The Potential Effect of the 21st Century Cures Act on Drug Development.

Abstract

Enacted in December 2016, the 21st Century Cures Act is a pivotal piece of legislation that will influence the FDA drug and device approval process for the foreseeable future. Although this legislation received overwhelming support by members of Congress for much needed budgetary increases for the FDA and other national health organizations, there is much controversy over certain provisions that potentially diminish the robustness of the evidence base required for drug and medical device approvals. This article discusses the key provisions from "Title III - Development" under "Division A - 21st Century Cures" related to drug development and, specifically, explores those aspects that address patient-focused drug development, use of surrogate endpoints in clinical trials, modernization of trial design, and use of real-world evidence for decision making and health care economic information. In addition, specific legislation on regulatory changes is discussed pertaining to antimicrobial stewardship. Essentially, summarized interpretations are presented here of the provisions covering the aforementioned topics, along with insights into potential intended and unintended consequences for U.S. health care payers, health care providers, government entities, and product manufacturers. No outside funding supported this writing. Goble is completing fellowship training sponsored by Novartis Pharmaceuticals.