OP03 Optimizing The Use Of RWE In HTA: Lessons From The ICER Summit

2018 ◽  
Vol 34 (S1) ◽  
pp. 1-2
Author(s):  
Grace Hampson ◽  
Chris Henshall ◽  
Adrian Towse ◽  
Bill Dreitlein ◽  
Steven Pearson
Keyword(s):  
Real World ◽  
Policy Decision ◽  
Drug Effectiveness ◽  
Real World Evidence ◽  
Coverage With Evidence Development ◽  
The Us ◽  
Policy Decision Making ◽  
Meta Analyses ◽  
Study Designs

Introduction:Real world evidence (RWE) is changing the overall data landscape and it has potential to advance the evaluation of real world performance (comparative effectiveness) of healthcare technologies by providing a greater quantity and quality of evidence. However, many are concerned that non-randomized RWE may be substituted for RCT data and thus increase uncertainty about effectiveness. This presentation sets out the opportunities and challenges for use of RWE by payers and HTA bodies to evaluate health care technologies.Methods:Current uses, opportunities and challenges were identified via a literature review and interviews with nine experts. Interim results were discussed at the 2017 ICER Policy Summit, which brought together leaders from payer and life sciences organizations, to develop specific and actionable recommendations for the use of RWE in drug coverage and policy decision-making.Results:RWE is utilized for multiple purposes in the US and globally, including: aiding design of drug development pathways; supporting regulatory approval decisions; monitoring safety; and informing HTA assessments and payer coverage decisions. Some stakeholders see great value in RWE and want to make greater use of these data sources, including for: drug effectiveness evaluations (including supplementing network meta-analyses); innovative study designs (including pragmatic trials); real time patient monitoring; and adaptive pathways or coverage with evidence development. However, others see numerous challenges, many of which are related to the quality and reliability of RWE sources. Acceptance of an expanded future role for RWE is not universal, and payers and developers must work together to find mutually beneficial strategies for progressing the development and use of RWE.Conclusions:Specific and actionable recommendations will be presented which highlight the role that each stakeholder group can play in overcoming the challenges and realizing the potential for RWE.

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

2021 ◽  
Vol 37 (1) ◽  
Author(s):  
Pedro Serrano-Aguilar ◽  
Iñaki Gutierrez-Ibarluzea ◽  
Pilar Díaz ◽  
Iñaki Imaz-Iglesia ◽  
Jesús González-Enríquez ◽  
...  
Keyword(s):  
Decision Making ◽  
Medical Devices ◽  
Real World ◽  
Policy Decision ◽  
Assessment Process ◽  
The European Union ◽  
Political Commitment ◽  
Real World Evidence ◽  
Health Policy Decision ◽  
Regional Health Authorities

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.

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

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.

scholarly journals Real-world evidence data on the monoclonal antibody erenumab in migraine prevention: perspectives of treating physicians in Germany

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Andreas Straube ◽  
Philipp Stude ◽  
Charly Gaul ◽  
Katrin Schuh ◽  
Mirja Koch
Keyword(s):  
Quality Of Life ◽  
Monoclonal Antibody ◽  
Clinical Trials ◽  
Real World ◽  
Treatment Patterns ◽  
Patient Profile ◽  
Real World Evidence ◽  
Patient Profiles ◽  
Treatment Responses

Abstract Background Erenumab, the first-in-class fully human monoclonal antibody targeting the calcitonin gene-related peptide receptor, was shown to be efficacious and safe for the prophylactic treatment of migraine in adults in randomized clinical trials. Large-scale, real-world evidence in multi-centre settings is still needed to confirm these results. Erenumab patient profiles outside clinical trials and physicians’ treatment patterns, as well as data from patients treated in Germany, a severely impacted population, are not published yet. Methods TELESCOPE was a multi-centre survey gathering real-world data from 45 German headache centres between July 2019 and December 2019. The project consisted of two parts. In the first part, treating physicians shared their experiences on current erenumab treatment with regard to patient profiles, treatment patterns and treatment responses. In the second part, a retrospective chart review was conducted of 542 migraine patients treated with erenumab for at least three months. Treatment responses focused on various aspects of patients’ quality of life. Results The analysis of 542 patients’ charts revealed that three-month treatment with erenumab significantly reduced monthly headaches, migraine and acute medication days. Furthermore, headache intensity and frequency were reduced in over 75 % and accompanying aura in 35 % of patients. The clinical global impression scale revealed a general improvement in 91 % of patients. According to the treating physicians’ professional judgement, 83 % of patients responded to erenumab and 80 % were satisfied with the treatment. Physicians evaluated restricted quality of life, the number of monthly migraine days and previous, prophylactic treatments as the main components of the current patient profile for monoclonal antibody recipients. Based on the assessment of physicians, erenumab reduced migraine symptoms in 65 % and increased quality of life in more than 75 % of their patients. Conclusions TELESCOPE confirms positive treatment responses with erenumab shown in clinical trials in a real-world multi-centre setting. The results show consistently positive experiences of physicians utilizing erenumab in clinical practice and underline that therapy with this monoclonal antibody is effective in migraine patients, particular in those, who have failed several prophylactic therapies.

scholarly journals PNS144 NETWORK META-ANALYSES OF REAL-WORLD EVIDENCE: A SYSTEMATIC REVIEW OF METHODS

2020 ◽  
Vol 23 ◽  
pp. S311
Author(s):  
S. Nambiar ◽  
S. Dehipawala ◽  
G. Miyasato ◽  
N. Hadker
Keyword(s):  
Systematic Review ◽  
Real World ◽  
Real World Evidence ◽  
Meta Analyses

scholarly journals The Expanding Role of Real-World Evidence Trials in Health Care Decision Making

2019 ◽  
Vol 14 (1) ◽  
pp. 174-179 ◽  
Author(s):  
David C. Klonoff
Keyword(s):  
Real World ◽  
Clinical Evidence ◽  
Real World Data ◽  
Medical Products ◽  
Advantages And Disadvantages ◽  
Randomized Controlled ◽  
Real World Evidence ◽  
Care Decision

Real-world evidence (RWE) is the clinical evidence about benefits or risks of medical products derived from analyzing real world data (RWD), which are data collected through routine clinical practice. This article discusses the advantages and disadvantages of RWE studies, how these studies differ from randomized controlled trials (RCTs), how to overcome barriers to current skepticism about RWE, how FDA is using RWE, how to improve the quality of RWE, and finally the future of RWE trials.

Unveiling the real-world outcomes of breast cancer (BC) patients with taxanes-induced neuropathy using a digital patient-powered network (PPN).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18248-e18248
Author(s):  
Raanan Berger ◽  
Lior Hasid ◽  
Irad Deutsch ◽  
Eliran Malki ◽  
Maytal Bivas-Benita
Keyword(s):  
Cancer Patients ◽  
Real World ◽  
Healthcare Costs ◽  
Patient Reported Outcomes ◽  
Early Stage ◽  
Treatment Decisions ◽  
Real World Evidence ◽  
Patient Reported ◽  
The Us ◽  
Disease Stages

e18248 Background: Taxanes-induced neuropathy is common in BC patients receiving taxanes, forcing dose reductions and treatment delays and posing serious challenges for the long-term patient QoL. Discovering neuropathy predictors in patients could guide better treatment decisions, improved QoL and reduce healthcare costs. Belong digital PPN is a social network for cancer patients and caregivers that supports disease management. In this study we used our artificial intelligence (AI) engine to classify the prevalence, characteristics and taxanes-induced neuropathy status of BC patients. Methods: We analyzed real-world patient-reported outcomes provided voluntarily and anonymously from users on the Belong PPN. Data from BC patients reporting treatment with taxanes was extracted and additional analysis segmented the data to those who experienced neuropathy and those who did not. Further validation of the data was performed by our research team to assure accuracy. Results: We evaluated 169 BC cancer patients from the US treated with taxanes. In the cohort 72% were Paclitaxel-treated and 28% Docetaxel-treated at various disease stages: 68% at early stage BC (0-2) and 32% at the advanced/metastatic stages (3-4). 83% of Paclitaxel-treated patients and 67% of Docetaxel-treated patients reported experiencing neuropathy in the Belong platform. These real-world reports indicated significantly higher incidence of taxane-induced neuropathy in comparison to literature summarizing data from clinical trials, suggesting neuropathy incidence of 27% for paclitaxel and 16% for docetaxel (grades 2-4). Conclusions: Real-world patient-reported outcomes from the Belong PPN captured the prevalence of taxanes-induced neuropathy in BC patients and correlated it to the specific drug in use. Evidence for higher incidence of taxanes-induced neuropathy may lead to lower patient QoL and higher healthcare costs and should stimulate better treatment decisions. Further exploration of the gap between controlled clinical studies and real-world evidence is urgently needed to understand the true patient outcomes and optimize healthcare accordingly.

Simulating the Foreign Policy Decision-Making Process in the Undergraduate Classroom

2009 ◽  
Vol 42 (02) ◽  
pp. 401-407 ◽  
Author(s):  
Julie A. Loggins
Keyword(s):  
Decision Making ◽  
Foreign Policy ◽  
Learning Process ◽  
Real World ◽  
Policy Decision ◽  
Decision Making Process ◽  
Multiple Factors ◽  
Political Events ◽  
Policy Decision Making ◽  
Concrete World

A simulation of the foreign policy decision-making process, as described in this article, can assist an instructor in linking students' abstract understanding of complex political events, circumstances, and decision making to the real-world interplay of the multiple factors involved in decision making. It is this type of active learning that helps bring a student's abstract understanding into the concrete world. Instead of being passive learners relying on an instructor's knowledge, students are active participants in the learning process.

Sign in / Sign up

Export Citation Format

Share Document