How Often Are Drugs Made Available Under the Food and Drug Administration's Expanded Access Process Approved?

Area of Science:

• Pharmacology and Drug Development
• Regulatory Science
• Patient Access to Investigational Drugs

Background:

• The US Food and Drug Administration (FDA) oversees an expanded access program for investigational drugs.
• This program allows patients with serious or life-threatening conditions to access drugs outside of clinical trials.
• Understanding the outcomes of these requests is crucial for patients, physicians, and sponsors.

Purpose of the Study:

• To evaluate the approval rates of individual patient expanded access requests.
• To assess the likelihood of marketing approval for drugs accessed via expanded access.
• To investigate potential product liability risks for sponsors related to expanded access.

Main Methods:

• Review of individual patient expanded access requests submitted to the Center for Drug Evaluation and Research (CDER) from FY2010 to FY2014.
• Analysis of marketing approval status for drugs obtained through expanded access.
• Search of legal and news databases for product liability cases linked to expanded access.

Main Results:

• 98% of individual patient expanded access requests were allowed to proceed.
• Within one year of submission, 24% of unique drugs and 20% of applications received marketing approval.
• Within five years, these approval rates increased to 43% for unique drugs and 33% for applications.
• No product liability cases were identified in legal or news databases arising from expanded access use.

Conclusions:

• The FDA's expanded access program effectively balances patient access with safety.
• Physicians and patients can expect a high rate of approval for expanded access requests.
• Expanded access does not appear to pose significant product liability risks to sponsors.
• The program supports the drug development process while providing access to potentially beneficial therapies.