Innovations

Peer-reviewed publications, award-winning methodology, and real-world evidence that advances healthcare research.

Awards & Grants

Research Grant

2025

GenZ-trials: Revolutionizing Clinical Trials to Optimize Assessment of Healthcare Innovations

Winner of 5-year research grant GenZ-trials to lead the integration of Digital Twins and Synthetic Control Arms for protocol optimization through in-silico simulation, enabling ethically sound alternatives to placebo groups using real-world data to construct robust comparators with fewer enrolled participants.

Learn More
Award Winner

2018

Global Scrip® Award — Best Use of Real-World Evidence, London

The Scrip Awards recognized RWE expertise with its inaugural award for “Best Use of Real-World Evidence.” The winning project aimed to overcome challenges faced when using real-world healthcare data to identify patients with specific subtypes of Multiple Sclerosis, identify high disease activity, evaluate disability by ICD-based EDSS/KFSS, and characterize relapse events, via validated algorithms.

Award Winner

2009

SafetyWorks® Innovation Award — Best Practices, Bio-IT World

Awarded for developing and validating the web-based application to detect and assess drug safety signals — winner of the Best Practices Award of the Bio-IT World, Boston.

Featured Case Study

Real-World Synthetic Control Arms in Action

Journal: Leukemia & Lymphoma (2023)

Study: Comparison of lisocabtagene maraleucel vs. conventional therapy in relapsed/refractory large B-cell lymphoma using a real-world synthetic control arm

Authors: Le HV, Van Naarden Braun K, Nowakowski GS, et al.

Significance: Demonstrated successful application of synthetic control arms for direct therapeutic comparison in a high-stakes oncology setting. This ECA was used as a case study in the EMA 2025 Workshop.

Kaplan-Meier survival curve comparing lisocabtagene maraleucel intervention versus synthetic control arm over 24 months

External Control Arms & Digital Twins

Blood Cancer Journal of Nature

KarMMa-RW: comparison of idecabtagene vicleucel with real-world outcomes in relapsed and refractory multiple myeloma

Jagannath S, Lin Y, Goldschmidt H, et al.

2021

DOI: 10.1038/s41408-021-00507-2

This ECA was used as a case study in the EMA 2025 Workshop

Leukemia & Lymphoma

Comparison of lisocabtagene maraleucel vs. conventional therapy in relapsed/refractory large B-cell lymphoma using a real-world synthetic control arm

Le HV, Van Naarden Braun K, Nowakowski GS, et al.

2023

DOI: 10.1080/10428194.2022.2160200

This ECA was used as a case study #3 in the EMA 2025 Workshop

EU Research Grant

GenZ-trials: Revolutionizing Clinical Trials to Optimize Assessment of Healthcare Innovations

Evinexus (Consortium Partner)

2025

View Project →
Epidemiologic Methods

Effect of designations of index date in externally controlled trials: an empirical example

Le HV, De Benedetti M, Yue L, et al.

2024

DOI: 10.1515/em-2023-0041
Advances in Therapy

Real-World Treatment Patterns and Clinical Outcomes Among Patients with Metastatic or Unresectable EGFR-Mutated Non-Small Cell Lung Cancer Previously Treated with Osimertinib and Platinum-Based Chemotherapy

Patel J, Meng J, Le HV, et al.

DOI: 10.1007/s12325-024-02936-4

Algorithm Development & Validation

Value in Health

Identifying relapsing-remitting multiple sclerosis (RRMS) in United States integrated delivery network healthcare claims and electronic health record (EHR) data

Le HV, Truong C, Kamauu A, et al.

DOI: 10.1016/j.jval.2018.06.014
Advances in Therapy

Creating a Real-World Data, United States Healthcare Claims-Based Adaptation of Kurtzke Functional Systems Scores for Assessing Multiple Sclerosis Severity and Progression

Truong C, Le HV, Kamauu A, et al.

2021

DOI: 10.1007/s12325-021-01858-9
BMC Medical Research Methodology

Effects of Aggregation of Drug and Diagnostic Codes on the Performance of the High Dimensional Propensity Score Algorithm: An Empirical Example

Le HV, Poole C, Brookhart AM, et al.

DOI: 10.1186/1471-2288-13-142

Data Labeling & Annotation for AI & RWE

Drug Safety

Evaluation of Facebook and Twitter Monitoring to Detect Safety Signals for Medical Products: An Analysis of Recent FDA Safety Alerts

Pierce CE, Bouri K, Pamer C, et al.

DOI: 10.1007/s40264-016-0491-0
BMJ Open

Identification of responders to inhaled corticosteroids in a COPD population using cluster analysis

Hinds DR, DiSantostefano RL, Le HV, et al.

DOI: 10.1136/bmjopen-2015-010099
ISPE 2025

Performances of the Deep Learning Approach to Nonparametric Propensity Score Estimation with Optimized Covariate Balance in Small Samples

Le HV, et al.

2025

Study Design, Analytics & Scientific Communication

Advances in Therapy

Real-World Treatment Patterns and Clinical Outcomes in Patients With Extensive-Stage Small Cell Lung Cancer Treated With First-Line Platinum-Based Chemotherapy and ≥ 2 Subsequent Lines of Therapy in the United States

Sankar K, Unni S, Eberl M, et al.

DOI: 10.1007/s12325-025-03408-z
Statistical Methods in Medical Research

Performance of a semi-automated approach for risk estimation using a common data model for longitudinal healthcare databases

Le HV, Beach JK, Powell G, et al.

DOI: 10.1177/0962280211403599
Antibiotics (Basel)

Hospital Admission Patterns in Adult Patients with Community-Acquired Pneumonia Who Received Ceftriaxone and a Macrolide by Disease Severity across United States Hospitals

Lodise PT, Le HV, LaPensee K.

DOI: 10.3390/antibiotics9090577

Medical Coding & Phenotype Definition

PLoS One

Risk of pneumonia with inhaled corticosteroid versus long-acting bronchodilator regimens in chronic obstructive pulmonary disease: a new-user cohort study

DiSantostefano RL, Sampson T, Le HV, et al.

2014

DOI: 10.1371/journal.pone.0097149
Journal of Chronic Obstructive Pulmonary Disease

Can assessment of disease burden prior to changes in initial COPD maintenance treatment provide insight into remaining unmet needs? A retrospective database study in UK primary care

Landis S, Wurst K, Le HV, et al.

DOI: 10.1080/15412555.2016.1240159
Respiratory Research

Patient characteristics and treatment patterns over 24 months among COPD patients initiating long-acting bronchodilator prescription in the UK primary care setting

Punekar YS, Landis S, Wurst K, et al.

DOI: 10.1186/s12931-015-0295-2
Respiratory Medicine

The Relationship Between Blood Eosinophils and Clinical Characteristics in a Cross-Sectional Study of a US Population-Based COPD Cohort

DiSantostefano RL, Le HV, Hinds, et al.

DOI: 10.1016/j.rmed.2016.01.013