Richard J. Willke

Finding the right tool for the specific task: navigating RWE tools and checklists

Richard J. Willke, Paul Henri Cottu, Andrew H. Briggs, Uwe Siebert et autres

Real-world evidence (RWE) is increasingly used to support product approvals and label expansions, as well as clinical and payer decision-making. Various tools (e.g. frameworks, checklists) have been developed to help inform and assess the robustness and quality of real-world study design and …

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Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Ryan A. Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard et autres

Purpose: Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). The objective of this study was to identify how value assessment …

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The Value of the Quality-Adjusted Life Years

Richard J. Willke, Laura T. Pizzi, Leah Z. Rand, Peter G. Neumann

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Medicare Drug Price Setting Provides An Opportunity To Generate Stronger Evidence On Treatment Effect Heterogeneity

Sachin J. Kamal‐Bahl, Justin T. Puckett, Jacquelyn McRae, Richard J. Willke

To build public trust in the Medicare drug price-setting process, CMS must implement a more defined and transparent approach to collecting and using real-world evidence of treatment heterogeneity for underserved populations, thereby affirming its commitment to health equity.

EUreccA 2025: A Multistakeholder Effort to Further Real-World Evidence in Healthcare Decision Making

Laura T. Pizzi, Richard J. Willke

The maturation of use of real-world evidence (RWE) in decision making requires substantiation and growth in several areas including data sources and catalogs, data quality, governance, analytic methods, reporting standards, transparency, replicability, and fit-for-purpose specifications.1-3 During the past few years, recent guidance …

Reproducibility of real-world evidence studies using clinical practice data to inform regulatory and coverage decisions

Shirley V. Wang, Sushama Kattinakere Sreedhara, Sebastian Schneeweiß, Jessica Myers Franklin et autres

Abstract Studies that generate real-world evidence on the effects of medical products through analysis of digital data collected in clinical practice provide key insights for regulators, payers, and other healthcare decision-makers. Ensuring reproducibility of such findings is fundamental to effective evidence-based decision-making. …

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Incidence of mental health conditions by seizure control among adults with epilepsy in the United States

Vernon F. Schabert, Sean Stern, Louis Ferrari, Clarence T. Wade et autres

BACKGROUND: Mental health conditions (MHCs) are frequent comorbidities among people with epilepsy; however, the influence of seizure control on the incidence of MHCs is not well reported. This retrospective observational cohort study based on claims data evaluated the effects of indicators of …

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Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process

Elisabeth M. Oehrlein, Silke C. Schoch, Mehmet Burcu, Jennifer L. Bright et autres

OBJECTIVES: The Joint ISPOR-ISPE Special Task Force on Real-World Evidence included patient/stakeholder engagement as a recommended good procedural practice when designing, conducting, and disseminating real-world evidence (RWE). However, there are no guidelines describing how patient experience data (PED) can be applied when …

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The History and Future of the “ISPOR Value Flower”: Addressing Limitations of Conventional Cost-Effectiveness Analysis

Peter J. Neumann, Louis P. Garrison, Richard J. Willke

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Valuing treatment in oncology: embracing a broader notion of value

Sachin J. Kamal‐Bahl, Justin T. Puckett, Amitabh Singh, Richard J. Willke

Over the past decade, we have witnessed unprecedented, groundbreaking innovation in pharmaceuticals. This has been particularly true in oncology, where new therapies have increased survival and at times offered clinical cure. However, the impact of these promising treatments has been attenuated by …

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CHEERS to Updated Guidelines for Reporting Health Economic Evaluations!

Richard J. Willke, Laura T. Pizzi

Comparative effectiveness and safety of pharmaceuticals assessed in observational studies compared with randomized controlled trials

Yoon Duk Hong, Jeroen Paul Jansen, John Guerino, Marc L. Berger et autres

BACKGROUND: There have been ongoing efforts to understand when and how data from observational studies can be applied to clinical and regulatory decision making. The objective of this review was to assess the comparability of relative treatment effects of pharmaceuticals from observational …

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