The impact of capping health system cost savings on the projected cost-effectiveness of etranacogene dezaparvovec compared with factor IX prophylaxis for the treatment of hemophilia B.

This viewpoint discusses cost-effectiveness estimates for EtranaDez, a gene therapy for hemophilia B, using the Institute for Clinical and Economic Review's (ICER) framework for single and short-term therapies (SSTs). EtranaDez offers long-term benefits from a single administration, in contrast to the high costs and frequent dosing required by current factor IX prophylaxis. However, the projected gains in health from EtranaDez are small relative to the cost implications of the therapy, and consequently, how the cost offsets associated with EtranaDez are counted has a substantial impact on assessing its cost-effectiveness.

Real-world antiseizure medication treatment outcomes in drug-resistant focal epilepsy patients.

Objective: To gather real-world evidence on antiseizure medications (ASMs) treatment patterns and related outcomes in patients with drug-resistant focal epilepsy.

Methods: Medical insurance claims from the start of 2014 till the end of 2019 were used. Patient selection criteria included International Classification of Diseases (ICD) codes followed by documented ASM use.

Comparison of Preferences and Data Quality between Discrete Choice Experiments Conducted in Online and Face-to-Face Respondents.

Introduction: Discrete choice experiments (DCE) are increasingly being conducted using online panels. However, the comparability of such DCE-based preferences to traditional modes of data collection (e.g.

Quantitative revenue estimates and qualitative assessments of innovative fundraising sources for treating rare diseases in Colombia.

Like many developing countries, Colombia faces difficulties in financing health-care services as well as programs for health promotion and health education and there is evidence that its health-care system is underperforming. To provide evidence-based estimates of potential funding levels and assess the strengths, weaknesses, and viability of innovative funding mechanisms with a focus on treating rare diseases in Colombia. The strategy involved evidence-based projections of potential funding levels and a qualitative viability assessment using an expert panel.

The effectiveness and value of gene therapy for hemophilia: A Summary from the Institute for Clinical and Economic Review's California Technology Assessment Forum.

Dr Tice and Mr Sarker received ICER grants during the conduct of the study. Dr Moradi, Ms Herce-Hagiwara, Dr Faghim, Dr Agboola, Dr Rind, and Dr Pearson reports grants from Arnold Ventures, grants from Blue Cross Blue Shield of MA, grants from California Healthcare Foundation, grants from The Commonwealth Fund, grants from The Peterson Center on Healthcare, during the conduct of the study; other from Aetna, other from America's Health Insurance Plans, other from Anthem, other from AbbVie, other from Alnylam, other from AstraZeneca, other from Biogen, other from Blue Shield of CA, other from Cambia Health Services, other from CVS, other from Editas, other from Express Scripts, other from Genentech/Roche, other from GlaxoSmithKline, other from Harvard Pilgrim, other from Health Care Service Corporation, other from Health Partners, other from Johnson & Johnson (Janssen), other from Kaiser Permanente, other from LEO Pharma, other from Mallinckrodt, other from Merck, other from Novartis, other from National Pharmaceutical Council, other from Premera, other from Prime Therapeutics, other from Regeneron, other from Sanofi, other from Spark Therapeutics, other from United Healthcare, other from HealthFirst, other from Pfizer, other from Boehringer-Ingelheim, other from uniQure, other from Evolve Pharmacy Solutions, other from Humana, other from Sun Life, outside the submitted work.

Mavacamten for hypertrophic cardiomyopathy: effectiveness and value.

Funding for this summary was contributed by Arnold Ventures, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Sun Life Financial, uniQure, and United Healthcare. Beinfeld, Nhan, Rind, and Pearson are employed by ICER.

Protocol for a randomized controlled trial of low-intensity physical activity for frail older adults: Promoting seniors' health with home care aides (Pro-Home).

Regular participation in physical activity benefits older adults physically and mentally. However, the availability and assessment of physical activity programs that are safe and appropriate for homebound older adults at risk for nursing home admission are limited. Here we describe the protocol for a randomized controlled trial that examines the effectiveness of a gentle physical activity program.

An initial evaluation of expanded hemodialysis on hospitalizations, drug utilization, costs, and patient utility in Colombia.

To examine new evidence linking expanded hemodialysis (HDx) using a medium cut-off (MCO) membrane with hospitalizations, hospital days, medication use, costs, and patient utility. This retrospective study utilized data from Renal Care Services medical records database in Colombia from 2017 to 2019. Clinics included had switched all patients from high flux hemodialysis (HD HF) to HDx and had at least a year of data on HD HF and HDx.

Comparison of online and face-to-face valuation of the EQ-5D-5L using composite time trade-off.

Objective: The aim of this study was to compare online, unsupervised and face-to-face (F2F), supervised valuation of EQ-5D-5L health states using composite time trade-off (cTTO) tasks.

Methods: The official EuroQol experimental design and valuation protocol for the EQ-5D-5L of 86 health states were implemented in interviewer-assisted, F2F and unsupervised, online studies. Validity of preferences was assessed using prevalence of inconsistent valuations and expected patterns of TTO values.

Understanding Student Preferences in the Selection of a Graduate Allied Health Program: A Conjoint Analysis Study.

Issue: As the healthcare landscape rapidly changes, graduate allied health programs must position themselves to educate the next generation of healthcare professionals in a highly competitive landscape. No studies have directly measured the relative importance of attributes in program selection by prospective healthcare students.

Methods: We surveyed graduate healthcare management program applicants in the 2018 admissions cycle (n=512) to determine which attributes were most important in program choice.

Increasing respondent engagement in composite time trade-off tasks by imposing three minimum trade-offs to improve data quality.

Background: Web-based surveys are increasingly utilized for health valuation studies but may be more prone to lack of engagement and, therefore, poor data validity. The objective of this study was to evaluate the effect of imposed engagement (i.e.

Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group.

Precision medicine is a dynamic area embracing a diverse and increasing type of approaches that allow the targeting of new medicines, screening programs or preventive healthcare strategies, which include the use of biologic markers or complex tests driven by algorithms also potentially taking account of patient preferences. The International Society for Pharmacoeconomics and Outcome Research expanded its current work around precision medicine to (1) describe the evolving paradigm of precision medicine with examples of current and evolving applications, (2) describe key stakeholders perspectives on the value of precision medicine in their respective domains, and (3) define the core factors that should be considered in a value assessment framework for precision medicine. With the ultimate goal of improving health of well-defined patient groups, precision medicine will affect all stakeholders in the healthcare system at multiple levels spanning the individual perspective to the societal perspective.

The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy.

Funding for this summary was contributed by Arnold Ventures, Commonwealth Fund, California Health Care Foundation, National Institute for Health Care Management (NIHCM), New England States Consortium Systems Organization, Blue Cross Blue Shield of Massachusetts, Harvard Pilgrim Health Care, Kaiser Foundation Health Plan, and Partners HealthCare to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Agboola, Fluetsch, Rind, and Pearson are employed by ICER.

Implementation Lessons From a Randomized Trial Integrating Community Asthma Education for Children.

This study characterized and compared the implementation of clinically integrated community health workers (CHWs) to a certified asthma educator (AE-C) for low-income children with asthma. In the AE-C arm (N = 115), 51.3% completed at least one in-clinic education session.

Estimating the Impact of Food and Drug Administration's Unapproved Drug Initiative on Drug Prices and Sales.

Background: The 2006 FDA's Unapproved Drug Initiative (UDI) aimed to improve safety and public health by decreasing the availability of drug products that never obtained FDA approval (unapproved drug products) in the market and incentivizing manufacturers to emphasize that these products must obtain FDA approval. The objective of this study was to measure changes in the prices, sales, and quantities sold of drug products approved under the FDA-UDI.

Methods: Drug products that obtained voluntary approval under FDA-UDI from 2006 to 2015 were identified and trends in prices, sales, and units sold were analyzed using the IQVIA National Sales Perspective database.

Evaluating a remote patient monitoring program for automated peritoneal dialysis.

Background: The benefits of automated peritoneal dialysis (APD) have been established, but patient adherence to treatment remains a concern. Remote patient monitoring (RPM) programs are a potential solution; however, the cost implications are not well established. This study modeled, from the payer perspective, expected net costs and clinical consequences of a novel RPM program in Colombia.

Racial and ethnic disparity in palliative care and hospice use.

Objectives: Prior research has demonstrated differences across race and ethnicity, as well as across geographic location, in palliative care and hospice use for patients near the end of life. However, there remains inconsistent evidence regarding whether these disparities are explained by hospital-level practice variation. The goals of this study were to evaluate whether inpatient palliative care consultation use and discharge to hospice differed by race/ethnicity and whether hospital-level variations explained these differences.

Adherence and Persistence with DPP-4 Inhibitors Versus Pioglitazone in Type 2 Diabetes Patients with Chronic Kidney Disease: A Retrospective Claims Database Analysis.

Background: Adherence and persistence with diabetes medication play an important role in glycemic control and may differ by medication class. However, there is a lack of research comparing diabetes medications in patients with renal impairment, despite the challenges and higher burden associated with managing this population.

Objective: To compare adherence and persistence among patients with type 2 diabetes mellitus (T2DM) and nondialysis chronic kidney disease (CKD) treated with dipeptidyl peptidase-4 (DPP-4) inhibitors versus pioglitazone.

A Multifaceted Intervention to Improve Patient Knowledge and Safe Use of Opioids: Results of the ED EMC Randomized Controlled Trial.

Objectives: Despite increased focus on opioid prescribing, little is known about the influence of prescription opioid medication information given to patients in the emergency department (ED). The study objective was to evaluate the effect of an Electronic Medication Complete Communication (EMC ) Opioid Strategy on patients' safe use of opioids and knowledge about opioids.

Methods: This was a three-arm prospective, randomized controlled pragmatic trial with randomization occurring at the physician level.