Veiligheidswaarschuwingen voor weesgeneesmiddelen in de Europese Unie en de Verenigde Staten

Abstract

Objective: To determine the frequency and nature of safety-related regulatory actions for orphan drugs in the United States and the European Union. Orphan drugs are often intended for serious or chronically debilitating diseases. None of the studies that have been conducted on safety-related regulatory actions for drugs focused on orphan drugs. Design: Cohort study. Methods: Orphan drugs approved in the United States and/or the European Union between January 2000 and December 2007 were identified (75 US, 44 EU, of which 24 in both regions). Nature, frequency and timing of safety-related regulatory actions were determined for each orphan drug, defined as safety withdrawals, black box warnings, and written communications to healthcare professionals issued by the FDA or the EMA between January 2000 and June 2008. Results: Of the 95 identified orphan drugs 10 (11%) received at least one safety-related regulatory action. No safety withdrawals, 4 black box warnings and 12 written orphan drugs were identified. Probability of a first safety-related regulatory action for orphan drugs was 20% after 8 years of follow-up. Orphan drugs approved by accelerated approval [relative risk (RR) 3.3; 95% CI 1.1 to 10.4), oncologic products (RR 7.8; 95% CI 1.0-63.8) and products for gastrointestinal and metabolism indications (RR 10.4; 95% CI 1.3-87.3) may have a higher risk for a safety-related regulatory action. Conclusion: The probability of a first safety-related regulatory action of an orphan drug was slightly lower than reported for biologicals and new molecular entities. However, detection of safety issues may be complicated by the limited experience with orphan drugs in practical use. Copyright Adis Data Information 2010. All rights reserved.