Postlaunch evidence-generation studies for medical devices in Spain: the RedETS approach to integrate real-world evidence into decision making

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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Do existing real-world data sources generate suitable evidence for the HTA of medical devices in Europe? Mapping and critical appraisal

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321000301 ◽

2021 ◽

Vol 37 (1) ◽

Author(s):

Benedetta Pongiglione ◽

Aleksandra Torbica ◽

Hedwig Blommestein ◽

Saskia de Groot ◽

Oriana Ciani ◽

...

Keyword(s):

Decision Making ◽

Medical Devices ◽

Real World ◽

Real World Data ◽

Data Accessibility ◽

World Data ◽

Transcatheter Aortic Valve ◽

Real World Evidence ◽

Hip And Knee Arthroplasty ◽

Valve Implantation

Abstract Aim Technological and computational advancements offer new tools for the collection and analysis of real-world data (RWD). Considering the substantial effort and resources devoted to collecting RWD, a greater return would be achieved if real-world evidence (RWE) was effectively used to support Health Technology Assessment (HTA) and decision making on medical technologies. A useful question is: To what extent are RWD suitable for generating RWE? Methods We mapped existing RWD sources in Europe for three case studies: hip and knee arthroplasty, transcatheter aortic valve implantation (TAVI) and mitral valve repair (TMVR), and robotic surgery procedures. We provided a comprehensive assessment of their content and appropriateness for conducting the HTA of medical devices. The identification of RWD sources was performed combining a systematic search on PubMed with gray literature scoping, covering fifteen European countries. Results We identified seventy-one RWD sources on arthroplasties; ninety-five on TAVI and TMVR; and seventy-seven on robotic procedures. The number, content, and integrity of the sources varied dramatically across countries. Most sources included at least one health outcome (97.5%), with mortality and rehospitalization/reoperation the most common; 80% of sources included resource outcomes, with length of stay the most common, and comparators were available in almost 70% of sources. Conclusions RWD sources bear the potential for the HTA of medical devices. The main challenges are data accessibility, a lack of standardization of health and economic outcomes, and inadequate comparators. These findings are crucial to enabling the incorporation of RWD into decision making and represent a readily available tool for getting acquainted with existing information sources.

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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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Are Real-World Data and Evidence Good Enough to Inform Health Care and Health Policy Decision-Making?

Decision Making in a World of Comparative Effectiveness Research ◽

10.1007/978-981-10-3262-2_8 ◽

2017 ◽

pp. 97-103 ◽

Cited By ~ 1

Author(s):

Marc L. Berger ◽

James Harnett

Keyword(s):

Health Care ◽

Decision Making ◽

Health Policy ◽

Real World ◽

Policy Decision ◽

Real World Data ◽

World Data ◽

Health Policy Decision ◽

Policy Decision Making

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Real‐world evidence to support regulatory decision making: New or expanded medical product indications

Pharmacoepidemiology and Drug Safety ◽

10.1002/pds.5222 ◽

2021 ◽

Author(s):

Jessica M. Franklin ◽

Kai‐Li Liaw ◽

Solomon Iyasu ◽

Cathy Critchlow ◽

Nancy Dreyer

Keyword(s):

Decision Making ◽

Real World ◽

Medical Product ◽

Regulatory Decision ◽

Real World Evidence

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Chemical Exposure: European Citizens’ Perspectives, Trust, and Concerns on Human Biomonitoring Initiatives, Information Needs, and Scientific Results

International Journal of Environmental Research and Public Health ◽

10.3390/ijerph18041532 ◽

2021 ◽

Vol 18 (4) ◽

pp. 1532

Author(s):

Maria Uhl ◽

Ricardo R. Santos ◽

Joana Costa ◽

Osvaldo Santos ◽

Ana Virgolino ◽

...

Keyword(s):

Decision Making ◽

Policy Development ◽

Information Needs ◽

Policy Decision ◽

Human Biomonitoring ◽

Chemical Exposure ◽

The European Union ◽

Cross Sectional ◽

The United Kingdom ◽

Scientific Results

Over the last few decades, citizen awareness and perception of chemical products has been a topic of interest, particularly concerning national and international policy decision makers, expert/scientific platforms, and the European Union itself. To date, few qualitative studies on human biomonitoring have analysed communication materials, made recommendations in terms of biomonitoring surveillance, or asked for feedback in terms of specific biomonitoring methods. This paper provides in-depth insight on citizens’ perceptions of knowledge of biomonitoring, impact of chemical exposure on daily life, and claims on how results of research should be used. Four semi-structured focus groups were held in Austria, Portugal, Ireland, and the United Kingdom (UK). The cross-sectional observational qualitative design of this study allows for better understanding of public concern regarding chemicals, application, and use of human biomonitoring. The main findings of this study include citizens’ clear articulation on pathways of exposure, the demand on stakeholders for transparent decision-making, and sensitivity in communication of results to the public. Validated and trustful communication is perceived as key to empowering citizens to take action. The results can be used to facilitate decision-making and policy development, and feeds into the awareness needs of similar and future projects in human biomonitoring. Furthermore, it also brings to light ideas and concepts of citizens’ in shaping collaborative knowledge between citizens’, experts, scientists, and policy makers on equal terms.

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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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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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