The Cost of Continuous Glucose Monitoring Versus Self-Monitoring of Blood Glucose in Type 1 Diabetes Pregnancies in the United States: A Cost-Consequences Analysis Using Real-World Evidence.

Data on the cost implications of continuous glucose monitoring (CGM) use in type 1 diabetes (T1D) pregnancies in the United States are sparse. Drawing on associations identified in real-world evidence from a retrospective chart review at the Barbara Davis Center for Diabetes, we conducted a cost-consequences analysis of CGM use versus self-monitoring of blood glucose (SMBG), inclusive of neonatal intensive care unit (NICU) spending. In the base-case analysis assuming per-label CGM use and per-guideline finger-stick frequency, the per-person cost was $16,254 for CGM versus $15,182 for SMBG.

How Do the Institute for Clinical and Economic Review's Assessments of Comparative Effectiveness Compare With the German Federal Joint Committee's Assessments of Added Benefit? A Qualitative Study.

Objectives: We compared the Institute for Clinical and Economic Review's (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee's (G-BA) added benefit ratings, and explored what factors may explain the disagreement between the 2 organizations.

Methods: We included drugs if they were assessed by ICER under its 2020 to 2023 Value Assessment Framework and had a corresponding assessment by G-BA as of January 2024 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER's proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA's certainty ratings.

An empirical ethics study of the coherence of NICE technology appraisal policy and its implications for moral justification.

Background: As the UK's main healthcare priority-setter, the National Institute for Health and Care Excellence (NICE) has good reason to want to demonstrate that its decisions are morally justified. In doing so, it has tended to rely on the moral plausibility of its principle of cost-effectiveness and the assertion that it has adopted a fair procedure. But neither approach provides wholly satisfactory grounds for morally defending NICE's decisions.

The ethical canary: narrow reflective equilibrium as a source of moral justification in healthcare priority-setting.

Healthcare priority-setting institutions have good reason to want to demonstrate that their decisions are morally justified-and those who contribute to and use the health service have good reason to hope for the same. However, finding a moral basis on which to evaluate healthcare priority-setting is difficult. Substantive approaches are vulnerable to reasonable disagreement about the appropriate grounds for allocating resources, while procedural approaches may be indeterminate and insufficient to ensure a just distribution.

Assessing clinical benefit in the Medicare Drug Price Negotiation Program: A 2-step approach for improving transparency, consistency, and meaningful patient engagement.

In early 2024, the Centers for Medicare & Medicaid Services (CMS) will send initial price offers to the manufacturers of the first 10 drugs selected for the Medicare Drug Price Negotiation Program, established under the Inflation Reduction Act. However, CMS has not specified exactly how it will adjust the starting point for an initial price offer based on assessment of a drug's clinical benefit. This article addresses unanswered questions relating to CMS' methods for assessing clinical benefit.

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).

Estimated Savings From Using Added Therapeutic Benefit and Therapeutic Reference Pricing in United States Medicare Drug Price Negotiations.

Objectives: US Medicare will begin negotiating prices for top-selling drugs in 2023. This study describes and estimates potential savings from a therapeutic reference pricing approach, linking comparative effectiveness with the costs of existing therapeutic alternatives, that Medicare could use to adjust the starting point for price negotiations.

Methods: First, we identified target drugs likely to be selected for Medicare negotiation.

Moral and Social Values in Evidence-Informed Deliberative Processes for Health Benefit Package Design Comment on "Evidence-Informed Deliberative Processes for Health Benefit Package Design - Part II: A Practical Guide".

An evidence-informed deliberative process (EDP) is defined as "a practical and stepwise approach for health technology assessment (HTA) bodies to enhance legitimate health benefit package design based on deliberation between stakeholders to identify, reflect and learn about the meaning and importance of values, informed by evidence on these values." In this commentary, I discuss some considerations for EDPs that arise from acknowledging the difference between social and moral values. First, the best practices for implementing EDPs may differ depending on whether the approach is grounded in moral versus social values.

Incorporating Added Therapeutic Benefit and Domestic Reference Pricing Into Medicare Payment for Expensive Part B Drugs.

Objectives: Identify expensive Part B drugs and evidence for each drug's added benefit and model a reimbursement policy for Medicare that integrates added benefit assessment and domestic reference pricing.

Methods: A retrospective analysis using a 20% nationally representative sample of 2015 to 2019 traditional Medicare Part B claims. Expensive drugs were defined as having average annual spending per beneficiary exceeding the average annual social security benefit ($17 532 in 2019).

Comparison of Out-of-Pocket Spending on Ultra-Expensive Drugs in Medicare Part D vs Commercial Insurance.

Importance: Little is known about how out-of-pocket burden differs between Medicare and commercial insurance for ultra-expensive drugs.

Objective: To investigate out-of-pocket spending for ultra-expensive drugs in the Medicare Part D program vs commercial insurance.

Design, Setting, And Participants: This was a retrospective, population-based cohort study of individuals using ultra-expensive drugs included in a 20% nationally random sample of prescription drug claims from Medicare Part D and individuals aged 45 to 64 years using ultra-expensive drugs included in a large national convenience sample of outpatient pharmaceutical claims from commercial insurance plans.

Integrating Health Technology Assessment and the Right to Health in South Africa: A Qualitative Content Analysis of Substantive Values in Landmark Judicial Decisions.

The World Health Assembly has encouraged WHO member-states to establish capacity in health technology assessment (HTA) as a support for achieving universal health coverage (UHC). Simultaneously, the WHO has stated that UHC is "a practical expression of the concern for health equity and the right to health." This has prompted questions about potential tensions between priority-setting efforts and the right to health on the road to UHC.

Association Between Drug Characteristics and Manufacturer Spending on Direct-to-Consumer Advertising.

Importance: Some drugs are heavily marketed through direct-to-consumer advertising.

Objective: To identify drug characteristics associated with a greater share of promotional spending on advertising directly to consumers.

Design, Setting, And Participants: Exploratory cross-sectional analysis of drug characteristics and promotional spending for the 150 top-selling branded prescription drugs in the US in 2020 as identified from IQVIA National Sales Perspectives data.

The Value of Explicit, Deliberative, and Context-Specified Ethics Analysis for Health Technology Assessment: Evidence From a Novel Approach Piloted in South Africa.

Objectives: This article explores the perceived value, including associated strengths and challenges, of using a context-specified ethics framework to guide deliberative health technology appraisals.

Methods: The South African Values and Ethics for Universal Health Coverage (SAVE-UHC) approach, piloted in South Africa, consisted of 2 phases: (1) convening a national multistakeholder working group to develop a provisional ethics framework and (2) testing the provisional ethics framework through simulated health technology assessment appraisal committee meetings (SACs). Three SACs each reviewed 2 case studies of sample health interventions using the framework.

Developing and piloting a context-specified ethics framework for health technology assessment: the South African Values and Ethics for Universal Health Coverage approach.

Objectives: While ethics has been identified as a core component of health technology assessment (HTA), there are few examples of practical, systematic inclusion of ethics analysis in HTA. Some attribute the scarcity of ethics analysis in HTA to debates about appropriate methodology and the need for ethics frameworks that are relevant to local social values. The "South African Values and Ethics for Universal Health Coverage" (SAVE-UHC) project models an approach that countries can use to develop HTA ethics frameworks that are specific to their national contexts.

Public opinion regarding U.S. Food and Drug Administration approval of aducanumab and potential policy responses: A nationally representative survey.

Background: Despite controversy among experts regarding aducanumab's approval by the U.S. Food and Drug Administration, little is known about public opinion on this matter.

Integrating health technology assessment and the right to health: a qualitative content analysis of procedural values in South African judicial decisions.

South Africa's move towards implementing National Health Insurance includes a commitment to establish a health technology assessment (HTA) body to inform health priority-setting decisions. This study sought to analyse health rights cases in South Africa to inform the identification of country-specific procedural values related to health priority-setting and their implementation in a South African HTA body. The focus on health rights cases is motivated in part by the fact that case law can be an important source of insight into the values of a particular country.

Seventy-two-hour Return Initiative: Improving Emergency Department Discharge to Decrease Returns.

Unlabelled: Unscheduled return visits within 72 hours of discharge account for 4% of pediatric emergency department (ED) visits each year and are a quality indicator of ED care. This project aimed to reduce the unexpected 72-hour return visit rate for a network of ED and urgent cares (UC) by improving discharge processes.

Methods: A multidisciplinary team conducted a quality improvement initiative in the EDs/UCs of a tertiary children's hospital network.

Assessing the Added Therapeutic Benefit of Ultra-Expensive Drugs.

Objectives: While the United States does not have a method for assessing the added therapeutic benefit of drugs, France, Canada, and Germany do. We examined the added therapeutic benefit of the most expensive drugs prescribed to Medicare Part D beneficiaries in the United States.

Methods: We identified ultra-expensive drugs with annual Medicare spending that exceeded $62 794 (United States GDP per capita in 2018) using Medicare Part D Prescription Drug Spending and Utilization Data.