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

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

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 Real‐world evidence to support regulatory decision‐making for medicines: Considerations for external control arms

2020 ◽  
Vol 29 (10) ◽  
pp. 1228-1235 ◽  
Author(s):  
Mehmet Burcu ◽  
Nancy A. Dreyer ◽  
Jessica M. Franklin ◽  
Michael D. Blum ◽  
Cathy W. Critchlow ◽  
...  
Keyword(s):  
Decision Making ◽  
Real World ◽  
External Control ◽  
Regulatory Decision ◽  
Real World Evidence

scholarly journals FDA Oncology Center of Excellence Review of Real World Data Submissions Supporting Drug Development in Hematologic Malignancies

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 5037-5037
Author(s):  
Donna R. Rivera ◽  
Jennifer J Lee ◽  
Melanie Royce ◽  
R. Angelo de Claro ◽  
Nicole J. Gormley ◽  
...  
Keyword(s):  
Decision Making ◽  
Overall Survival ◽  
Real World ◽  
Response Rate ◽  
Hematologic Malignancies ◽  
Real World Data ◽  
World Data ◽  
Regulatory Decision ◽  
Real World Evidence ◽  
Regulatory Submissions

Abstract Background Aligning with 21st Century Cures legislation, FDA is exploring methodologies to advance appropriate uses of Real World Data (RWD) to generate Real World Evidence (RWE). RWD to support regulatory decision making has markedly increased in oncology. This review specifically focused on the analysis of RWD containing submissions for medical products in development for the treatment of hematological malignancies and associated treatment related conditions (e.g., Cytokine Release Syndrome (CRS), Graft Versus Host Disease (GVHD). Methods A systematic search was conducted using internal FDA databases to identify RWD submissions from 2010 to 2020. Search terms included: real world evidence, real world data, electronic health record, cancer registry, administrative claims, external control arm, observational cohort, historical control arm, real world Overall Survival (rwOS) , real world Response Rate (rwRR), real world Overall Response Rate rwORR and real world Complete Response (rwCR). Regulatory submissions specific to malignant hematology and associated treatment related conditions were reviewed, and pre-defined common data elements were extracted and validated by independent dual review. Descriptive statistics were calculated. Results A total of 142 regulatory submissions included RWD from 2011-2020. A subset of 94 RWD submissions met the criteria for further evaluation, of which 20 (21%) submissions corresponding with 14 molecular entities were for hematologic malignancies or treatment related conditions (e.g., CRS, GVHD). RWD submissions increased substantially over time, with 14 (70%) of submissions received between 2019-2020. Specific evaluation for pediatric indications was referenced in 15% of submissions. The most commonly referenced RWD source was EHR data (55%), followed by use of multiple sources (20%), and registry data (15%). Approximately 90% of the submissions aimed to support treatment effectiveness. Primary RWD study objectives included supporting approval of a new molecular entity (NME) (40%), expanding an approved indication (25%), conversion from accelerated to regular approval (15%), and providing data to inform postmarketing safety evaluation (20%). Among RWD submissions, response endpoints (e.g., rwORR, rwCR, rwPR, Partial Response) and overall survival (e.g., rwOS) were most frequently selected as primary outcomes for 50% and 20% of proposals respectively; however, these outcomes were included as any endpoint in 65% and 75% of submissions. Conclusion This review demonstrates increasing use of various RWD sources to support evidence generation for drug development in hematologic malignancies and associated treatment related conditions with the primary objective of supporting demonstration of effectiveness using rwOS or real world response measures as primary endpoints. Given the increased inclusion of RWD in regulatory submissions, further methodological development is needed, including in the selection and validation of rwEndpoints. Appropriate study design must be aligned with a clear regulatory objective to ensure that RWD can be adequately evaluated. Additionally, the development of standardized metrics for data characterization and transparency in reporting of RWD are foundational steps to the evaluation of fit for purpose RWD to support regulatory decision making. Disclosures No relevant conflicts of interest to declare.

scholarly journals EU-funded initiatives for real world evidence: descriptive analysis of their characteristics and relevance for regulatory decision-making

BMJ Open ◽  
2018 ◽  
Vol 8 (6) ◽  
pp. e021864 ◽  
Author(s):  
Kelly Plueschke ◽  
Patricia McGettigan ◽  
Alexandra Pacurariu ◽  
Xavier Kurz ◽  
Alison Cave
Keyword(s):  
Decision Making ◽  
Real World ◽  
Descriptive Analysis ◽  
Regulatory Decision ◽  
Real World Evidence

FDA Oncology Center of Excellence landscape analysis of real-world data submissions for oncology drugs.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18787-e18787
Author(s):  
Donna Rivera ◽  
Jennifer J. Lee ◽  
Melanie E Royce ◽  
Paul Gustav Kluetz
Keyword(s):  
Decision Making ◽  
Real World ◽  
Landscape Analysis ◽  
Real World Data ◽  
World Data ◽  
Regulatory Decision ◽  
Real World Evidence ◽  
Regulatory Submissions ◽  
Oncology Center ◽  
Oncology Drug Development

e18787 Background: Aligning with 21st Century Cures legislation, the FDA is exploring trial design modernization and methodology to advance appropriate uses of Real World Data (RWD) to generate Real World Evidence (RWE). The Oncology Center of Excellence RWE Program was established in 2020 to advance RWE efforts specific to oncology drug development. Inclusion of RWD to support regulatory decision making has increased in oncology, and a landscape analysis was conducted to characterize the RWD included in submissions. Methods: A systematic search was conducted using internal FDA databases to identify RWD submissions from 2010 to 2020. Search terms included: real world evidence, real world data, electronic health record, cancer registry, administrative claims, external control arm, observational cohort, historical control arm, rwOS, rwRR, rwCR, and rwORR. Relevant regulatory submissions were reviewed, and pre-defined common data elements were extracted. A team of FDA reviewers assessed agreement through subset validation (20%). Descriptive statistics were calculated. Results: A total of 142 regulatory submissions included RWD from 2011 to 2020. A subset of 94 submissions met the criteria for evaluation, consisting of 78 unique studies evaluating 56 molecular entities. RWD submissions increased substantially over time, with 28 submissions in 2020. Nearly half of the RWD submissions were for solid tumor indications (68%), with lung cancer being the most predominant site. More than one third of the RWD submissions (37%) were for rare indications. The most common primary RWD study objective was effectiveness (62%) and the most commonly referenced RWD source was EHR/clinical data (54%). The most frequently used primary RWD endpoints were survival (rwOS, 35%) and response (rwORR/PR/BTR, 31%) outcomes (Table). Conclusions: Our review demonstrates a dramatic increase in RWD submissions to support FDA oncology drug development programs. Submissions included a variety of study objectives, data sources, and endpoints. While this landscape analysis provides a picture of potential regulatory objectives, the adequacy of each proposal to support regulatory decision making was not evaluated. Establishing a set of clear regulatory objectives can help advance the development of metrics for robust data characterization and outcome validation to ensure that RWD can be appropriately evaluated and provide the rigor necessary to be considered adequate RWE.[Table: see text]

Nonrandomized Real‐World Evidence to Support Regulatory Decision Making: Process for a Randomized Trial Replication Project

2020 ◽  
Vol 107 (4) ◽  
pp. 817-826 ◽  
Author(s):  
Jessica M. Franklin ◽  
Ajinkya Pawar ◽  
David Martin ◽  
Robert J. Glynn ◽  
Mark Levenson ◽  
...  
Keyword(s):  
Decision Making ◽  
Randomized Trial ◽  
Real World ◽  
Decision Making Process ◽  
Regulatory Decision ◽  
Real World Evidence

Role of real world data and real world evidence in regulatory decision making

2021 ◽  
Vol 14 (2) ◽  
pp. 1177-1182
Author(s):  
B. C. Bharath ◽  
V. Balamuralidhara ◽  
M. P. Gowrav
Keyword(s):  
Decision Making ◽  
Real World ◽  
Real World Data ◽  
World Data ◽  
Regulatory Decision ◽  
Real World Evidence

scholarly journals PNS26 Real-World Evidence in Regulatory Decision-Making: Parallel between FDA in the US and Pmda in JAPAN

2020 ◽  
Vol 22 ◽  
pp. S85
Author(s):  
A. Vladimirova ◽  
Y. Onishi ◽  
K. Kloc ◽  
C. Rémuzat ◽  
M. Toumi
Keyword(s):  
Decision Making ◽  
Real World ◽  
Regulatory Decision ◽  
Real World Evidence ◽  
The Us

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.

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