Patient Safety and Toxicology Modernization Act of 2016

Preclinical testing serves a fundamental role in characterizing the potential risks and benefits associated with regulated medicines and products.

Critical gaps remain in the understanding of the relationship between patient response and preclinical findings.

Serious, rare, and unexpected adverse events may be observed in clinical trials or postapproval, particularly toxicology effects not identified in animals that may harm human organs.

Patient efficacy, safety, dosage information, and speedier access to new medicines will benefit from models that are more predictive than animals and that mimic key elements of human organs.

A 2011 report by the Food and Drug Administration, entitled “Advancing Regulatory Science at FDA”, prioritized toxicology testing and the development of models of human adverse response as one of the areas of regulatory science where new or enhanced engagement by the agency is essential to the continued success of the public health and regulatory mission of the Food and Drug Administration.

The Food and Drug Administration’s 2016 draft commitment letter concerning the reauthorization of fees relating to drugs under part 2 of subchapter C of chapter VII of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 379g et seq.) proposes a process to add new preclinical models that will not be finalized until at least 2021.

Peer-reviewed data is readily available to illustrate the benefits of commercially available human tissue models to improve the drug discovery process by replicating key elements of living human tissue.

The Food and Drug Administration should take immediate steps to validate new models, including three-dimensional human tissue models, that improve regulatory decisionmaking in preclinical, clinical, labeling, and postmarket safety and efficacy testing, or other uses by product sponsors.

In general— Not later than December 31, 2018, the Secretary of Health and Human Services, acting through the Commissioner of Food and Drugs, shall issue guidance addressing—

Periodic updates— The Secretary shall periodically update the guidance issued under subsection (a).

obtaining approval or licensure of a drug or biological product, including a combination product, under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) or section 351 of the Public Health Service Act (42 U.S.C. 262); or

meeting the requirements of a regulatory decision issued by the Secretary of Health and Human Services.

Biological product— The term “biological product” has the meaning given such term in section 351(i) of the Public Health Service Act (42 U.S.C. 262(i)).

Combination product— The term “combination product” means a combination product described in section 503(g) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 353(g)).

Drug— The term “drug” has the meaning given such term in section 201 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 321).

Three-dimensional human tissue model— The term “three-dimensional human tissue model” means a three-dimensional model that—