Background: The societal costs and health benefits of tissue plasminogen activator (tPA) for ischemic stroke can be modeled and extended to the US population. Similarly, the societal impact of new thrombolytics with improved efficacy or safety or extending eligibility can also be modeled.
Methods: We previously modeled the impact of tPA on societal costs and health in the United States. Pertinent publications on utilization, societal cost, and health impact were identified by systematic review, updated to include studies describing the impact of extending the tPA time window. Information on utilization of tPA was integrated with published per-use data on costs and health impact (converted to 2004 dollars) to generate annual projections for the US population. Model inputs were modified to reflect various characteristics of new thrombolytics.
Results: At its current price, tPA saves $6074 and adds 0.75 quality-adjusted life year (QALY) per use. If tPA were priced at $50,000/QALY, a standard benchmark for cost-effectiveness, it would cost $45,800 per dose and would be expected to generate $458 million in revenue annually for its manufacturer. Thrombolytics for stroke that extended the time window or improved efficacy could generate greater revenue if priced at an accepted level of cost-effectiveness. Given current clinical development costs, development of candidate drugs with 2.8% to 5.7% probability of ultimate FDA approval would be justified.
Conclusions: tPA produces substantial health and economic benefits in the United States. Better thrombolytics for stroke could have substantial impact on society, and potential returns to developers would appear to justify greater investment in new candidates.