HR 1184 — Helping Experts Accelerate Rare Treatments Act of 2021

(a) Submission of proposed strategy (1) Initial approval If the Secretary, in consultation with the office responsible for reviewing the drug and the office responsible for postapproval safety with respect to the drug, determines that a risk evaluation and mitigation strategy is necessary to ensure that the benefits of the drug outweigh the risks of the drug, and informs the person who submits such application of such determination, then such person shall submit to the Secretary as part of such application a proposed risk evaluation and mitigation strategy. In making such a determination, the Secretary shall consider the following factors: (A) The estimated size of the population likely to use the drug involved. (B) The seriousness of the disease or condition that is to be treated with the drug. (C) The expected benefit of the drug with respect to such disease or condition. (D) The expected or actual duration of treatment with the drug. (E) The seriousness of any known or potential adverse events that may be related to the drug and the background incidence of such events in the population likely to use the drug. (F) Whether the drug is a new molecular entity. (2) Postapproval requirement (A) In general If the Secretary has approved a covered application (including an application approved before the effective date of this section) and did not when approving the application require a risk evaluation and mitigation strategy under paragraph (1), the Secretary, in consultation with the offices described in paragraph (1), may subsequently require such a strategy for the drug involved (including when acting on a supplemental application seeking approval of a new indication for use of the drug) if the Secretary becomes aware of new safety information and makes a determination that such a strategy is necessary to ensure that the benefits of the drug outweigh the risks of the drug. (B) Submission of proposed strategy Not later than 120 days after the Secretary notifies the holder of an approved covered application that the Secretary has made a determination under subparagraph (A) with respect to the drug involved, or within such other reasonable time as the Secretary requires to protect the public health, the holder shall submit to the Secretary a proposed risk evaluation and mitigation strategy. (3) Abbreviated new drug applications The applicability of this section to an application under section 355(j) of this title is subject to subsection (i). (4) Non-delegation Determinations by the Secretary under this subsection for a drug shall be made by individuals at or above the level of individuals empowered to approve a drug (such as division directors within the Center for Drug Evaluation and Research). (b) Definitions For purposes of this section: (1) Adverse drug experience The term “adverse drug experience” means any adverse event associated with the use of a drug in humans, whether or not considered drug related, including— (A) an adverse event occurring in the course of the use of the drug in professional practice; (B) an adverse event occurring from an overdose of the drug, whether accidental or intentional; (C) an adverse event occurring from abuse of the drug; (D) an adverse event occurring from withdrawal of the drug; and (E) any failure of expected pharmacological action of the drug, which may include reduced effectiveness under the conditions of use prescribed in the labeling of such drug, but which may not include reduced effectiveness that is in accordance with such labeling. (2) Covered application The term “covered application” means an application referred to in section 355(p)(1)(A) of this title. (3) New safety information The term “new safety information”, with respect to a drug, means information derived from a clinical trial, an adverse event report, a postapproval study (including a study under section 355(o)(3) of this title), or peer-reviewed biomedical literature; data derived from the postmarket risk identific...

(a) In general For the purpose of promoting the efficiency of and informing the review by the Food and Drug Administration of new drugs and biological products for rare diseases and drugs and biological products that are genetically targeted, the following shall apply: (1) Consultation with stakeholders Consistent with sections X.C and IX.E.4 of the PDUFA Reauthorization Performance Goals and Procedures Fiscal Years 2013 through 2017, as referenced in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012, the Secretary shall ensure that opportunities exist, at a time the Secretary determines appropriate, for consultations with stakeholders on the topics described in subsection (b). (2) Consultation with external experts (A) In general 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 issues, including topics described in subsection (b). The Secretary may, when appropriate to address a specific regulatory question, consult such external experts on issues related to the review of new drugs and biological products for rare diseases and drugs and biological products that are genetically targeted, including the topics described in subsection (b), when such consultation is necessary because the Secretary lacks the specific scientific, medical, or technical expertise necessary for the performance of the Secretary’s regulatory responsibilities and the necessary expertise can be provided by the external experts. (B) External experts For purposes of subparagraph (A), external experts are individuals who possess scientific or medical training that the Secretary lacks with respect to one or more rare diseases. (b) Topics for consultation Topics for consultation pursuant to this section may include— (1) rare diseases; (2) the severity of rare diseases; (3) the unmet medical need associated with rare diseases; (4) the willingness and ability of individuals with a rare disease to participate in clinical trials; (5) an assessment of the benefits and risks of therapies to treat rare diseases; (6) the general design of clinical trials for rare disease populations and subpopulations; (7) the demographics and the clinical description of patient populations; and (8) the science of small population studies. (c) Classification as special government employees The external experts who are consulted under this section may be considered special government employees, as defined under section 202 of title 18. (d) Protection of confidential information and trade secrets (1) Rule of construction Nothing in this section shall be construed to alter the protections offered by laws, regulations, and policies governing disclosure of confidential commercial or trade secret information, and any other information exempt from disclosure pursuant to section 552(b) of title 5 as such provisions would be applied to consultation with individuals and organizations prior to July 9, 2012. (2) Consent required for disclosure The Secretary shall not disclose confidential commercial or trade secret information to an expert consulted under this section without the written consent of the sponsor unless the expert is a special government employee (as defined under section 202 of title 18) or the disclosure is otherwise authorized by law. (e) Other consultation Nothing in this section shall be construed to limit the ability of the Secretary to consult with individuals and organizations as authorized prior to July 9, 2012. (f) No right or obligation (1) No right to consultation Nothing in this section shall be construed to create a legal right for a consultation on any matter or require the Secretary to meet with any particular expert or stakeholder. (2) No altering of goals Nothing in this section shall be construed to alter agreed upon goals and procedures identified in the letters described in section 101(b) of the Prescription Dru...