Leveraging Real-World Evidence in NMIBC Therapy Evaluations

EP: 1.Evolving Guidelines: Impact of Gene Therapy and BCG Failure Redefinitions on High-Risk NMIBC Patient Management

EP: 2.Applying Evidence Standards to Guide Gene Therapy Use in BCG-Unresponsive NMIBC

EP: 3.Impact of Real-World Treatment Experience on Gene Therapy Uptake in NMIBC

EP: 4.Navigating Challenges and Impact on Workflow with New NMIBC Therapies in Practice

EP: 5.Effective Strategies for Clinics Preparing to Implement Gene Therapy Into NMIBC Treatment Workflows

EP: 6.Reducing Clinical Resource Burden: Impact of Less Frequent NMIBC Therapy Administration

EP: 7.How Community-Based Gene Therapy Is Changing Treatment and Improving Quality of Life for Patients With BCG-Unresponsive NMIBC

EP: 8.Understanding NMIBC and Its Risk Factors

EP: 9.Navigating Treatment Burden in NMIBC

EP: 10.When BCG Falls Short: What’s Next for NMIBC Patients?

EP: 11.A New Era: The Rise of Gene Therapy in NMIBC

EP: 12.Where Do We Go From Here? Overcoming Barriers in NMIBC Care

EP: 13.Bridging the Gap Between Clinical Trial Evidence and Real-World Practice in NMIBC

EP: 14.Coverage Criteria and Clinical Evidence Used to Shape Health Plan Formulary Decisions in BCG-Unresponsive NMIBC

EP: 15.Prioritizing Safety and Efficacy: Role of Guidelines in NMIBC Formulary Decisions

EP: 16.Evaluating Cost-Effectiveness of NMIBC Therapies From a Health Plan Perspective

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EP: 17.Leveraging Real-World Evidence in NMIBC Therapy Evaluations

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Real-world evidence (RWE) currently plays a limited role in health plan decision-making for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) treatments, primarily due to the absence of standardized evaluation frameworks and the legal requirement for payers to defend coverage decisions with established clinical evidence. Health plans view RWE as supplementary “nice-to-have” information rather than primary decision-making data, particularly for newly launched therapies where limited real-world usage data exists. The reliance on controlled clinical trial data remains predominant, as these studies provide the regulatory foundation necessary for formulary coverage justification.

The landscape for RWE utilization is rapidly evolving through initiatives led by the Academy of Managed Care Pharmacy (AMCP), which is developing comprehensive frameworks with specific protocols for incorporating real-world data into coverage decisions. This framework addresses different drug life cycle phases, from preapproval through postlaunch periods, establishing data requirements for each stage. At product launch, the framework emphasizes pragmatic clinical trials, external control arms and economic modeling, focusing on primary end points that demonstrate value versus standard of care and address unmet medical needs.

For established therapies like pembrolizumab with extensive postmarket experience, abundant real-world data provide high confidence levels for formulary evaluation, contrasting sharply with newly approved treatments lacking comprehensive real-world utilization patterns. The framework considers specific outcomes relevant to NMIBC, including complete response rates, cystectomy-free survival, adverse events and comparative analyses between treatment options. As RWE standards mature, health plans anticipate greater integration of real-world outcomes data into formulary management, potentially revolutionizing how coverage decisions are made for complex oncology treatments like those used in BCG-unresponsive bladder cancer management.