Public Law 117-79 passed December 23, 2021

Accelerating Access to Critical Therapies for ALS Act

Secretary of Health and Human Services will award grants to entities for scientific research on investigational drugs for individuals who are NOT eligible for clinical trials for the preventions, diagnosis, mitigation, treatment or cure of (ALS) amyotrophic lateral sclerosis.

To qualify for this grant an expanded access request must be submitted. The Secretary of Health and Human Services must allow the proceeding application under the Federal Food, Drug and Cosmetic Act before the application is submitted.

Under the application the entity must describe how data generated though an expanded access request with respect to the investigational drug involved will be used to support research, development, prevention, diagnosis, mitigation treatment or cure of ALS.

Applicants must include a description of how the program will be designed as to NOT interfere with patient enrollment in ongoing clinical trials.

Under the Public Health Services Act the Secretary shall award the grant used to support a scientific research objective and shall NOT effect the eligibility of impending enrollments for ongoing clinical trials by determining that individuals who receive expanded access to investigational drugs through such a grant are NOT eligible for enrollment in ongoing clinical trials registered on ClinicalTrials.gov.

Clinical trials under the exemption of the Federal Food, Drug and Cosmetic Act has been granted by the Food and Drug Administration expected to begin enrollments within one year and the resulting project funded will allow for equitable access to investigational drugs by minority and underserved populations.

Funds to pay the manufacturer for direct costs of the investigational drug subject of an expanded access request such costs need to be justified as part of peer review of the grant.

“Participating entities” are clinical trial sites sponsored by a small business under the Small Business Act and that small business is the sponsor of an investigational drug under the federal Food, Drug and Cosmetic Act.

“Participating clinical trial” a phase 3 clinical trial conducted pursuant to an exemption under the Federal Food, Drug and Cosmetic Act or the Public Health Service Act.

“Participating Clinical Trial Site” a health care facility or network facility participating in a clinical trial receiving investigational drugs through such trial.

Expires September 30, 2026

No later than one year of this Act the Secretary of HHS shall implement a Public-Private Partnership for Neurodegenerative Diseases between the National Institutes of Health, the Food and Drug Administration and other eligible entities upon agreements and contracts.

The Partnership shall establish partnerships with other public, private and individuals with expertise in ALS and other rare neurodegenerative diseases. Focus on advancing regulatory science and scientific research that will support the development of drugs for patients with ALS. Foster the development of effective drugs that improve the lives of people with ALS and other rare neurodegenerative disease.

“Eligible entity” an institution of higher education defined by the Higher Education Act of 1965 or an organization described in the Internal Revenue Code of 1986 and exempt from tax.

Eligible entities must have experienced personnel with clinical and technical expertise in the field of biomedical sciences and demonstrate the link to the patient population. Per the Secretary’s satisfaction, be able to identify and establish collaborations between public, private and individual expertise in neurodegenerative diseases to facilitate development and critical evaluation of tools methods and processes to characterize their natural history. Identify molecular targets and increase efficiency, predictability and productivity of clinical development including advancement of rational therapeutic development and establishment of clinical trial networks and securing funding for Partnership from Federal and non-Federal governmental sources, foundations and private individuals.

Must provide assurance that the entity will NOT accept funding for Partnership project from any organization that manufactures or distributes products regulated by the Food and Drug Administration UNLESS the entity provides assurance in agreement with the Secretary that the results of the project will NOT influence any source of funding.

The Partnership may solicit and accept gifts, grants, and other donations, establish accounts, invest and expand funds in support of basic research in association with phase 3 clinical trials subject to expanded access requests under Federal Food, Drug, and Cosmetic Act.

6 months after the enactment of this Act, the Commissioner of the FDA will publish on the website describing actions they tend to take during the 5 year period following publication of program enchantment, policy development, regulatory science initiatives and other initiatives. Foster the development of safe and effective drugs that extend a persons life living with ALS and facilitate access to investigational drugs.

Initial action plan published shall identify representation within the FDA. Facilitate interactions and collaboration between FDA including review centers and stakeholders also including patients, sponsors, and external biomedical research community. Consider cross-cutting clinical and regulatory policy including regulatory advice and decision making. Identify key regulatory science and policy issues critical to advancing development of safe and effective drugs. Enhance collaboration of centers and office of the FDA with other operating divisions within Department of Health and Human Services and other neurodegenerative disease communities.

Secretary of Health and Human Services acting though the FDA will award grants and contracts to the public and private entities to cover costs of research, development of intervention, prevent, diagnose, mitigate, treat or cure ALS and other neurodegenerative disease in adults and children.

No later than 4 years after the date of enactment of this Act the Comptroller General of the United States will submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor and Pensions of the Senate.

The report shall contain an analysis on the research or development, data on how many grants were given out, whom they were given too, the value of each grant, description of the research, number of patients who received investigational drugs, average days between grant application and grant awarded and analysis of impact on research and development related to ALS. Finally, analysis on what is known about the grants to increase efficiency and productivity of the clinical development of therapies including common master protocols or add-on clinical trial design.

$100,000,000.00 has been authorized for each fiscal year 2022-2026.