drug Administration has formally put in place 
                and utilized to gather external expertise (including 
                patients, patient groups, and physicians) on specific 
                applications for diseases or conditions affecting 
                20,000 or fewer patients in the United States and 
                specific applications for diseases or conditions 
                affecting 200,000 or fewer patients in the United 
                States;
                    (B) examine tools or mechanisms to improve efforts 
                and initiatives of the Food and Drug Administration to 
                collect and consider such external expertise with 
                respect to applications for diseases or conditions 
                affecting 20,000 or fewer patients in the United States 
                compared to applications for diseases or conditions 
                affecting 200,000 or fewer patients in the United 
                States throughout the application review and approval 
                or licensure processes, including within internal 
                benefit-risk assessments, advisory committee processes, 
                and postapproval safety monitoring; and
                    (C) examine processes or alternatives to address or 
                resolve conflicts of interest that impede the Food and 
                Drug Administration in gaining external expert input on 
                rare diseases or conditions with a limited set of 
                clinical and research experts.
            (3) Report.--Not later than 2 years after the date of 
        enactment of this Act, the Comptroller General of the United 
        States shall--
                    (A) complete the study under paragraph (1);
                    (B) submit a report on the results of such study to 
                the Congress; and
                    (C) include in such report recommendations, if 
                appropriate, for changes to the processes and 
                authorities of the Food and Drug Administration to 
                improve the collection and consideration of external 
                expert opinions of patients, patient groups, and 
                physicians with expertise in rare diseases or 
                conditions, including any specific recommendations for 
                diseases or conditions affecting 20,000 or fewer 
                patients in the United States.
    (f) Definition.--In this section, the term ``rare disease or 
condition'' means a disease or condition affecting fewer than 200,000 
persons in the United States.
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Helping Experts Accelerate Rare Treatments Act of 2022
To amend the Federal Food, Drug, and Cosmetic Act to improve the treatment of rare diseases and conditions, and for other purposes.
Helping Experts Accelerate Rare Treatments Act of 2022
Rep. Tonko, Paul