The US Food and Drug Administration's Real‐World Evidence Framework: A Commitment for Engagement and Transparency on Real‐World Evidence

2019 ◽  
Vol 106 (1) ◽  
pp. 33-35 ◽  
Author(s):  
M. Khair ElZarrad ◽  
Jacqueline Corrigan‐Curay
Keyword(s):  
Real World ◽  
Evidence Framework ◽  
Real World Evidence ◽  
The Us

scholarly journals A Real‐World Evidence Framework for Optimizing Dosing in All Patients With COVID‐19

2020 ◽  
Vol 108 (5) ◽  
pp. 921-923 ◽  
Author(s):  
Richard W. Peck ◽  
Daniel Weiner ◽  
Jack Cook ◽  
J. Robert Powell
Keyword(s):  
Real World ◽  
Evidence Framework ◽  
Real World Evidence

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.

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 Integrating Real-World Evidence in the Regulatory Decision-Making Process: A Systematic Analysis of Experiences in the US, EU, and China Using a Logic Model

2021 ◽  
Vol 8 ◽  
Author(s):  
Meng Li ◽  
Shengqi Chen ◽  
Yunfeng Lai ◽  
Zuanji Liang ◽  
Jiaqi Wang ◽  
...  
Keyword(s):  
Decision Making ◽  
Real World ◽  
Decision Making Process ◽  
Real World Data ◽  
Systematic Analysis ◽  
Regulatory Decision ◽  
Real World Evidence ◽  
The Us ◽  
Post Market ◽  
New Treatment

Real world evidence (RWE) and real-world data (RWD) are drawing ever-increasing attention in the pharmaceutical industry and drug regulatory authorities (DRAs) all over the world due to their paramount role in supporting drug development and regulatory decision making. However, there is little systematic documentary analysis about how RWE was integrated for the use by the DRAs in evaluating new treatment approaches and monitoring post-market safety. This study aimed to analyze and discuss the integration of RWE into regulatory decision-making process from the perspective of DRAs. Different development strategies to develop and adopt RWE by the DRAs in the US, Europe, and China were reviewed and compared, and the challenges encountered were discussed. It was found that different strategies on development of RWE were applied by FDA, EMA, and NMPA. The extent to which RWE was adopted in China was relatively limited compared to that in the US and EU, which was highly related to the national pharmaceutical environment and development stages. A better understanding of the overall goals, inputs, activities, outputs, and outcomes in developing RWE will help inform actions to harness RWD and leverage RWE for better health care decisions.

scholarly journals 855. Persistence on F/TAF versus F/TDF for HIV Pre-Exposure Prophylaxis: A Real-World Evidence Analysis in the United States

2021 ◽  
Vol 8 (Supplement_1) ◽  
pp. S518-S519
Author(s):  
Li Tao ◽  
Valentina Shvachko ◽  
Moupali Das ◽  
Christoph C Carter ◽  
Jared Baeten ◽  
...  
Keyword(s):  
United States ◽  
Real World ◽  
The United States ◽  
Odds Ratios ◽  
Preexposure Prophylaxis ◽  
Hazard Ratios ◽  
Real World Evidence ◽  
The Us ◽  
Evidence Analysis ◽  
Exposure Prophylaxis

Abstract Background Persistence to preexposure prophylaxis (PrEP) is an important determinant of its efficacy, but evidence on real-world persistence is lacking. This study assesses adherence to F/TDF and F/TAF for PrEP both in terms of discontinuation and re-initiation patterns. Methods We identified HIV-negative individuals in the United States who initiated F/TDF or F/TAF for PrEP between October 2019 and December 2020 from a de-identified prescription claims database; users taking generic F/TDF were excluded. Non-persistence was defined as a prescription fill gap of >30 days; discontinuation included switch from F/TDF to F/TAF or F/TAF to F/TDF. We used survival analyses to estimate persistence, Cox regressions to compare the hazard ratios (HR) of discontinuation, and logistic regression to compare the odds ratios of re-initiation after discontinuation. Results Among F/TAF users (N=82,402) median age at PrEP initiation was 35 years (interquartile range [IQR] 28−47) and median PrEP persistence was 4 months (IQR 1.8-8.9), compared to 31 years (IQR 25−40) and 2 months (IQR 1.0-3.8) for F/TDF users (N=48,501). PrEP persistence at 60 and 90 days was higher among F/TAF users than F/TDF users (Figure). F/TDF users were 2.5 times more likely to discontinue than F/TAF users, with more marked differences in older users than that in younger users (p for interaction between discontinuation and age group < 0.01, Table). We also observed a higher rate of discontinuation of F/TDF versus F/TAF if PrEP was prescribed by internal medicine or infectious disease physicians than by family medicine physicians (data not shown). After discontinuation, F/TAF users were 1.7 times more likely than F/TDF users to re-initiate PrEP; the association was not different by age. Persistence rates of F/TAF and F/TDF for PrEP by time of PrEP initiation Hazard ratios (HR) and corresponding 95% confidence intervals (CI) of non-persistence and odds ratios (OR) of re-initiation after discontinuation for users of F/TAF and F/TDF for PrEP in the US, Oct 2019 – Dec 2020 Conclusion In this real-world analysis, the F/TAF for PrEP regimen was associated with higher persistence and re-initiation than F/TDF for PrEP. These findings underscore the dynamic nature of PrEP utilization in the real-world and the importance of interventions aimed at improving PrEP persistence and re-initiation in people who would benefit from PrEP. Disclosures Li Tao, MD, PhD, Gilead Sciences Inc (Employee, Shareholder) Valentina Shvachko, MS, Gilead Sciences Inc (Employee, Shareholder) Moupali Das, MD, Gilead Sciences Inc. (Employee, Shareholder) Christoph C. Carter, MD, Gilead Sciences Inc. (Employee, Shareholder) Jared Baeten, MD, PHD, Gilead Sciences Inc. (Employee, Shareholder) David Magnuson, PharmD, Gilead Sciences Inc (Employee, Shareholder)

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