Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits

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.

KEYWORDS:

• Cancer
• efficacy
• enhanced access
• expanded access
• FDA
• investigational
• off-label
• oncology
• personalized medicine
• precision medicine

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Corrigendum

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• Expanded access is the use of an investigational product by a patient with a serious and life-threatening condition who cannot participate in a clinical trial.

• Expanded access to an investigational drug offers a treatment option to a patient with unmet needs, and requires approval by the drug manufacturer and regulatory agencies.

• Pharmaceutical companies, treating physicians, patient advocates, regulatory agencies, and institutional review boards are involved in the complicated process of expanded access.

• Ongoing efforts focus on overcoming the safety, regulatory, legal, and ethical challenges associated with expanded access.

• Global scientific communities, governmental and regulatory agencies, and patient advocates are committed to improve expanded access procedures.

• Optimization of laws and policies, development of specialized regulatory agencies, harmonization of policies among regulatory agencies, and broadening of inclusion criteria in clinical studies are expected to accelerate expanded access to investigational drugs.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Additional information

Funding

This work was supported by the NIH/NCI, award number P30 CA016672.