Leveraging Existing Data for Clinical and Observational Research

 The abundance of data generated during routine health care is growing in significance and should be re-used for clinical and observational research. Patient electronic records, registries, insurance claims, data from pharmacy and social media, and electronic patient-reported outcomes have been increasingly used as eSources. This process requires strategizing, utilizing novel data technologies, as well as close collaboration between pharmaceutical companies and organizations that possess the data. CHI’s Inaugural “Leveraging Existing Data for Clinical and Observational Research” will discuss challenges and solutions with secondary use of existing healthcare data for assessing the effectiveness and safety of medical products.

Wednesday, February 24

12:10 pm Bridging Luncheon Presentation:
Pharmacoeconomic Assessment through Market Approval and Beyond: Theory and Operations

Matthew Page, Epidemiologist, Biometrics, Medpace

12:50 Coffee and Dessert in the Exhibit Hall

1:30 Plenary Keynotes - View Details

3:00 Refreshment Break in the Exhibit Hall (Last Chance for Viewing)

REAL WORLD DATA SOURCES, ANALYTICS AND APPLICATIONS

4:00 Chairperson’s Remarks

Adam Wilcox, Ph.D., Medical Informatics Director, Intermountain Healthcare

4:30 Do Patients Defined in My Clinical Trial Protocol Really Exist in Real-World Healthcare Settings?

Irene Cosmatos, M.Sc., Database Analytics Automation, UBC: An Express Scripts Company

An evidence-based approach to understanding clinical and demographic patient profiles early in a trial’s design can inform selection criteria before a protocol is finalized. Benefits include improved patient recruitment and a reduced need for costly protocol amendments. Through scenarios using real world healthcare data, attendees will learn how observational data can inform clinical teams of the impact that certain patient selection criteria may have on recruitment, using visualizations that highlight problem criteria.

4:55 Novel Ways to Design and Execute Prospective Observational Studies Using EMR and Linked Data Network

Hui Cao, M.D., Ph.D., Executive Director, Real-world Evidence, COE for RWE, Global Medical Affairs, Novartis Pharmaceuticals Corporation

Technologies such as EHR, mobile health, wearable devices and linked data networks are providing new ways to conduct observational studies. EHR not only enables secondary uses for retrospective studies, but also provides a new platform to observe patients prospectively. We will discuss with the audience the examples of novel prospective studies that leverage EHR and m-Health technologies to maximize the data collection and to follow up patients beyond the study period.

5:20 Panel Discussion: Blurring the Lines between Primary and Secondary Use of Medical Data

Usman Iqbal M.D., Senior Medical Affairs Leader, Neuroscience, Global Medical Affairs, AstraZeneca

Panelists:
Sebastian Schneeweiss, M.D., Sc.D., Vice Chief of the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine of the Brigham and Women’s Hospital, Professor of Medicine, Harvard Medical School, Professor in Epidemiology, the Harvard School of Public Health

Hui Cao, M.D., Ph.D., Executive Director, Real-world Evidence, COE for RWE, Global Medical Affairs, Novartis Pharmaceuticals Corporation

Adam Wilcox, Ph.D., Medical Informatics Director, Intermountain Healthcare

Khurram Nasir, M.D., Director Center for Healthcare Advancement & Outcomes at Baptist Health South Florida

6:15 Reception Hosted by Exostar

7:15 Close of Day

Thursday, February 25

7:15 am Registration

7:45 Breakfast Presentation: How One (Golden) Number
Can Transform Clinical Trials

Elisa Cascade, MBA, President, Data Solutions, DrugDev

For years pharmaceutical companies and CROs have struggled to collect, clean and collate site data from disparate sources so they can make better decisions about site feasibility and selection. With the DrugDev Golden Number - a universal identifier for Investigators and sites - individual pharma companies and CROs easily match and master data, and share data across collaborations like the Investigator Databank and TransCelerate. While the Golden Number began as a method for data sharing, innovators are using it to drive operational efficiencies, collaboration and integrated reporting as well. We expect it also will give regulators a more detailed view into the global investigator community, which ultimately can improve patient safety. Join this session for a discussion on the Golden Number and how it can help pharma, CROs and sites do more trials.

PRAGMATIC TRIALS

8:35 Chairperson’s Remarks

8:40 Big Clinical Data: Challenges and Approaches in Using Electronic Health Record Data for Research Studies

Adam Wilcox, Ph.D., Medical Informatics Director, Intermountain Healthcare

With the widespread adoption of electronic health records, there is a great opportunity to use the data from these records to advance research studies. However, there are important differences between data from EHRs and data collected for research studies. This presentation discusses these challenges, and outlines approaches and methods to address them, with lessons learned from managing clinical data warehouses and with the Patient Centered Outcomes Research Institute.

9:05 Designing Cardiovascular Pragmatic Clinical Trials: Learning Healthcare System at Work

Khurram Nasir, M.D., Director, Center for Healthcare Advancement & Outcomes at Baptist Health South Florida

Clinical trials have been the main tool used by the health sciences community to test and evaluate interventions. Trials can fall into two broad categories: pragmatic and explanatory. Pragmatic trials are designed to evaluate the effectiveness of interventions in real-life routine practice conditions, whereas explanatory trials aim to test whether an intervention works under optimal situations. Pragmatic trials produce results that can be generalized and applied in routine practice settings.

9:30 Utilizing Pragmatic Clinical Trials in Clinical Development Programs

Usman Iqbal, M.D., Senior Medical Affairs Leader, Neuroscience, Global Medical Affairs, AstraZeneca

The presentation will focus on understanding/using the concept of patient journey through the lens of BIG DATA & secondary databases to identify patient centric unmet needs and gaps in care, and leveraging the related output to design pragmatic trials. A real world case study will be presented in this regard walking the audience through the methodology of patient journey assessment and systematic application of related evidence and knowledge towards pragmatic trial design, patient populations, end point strategy and strategic clinical decision support.

9:55 Leveraging the Electronic Medical Records (EMR) Based Analytics to Optimize Design of a Prospective Observation Study for Asthma and COPD (Chronic Obstructive Pulmonary Disease)

Xia Wang, Ph.D., Principal Health Informatics Scientist, Informatics Lead RIA, AstraZeneca

This talk relates an EMR based analysis to optimize the design of a 3-year, multi-country prospective observational study focusing on chronic obstructive lung disease. This study aims to enroll patients from a non-controlled ("real-world") setting and will be the largest coordinated global study of its kind. The analysis of 11 country-specific EMR data sources provides unique insight of clinical practices and patient profiles, promotes optimized design and enables novel data collection workflow.

10:20 Coffee Break

CROSS-LINKING DATA SOURCES

10:35 Chairperson’s Remarks

Randy Ramin-Wright, Head, Quality Risk Management, Clinerion

10:40 Improving Transparency, Efficiency, and Quality in Patient Registry-Based Research: AHRQ Strategy and Initiatives

Elise Berliner, Ph.D., Director, The Technology Assessment Program, AHRQ

This presentation will share our work to improve validity of registry studies through three related methods projects to improve the development of registries, a major activity of AHRQ’s Effective Health Care Program:

• Framework for record linkage of registry to other data sources.
• Prospectively measuring physicians’ treatment allocation decision process to minimize confounding by indication.
• Prospective collection of registry information for deriving instrumental variables.

11:05 Broadening Insight into Cancer Patient Characteristics through Matching Secondary Data Sources

Elizabeth A. MacLean, Pharm.D., Director, Global Health and Value/Outcomes & Evidence, Pfizer

Use of administrative claims data to address questions related to cancer patient treatment experience may yield limited information. However, match of administrative claims to corresponding medical record data can broaden insight into patients' clinical and disease characteristics. This presentation will describe the experience of conducting a two-part study to examine treatment experience of patients with non-small cell lung cancer.

11:30 Evaluating an Innovative Approach to a Prospective Patient Registry Design using Electronic Medical Record (EMR) Data

Susan Fish, Senior Statistical Programmer Analyst, Genentech

Registries today are more complex and expensive to implement than in past years due to increased regulatory reporting requirements and compliance concerns. This presentation will describe a potential solution for registry data collection using an innovative external partnership model that is both cost effective and collaborative, and present results from a pilot study using EMR based data collection vs traditional CRF data for a lung cancer registry.

11:55 Safety Monitoring for Newly Marketed Products Using a Claims Database

Rui Jiang, M.D., DrPH, Associate Director, Pharmacovigilance & Epidemiology, Gilead Sciences

Prospectively collected electronic healthcare data are more and more used for drug-safety research in complement to passive safety monitoring system such as FDA adverse event reporting system (AERS). The goal of active drug safety monitoring is to detect serious adverse reactions (pre-specified outcomes) as early as possible without too many false alarms. We are applying this method to the study design for analyzing the IMS claims data to monitoring of adverse reactions among patients treated with a newly approved oncology product Zydelig, a treatment for relapsed/refractory chronic lymphocytic leukemia and indolent NHL, from 2014 to 2018.

12:20 INTERACTIVE PANEL: Can We Shift Investigators from “1 and Done” to Repeat Performers

Claire Sears, Ph.D., Director, Investigator Engagement, SiteStart, DrugDev

Immo Zadezensky, PharmD, Ph.D., Clinical Pharmacologist, Professional Affairs and Stakeholder Engagement (PASE), Office of the Center Director, CDER, FDA

Christine Pierre, Founder & President, Society for Clinical Research Sites (SCRS)

Jeffrey Rosen, M.D., Medical Director, Clinical Research of South Florida; Associate Professor, University of Miami Miller School of Medicine

In this session we will discuss the problem of investigator turnover – in particular, the large ‘1 and done’ population. Tufts will present information from the FDA 1572 database on the trends, characteristics and size of the ‘1 and done’ population. This information will be supplemented by a presentation of findings from the Investigator Databank related to characteristics of approximately 70,000 ‘1 and done’ sites including site level performance amongst other factors (e.g., geography, pediatrics, therapeutic area). It is clear that the high rate of attrition of investigators and the need to initiate new investigators is detrimental to site and overall trial performance, and is costly and time-consuming for sponsors. The session will conclude with a commentary on the data from the Site perspective regarding what can be done (if anything) to decrease the rate of investigator turnover. The learning objectives for this session are:

• Learn detailed characteristics of the global population of investigators who have only participated in one clinical trial
• Identify factors that can be gleaned from these detailed analyses that help identify investigators at risk of only participating in a single clinical trial
• Based on these insights, discuss strategies/actions that can help reduce the likelihood of future site turnover

12:45 Closing Remarks

12:50 pm SCOPE 2016 Conference Adjourns (see you in Miami for 2017!)