Outcomes Research
Gracias al Convenio de Colaboración AZ-CRES 2022 ofrecemos en abierto este segundo lanzamiento. Se origina en un rastreo de una cincuentena de revistas de las que se seleccionan las contribuciones más relevantes del PharmaEconomics juzgado como el más puntero de la investigación en Economía de la Salud.
Siguiendo las pautas de nuestro think tank (véase en nuestra web los procedimientos seguidos) hemos dirigido esta vez la atención a los aspectos de outcomes research más novedosos. He aquí algunos de ellos seleccionados en una primera lista de dieciséis de entre los más de setenta revisados (listados a continuación en el apartado de Listado de revistas y artículos seleccionados). De ellos, ocho son objeto de comentario específico por el equipo editorial, constando de un resumen y evaluación crítica, sobre todo desde la óptica de su relevancia para el policy-making del sector.
La secuencia es como sigue, tras la búsqueda inicial (I Oliva). Llamamos la atención sobre el artículo de Feenstra et al en Pharmaeconomics acerca de los nuevos aspectos que inciden en la evaluación económica desde la perspectiva de la validación de modelos y cómo cambian el ‘frame’ de la evaluación algunos de los cambios globalmente disruptivos que se están produciendo. A todo ello cabe añadir el texto de Hofmann en Health Economics Policy and Law sobre las barreras internas a la eficiencia que chocan en la gestión del cambio y su difícil traducción en las políticas de ‘desinversión’. Son textos-contextuales de interés que se acompañan con los comentados por V Peiró sobre el alcance y límites de la Real World Evidence, Real World Data anexos y similares cuando la posibilidad de valoración de outcomes no puede ser otra que la composición ‘sintética’. El contexto de la incertidumbre en la apreciación de resultados en salud y como ello ha de marcar las estrategias de negociación eficiente para terapias innovadoras (véase aquí el texto del International Journal of Technology Assessment in Health Care) es objeto de revisión y comentario de la mano de un experimentado T Gilabert. La parte más económica de este segundo lanzamiento es vista por M Jofre, que comenta los textos de Gonçalves en el European Journal of Health Economics sobre precios basados en el valor en terapias avanzadas, y de Moye-Holtz, en Applied Health Economics sobre divergencias de precios en cáncer entre países (España entre ellos). Finalmente, P Ibern focaliza su análisis en modelos de financiación Payment Models for Pharmaceuticals, del European Journal of Health Economics y el texto de Bohn et al de Outcomes based contracts en Pharmaceuticals.
Textos adicionales de lectura de interés se consideran igualmente los de Schlander et al de revisión de los costes de I+D por áreas terapéuticas, el de precios y cobertura en Francia (en Value in Health), de Applied Health Economics sobre el coste efectividad de los medical devices y el de Kalf et al sobre la calidad en los tratamientos oncológicos. Y apuntar también el texto de Rejon et al de revisión sobre lo que se entiende por innovación y el de Reyes-Trave at al sobre las experiencias de risk-sharing en Cataluña, en Pharmaeconomics.
El texto editorial va en esta ocasión a cargo de V Peiró en su libre apreciación de dos ejes relevantes en el momento actual en la evaluación de fármacos: el de como valorar la gravedad (intenso y no necesariamente extenso) y la cronicidad (donde el alcance en el tiempo se muestra más que en su severidad), con el modelo Netflix en precios para la diabetes (Cherca et al.) como artículo de referencia y el de Schurer et al para el caso de Holanda para la valoración de la gravedad.
Espero como responsable del grupo de Antenas y Difusión que la lectura sea útil.
Guillem López i Casasnovas
Barcelona 31 de Mayo 2022
Listado de revistas y artículos seleccionados
- American Journal of Public Health
- Annals of International Medicine
- Potential for Point-of-Care Ultrasonography to Improve Patient Care in Diagnosis of Dyspnea
- Residual Confounding in Health Plan Performance Assessments: Evidence From Randomization in Medicaid
- Invitation to a single FS screening vs. usual care reduced colorectal cancer mortality and incidence at >15 y
- Characteristics and Outcomes of Hospitalized Pregnant Women With Influenza, 2010 to 2019: A Repeated Cross-Sectional Study
- When and How Would You Screen This Patient for Cervical Cancer? Grand Rounds Discussion From Beth Israel Deaconess Medical Center
- Cost-Effectiveness of Screening Mammography Beyond Age 75 Years: A Cost-Effectiveness Analysis
- Long-Term Follow-up of the Italian Flexible Sigmoidoscopy Screening Trial
- A Primary Care–Based Cognitive Behavioral Therapy Intervention for Long-Term Opioid Users With Chronic Pain: A Randomized Pragmatic Trial
- Colorectal Cancer Screening: Randomized Trials Are Essential to Support Recommendations
- Annals of Oncology
- Conference abstract: Core variables for managed entry agreements, regarding clinical outcomes and patient reported measures, in càncer
- Anti-cancer drug price regulation in India: Financial implications and annual savings to patients
- The association between prices and clinical benefit of approved drugs for solid tumors in Japan
- Comparison of long-term survival and cost-effectiveness (CE) of first-line (1L) treatment options in advanced renal cell carcinoma (aRCC) with intermediate or poor (I/P) prognostic risk
- Applied Health Economics and Health Policy
- Price Reduction of Anticancer Drugs from 2007 to 2019 in South Korea: The Impact of Pharmaceutical Cost-Containment Policies
- How to Cost the Implementation of Major System Change for Economic Evaluations: Case Study Using Reconfigurations of Specialist Cancer Surgery in Part of London, England
- From Spreadsheets to Script: Experiences From Converting a Scottish Cardiovascular Disease Policy Model into R
- PopulationBased Testing for Undiagnosed Hepatitis C: A Systematic Review of Economic Evaluations
- Are Medical Devices CostEffective?
- Effect of Competition on Generic Drug Prices
- Avoiding Opportunity Cost Neglect in CostEffectiveness Analysis for Health Technology Assessment
- Comparison of Prices and Affordability of Cancer Medicines in 16 Countries in Europe and Latin America
- Economic Consequences of Anti‑HCV Treatment of Patients Diagnosed Through Screening in Italy: A Prospective Modelling Analysis
- Financing and Reimbursement Models for Personalised Medicine: A Systematic Review to Identify Current Models and Future Options
- Atención Primaria
- British Medical Journal (BMJ)
- Canadian Medical Association Journal
- Canadian Respiratory Journal:Stakeholder views regarding patient discharge from intensive care: Suboptimal quality and opportunities for improvement
- Environmental Research and Public Health: Characteristics and Service Utilization by Complex Chronic and Advanced Chronic Patients in Catalonia: A Retrospective Seven-Year Cohort-Based Study of an Implemented Chronic Care Program
- Cancer Discovery
- Circulation
- Clinical Pharmacology Therapeutics
- A New Comprehensive Approach to Assess the Probability of Success of Development Programs Before Pivotal Trials (No free access)
- Japan’s Drug Regulation During the COVID-19 Pandemic: Lessons From a Case Study of Favipiravir
- Machine Learning Application in a Phase I Clinical Trial Allows for the Identification of Clinical-Biomolecular Markers Significantly Associated With Toxicity (No free access)
- Innovative Pediatric Development for Secukinumab in Psoriasis: Faster Patient Access, Reduction of Patients on Control (No free access)
- Real-world Overall Survival Using Oncology Electronic Health Record Data: Friends of Cancer Research Pilot
- The “Coming of Age” of Real-World Evidence in Drug Development and Regulation
- Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value
- Use of Real-World Data and Evidence in Drug Development of Medicinal Products Centrally Authorized in Europe in 2018–2019
- Evolving Acceptance and Use of RWE for Regulatory Decision Making on the Benefit/Risk Assessment of a Drug in Japan
- When Can We Trust Real-World Data To Evaluate New Medical Treatments?
- Cochrane Library
- Diabetes Cares
- EMBO Journal
- Epidemiology
- European Journal of Health Economics
- Income and conversion handicaps: estimating the impact of child chronic illness/disability on family income and the extra cost of child chronic illness/child disability in Ireland using a standard of living approach
- The impact of inpatient bed capacity on length of stay
- Using fees to reduce bed-blocking: a game between hospitals and long-term care providers
- Long-term cost of breast cancer treatment to the United States Medicare Program by stage at diagnosis
- A guide to extending and implementing generalized risk-adjusted cost-effectiveness (GRACE)
- How the COVID-19 pandemic can distort risk adjustment of health plan payment
- Value-based pricing for advanced therapy medicinal products: emerging affordability solutions
- The economics of alternative payment models for pharmaceuticals
- How Managed Entry Agreements can improve allocation in the public health system: a mechanism design approach
- European Journal of Hospital Pharmacy. Science and Practice
- European Journal of Public Health
- Gaceta Sanitaria
- Health Affairs
- Many Medicare Beneficiaries Do Not Fill High-Price Specialty Drug Prescriptions
- Trends In Hospital Prices Paid By Private Health Plans Varied Substantially Across The US
- Value-Based Payment Models In The Commercial Insurance Sector: A Systematic Review
- The Association Of HIV With Health Care Spending And Use Among Medicare Beneficiaries
- Multicancer Screening Tests: Anticipating And Addressing Considerations For Payer Coverage And Patient Access
- Health Economics
- How to deal with persistently low/high spenders in health plan payment systems?
- Eliciting risk preferences that predict risky health behavior: A comparison of two approaches
- Reforming the provision of cross-border medical care: Evidence from Spain
- Utility maximization versus regret minimization in health choice behavior: Evidence from four datasets
- Multivariate risk preferences in the quality-adjusted life year model
- Not all respondents use a multiplicative utility function in choice experiments for health state valuations, which should be reflected in the elicitation format (or statistical analysis)
- The effects of drug safety warnings on drug sales and share prices
- Health Economics Review
- Health Economics, Policy and Law
- Broadening the application of health technology assessment in the Netherlands: a worthwhile destination but not an easy ride?
- Internal barriers to efficiency: why disinvestments are so difficult. Identifying and addressing internal barriers to disinvestment of health technologies
- The ‘Netflix plus model’: can subscription financing improve access to medicines in low- and middle-income countries?
- Exploring differences between private and public prices in the English care homes market
- Physicians' attitudes towards accelerated access to medicines
- State strategies to address medicaid prescription spending: negotiated pricing vs price transparency
- Excessive pricing in the pharmaceutical industry: adding another string to the bow of EU competition law
- Should health authorities offer risk-sharing contracts to pharmaceutical firms? A theoretical approach
- Conceptualising equity in the impact evaluation of chronic disease management programmes: a capabilities approach
- (Re)defining legitimacy in Canadian drug assessment policy? Comparing ideas over time
- Health Services Research
- International Journal of Technology Assessment in Health Care
- Faciliating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework
- Bridging health technology assessment and healthcare quality improvement using international consortium of health outcomes measurement standard sets
- Application of a health technology assessment framework to digital health technologies that manage chronic disease: a systematic review
- International Journal on Quality in Health Care
- Journal of American Medical Association (JAMA) / JAMA Oncology
- Aspirin Use to Prevent Cardiovascular Disease and Colorectal Cancer
- Drug Pricing With Evidence Development
- Evaluating Long-term Efficacy of Neoadjuvant Chemoradiotherapy Plus Surgery for the Treatment of Locally Advanced Esophageal Squamous Cell Carcinoma
- Treatment and Survival Outcomes Associated With Platinum Plus Low-Dose, Long-term Fluorouracil for Metastatic Nasopharyngeal Carcinoma
- Association of Complex Multimorbidity and Long-term Survival After Emergency General Surgery in Older Patients With Medicare
- Long-term Outcomes of Transcatheter Arterial Chemoembolization Combined With Radiofrequency Ablation as an Initial Treatment for Early-Stage Hepatocellular Carcinoma
- Looking to Long-term Survivors for Improved Pancreatic Cancer Treatment
- Joint Commission Journal on Quality Improvement
- Journal of Clinical Epidemiology
- Journal of Clinical Oncology
- Risk of Adverse Financial Events in Patients With Cancer: Evidence From a Novel Linkage Between Cancer Registry and Credit Records
- Response Rate, Event-Free Survival, and Overall Survival in Newly Diagnosed Acute Myeloid Leukemia: US Food and Drug Administration Trial-Level and Patient-Level Analyses
- Long-Term Outcomes and Genomic Correlates of Response and Survival After Neoadjuvant Chemotherapy With or Without Carboplatin and Bevacizumab in Triple-Negative Breast Cancer
- Five-Year Survival Outcomes From the PACIFIC Trial: Durvalumab After Chemoradiotherapy in Stage III Non–Small-Cell Lung Cancer
- Valuing Chimeric Antigen Receptor T-Cell Therapy: Current Evidence, Uncertainties, and Payment Implications
- Journal of Comparative Effectiveness Research
- Journal of Epidemiology & Community Health
- Prospective association between receipt of the economic impact payment and mental health outcome
- Variation in colon cancer survival for patients living and receiving care in London, 2006–2013: does where you live matter?
- Long-term trends in population-based hospitalisation rates for myocardial infarction in England: a national database study of 3.5 million admissions, 1968–2016
- Journal of General Internal Medicine
- Understanding the Influence and Impact of Stakeholder Engagement in Patient-centered Outcomes Research: a Qualitative Study
- Co-creating the Patient Partner Guide by a Multiple Chronic Conditions Team of Patients, Clinicians, and Researchers: Observational Report
- Association of Post-discharge Service Types and Timing with 30-Day Readmissions, Length of Stay, and Costs
- Perceptions of Hospital-at-Home Among Stakeholders: a Meta-synthesis
- The Costs of Delayed Generic Drug Entry: Evidence from a Controversial Prostate Cancer Drug Patent
- Estimating Discounts for Top Spending Drugs in Medicare Part D
- Predictors of Success in the Bundled Payments for Care Improvement Program
- Physician Practice Leaders’ Perceptions of Medicare’s Merit-Based Incentive Payment System (MIPS)
- “I’m a Survivor”: Perceptions of Chronic Disease and Survivorship Among Individuals in Long-Term Remission from Opioid Use Disorder
- Community Health Workers as Healthcare Navigators in Primary Care Chronic Disease Management: a Systematic Review
- Association Between Cost-Saving Prescription Policy Changes and Adherence to Chronic Disease Medications: an Observational Study
- Journal of Health Economics
- The effect of co-payments on the take-up of prenatal tests
- Information shocks and pharmaceutical firms’ marketing efforts: Evidence from the Chantix black box warning removal
- Disruptions to the patient-provider relationship and patient utilization and outcomes: Evidence from medicaid managed care
- Price and income effects of hospital reimbursements
- Does the framing of patient cost-sharing incentives matter? the effects of deductibles vs. no-claim refunds
- Alternative payment models and physician treatment decisions: Evidence from lower back pain
- Optimal hospital payment rules under rationing by waiting
- Data transformations to improve the performance of health plan payment methods
- The Effects of Chronic Disease Management in Primary Health Care: Evidence from Rural China
- Long-term care spending and hospital use among the older population in England
- Paying for pharmaceuticals: uniform pricing versus two-part tariffs
- Journal of Healthcare Quality Research
- Journal of Public Health
- The Lancet (UK)
- Synthetic patient data in health care: a widening legal loophole
- Disease-modifying anti-asthmatic drugs
- Martin Landray: harnessing the power of good clinical trials
- Vaccine approval before phase 3 trial results: a consequence of vaccine access inequity
- Comparative efficacy and tolerability of 32 oral and long-acting injectable antipsychotics for the maintenance treatment of adults with schizophrenia: a systematic review and network meta-analysis
- Lancet Oncology
- Medical Care
- Medical Care Research and Review
- Medical Decision Making
- Generating, Presenting, and Interpreting Cost-Effectiveness Results in the Context of Uncertainty: A Tutorial for Deeper Knowledge and Better Practice
- Assessing Interventions That Prevent Multiple Infectious Diseases: Simple Methods for Multidisease Modeling
- Personalization of Medical Treatment Decisions: Simplifying Complex Models while Maintaining Patient Health Outcomes
- Expected Value of Sample Information to Guide the Design of Group Sequential Clinical Trials
- Relationships among Antecedents, Processes, and Outcomes for Shared Decision Making: A Cross-Sectional Survey of Patients with Lumbar Degenerative Disease
- Estimating Treatment-Switching Bias in a Randomized Clinical Trial of Ovarian Cancer Treatment: Combining Causal Inference with Decision-Analytic Modeling
- Choosing a Metamodel of a Simulation Model for Uncertainty Quantification
- Medicare’s Hospital Value-Based Purchasing Program Values Quality over QALYs
- Medicina Clínica (Barcelona)
- Nature
- New England Journal of Medicine
- Pharmacoeconomics
- Four Aspects Affecting Health Economic Decision Models and Their Validation
- How Much Does It Cost to Research and Develop a New Drug? A Systematic Review and Assessment
- Characterization of the Pharmaceutical Risk-Sharing Arrangement Process in Catalonia
- Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel
- The Challenges of OutcomesBased Contract Implementation for Medicines in Europe
- State of the ART? Two New Tools for Risk Communication in Health Technology Assessments
- Is an Orphan Drug’s Cost-Effectiveness Associated with US Health Plan Coverage Restrictiveness?
- Plos Medicine
- Long-Term Survival and Dialysis Dependency Following Acute Kidney Injury in Intensive Care: Extended Follow-up of a Randomized Controlled Trial: e1001601
- Cardiovascular disease (CVD) and chronic kidney disease (CKD) event rates in HIV-positive persons at high predicted CVD and CKD risk: A prospective analysis of the D:A:D observational study
- Precision and Future Medicine
- Revista Española de Salud Pública
- Revista Española Economía de la Salud
- Social Science & Medicine
- The Office of Health Economics
- It Takes Two to Tango: When do Conditional Reimbursement Risk-Sharing Schemes Work for Both Parties?. OHE Consulting Report.
- Making Outcome-Based Payment a Reality in the NHS. Phase Two: Practical Considerations. OHE Research Paper
- Payment Models for Multi-Indication Therapies. OHE Consulting Report
- The Lower Drug Costs Now Act and Pharmaceutical Innovation. OHE Consulting Report
- Why we need a new Outcomes-based Value Attribution Framework for Combination Regimens in Oncology. OHE Consulting Report
- It Takes Two to Tango: When do Conditional Reimbursement Risk-Sharing Schemes Work for Both Parties?. OHE Consulting Report
- Therapeutics and Clinical Risk Management
- Value in Health
- The Potential Cost-Effectiveness of a Machine Learning Tool That Can Prevent Untimely Intensive Care Unit Discharge
- Estimation of the Width of Uncertainty in Care Consumption and Costs When Using Common Data Collection Tools in Economic Evaluations: A Benchmark for Sensitivity Analyses
- Implementing Risk-Sharing Arrangements for Innovative Medicines: The Experience in Catalonia (Spain)
- Coverage and Pricing Recommendations of the French National Health Authority for Innovative Drugs: A Retrospective Analysis From 2014 to 2020
- Improving survival and local control in rectal cancer in Catalonia (Spain) in the context of centralisation: A full cycle audit assessment
- Rationalizing the introduction and use of pharmaceutical products: The role of managed entry agreements in Central and Eastern European countries
- Determining the Comparative Value of Pharmaceutical Risk-Sharing Policies in Non–Small Cell Lung Cancer Using Real-World Data
- Predictors of chronic medication nonadherence in cancer survivors: a systematic literature review
- Varying Willingness to Pay Based on Severity of Illness: Impact on Health Technology Assessment Outcomes of Inpatient and Outpatient Drug Therapies in The Netherlands
- A Framework for Using Real-World Data and Health Outcomes Modeling to Evaluate Machine Learning–Based Risk Prediction Models
- Is an Early Access Program for an Oncology Drug an Asset in the French Pricing and Reimbursement Pathway?
- ICER
- Unsupported Price Increases Occurring in 2020
- ICER Publishes First Annual Assessment of Barriers to Fair Access Within US Commercial Insurance Prescription Drug Coverage
- Unsupported Price Increases Occurring in 2020 in California
- Multiple Sclerosis: CIS, RRMS, and SPMS
- Evaluating Reforms to Orphan Drug Development, Pricing, and Coverage
- NICE