S. 4472 — what changed

added Section 2 of the Accelerating Access to Critical Therapies for ALS Act (21 U.S.C. 360ee note) is amended—

removed “(4) Clinical trial status review

removed “(A) In general—In reviewing applications for renewals of a grant awarded under this section with respect to an investigational drug, the Secretary shall assess the status of a clinical trial carried out for such drug with respect to data on enrollment of patients in such clinical trial.

removed “(B) Interim clinical trial data—To enable the Secretary to make the assessment under subparagraph (A) with respect to an investigational drug, the Secretary shall request that the manufacturer of the investigational drug share interim clinical trial data with respect to such drug with the Secretary.”

added “(b) Application—A participating”

added “(1) a description of how data generated through the proposed expanded access grant will be used to support research or development related to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis;”

added “(3) an assurance that such entity will promptly report to the Secretary available safety data from any ongoing clinical trial of the investigational drug as set forth in the terms and conditions of the grant.”

added “(1) confirm that—”

added “(2) in the case of a renewal of such a grant, request from the sponsor of the investigational new drug application involved, and assess, the enrollment, safety, and any available efficacy data of the drug related to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis.”

added “(4) The term phase 3 clinical trial includes a phase 2/3 combined trial and a planned phase 3 clinical trial that is not yet enrolling participants.”

removed “(4) The term phase 3, with respect to a clinical trial, includes a phase 2/3 combined trial and a planned phase 3 clinical trial that is not yet enrolling participants.”

changed Section 4 of the Accelerating Access to Critical Therapies for ALS Act (21 U.S.C. 360aa note) is amended by adding at the end the following:amended—

added “(4) for each action plan published after the date of enactment of the Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026, include a description of—

added “(A) previous actions taken by the Food and Drug Administration to implement the previous action plan published under subsection (a);

added “(B) any other planned actions to implement such action plan; and

added “(C) any barriers to implementing such action plan and related recommendations, which may include an estimate of resources necessary to address such barriers.”

removed “(c) Report on ALS and other rare neurodegenerative disease action plans—Not later than one year after the date of enactment of the Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026, the Commissioner of Food and Drugs shall publish on the website of the Food and Drug Administration a report that contains—

removed “(1) an updated action plan, including—

removed “(A) a description of the actions the Food and Drug Administration intends to take during the 5-year period following publication of the plan with respect to the program enhancements, policy development, regulatory science initiatives, and other appropriate initiatives described in subsection (a);

removed “(B) a description of the resources necessary to implement each section of the plan within such 5-year period; and

removed “(C) specific approaches the Commissioner will take to improve coordination of implementation of the plan with rare neurodegenerative disease communities that are not specifically ALS communities; and

removed “(2) with respect to the Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) published by the Food and Drug Administration on June 23, 2022 (referred to in this section as the “2022 Action Plan”), a description of—

removed “(A) the actions taken by the Food and Drug Administration under the 2022 Action Plan;

removed “(B) the effect of the implementation of the 2022 Action Plan on the development of therapies and regulatory consideration of therapies for ALS and other rare neurodegenerative diseases;

removed “(C) any programs and initiatives that established or carried out as part of the implementation of the 2022 Action Plan; and

removed “(D) the extent to which the 2022 Action Plan was implemented with respect to rare neurodegenerative diseases that are not amyotrophic lateral sclerosis.”

changed Section 6 of the Accelerating Access to Critical Therapies for ALS Act (Public Law 117–79) is amended, in the matter preceding paragraph (1)—amended—

added “(a) GAO report—Not later than”

added “(b) HHS report—Not later than 4 years after the date of enactment of the Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026, the Secretary of Health and Human Services shall, in a manner that does not duplicate the information described in the action plan published pursuant to section 4, submit to the Committee on Health, Education, Labor, and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives a report assessing the effectiveness of the activities carried out under sections 2, 3, and 5 and making recommendations to improve such activities.”