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).

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.

Pharmacogenetic testing among patients with depression in a US managed care population.

Actionable drug-gene pairs relevant to depression treatment include CYP2D6 and CYP2C19 with specific antidepressants. While clinical use of pharmacogenetic testing is growing, little is known about pharmacogenetic testing for depression treatment in managed care. We determined the incidence of single-gene CYP2D6 and CYP2C19 testing following a new depression episode among US managed care patients, and described characteristics and antidepressant use of patients receiving tests.

Criteria and Scoring Functions Used in Multi-criteria Decision Analysis and Value Frameworks for the Assessment of Rare Disease Therapies: A Systematic Literature Review.

Background: Traditionally, the economic value of health technologies is assessed with cost-effectiveness (CE) and budget impact (BI) analyses. However, the evaluation of rare disease therapies often considers novel value criteria. Multi-criteria decision analysis (MCDA) is a promising tool in the assessment of value criteria that typically cannot be captured with traditional approaches.

Prevalence of Total, Diagnosed, and Undiagnosed Diabetes Among Adults: United States, 2013-2016.

Diabetes is a major cause of morbidity and mortality in the United States (1-3). Diabetes can be present but undiagnosed, meaning that a person can have diabetes but not report having ever been told by a doctor or health professional that they have the condition. Type 2 diabetes can progress over an extended time period with gradual, often unnoticed, changes occurring before diagnosis.