Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits. (1st February 2018)

• Record Type: Journal Article
• Authors: Fountzilas, Elena; Said, Rabih; Tsimberidou, Apostolia M.
• Is Part Of: Expert opinion on investigational drugs. Volume 27:Number 2(2018)
• Page Start: 155
• Page End: 162
• Publication Date: 2018-02-01
• Subjects: Cancer -- efficacy -- enhanced access -- expanded access -- FDA -- investigational -- off-label -- oncology -- personalized medicine -- precision medicine; Drugs -- Design -- Periodicals; Drugs, Investigational -- Bibliography; Drugs, Investigational -- Periodicals; 615.1
• DOI: 10.1080/13543784.2018.1430137
• Languages: English
• ISSNs: 1354-3784

Abstract

ABSTRACT: Introduction : Expanded access is the use of an investigational product by patients with serious medical conditions without participation in a clinical trial. It is a complicated process involving the collaboration of many parties and pharmaceutical companies. Ongoing efforts focus on accelerating expanded access procedures in the best interest of patients with cancer. Areas covered : We review the regulatory and ethical challenges encountered in efforts to optimize expanded access. Expert opinion : In the era of personalized medicine, patients may benefit from novel therapeutic agents that demonstrate encouraging results in early studies. However, drug approval is a lengthy and cumbersome procedure that might exceed the time frame of a life-threatening disease. Expanded access provides options to patients with unmet needs. It may provide informative safety and efficacy data to the manufacturers and the scientific and regulatory organizations. Ongoing efforts are being made by global governmental and scientific committees, regulatory agencies, and patient organizations to address the ethical and regulatory issues and to optimize the expanded access process. Their goal is to expand access to promising novel drugs for individual patients and to accelerate the necessary procedures while preserving patient safety.