Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid.

Objective: High upfront costs and long-term benefit uncertainties of gene therapies challenge Medicaid budgets, making value-based contracts a potential solution. However, value-based contract design is hindered by cost-offset uncertainty. The aim of this study is to determine actual cost-offsets for valoctocogene roxaparvovec (hemophilia A) and etranacogene dezaparvovec (hemophilia B) from Colorado Medicaid's perspective, defining payback periods and its uncertainty from the perspective of Colorado Medicaid.

Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program.

Introduction: The United States has begun assessing the value of pharmaceuticals to inform negotiated prices in the Medicare program. Given strong political objections in the United States to the use of QALYs, Medicare will need to adopt an alternative approach to measuring value.

Areas Covered: In this narrative review, we identified six alternative approaches to measuring value (equal value life-years, health years in total, generalized risk-adjusted cost-effectiveness, severity weighting based on absolute or proportional shortfall, comparative effectiveness based on conventional clinical endpoints, and comparative effectiveness based on both conventional endpoints and patient-centric value elements) and five criteria for assessing these approaches (responsiveness to concerns about discrimination, feasibility, transparency, flexibility, and the ability to incorporate factors beyond traditional value elements).

Multistakeholder Perceptions of Additional Value Elements for United States Value Assessment of Health Interventions.

Objectives: Limitations in conventional cost-effectiveness methods have led to calls for incorporation of additional value elements in assessments of health technologies. However, gaps remain in how additional value elements may inform decision making. This study aimed to prioritize additional value elements from the perspective of healthy individuals without a specific condition or indicated for a specific treatment in the United States among a multistakeholder panel and compare the importance of perspective-specific value elements.

Framework for Patient Experience Value Elements in Rare Disease: A Case Study Demonstrating the Applicability of Combined Qualitative and Quantitative Methods.

Background And Objective: Several novel methods have been suggested to extend a conventional value assessment to capture a more comprehensive perspective of value from a patient perspective. The objective of this research was to demonstrate a framework for implementing a combined qualitative and quantitative method to elicit and prioritize patient experience value elements in rare diseases. Neuromyelitis optica spectrum disorder was used as a case study.

Stakeholder perception of pharmaceutical value: A multicriteria decision analysis pilot case study for value assessment in the United States.

Recent attention to value frameworks has highlighted limitations of current conventional value and health technology assessment (V/HTA) methods (eg, cost-effectiveness). Multicriteria decision analysis (MCDA) has potential as a supplemental tool to incorporate additional value criteria into conventional value assessment. To conduct a pilot study to illustrate the impact of an MCDA approach on the value perceptions of hypothetical treatment profiles from a multistakeholder panel.