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Implementation of 21st Century Cures Act Expanded Access Policies Requirements
Author(s) -
Kang Sukhun,
Chang Sungyong,
Ross Joseph S.,
Miller Jennifer E.
Publication year - 2021
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.2401
Subject(s) - biopharmaceutical , food and drug administration , expanded access , business , drug approval , clinical trial , drug , closing (real estate) , drug development , medicine , pharmacology , finance , oncology , risk analysis (engineering) , genetics , biology
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.