Helping Experts Accelerate Rare Treatments Act of 2021
This bill addresses rare diseases, including by requiring the Food and Drug Administration to ensure the inclusion of rare disease experts and staff members in various parts of the drug review process.
The bill also requires certain reports to address issues related to rare diseases and drugs for treating such diseases (sometimes referred to as orphan drugs if the rarity of the disease makes it difficult for the drug to be profitable).
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 2021''.

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 
2021, and not less than annually thereafter, the Secretary shall submit 
to the Congress a report summarizing the activities of the Food and 
Drug Administration for 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 
        sections 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 considered by each Food and Drug 
        Administration division, and the number of such applications 
        pending and approved;
            ``(2) the number of applications described in paragraph (1) 
        disaggregated by--
                    ``(A) the disease or condition to be addressed; and
                    ``(B) the target subpopulations identified in such 
                applications;
            ``(3) the prevalence in the United States of each disease 
        or condition addressed by an application described in paragraph 
        (1);
            ``(4) integration of Food and Drug Administration staff 
        with expertise in rare diseases and conditions in each review 
        of an application described in paragraph (1);
            ``(5) identification of all external experts with expertise 
        in rare diseases and conditions who are consulted in connection 
        with, or who otherwise participate in, each review of an 
        application described in paragraph (1); and
            ``(6) the status of each application described in paragraph 
        (1).
    ``(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.''.
    (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 conduct a study on European Union safety and 
        efficacy reviews of drugs for rare diseases and conditions, 
        including--
                    (A) any differential prevalence-based mechanisms; 
                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.
            (2) Report.--Not later than 1 year after the date of 
        enactment of this Act, the Comptroller General 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 for 
                changes to the processes and authorities of the Food 
                and Drug Administration to facilitate development of, 
                and access to, treatments for rare diseases and 
                conditions.
            (3) Public availability.--The Comptroller General of the 
        United States shall make each report under paragraph (2) 
        available to the public, including by posting the report on the 
        website of the Government Accountability Office.
    (c) Review Process.--Subparagraph (A) of section 569(a)(2) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb-8(a)(2)) is 
amended to read as follows:
                    ``(A) In general.--
                            ``(i) List.--The Secretary shall develop 
                        and maintain a list of external experts who, 
                        because of their special expertise, are 
                        qualified to provide advice on rare disease and 
                        condition issues, including topics described in 
                        subsection (b).
                            ``(ii) Availability at meetings.--The 
                        Secretary shall ensure availability, for 
                        consultation purposes, of an external expert on 
                        rare diseases, as described in subparagraph 
                        (B), in connection with each drug product 
                        advisory committee meeting concerning a drug or 
                        biological product for a rare disease or 
                        condition.
                            ``(iii) Review of applications.--The 
                        Secretary shall ensure that each review of an 
                        application submitted under section 505 of this 
                        Act or section 351 of the Public Health Service 
                        Act for an indication associated with a rare 
                        disease or condition includes Food and Drug 
                        Administration staff with expertise on rare 
                        diseases and conditions as an integral part of 
                        the review team.''.
    (d) Risk Evaluation and Mitigation Strategy Elements.--Subsection 
(a) of section 505-1 of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355-1) is amended by adding at the end the following new 
paragraph:
            ``(5) Orphan drugs.--In making any determination regarding 
        the inclusion, in the risk evaluation and mitigation strategy 
        for a drug that has been designated as a drug for a rare 
        disease or condition pursuant to section 526, of an element 
        that requires patient action, restrictions on use of the drug, 
        or patient participation, the Secretary shall consult with 
        patients impacted by the rare disease or condition in 
        considering the burden of participation and likely patient 
        compliance.''.
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Helping Experts Accelerate Rare Treatments Act of 2021
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 2021
Rep. Tonko, Paul