Priority Review Drugs Approved by the Food and Drug Administration and the European Medicines Agency: Differences and Policy Implications
Methods: Regulatory information on drug applications, approvals, indications, and orphan designations data were derived from FDA and EMA websites for the period 1999-2011. Descriptive statistics and-t-tests and Wilcoxon tests were performed.
Results: Overall, 100 FDA-priority review new molecular entities and new therapeutic biologics were approved by both agencies; 87.0% of the products were first approved by the FDA. The average FDA review time (9.16±8.39 months) was significantly lower than the EMA average review time (14.60±3.97 months) (p<0.0001). The FDA and the EMA granted orphan designation to 43% and 33%, respectively of the applications. There were differences in the administration route (1% of all products), dosage (8.0%), strength (23%), posology (51.0%), indications (30.0%), restrictions of use (52.0%), limitations of use (19.0%), and outcomes limitations (28.0%) approved by both regulatory agencies.
Conclusion: Significant differences exist in the characteristics of the priority review drugs approved by the FDA and the EMA.
Learning Areas:Biostatistics, economics
Provision of health care to the public
Public health administration or related administration
Public health or related laws, regulations, standards, or guidelines
Public health or related organizational policy, standards, or other guidelines
Public health or related research
Assess the characteristics (i.e. approvals, review time, and procedure and orphan designation) of FDA priority review new molecular entities (NME) and new therapeutic biologics (BLA) approved by the FDA and the EMA Evaluate differences between the US and European regulatory agencies in the indications, route of administration, dosage form, strength, and posology of pharmaceuticals approved by both agencies Discuss policy implications of differences may exist on clinical guidelines, public funding, pricing policies, and drug utilization
Keyword(s): Economic Analysis, Evaluation
Qualified on the content I am responsible for because: I have fifteen years of research experience in the area of pharmaceutical economics and policy. I have collected and analyzed data, and drafted manuscript in collaboration with co-authors.
Any relevant financial relationships? No
I agree to comply with the American Public Health Association Conflict of Interest and Commercial Support Guidelines, and to disclose to the participants any off-label or experimental uses of a commercial product or service discussed in my presentation.