The Complexities of Medicare Directly Negotiating Drug Prices
This article provides a short history of the concept of allowing Medicare to negotiate drug prices, describes various approaches and assessments of their potential savings from the Congressional Budget Office, and considers the prospects for action in the future.
While some European countries have developed schemes that allow them to obtain confidential discounts, resulting patterns of price differences and access limitations are falling far short of what a coherent approach might provide.
The Increasing Role of Qualitative Evidence on Health Preferences for Regulatory Decision Making
Kathleen Beusterien, Benjamin M. Craig
The U.S. FDA is actively promoting the submission of health preference research under the title “patient preference information” (PPI).
Measuring Multiple Medication Adherence–Which Measure When?
Priti Pednekar, Mia (Maria) Malmenas, Tamás Ágh, Bryan Bennett, Andrew Peterson on behalf of the Leadership Members of the Multiple Medication Adherence Measurement Working Group of the ISPOR Medication Adherence and Persistence Special Interest Group
In this article, the authors review different methods to measure multiple medication adherence.
The Good, the Bad, and the Ugly: Reminders About Propensity Scores
Lauren Strand, Blythe Adamson, Joseph Delaney, Anirban Basu
Managing the Winds of Change in Real-World Evidence Studies
Alison Bourke, Andrew Bate, Brian Sauer, Jeffrey Brown, Gillian C. Hall
In this article, Bourke et al acknowledge the rapidly evolving health care data infrastructure and the impact change can have on scientific reproducibility. Necessitating forward planning to anticipate, document, and where possible, mitigate the effects of such change is becoming increasingly important.
Maximizing the Utility of Real-World Evidence: Integration of Structured Electronic Medical Record (EMR) Data, Unstructured EMR Data, and Billing Data for Economics and Outcomes Research in Oncology
Electronic medical records are a source of rich clinical data that can address a wide range of HEOR questions not easily answered with other real world data sources.
The Current Biosimilars Landscape and Methodological Considerations for Real-World Evidence Generation
Jaclyn L. F. Bosco, Allison Bryant, Nancy A. Dreyer
This article by Bosco et al describes design and methodologic considerations in the context of specific study goals for biologics and biosimilars.
A Highlight of Similarities and Differences Between the HTA Core Model 3.0 and the AMCP Format 4.0
Elisabeth M. Oehrlein, Maya L. Hanna, Priyanka Gaitonde, Eleanor M. Perfetto
In this piece, the authors introduce and compare the HTA Core Model 3.0 and the AMCP Format 4.0.
This article explores both sides of the controversial debate between speed versus safety in bringing new medicines to market for patients with rare diseases.
Thoughts on the Real-World Evidence Navigator
Kristijan Kahler, RPh, PhD
In this commentary, Kahler et al. share a perspective from the Real World Evidence Center of Excellence at Novartis on how the RWE Navigator can be used to inform overall evidence generation to support data driven decision making.
GetReal: Real-World Evidence Navigator: Beyond the Hype
Heather Stagenga, MSc
This article provides an introduction to the freely available RWE Navigator, a tool designed to address an unmet need for guidance on the role of real-world evidence in health care decision making.
Learning by Listening, Digitally
Marilyn Metcalf, PhD
The authors explore the value of anonymized social media data for insights into patients' use of medicines.
This article explains what manufacturer’s should know about the new draft guidelines, Drug and Device Manufacturer Communications with Payers, Formulary Committees, and Similar Entities – Questions and Answers.
In this article, Dr. Sapède discusses how, under adaptive pathways, early and continuous scientific dialogue and adaptive payment models could help plan and manage evidence expectations, inform stakeholders’ decisions and ultimately enable patient access to selected promising medicines.
This article highlights different perspectives on the challenges in assessing the value of medical devices.
An Introduction to Risk Adjustment in Outcomes Research Comparison of the Strength of Associations between Variants of the Charlson Comorbidity Index and Patient-Reported Health Outcomes
In conducting outcomes research studies that compare populations or intervention groups, it is not always possible to control for differences in risk factors using randomization; therefore, it is essential to have methods to standardize risk factors when conducting observational studies.
The CER Collaborative Initiative—a joint effort by ISPOR, AMCP, and NPC—is providing key guidance for advancing appropriate use of comparative effectiveness research (CER) to improve patient health outcomes, including a free web-based user-friendly toolkit for assessing CER studies.
In this article, the authors explore the potential impact of ICER assessments beyond the United States, with global health care decision makers from various HTA/payer organizations.
The authors examine the pros and cons of seeking parallel regulatory-HTA guidance during clinical development and discusses the importance of scientific advice in ensuring regulatory approval and HTA acceptance.
Edward Witt and Amir Goren provide data-supported recommendations regarding when and how to control for unrelated comorbidities in models examining the potential impact of diseases and other variables on health outcomes.
An Introduction to Competing Risks
Beenish S. Manzoor
Survival analysis is a cornerstone of health outcomes research, and is based on the assumption of non-informative, or independent, censoring. However, in the presence of competing risks, this assumption may not hold.
Visualizing Data for Hypothesis Generation Using Large-Volume Claims Data
In this article, Eberechukwu Onukwugha et al. illustrate the potential for insight and hypothesis generation from observational data via the use of EventFlow and the Grouping Algorithm for Cancer Data.
Conceptual Overview of Computerized Adaptive Testing
Michael C. Edwards
Michael Edwards provides a high-level overview of computerized adaptive testing and its possible benefits in clinical outcome assessment.
In this article, Jenny Retzler explains how consideration of PRO strategies from the very beginning of clinical product development can not only ensure optimal collection of PRO data for the demonstration of treatment benefits, but also generate evidence to inform trial design, product development, and marketing decisions.
Patient-Centeredness in Clinical Outcomes Assessment
Elisabeth M. Oehrlein
In this article, Elisabeth Oehrlein et al. highlight several key points to consider when evaluating the patient-centeredness of outcome measures used in past research or determining endpoints for future studies.
Simu Thomas highlights the specific considerations we need to make to foster development and access to lifesaving innovations in regenerative medicine.
Noting that 80% of large companies provide incentives to their employees to practice better health behaviors, Dr. Volpp discusses several case studies in which behavioral economists and corporate leaders have together tried to maximize employee participation in health behavior improvement programs through incentives.
Irrationality in Health Care
Douglas E. Hough
Dr. Hough discusses the work of Daniel Kahneman, a behavioral psychologist, and how Kahneman’s observations and concepts may be used in behavioral economics to help explain rationality and irrationality in peoples’ health care decision making.
Although it is difficult to predict the future, performance-based managed entry agreements are bound to be used more often based on the need to manage access to innovative therapies with immature data at launch, emerging appetite of payers for real-world evidence, integration of databases and improving sophistication of pharma market access teams.
In this article, the authors report the perception of anti-TNF biosimilars among payer stakeholders in the big-5 European countries, their rating of level of comfort with extrapolation of clinical data, quality of clinical and economic data available for novel compound evaluations and the perception of suitable target population for biosimilars.
A Primer on Latent Class Analysis
Earnest H. Law
This article introduces latent class analysis, an increasingly popular method for clustering individuals, and provides an overview of its theory and applications in outcome research.
This article discuss the analytic issues associated with using big data for health care research and clinical decision making.
Multivariate Meta-Analysis: Concepts and Applications
Sonya J. Snedecor
This article discuss the principles and issues associated with multivariate meta-analyses.
The ISPOR Patient Engagement in Research Working Group demonstrates the landscape of patient engagement and aims to define new methods to facilitate and measure successful patient engagement through collaborative efforts in research.
This article is a brief methods piece on techniques for revalidating patient-reported outcome measures (PROs). It covers common types of validated PROs and reviews situations in which they may need revalidation.
A Roadmap for Sustainable Health Care: Avoiding the ‘Perfect Storm’
In this article, the author proposes practical recommendations for different stakeholders and decision makers, which would enable them to survive the perfect storm in health care and be ready for a health care transformation in a cost-efficient, effective, and sustainable manner.
How Can We Enhance the Practical Application of Outcomes Research?
In this article, the author identifies three key concepts that enhance the practical application of outcomes research.
This article describes why some technologies may fail to demonstrate cost effectiveness even when the acquisition price is set to zero, and why the evaluation of these technologies raises both methodological and ethical questions for HTA agencies.
The Evaluation of Ultra-Innovative Drugs: A Broader Perspective
Mark Nuijten, PhD, MD, MBA
This article explores the application of economic valuation methods to justify a drug price, when the incremental cost-effectiveness ratio (ICER) exceeds the threshold.
This article explores the framework and differences in the assessment of innovations for Medical Devices in Germany and the UK.
In this article, the authors highlight the different perspectives HTA decision makers may have on the statistical methods used to adjust for treatment switching bias, and review the challenges this creates for pharmaceutical manufacturers.
From Proof of Concept to Basis for Initial Marketing Authorization, HTA Appraisal and Reimbursement: Clinical Phase II in the Context of Adaptive Pathways
This article examines the role of phase II in clinical drug development in the context of the adaptive pathways approach.
A Life-Span Approach to Decision Making: Why EU Collaboration Is a Sine Qua Non
This article examines an adaptive pathways approach that would be needed to enable translation of helpful innovation to current and future patients.
The Current and Future Lifecycle Approach to Decision Making on Health Technologies
Finn Bø Kristensen
This article presents EUnetHTA’s approach to developing and providing timely, transparent, and reliable information about the value of a drug, device, or intervention by way of applying the HTA Core Model.
Evidence Development and Decision Making for Medical Devices
This article compares health technology assessments and regulation for medical devices versus the assessment and regulation policies for pharmaceuticals. The author notes that medical devices do not require high levels of clinical evidence; instead “safety and efficacy” are often enough to satisfy regulators.
An Introduction to Unit-of-Analysis Error
Maya Hanna, Elisabeth Oehrlein, Eleanor Perfetto
This article emphasizes the importance of determining whether unit of analysis errors are present when using study results to inform health care decision making.
Horizon Scanning: Identifying and Estimating Future Impact of Emerging Innovations on US Health Care
In this article, ECRI Institute staff describes the AHRQ Healthcare Horizon Scanning System and recently commissioned cost analyses as presented at last year’s ISPORs 20th Annual International Meeting in Philadelphia, PA, USA.
A Real-World Research Perspective for Biosimilars
This article by Jacklyn L.F. Bosco, is based on a presentation given during the “Distinguishing Biosimilarity: How Can We Generate Real-World Evidence to Support Decision-Making” issue panel at the ISPOR 20th Annual International Meeting in Philadelphia, PA, USA.
Time-Dependent Confounders: Are They All the Same?
In this article, which is intended for students and early-career researchers, Mehmet Burcu et al. briefly describe the concepts of confounding, treatment assignment, and selection bias in observational research, and distinguish two distinct types of time-dependent confounding: 1) time-dependent confounding not affected by prior treatment, and 2) time-dependent confounding affected by prior treatment.
How Do U.S. Payers Use Economic Models Submitted by Life Sciences Organizations?
This article by Laurie Fazio et al. helps us gain a better understanding of how payers use industry provided models and where opportunities exist for greater collaboration between payers and life sciences organizations.
Ready for Risk Sharing? Challenges and Implications for Manufacturers
Kimberly White explores the challenges of risk sharing by manufacturers and why this is gaining attention in the United States.
The Nordic Health Care Registries–Real Improvement to Outcomes Research?
This article by Lars Pedersen describes the unique features of the Nordic Health Care Registries and possible applications in outcomes research.
Are We Moving Towards Collaborative European Rapid Relative Effectiveness Assessments? Insights Gleaned from the EUnetHTA Joint Assessment of Canagliflozin–Approaching Joint Relative Effectiveness Assessments
In this article, Simone Warren et al. explore whether Europe is moving towards collaborative HTA based on the lessons learned from the assessment of canagliflozin, the second pilot of European cross border collaboration in joint HTA as part of the EUnetHTA 2nd Joint Action.
Patient Registries as Instruments for HTA Outcomes Research: A European Perspective
In this article Persephone Doupi et al. examine EU-level activities towards developing patient registries into reliable and high-quality data sources for HTA analyses of pharmaceuticals and medical devices, thus supporting health policy-relevant research.
How an Early Network Meta-Analysis Can Inform Clinical Trial Design and Help with Health Technology Assessment Submissions
In this article, Yingxin Xu et al. show several examples illustrating the value that performing an early NMA brings to phase III trial planning
Novel Methods for Indirect Comparison of Treatments: When Are They Needed and How Do They Work?
Traditional approaches to indirect or mixed comparison of treatments may not always be appropriate or feasible; novel approaches like simulated treatment comparison and matching adjusted indirect comparisons can overcome challenges like heterogeneity in study populations or incomplete or interrupted evidence networks. Traditional approaches to indirect or mixed comparison of treatments may not always be appropriate or feasible; novel approaches like simulated treatment comparison and matching adjusted indirect comparisons can overcome challenges like heterogeneity in study populations or incomplete or interrupted evidence networks.
Instrumental Variables: A Tool to Reduce Bias in Non-Randomized Studies
In this article, Mark Bounthavong et al. explain how and why to use instrumental variables in outcomes research to tackle the problem of endogeneity sometimes biasing non-randomized studies.
Toward a True Rapid Learning Health Care System: How Can We Build Both the Will and the Skill?
Sarah Greene discusses the fast-pace learning health care system and how we can build on it.
Health Care Transformation and the Learning Health System: Where Are We and What’s Next?
In this article, Lewis Sandy follows Sarah Greene with a piece on the transformation on the learning health care system and where we go from here.
The Learning Health System: An Optimist’s Perspective
Sachin H. Jain gives an optimist’s perspective on the leaning health system.
How Should We Value Disease Eradication?
In this article, Dana Goldman demonstrates – through modeling of alternate treatment scenarios – how investing in broader treatment strategies for hepatitis C results in significant cost savings as well as a dramatic reduction in prevalence and new infections.
Assessing the Impact of Morbidity in Populations of North London Clinical Commissioning Groups on Patient Admission Rates
As Clinical Commissioning Groups are faced with the challenges of austerity within the NHS, UK, Zoe Groom set out to ascertain whether disease prevalence alone could explain elective and non-elective patient admission rates in 20 North London CCGs in order to aid decision-making processes.
Pills, People, and Preferences: Evaluating Real-Life Practice in Pragmatic Trials
Hillary Pinnock discusses the potential of real-life studies and implementation trials to inform clinical practice.
Principles of Highly Effective Outcomes Research Consulting Teams
In this article, Steve Arcona, PhD, discusses that outcomes research (OR) consulting teams become highly effective by establishing a priori communication rules, sharing an identifiable communication pattern, and operating within a speak-up culture, along with clearly agreed upon goals and performance objectives and an aligned and shared team vision.
Evidence from Direct-to-Subject Study Designs for Health (outcomes) Research and Pharmaco(epidemiology)
In this article, Florian Eichmann et al. outlines how direct-to-subject study approaches can provide valid evidence for regulatory as well as non-regulatory purposes.
Ethical and Legal Issues Concerning the Use of Social Media to Get to the "Real World"
Andrew Cox et al. raise and explore the need for professional guidelines covering the use of patient social media as a data source.
Working with Oncology Electronic Medical Record (EMR) Data in Outcomes Research
In this article, Foley et al. discuss the distinct challenges of working with electronic medical record (EMR) data in oncology outcomes research and provide several recommendations for handling common challenges.
Eliciting Beliefs in Health Technology Assessment to Characterize Uncertainties
In this article, Marta Soares describes the use of formal approaches in health technology assessment (HTA); specifically those that aim to elicit uncertain judgments.
Biases Related to Prescribing Decisions in Retrospective Database Research in Diabetes
Ayad Ali describes four types of prescribing-related biases that are introduced in real-world evidence database research involving disease states with stepwise drug treatment. Diabetes treatment is used as an example to illustrate these biases.
Current Status of Health Technology Assessment in Argentina
Lautaro Renati provides the current state of health technology assessment (HTA) in Argentina.
Big Data in Health Care Decisions: Where Are We Now and What Does the Future Hold?
In this article, Marcus Wilson outlines the implications of harnessing big data and discusses the interdependence of descriptive, predictive, and prescriptive analytics in powering health care decision making in the future.
Dynamic Simulation Modeling to Evaluate Complex System Interventions for Health Care Delivery Research – What Methods for What Problems?
William Padula et al. investigate the dynamic simulation modeling methods factor in unintended consequences resulting from human behavior, person-to-person interactions, and feedback on multiple system levels to more realistically depict health care delivery systems that span diverse populations.
Outcomes Research and Machine Learning: Complementary or Incompatible?
In this article, Lesley Curtis describes the key elements of the outcomes research process and how these elements apply to a variety of methodological approaches including machine learning.
Hepatitis C Viral Infection Disease Burden in Asia
Lai Wei, et al. examine the public health issues in Asia related to hepatitis C.
Perception of Country-Specific Health Care Reform and Consideration of Real World Evidence in Routine Practice: Survey of Oncologists in Europe, the United States, China, and Brazil
In this article, Siva Narayanan examines the perspectives of oncologists regarding health care reform in their respective countries as well as their attitudes toward real world evidence, cost and patient affordability, and patient health-related quality-of-life (HRQoL) when making treatment decisions.
An EMA Perspective
Shelia Killalea explains the procedures and key benefits of engaging with the EMA for Scientific Advice, with a focus on the parallel HTA/EMA procedure.
A NICE Perspective
In this article, Leeza Osipenko gives a brief overview of NICE Scientific Advice service.
The EUnetHTA Perspective
In this article, Mira Pavlovic will present the EUnetHTA perspective on the early dialogues between manufacturers, health technology assessment (HTA) bodies and regulators on initial evidence generation for relative effectiveness and cost effectiveness assessment.
Question is Not Whether but How to use MCDA
In this article, Rob Baltussen outlines the present use of various criteria in reimbursement decisions on health technologies, and evaluates the potential of MCDA to guide these decisions.