A Systems Evaluation Model for the Development of Companion Diagnostics and Associated Molecularly Targeted Therapies

Abstract

Abstract Purpose Monoclonal antibodies (mAbs) are important active ingredients of molecularly targeted drugs, which are only effective for specific patient groups. Early assessment of their effectiveness is important for more efficient use of time and resources. Companion diagnostics (CDx) are medical devices or tests to identify groups of promising patients based on specific biomarkers. This work offers a systems evaluation model and a comprehensive assessment from multiple stakeholder perspectives. Methods This work introduces a new systems model for assessing available treatment options. Process system diagrams, consisting of independently defined unit structures, are applied to represent the expected decision points and outcomes. A sensitivity analysis is conducted to identify the critical requirements for achieving cost-effectiveness. The model was applied to a case of terminal colorectal cancer treatment to compare mAb drugs to standard therapy. Results The results showed that from the payers’ perspective, the cost and response rates of the mAb drug were critical parameters to improve for achieving the target cost-effectiveness. The results give quantitative guidance for the required improvement. Conclusion This work represents an important step towards a fair and systematic assessment of treatment alternatives and serves as a guideline for future CDx and therapy technology development efforts.