Managing Late Stage Research and Observational Studies

Non-interventional studies are an integral part of clinical development programs and product development plans. Product safety profiles, comparative effectiveness data as well as health economic evidence obtained from non-interventional studies, are essential for multiple stakeholders. These stakeholders include but are not limited to the following: regulatory agencies, payers, health care management organizations, formulary inclusion decision makers, healthcare professionals, and patients. Cambridge Healthtech Institute’s Fifth Annual “Managing Late Stage Research and Observational Studies" conference is designed to facilitate knowledge exchange around all aspects of observational research from the designing and managing of post-approval studies, to applying the data obtained to pivotal business and medical decisions.

Monday, February 22

7:00 – 9:00 pm Welcome and Networking Happy Hour on the Riverwalk hosted by CHI, DrugDev and Praxis

Tuesday, February 23

7:15 am Registration and Morning Coffee

8:25 Opening Plenary Keynotes - View Details

Biomedical Systems9:45 Grand Opening Coffee Break in the Exhibit Hall



10:45 Chairperson’s Remarks

Christopher-Paul Milne, Ph.D., Director, Research, Tufts CSDD, Tufts University School of Medicine

10:50 Building and Leveraging Data Resources and Capabilities: Patients, Partners and Platforms!

Cathy_CritchlowCathy Critchlow, Ph.D., Executive Director, Amgen Center for Observational Research

We have built a platform to meet increasing demand for real world data to support drug development and portfolio prioritization. Insurance claims and electronic medical record databases are put into a ‘data lake’, converted to a common data model, and powered by an architecture enabling rapid, simultaneous processing of large datasets. Creation of patient cohorts in conjunction with design, analytic and visualization tools enables broad, enterprise-wide access to data resources to proactively address key questions for internal and external stakeholders.

11:15 Demonstrating Effectiveness with Real World Evidence

Boxiong_TangBoxiong Tang, M.D., Ph.D., Senior Director, Growth Markets/GHEOR, Teva Pharmaceuticals

Real-world studies provide a different perspective on value than randomized clinical trials (RCTs). They reflect real life utility of drugs and for that reason they are increasingly viewed as viable alternative and complement to RCTs by many decision-makers, parituclarly payers. This presentation will share Teva’s best practices and strategies to study effectiveness of therapeutics with real world evidence.

11:40 Impact of Post-Approval Evidence Generation on the Biopharmaceutical Industry

Christopher-Paul_MilneChristopher-Paul Milne, Ph.D., Director, Research, Tufts CSDD, Tufts University School of Medicine

Meeting marketplace demands for proving the value of new products requires more data than the industry has previously had to routinely produce for a variety of post-approval decisions and an array of health care system stakeholders. These data include evidence from comparative effectiveness research (CER), including randomized, controlled trials, pragmatic clinical trials, observational studies, and meta-analyses. We report the findings of an industry working group that examined the burden of growing demands for CER evidence, the acceptability of post-approval study types, payer-specific issues related to CER, communication of data being genegenerated post-approval, and methods used for facilitating post-approval evidence generation.

12:05 pm Physician Participation in Real World Research – A Global Case Study

Kathleen MandziukKathleen Mandziuk, Senior Director, Scientific Affairs, PRA Health Sciences, Late Phase Services

One of the most challenging aspects of non-interventional research is gaining and retaining physician interest in participation. This presentation will cover the key aspects of site recruitment for observational studies, and the impact of various factors such as outreach methods/messaging, study design, target indication and country differences. A variety of case studies and examples will be outlined and presented.

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

Biomedical Systems1:20 Coffee and Dessert in the Exhibit Hall



2:00 Chairperson’s Remarks

Jeff Luross, Senior Director, Operations, Late Phase Services, PRA Health Sciences

2:05 Strengths and Limitations of Various Types of Existing Databases

Sean_ZhaoSean Zhao, M.D., Head, US Patient Safety Surveillance, AstraZeneca

Fully understanding the strengths and limitations of various types of these existing databases will help pharmaceutical companies and academic researchers to find the best suitable database and reasonable study design to address clinical research questions with high quality. In some cases, it is necessary to combine two or more types of existing databases or databases from multiple countries to address complex research issues. The presentation will discuss considerations in database selection, study design and analytic approaches, study outcome ascertainment, reduction of selection bias and information bias, and observational research activities based on multiple databases.

2:30 Identifying Drug Use in Hospital Settings in Denmark: A Challenge and a Promise.

Uffe_Heide-JørgensenUffe Heide-Jørgensen, Ph.D., Biostatistician, Clinical Epidemiology, Aarhus University

The unique constellation of population-based registries, universal health care, and individual-level linkage have placed Denmark and other Nordic countries at the forefront of clinical and observational research. As in most routine databases, identification of drugs dispensed in hospitals remains a challenge in Denmark, as those dispensations do not feed into the traditional outpatient dispensation registries. Harnessing existing routine registration mechanisms, including electronic health records, may help overcome this limitation in the near future.

2:55 PANEL DISCUSSION: Meeting the Evidentiary Needs of Multiple Stakeholders by Better Non-Interventional Studies

Christopher-Paul_MilneChristopher-Paul Milne, Ph.D., Director, Research, Tufts CSDD, Tufts University School of Medicine

Topics to be discussed include but are not limited to the following:

  • What are key considerations and approaches to balance scientific and commercial values of non-interventional studies
  • What are common utilization of non-interventional studies in supporting clinical development program
  • How can evidences generated from non-interventional studies be used in discussions with regulatory agencies during product development and post marketing in support of establishing product benefit risk profile, continual safety monitoring, and risk management and mitigation activities, as well as fulfilling regulatory post marketing safety requirement (PMRs and FUMs)

Icon Corporation3:20 Observational Studies; an Optimal Cost Effective Operational Approach

Hady KhouryHady Khoury, Vice President, Research & Alliance Support Services, PA&OR Project Management Commercialisation & Outcomes, ICON
As regulators, payers, and patients look more to observational research to better collect and assess long-term data on product safety and effectiveness, as well as product value, sponsors must ensure that these studies are designed and conducted in the most cost-effective and efficient manner possible. This session will provide an overview of the current observational research landscape, including a discussion of key operational and cost challenges. You will learn strategies for balancing operations and risk, and ways to implement more holistic operational oversight.

3:35 Sponsored Presentation (Opportunity Available)


3:55 Find Your Table and Meet Your Moderator

4:00 Interactive Breakout Discussion Groups

Concurrent breakout discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss some of the key issues presented earlier in the day’s sessions. Delegates will join a table of interest and become an active part of the discussion at hand. To get the most out of this interactive session and format please come prepared to share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and, most importantly, participate in active idea sharing.

Complete list of Breakout Discussion Groups

5:00 Welcome Reception in the Exhibit Hall

6:30 Close of Day

Wednesday, February 24

7:15 am Registration

QUINTILES7:45 Breakfast Presentation: Operational Excellence in Real-World Research: Observational Studies & Registries

Radha Puri, Associate Director, Americas, Real-World Evidence Strategy Unit, Real-World Late Phase Research, Quintiles

Obtaining and maintaining operational excellence in real-world research requires connecting insights for better outcomes. Understand the challenges to mitigating risks while maximizing data quality and hear how to achieve and maintain quality standards in observational studies and registries while minimizing site burdens.

Streamlining Operational Activities

8:25 Chairperson’s Remarks

Alicia Gilsenan, Ph.D., Senior Director, Epidemiology, RTI Health Solutions

8:30 Similarities and Differences in Operationalizing Observational Studies Versus Clinical Trials

Alicia_GilsenanAlicia Gilsenan, Ph.D., Senior Director, Epidemiology, RTI Health Solutions

While many aspects regarding the implementation of clinical trials and observational studies are similar, there are several aspects that require additional consideration. This talk will highlight strategies for the implementation and management of observational studies as compared to clinical trials from start-up activities through data collection and reporting to minimize selection bias, information bias and impact of confounding on the final results.

8:55 Investigator Initiated Trial Management – A Medical Affairs Perspective 

Lynn BassLynn Bass, ‎Director, Medical Affairs and Global Grants Manager, Jazz Pharmaceuticals

Many Investigator Initiated Trial (IIT) programs initiate with a concept which is received from the field by a medical affairs staff member.   From here, the concept is facilitated by the research grant manager through a series of reviews and audits from a variety of medical, clinical, and research personnel.  This session will focus on a review of the complexities, communications, and best practices from the receipt of an IIR concept through the completion of the study. Session objectives include:


  • Review the role of the research grant manager and the field medical role in the IIT process
  • Review how the concept is reviewed by a cross-functional set of stakeholders
  • Review the differnet role of the investigator in company sponsored research verses IIT



9:20 Better Data Faster – Using Claims Data to Accelerate Observational Research
Donny_ChenDonny Chen, MBA, Director, Medical Affairs Research Operations, PPD
As various stakeholders seek to substantiate the effectiveness, safety, quality-of-life, and economic benefits of new treatments, there's growing pressure to find the optimal observational study design to meet this need. This talk will highlight how a robust claims database can be a powerful complement to a prospective observational study by accelerating site/patient identification, minimizing data collection burden, and linking resource utilization and cost data to clinical endpoints.

Bio Clinica Logo9:45 Patient Centric Post Approval Research – It’s Time to Rethink How Post Approval Studies Are Done

Nayan NanavatiNayan Nanavati, COO, Post Approval Services, Bioclinica

Post approval research is an evolving science. Over the last decade, post approval studies have evolved, in their objectives, complexities and costs. There have been significant changes in regulatory requirements as well. Yet, the approaches and tactics used to conduct post approval studies has remained virtually the same. With an evolving role and complexities of post approval studies, and a shift toward patient centric outcomes, it’s time to rethink the cookie cutter approach and implement innovative, technology-driven research to increase efficiencies, maximize outcomes, and address data collection from the patient perspective.

Alpha Clinical Systems10:10 Coffee Break in the Exhibit Hall



11:10 Chairperson’s Remarks

11:15 Leveraging the Patient Perspective to Uncover What Matters Most

Megan_Leone-PerkinsMegan Leone-Perkins, Ph.D., Chief Scientist, Corporate, HealthiVibe, LLC

The sterile world of research can sometimes leave even the most experienced and educated scientists operating inside of a vacuum. The clinical knowledge of a disease does not always reveal issues that matter most from a patient perspective and collecting standard PRO data during a Phase III study does not always reveal outcomes that are meaningful to patients. Harnessing real-world insights from those who live with a disease or condition can result in unexpected outcomes and newly defined endpoints. Engaging in a real world research effort collecting insights directly from patients can help sponsors better understand patient perceptions.

11:40 Developing Quality Tools for Patient-Reported Prospective Data Collection

Mark_PriceMark A. Price, MA, MEd, Senior Director, Surveys and Observational Studies, RTI Health Solutions

In designing patient-centered prospective observational research, it is critical to take the necessary time to develop solid survey and diary tools. Adequate pre-planning and cognitive testing (including input directly from patients at the design stage) ensures that quality data meeting a study's objectives emerge during implementation.

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

Matthew PageMatthew Page, Epidemiologist, Biometrics, Medpace

Alpha Clinical Systems12:50 Coffee and Dessert in the Exhibit Hall


1:30 Close of Conference

Suggested Event Package*

2:00 – 5:30 pmSC2: Visual Analytics - An Immersive Workshop for Clinical Researchers in Pharma & Device Trials - View Details

February 23-24: Managing Late Stage Research and Observational Studies Conference

February 24-25: Leveraging Existing Data for Clinical and Observational Research Conference

* Separate registration required