Commercial coverage of specialty drugs, 2017-2021.

Health plans guide their enrollees' access to specialty drugs through coverage policies. We examined a set of health plan policies to determine if they have become more or less stringent over time. We did so by comparing the consistency of policies with Food and Drug Administration (FDA) label indications.

Documenting the Full Value of Vaccination: A Systematic Review of Value Frameworks.

Objectives: Economic evaluations of vaccination may not fully account for nonhealth patient impacts on families, communities, and society (ie, broader value elements). Omission of broader value elements may reflect a lack of established measurement methodology, lack of agreement over which value elements to include in economic evaluations, and a lack of consensus on whether the value elements included should vary by vaccination type or condition. We conducted a systematic review of value frameworks to identify broader value elements and measurement guidance that may be useful for capturing the full value of vaccination.

Cost-effectiveness of emicizumab prophylaxis for haemophilia A with inhibitors: an adaptive health technology assessment for the Indian setting.

Objective: To assess the cost-effectiveness of emicizumab prophylaxis for patients having haemophilia A with inhibitors in the Indian context using an adaptive health technology assessment (aHTA) methodology.

Design: Economic evaluation using multiple approaches aimed at adjusting previously generated cost-effectiveness results based on (1) price differences only ('simple') and (2) differences in cost and expected treatment duration ('moderate') and differences in cost, inflation and life expectancy ('complex').

Setting: Typical haemophilia care in India.

Harnessing Health Technology Assessment in Latin America and the Caribbean: Keeping the Region on Course.

Latin America and the Caribbean has made significant progress toward universal health coverage (UHC), but health spending efficiency, equity, and sustainability remain major challenges-and progress is hindered by the difficult macroeconomic context. Health technology assessment (HTA) can make resource allocation more efficient and equitable when systematically used to inform coverage decisions. We highlight five considerations that need to be taken into account to realize the full potential of HTA in the LAC region: i) explicitly link HTA to decision-making and anchor it in legal frameworks, ii) systematically incorporate the opportunity cost as a core principle into HTA activities informing coverage decisions, iii) make the internationally available evidence more fit for purpose for low- and middle-income countries (LMICs), iv) incorporate pragmatism as a key principle of HTA activities in the region, and v) institutionalize the monitoring of HTA processes and results.

Economic Evaluations of Chimeric Antigen Receptor T-Cell Therapies for Hematologic and Solid Malignancies: A Systematic Review.

Objectives: This study aimed to systematically review evidence on the cost-effectiveness of chimeric antigen receptor T-cell (CAR-T) therapies for patients with cancer.

Methods: Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments, and economic evaluations that compared costs and effects of CAR-T therapy in patients with cancer were included.

Geographic disparity in the distribution of cancer clinical trials in the United States and the associated factors.

Background: Little is known regarding the geographic disparity in the distribution of phase 1-3 clinical trials of new cancer treatments in the US and the associated factors.

Objective: To examine county-level variation in the number of phase 1-3 cancer clinical trials and the associations between county characteristics and having phase 1-3 cancer clinical trials.

Methods: We identified phase 1-3 cancer clinical trials started in the US between January 2008 and December 2022 from the Aggregate Analysis of ClinicalTrials.

Do Quality-Adjusted Life Years Discriminate Against the Elderly? An Empirical Analysis of Published Cost-Effectiveness Analyses.

Objectives: Critics of quality-adjusted life-years argue that it discriminates against older individuals. However, little empirical evidence has been produced to inform this debate. This study aimed to compare published cost-effectiveness analyses (CEAs) on patients aged ≥65 years and those aged <65 years.

Racial and ethnic differences in disease course Medicare expenditures for beneficiaries with dementia.

Background: Research on racial and ethnic disparities in costs of care during the course of dementia is sparse. We analyzed Medicare expenditures for beneficiaries with dementia to identify when during the course of care costs are the highest and whether they differ by race and ethnicity.

Methods: We analyzed data from the 2000-2016 Health and Retirement Study (HRS) linked with corresponding Medicare claims to estimate total Medicare expenditures for four phases: (1) the year before a dementia diagnosis, (2) the first year following a dementia diagnosis, (3) ongoing care for dementia after the first year, and (4) the last year of life.

It Is Time to Reconsider the 3% Discount Rate.

Objectives: Health technology assessment (HTA) guidance often recommends a 3% real annual discount rate, the appropriateness of which has received limited attention. This article seeks to identify an appropriate rate for high-income countries because it can influence projected cost-effectiveness and hence resource allocation recommendations.

Methods: The author conducted 2 Pubmed.

Incorporating Real Option Value in Valuing Innovation: A Way Forward.

Background: Considerable progress has been made in defining and measuring the real option value (ROV) of medical technologies. However, questions remain on how to estimate (1) ROV outside of life-extending oncology interventions; (2) the impact of ROV on costs and cost effectiveness; and (3) potential interactions between ROV and other elements of value.

Methods: We developed a 'minimal modeling' approach for estimating the size of ROV that does not require constructing a full, formal cost-effectiveness model.

The value and impact of health technology assessment: discussions and recommendations from the 2023 Health Technology Assessment International Global Policy Forum.

Health technology assessment (HTA) programs inform decision making about the value and reimbursement of new and existing health technologies; however, they are under increasing pressure to demonstrate that they are a cost-effective use of finite healthcare resources themselves. The 2023 HTAi Global Policy Forum (GPF) discussed the value and impact of HTA, including how it is assessed and communicated, and how it could be enhanced in the future. This article summarizes the discussions held at the 2023 HTAi GPF, where the challenges and opportunities related to the value and impact of HTA were debated.

Patient Welfare Implications of Indication-Specific Value-Based Pricing of Multi-Indication Drugs.

Objectives: Indication-specific value-based pricing (ISVBP) is a mechanism that allows the prices of multi-indication drugs to vary across indications by aligning the drug prices with value. However, the overall impact of ISVBP on patients across indications is uncertain. This study examines the theoretical welfare effects of ISVBP for multi-indication drugs and compares consumer surplus under ISVBP and single pricing, the latter of which is based on the weighted average value.

Estimating prevalence of early symptomatic Alzheimer's disease in the United States.

Introduction: Understanding the prevalence of treatment-eligible Alzheimer's disease (AD) is crucial for policy planning.

Methods: We used a comprehensive literature review and population cascade approach to estimate the number of amyloid-positive, clinically diagnosed patients with mild cognitive impairment (MCI) or mild dementia due to AD in the United States.

Results: An estimated 666,646 individuals were identified as having MCI due to AD (range: 351,926-1,227,776) and 620,850 individuals as having mild dementia due to AD (range: 445,082-820,339).

Variation in Medicaid and commercial coverage of cell and gene therapies.

Growth in the availability of cell and gene therapies (CGTs) promises significant innovation in the treatment of serious diseases, but the high cost and one-time administration of CGTs has also raised concern about strain on health plan budgets and inequity in access. We used coverage information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database for 18 large commercial health plans in the US and information from state Medicaid websites to examine variation in coverage of 11 CGTs in August 2021. We found that US commercial and Medicaid health plans imposed restrictions in 53.

The Impact of Biosimilar Use on Total Cost of Care and Provider Financial Performance in the Medicare Oncology Care Model: A Population-Based Simulation Study.

Introduction: Payment for oncology care is increasingly moving from fee-for-service to value-based payment (VBP). VBPs are agreements in which providers are held accountable for total cost of care (TCOC) through risk-sharing arrangements with payers that tie reimbursement levels to TCOC benchmarks. Oncology biosimilars may play an important role in managing financial risk in the VBPs like Medicare's Oncology Care Model (OCM), but there has been limited research in this area.

Life-Sustaining Treatments Among Medicare Beneficiaries with and without Dementia at the End of Life.

Background: Older adults with dementia including Alzheimer's disease may have difficulty communicating their treatment preferences and thus may receive intensive end-of-life (EOL) care that confers limited benefits.

Objective: This study compared the use of life-sustaining interventions during the last 90 days of life among Medicare beneficiaries with and without dementia.

Methods: This cohort study utilized population-based national survey data from the 2000-2016 Health and Retirement Study linked with Medicare and Medicaid claims.

Attitudes on Equal Health Care Access versus Efficient Clinical Decisions across a Not-for-Profit Health Care System.

Background: Professional roles within a hospital system may influence attitudes behind clinical decisions.

Objective: To determine participants' preferences about clinical decisions that either value equal health care access or efficiency.

Design: Deidentified survey asking participants to choose between offering a low-cost screening test to a whole population ("equal access") or a more sensitive, expensive test that could be given to only half of the population but resulting in 10% more avoided deaths ("efficient").

Varied use of step therapy among Medicare Advantage plans.

Objectives: First, to examine 7 large Medicare Advantage (MA) plans' use of step therapy. Second, to compare step therapy that health plans imposed in their MA and commercial (employer) drug coverage policies.

Study Design: Database analysis.

Improving Food and Drug Administration-Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval.

Unlabelled: Policy Points The increasing number of drugs granted accelerated approval by the Food and Drug Administration (FDA) has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We recommend several administrative and legislative approaches for improving FDA-Centers for Medicare and Medicaid Services (CMS) coordination around accelerated-approval drugs, including promoting earlier discussions among the FDA, the CMS, and drug companies; strengthening Medicare's coverage with evidence development program; linking Medicare payment to evidence generation milestones; and ensuring that the CMS has adequate staffing and resources to evaluate new therapies. These activities can help improve the integrity; transparency; and efficiency of approval, coverage, and payment processes for drugs granted accelerated approval.

Assessing the impact of grief on quality of life, work productivity, and health outcomes for parents bereaved from SMA: A study protocol.

Background: U.S. cost-effectiveness recommendations suggest that analyses should include all costs and effects relevant to the decision problem [1].