Real-World Evidence: Useful in the Real World of US Payer Decision Making? How? When? And What Studies?

Abstract The Monitoring Studies (MS) program, the approach developed by RedETS to generate postlaunch real-world evidence (RWE), is intended to complement and enhance the conventional health technology assessment process to support health policy decision making in Spain, besides informing other interested stakeholders, including clinicians and patients. The MS program is focused on specific uncertainties about the real effect, safety, costs, and routine use of new and insufficiently assessed relevant medical devices carefully selected to ensure the value of the additional research needed, by means of structured, controlled, participative, and transparent procedures. However, despite a clear political commitment and economic support from national and regional health authorities, several difficulties were identified along the development and implementation of the first wave of MS, delaying its execution and final reporting. Resolution of these difficulties at the regional and national levels and a greater collaborative impulse in the European Union, given the availability of an appropriate methodological framework already provided by EUnetHTA, might provide a faster and more efficient comparative RWE of improved quality and reliability at the national and international levels.

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Systematic review of the real-world evidence of adalimumab safety in psoriasis registries

Journal of the European Academy of Dermatology and Venereology ◽

10.1111/jdv.15203 ◽

2018 ◽

Vol 32 (12) ◽

pp. 2126-2133 ◽

Cited By ~ 5

Author(s):

B. Strober ◽

J. Crowley ◽

R.G. Langley ◽

K. Gordon ◽

A. Menter ◽

...

Keyword(s):

Systematic Review ◽

Real World ◽

The Real ◽

Real World Evidence

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PP205 The Use Of Real-World Evidence To Support National Institute For Health And Care Excellence Medical Technology Submissions

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321001306 ◽

2021 ◽

Vol 37 (S1) ◽

pp. 26-26

Author(s):

Scott Gibson ◽

Sita Saunders ◽

Maximilian Blüher ◽

Amanda Hansson Hedblom ◽

Rafael Torrejon Torres ◽

...

Keyword(s):

Decision Making ◽

Real World ◽

Trial Design ◽

Clinical Evidence ◽

Patient Characteristics ◽

The United Kingdom ◽

Real World Evidence ◽

Guidance Documents ◽

Meta Analyses ◽

Positive Recommendation

IntroductionAlthough randomized controlled trials (RCTs) are recognized as providing the highest level of clinical evidence, few medical device RCTs are available due to underfunding or inherent challenges associated with trial design. This study examines the extent to which real-world evidence (RWE) supports the recommendations made by the National Institute for Health and Care Excellence Medical Technologies Evaluation Programme (MTEP).MethodsAll MTEP guidance documents published online prior to October 2020 were reviewed. The “case for adoption” recommendation, type of clinical data, and clinical critiques for each MTEP submission were extracted and categorized. RWE was defined as studies with neither blinding nor prospective selection or control of patient characteristics.ResultsOf the MTEP submissions reviewed, 34 of 45 (76%) received a positive recommendation. Independent of outcome, all submissions included RWE, but only 19 (42%) utilized RCT evidence (15 were recommended and four were not). Meta-analyses of RWE were used whenever possible. The most common clinical critiques in unsuccessful submissions were the following: (i) not generalizable to the United Kingdom National Health Service (NHS); (ii) low quality; (iii) likelihood of bias; (iv) trial design faults; (v) uncertain benefit; and (vi) evidence unrelated to scope.ConclusionsThis study suggests that while the use of RCTs has not always led to a positive recommendation, RWE can be valuable in decision-making. Evidence that is generalizable to the NHS, is related to the scope, and shows clear indication of benefit is more likely to positively influence MTEP decision-making.

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Effectiveness of golimumab in ulcerative colitis: A review of the real world evidence

Digestive and Liver Disease ◽

10.1016/j.dld.2018.11.002 ◽

2019 ◽

Vol 51 (3) ◽

pp. 327-334 ◽

Cited By ~ 6

Author(s):

Pablo Olivera ◽

Silvio Danese ◽

Lieven Pouillon ◽

Stefanos Bonovas ◽

Laurent Peyrin-Biroulet

Keyword(s):

Ulcerative Colitis ◽

Real World ◽

The Real ◽

Real World Evidence

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Awareness, Knowledge, and Utility of RCT Data vs RWE: Results From a Survey of US Cardiologists: Real-world Evidence in Clinical Decision Making

Clinical Medicine Insights Cardiology ◽

10.1177/1179546820953410 ◽

2020 ◽

Vol 14 ◽

pp. 117954682095341 ◽

Cited By ~ 1

Author(s):

Todd C Villines ◽

Mark J Cziraky ◽

Alpesh N Amin

Keyword(s):

Decision Making ◽

Clinical Practice ◽

Real World ◽

Randomized Clinical Trials ◽

Clinical Decision Making ◽

Clinical Decision ◽

The Body ◽

Representative Sampling ◽

Real World Evidence ◽

Wide Range

Real-world evidence (RWE) provides a potential rich source of additional information to the body of data available from randomized clinical trials (RCTs), but there is a need to understand the strengths and limitations of RWE before it can be applied to clinical practice. To gain insight into current thinking in clinical decision making and utility of different data sources, a representative sampling of US cardiologists selected from the current, active Fellows of the American College of Cardiology (ACC) were surveyed to evaluate their perceptions of findings from RCTs and RWE studies and their application in clinical practice. The survey was conducted online via the ACC web portal between 12 July and 11 August 2017. Of the 548 active ACC Fellows invited as panel members, 173 completed the survey (32% response), most of whom were board certified in general cardiology (n = 119, 69%) or interventional cardiology (n = 40, 23%). The survey results indicated a wide range of familiarity with and utilization of RWE amongst cardiologists. Most cardiologists were familiar with RWE and considered RWE in clinical practice at least some of the time. However, a significant minority of survey respondents had rarely or never applied RWE learnings in their clinical practice, and many did not feel confident in the results of RWE other than registry data. These survey findings suggest that additional education on how to assess and interpret RWE could help physicians to integrate data and learnings from RCTs and RWE to best guide clinical decision making.

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Feminist Judging in the 'Real World'

Oñati Socio-legal Series ◽

10.35295/osls.iisl/0000-0000-0000-0995 ◽

2018 ◽

Vol 8 (9) ◽

pp. 1275-1306 ◽

Cited By ~ 1

Author(s):

Rosemary Hunter

Keyword(s):

Decision Making ◽

Empirical Research ◽

Real World ◽

Interview Data ◽

Feminist Perspective ◽

Decision Making Process ◽

The Real ◽

Judicial Practice ◽

Tools And Techniques

The various feminist judgment projects (FJPs) have explored through the imagined rewriting of judgments a range of ways in which a feminist perspective may be applied to the practice of judging. But how do these imagined judgments compare to what actual feminist judges do? This article presents the results of the author’s empirical research to date on ‘real world’ feminist judging. Drawing on case study and interview data it explores the how, when and where of feminist judging, that is, the feminist resources, tools and techniques judges have drawn upon, the stages in the hearing and decision-making process at which these resources, tools and techniques have been deployed, and the areas of law in which they have been applied. The article goes on to consider observed and potential limits on feminist judicial practice, before drawing conclusions about the comparison between ‘real world’ feminist judging and the practices of FJPs. Los proyectos de sentencias feministas, a través de la reelaboración imaginaria de sentencias judiciales, han explorado multitud de vías en las que las perspectivas feministas se podrían aplicar a la práctica judicial. Pero ¿qué resulta de la comparación entre dichas sentencias y la práctica real de las juezas feministas? Este artículo presenta los resultados de la investigación empírica de la autora. Se analiza el cómo, el cuándo y el dónde de la labor judicial feminista, es decir, los recursos, herramientas y técnicas feministas que las juezas han utilizado, las fases de audiencia y toma de decisión en las que se han utilizado y las áreas del derecho en que se han aplicado. Además, se toman en consideración los límites observados y potenciales de la práctica judicial feminista, y se extraen conclusiones sobre la comparación entre la labor judicial feminista en el “mundo real” y la práctica de los proyectos de tribunales feministas.

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Decision letter for "Transparency in real-world evidence (RWE) studies to build confidence for decision making: Reporting RWE research in diabetes"

10.1111/dom.13918/v1/decision1 ◽

2019 ◽

Keyword(s):

Decision Making ◽

Real World ◽

Real World Evidence

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Review for "Transparency in real-world evidence (RWE) studies to build confidence for decision making: Reporting RWE research in diabetes"

10.1111/dom.13918/v2/review1 ◽

2019 ◽

Author(s):

Michael A Adena

Keyword(s):

Decision Making ◽

Real World ◽

Real World Evidence

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Real-World Evidence in the Real World: Beyond the FDA

American Journal of Law & Medicine ◽

10.1177/0098858818789423 ◽

2018 ◽

Vol 44 (2-3) ◽

pp. 161-179 ◽

Cited By ~ 6

Author(s):

Joan H. Krause ◽

Richard S. Saver

Keyword(s):

Health Care ◽

Health Care System ◽

Real World ◽

Regulatory Oversight ◽

The Real ◽

Health Care Fraud ◽

Fda Approval ◽

Real World Evidence ◽

Potential Benefits ◽

Care System

The 21st Century Cures Act (“Cures Act”) relies on the concept of real-world evidence (“RWE”) to improve the Food and Drug Administration (“FDA”) approval process. This has amplified interest and furthered momentum in applying RWE more broadly, beyond FDA regulation. In this article, we discuss the understandable appeal of RWE's pragmatic application and its many potential benefits. But we also caution that claims about RWE's wide-ranging, ameliorative impact on the health care system are likely overstated.The real world of RWE is messy and uncertain. Successfully incorporating RWE into regular health care system decision-making, beyond the FDA, faces considerable obstacles and limitations. We review the reasons to be wary about RWE as a game-changer. These concerns including data reliability, insufficient incentives for stakeholders to generate and engage with high-quality RWE, and lack of comprehensive regulatory oversight. In addition, the push for RWE may impact the enforcement of the health care fraud and abuse laws, perhaps not in necessarily positive ways. Increased reliance on RWE may have significant implications for off-label fraud enforcement, further conflating the distinction between claims that are false for reimbursement rather than for scientific purposes.

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