Evidence generation on benefits and risks of medicines and its impact on regulatory and downstream decision-making

Multidimensional Evidence Generation and FDA Regulatory Decision Making

JAMA ◽

10.1001/jama.2017.9991 ◽

2017 ◽

Vol 318 (8) ◽

pp. 703 ◽

Cited By ~ 88

Author(s):

Jonathan P. Jarow ◽

Lisa LaVange ◽

Janet Woodcock

Keyword(s):

Decision Making ◽

Regulatory Decision ◽

Evidence Generation

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Evaluation, Acceptance, and Qualification of Digital Measures: From Proof of Concept to Endpoint

Digital Biomarkers ◽

10.1159/000514730 ◽

2021 ◽

pp. 53-64

Author(s):

Jennifer C. Goldsack ◽

Ariel V. Dowling ◽

David Samuelson ◽

Bray Patrick-Lake ◽

Ieuan Clay

Keyword(s):

Decision Making ◽

Critical Role ◽

Medical Product ◽

Clinical Validation ◽

Proof Of Concept ◽

Participant Engagement ◽

Key Concepts ◽

The Right ◽

Evidence Generation

To support the successful adoption of digital measures into internal decision making and evidence generation for medical product development, we present a unified lexicon to aid communication throughout this process, and highlight key concepts including the critical role of participant engagement in development of digital measures. We detail the steps of bringing a successful proof of concept to scale, focusing on key decisions in the development of a new digital measure: asking the right question, optimized approaches to evaluating new measures, and whether and how to pursue qualification or acceptance. Building on the V3 framework for establishing verification and analytical and clinical validation, we discuss strategic and practical considerations for collecting this evidence, illustrated with concrete examples of trailblazing digital measures in the field.

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OP188 Post-Launch Evidence Generation Studies For Medical Devices In Spain: Integrating Real World Evidence Into Decision-Making

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321000751 ◽

2021 ◽

Vol 37 (S1) ◽

pp. 4-5

Author(s):

Inaki Imaz-Iglesia ◽

Pedro Serrano ◽

Eugenia Orejas ◽

Anai Moreno ◽

Janet Puñal-Ruibóo ◽

...

Keyword(s):

Decision Making ◽

Cost Effectiveness ◽

Medical Devices ◽

Real World ◽

Left Ventricular ◽

Ventricular Assist Devices ◽

Repair System ◽

Left Ventricular Assist Devices ◽

Real World Evidence ◽

Evidence Generation

IntroductionA national act (Order SSI/1356/2015) regulating Post-Launch Evidence Generation (PLEG) studies was set in Spain in 2015. These PLEG studies are to inform decisions about technologies already included in the Benefit Portfolio of the Spanish National Health System (SNHS) in order to confirm/exclude/modify their terms of use. Once a PLEG is established the selected hospitals provide the technology according to a common protocol and register outcomes until the required sample size is reached.MethodsThe PLEG studies are prospective, observational and single arm studies on safety, effectiveness and cost-effectiveness of a technology in real practice. The technology is selected because of the identification of an evidence gap, usually through a health technology assessment (HTA) report made by an agency of the Spanish Network of HTA Agencies (RedETS). The execution of a PLEG is assigned to one of the RedETS Agencies, which is responsible of delivering annual reports and a final report when the objectives are reached.ResultsThe following six PLEG studies, all of them on medical devices, have been launched in Spain so far, i) Endobronchial valve for patients with persistent air leak; ii) Biodegradable esophageal stent; iii) Percutaneous mitral valve repair system by clip; iv) Left Atrial Appendage Closure Device; v) Sensor-based glucose monitoring systems for children with type 1 diabetes mellitus; vi) Left ventricular assist devices for destination therapy. Five studies will finish their data collection by the end of 2020 or during 2021.ConclusionsA new national procedure using PLEG has been made available in Spain facilitating the use of real-world evidence to inform national decision-making on the financing of selected technologies due to uncertainties about their effectiveness, safety, cost-effectiveness and organizational impact. The studies are requiring a high amount of coordination tasks, as they are involving an average of 21 hospitals each. The usefulness and suitability of this procedure to achieve its objectives must be evaluated once their results are available.

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The Nationwide Osmed Health-Db Database. A Tool To Support Healthcare Decision-Making And Real-World Evidence Generation

Value in Health ◽

10.1016/j.jval.2015.09.2577 ◽

2015 ◽

Vol 18 (7) ◽

pp. A693 ◽

Cited By ~ 4

Author(s):

L Degli Esposti ◽

S Saragoni ◽

D Sangiorgi ◽

S Buda ◽

A Cangini ◽

...

Keyword(s):

Decision Making ◽

Real World ◽

Healthcare Decision ◽

Healthcare Decision Making ◽

Real World Evidence ◽

Evidence Generation

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Discarding optimality: Throwing out the baby with the bathwater?

Behavioral and Brain Sciences ◽

10.1017/s0140525x18001401 ◽

2018 ◽

Vol 41 ◽

Cited By ~ 1

Author(s):

Patrick Simen ◽

Fuat Balcı

Keyword(s):

Decision Making ◽

Perceptual Decision Making ◽

Reward Rate ◽

Perceptual Decision ◽

Standard Observer ◽

Rate Maximization ◽

Reward Rate Maximization ◽

Descriptive Standard ◽

Observer Model

AbstractRahnev & Denison (R&D) argue against normative theories and in favor of a more descriptive “standard observer model” of perceptual decision making. We agree with the authors in many respects, but we argue that optimality (specifically, reward-rate maximization) has proved demonstrably useful as a hypothesis, contrary to the authors’ claims.

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LPCD framework: Analytical tool or psychological model?

Behavioral and Brain Sciences ◽

10.1017/s0140525x18001383 ◽

2018 ◽

Vol 41 ◽

Author(s):

David Danks

Keyword(s):

Decision Making ◽

Analytical Tool ◽

Mathematical Framework ◽

Bayesian Decision ◽

Perceptual Decision Making ◽

Psychological Model ◽

Psychological Reality ◽

Psychological Theories ◽

Target Article ◽

Bayesian Decision Making

AbstractThe target article uses a mathematical framework derived from Bayesian decision making to demonstrate suboptimal decision making but then attributes psychological reality to the framework components. Rahnev & Denison's (R&D) positive proposal thus risks ignoring plausible psychological theories that could implement complex perceptual decision making. We must be careful not to slide from success with an analytical tool to the reality of the tool components.

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Elaborating the role of reflection and individual differences in the study of folk-economic beliefs

Behavioral and Brain Sciences ◽

10.1017/s0140525x18000274 ◽

2018 ◽

Vol 41 ◽

Author(s):

Kevin Arceneaux

Keyword(s):

Decision Making ◽

Individual Differences ◽

Cognitive Processes ◽

Evolutionary History ◽

Behavioral Responses ◽

History Of ◽

Economic Beliefs

AbstractIntuitions guide decision-making, and looking to the evolutionary history of humans illuminates why some behavioral responses are more intuitive than others. Yet a place remains for cognitive processes to second-guess intuitive responses – that is, to be reflective – and individual differences abound in automatic, intuitive processing as well.

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