Implementation of 21st Century Cures Act Expanded Access Policies Requirements

Clin Pharmacol Ther. 2021 Dec;110(6):1579-1584. doi: 10.1002/cpt.2401. Epub 2021 Sep 12.


The US Food and Drug Administration (FDA) expanded access pathway allows patients with life-threatening or serious conditions to access investigational drugs outside of trials, under certain conditions. The 21st Century Cures Act ("Cures Act") requires certain drug companies to publicly disclose their expanded access policies. We characterized the proportion of applicable US biopharmaceutical companies, with an oncology related drug, implementing Cures Act requirements for expanded access policies and whether available policies contain the information described in the Act. We found about one-third of applicable biopharmaceutical companies (32%, 140/423) implemented the Cures Act requirement to have a public expanded access policy. Less than one-third of public policies contained all described information (31%, 44/140). Larger companies and those with at least one drug receiving an FDA expedited designation (59% vs. 21%; P < 0.001), or at least one FDA-approved drug (57% vs. 28%; P < 0.001) were more likely to have a public policy. Our results suggest the Cures Act may be having a limited impact on its goals of supporting timely medical decisions and closing informational gaps for patients and doctors around expanded access to investigational oncology therapies, especially for products sponsored by smaller and newer companies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Compassionate Use Trials / legislation & jurisprudence*
  • Compassionate Use Trials / methods
  • Databases, Factual*
  • Drug Approval / legislation & jurisprudence*
  • Drug Approval / methods
  • Drug Industry / legislation & jurisprudence*
  • Drug Industry / methods
  • Drugs, Investigational / therapeutic use*
  • Health Policy / legislation & jurisprudence*
  • Humans
  • Neoplasms / drug therapy
  • Neoplasms / epidemiology
  • United States


  • Antineoplastic Agents
  • Drugs, Investigational