Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases.

The benefits of preventive interventions lack comprehensive evaluation in standard health technology assessments (HTA), particularly for rare and transmissible diseases. To identify possible considerations for future HTA using analogies between the treatment and prevention of rare diseases. An Expert panel meeting assessed whether one HTA assessment framework can be applied to assess both rare disease treatments and preventive interventions.

The impact of external reference pricing on pharmaceutical costs and market dynamics.

Growth in the cost of prescription drugs in the US has generated significant interest in the use of external reference pricing (ERP) to tie prices paid for drugs to those in other countries. We used data from the Pricentric ONE™ database, an international drug pricing database, to examine product launch timing, launch price, and price changes from January 2010 - October 2021 in both ERP and non-ERP settings, with a focus on 100 high-priced drugs of interest to Medicare and Medicaid. We found that ERP policies were associated with a 73% reduction in the likelihood of drug launch within 9 months of regulatory approval relative to non-ERP settings.

Specialty drug coverage varies between health plans' medical and pharmacy benefit policies.

In an effort to control drug spending, health plans are increasingly shifting specialty drugs from their medical benefit to the pharmacy benefit. One consequence of this trend is that some health plans have both a medical and a pharmacy coverage policy for the same drug. To examine how frequently health plans issue medical and pharmacy benefit policies for the same specialty drug and to evaluate the concordance between plans' medical and pharmacy policies when plans issue both policy types.

A review of HTA guidelines on societal and novel value elements.

Objectives: Health technology assessment (HTA) organizations vary in terms of how they conduct assessments. We assess whether and to what extent HTA bodies have adopted societal and novel elements of value in their economic evaluations.

Methods: After categorizing "societal" and "novel" elements of value, we reviewed fifty-three HTA guidelines.

Recommendations for economic evaluations of cell and gene therapies: a systematic literature review with critical appraisal.

Objective: No consensus exists on the ideal methodology to evaluate the economic impact and value of new, potentially curative gene therapies. We aimed to identify and describe published methodologic recommendations for the economic evaluation of gene therapies and assess whether these recommendations have been applied in published evaluations.

Methods: This study was conducted in three stages: a systematic literature review of methodologic recommendations for economic evaluation of gene therapies; an assessment of the appropriateness of recommendations; and a review to assess the degree to which the recommendations were applied in published evaluations.

Factors Associated with Biosimilar Exclusions and Step Therapy Restrictions Among US Commercial Health Plans.

Background: Biosimilars have been introduced with the goal of competing with high-priced biologic therapies, yet their adoption has been slower than expected and resulted in limited efficiency gains. We aimed to explore factors associated with biosimilar coverage relative to their reference products by commercial plans in the United States (US).

Methods And Data: We identified 1181 coverage decisions for 19 commercially available biosimilars, corresponding to 7 reference products and 28 indications from the Tufts Medical Center Specialty Drug Evidence and Coverage database.

The association between US Food and Drug Administration-expedited review designations and health plan specialty drug coverage.

The US Food and Drug Administration (FDA) speeds approval of important clinical advancements through 4 expedited review programs: Priority Review, Accelerated Approval, Fast Track, and Breakthrough Therapy. Whether health plans prioritize coverage of expedited drugs relative to drugs that the FDA determined did not qualify from these programs is unclear. To investigate how fast US commercial health plans issued coverage policies for drugs included in different numbers of FDA-expedited programs.

The economic and humanistic burden of bipolar disorder in adults in the United States.

Background: Bipolar disorder is associated with functional impairment and diminished health-related quality of life (HRQoL). The purpose of this study was to estimate the annual per patient direct healthcare costs, indirect costs, and HRQoL of patients with bipolar disorder by depressive symptom severity and overall compared to the general population in the US.

Methods: This cross-sectional study used self-reported data from the 2020 US National Health and Wellness Survey.

Commercial health plans use of patient subgroup restrictions: An analysis of orphan and US Food and Drug Administration-expedited programs.

Health plans apply utilization management criteria to guide their enrollees' access to prescription drugs. Patient subgroup restrictions (ie, clinical prerequisites for drug coverage) are a form of utilization management that have not been thoroughly investigated. To examine the frequency with which large US commercial health plans impose patient subgroup restrictions beyond the US Food and Drug Administration (FDA) label in their coverage policies for orphan drugs and for drugs included in 1 or more FDA-expedited programs.

Variation in Medicaid and commercial payer coverage of aducanumab for Alzheimer's disease.

Introduction: Ever since the United States Food and Drug Administration (FDA) approved aducanumab and Centers for Medicare & Medicaid Service (CMS) restricted coverage for the drug, a crucial question has been how other payers will behave. This study examined how Medicaid and commercial plans cover aducanumab.

Methods: We created a database of aducanumab coverage policies issued by Medicaid fee-for-service programs (50 states and DC) and 35 of the largest commercial plans (covering ∼ 84% of the commercially insured population).

Development and Preliminary Validation of an Instrument to Measure Symptoms and Impacts in Patients with Proliferative Diabetic Retinopathy.

Introduction: Following a review of patient-reported outcome (PRO) instruments in the literature, existing PRO instruments may not adequately capture the experience of receiving treatment for proliferative diabetic retinopathy (PDR). Therefore, this study aimed to develop a de novo instrument to comprehensively assess the patient experience of PDR.

Methods: This qualitative, mixed-methods study comprised item generation for the Diabetic Retinopathy-Patient Experience Questionnaire (DR-PEQ), content validation in patients with PDR, and preliminary Rasch measurement theory (RMT) analyses.

Examining US commercial health plans' use of The Institute for Clinical and Economic Review's reports in specialty drug coverage decisions.

The Institute for Clinical and Economic Review (ICER) has emerged in a visible role in US health care. However, it is unclear to what extent US commercial health plans use ICER value assessments in their specialty drug coverage decisions. To evaluate the relationship between ICER's reported cost-effectiveness ratios (CERs) and coverage restrictiveness.

Patient and Caregiver Views on Measures of the Value of Health Interventions.

Purpose: We aimed to investigate patient and caregiver views on the relative importance of traditional and nontraditional domains of value, and to determine if these views differed according to key demographic characteristics.

Patients And Methods: We conducted a modified Delphi approach using a web-based survey of adult patients managing a chronic condition or caregivers of a patient with chronic illness who were recruited using purposive sampling focused on demographic and clinical characteristics. The first survey round asked participants to rate the 13 domains of value on a 5-point Likert scale and rank each domain that they rated as important or very important.

The Value of New: Consideration of Product Novelty in Health Technology Assessments of Pharmaceuticals.

Background: Efforts to understand how treatments affect patients and society have broadened the criteria that health technology assessment (HTA) organizations apply to value assessments. We examined whether HTA agencies in eight countries consider treatment novelty in methods and deliberations.

Methods: We defined a novel pharmaceutical product to be one that offers a new approach to treatment (e.

Toward comprehensive value assessment for Alzheimer's disease innovations.

Introduction: Assessing medical technologies for Alzheimer's disease (AD) creates challenges for current methods of value assessment. New value assessment approaches for AD are also needed.

Methods: We adapted concepts from health economics to help guide decision makers to more informed decisions about AD therapies and diagnostics.