Cambridge Healthtech Institute’s Fourth Annual 

Managing Late Stage Research, Observational Studies and Registries: 

Overcoming Operational Challenges
February 24-25, 2015 | Hyatt Regency Orlando | Orlando, FL 

Non-interventional studies are an integral part of clinical development programs and product development plans. Product benefit risk profile, comparative effectiveness data, and health economic evidences obtained from non-interventional studies are essential for multiple stakeholders, including regulatory agencies, payers, health care management organizations, formulary inclusion decision makers, healthcare professionals, as well as patients. Cambridge Healthtech Institute’s Fourth Annual Managing Late Stage Research, Observational Studies and Registries conference is designed to facilitate knowledge exchange around all aspects of observational research, from design of non-interventional studies and their management to application of the obtained data to business and stakeholder decisions.

Tuesday | Wednesday | Track Brochure | Full Event Brochure
Stay on for Part 2: Pharmacovigilance 

Tuesday, February 24

7:30 am Registration and Morning Coffee

8:25 Opening Plenary Keynotes

9:45 Grand Opening Coffee Break in the Exhibit Hall


10:45 Chairperson’s Remarks

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

10:50 KEYNOTE PRESENTATION: Designing, Managing and Applying Observational Studies and Registries. 

Charles BarrCharles Barr, M.D., Head, Evidence Science & Innovation, Group Medical Director, US Medical Affairs, Genentech, Inc.


11:15 Collaborating with a regulator: EMA-commissioned risk-minimization studies 

VeraEhrensteinVera Ehrenstein, MPH, DSc, Associate Professor, Department of Clinical Epidemiology, Institute of Clinical Medicine, Aarhus University

In 2006, the European Medicines Agency (EMA) established the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) to facilitate assessment of drug utilization and safety in Europe. Collaboration with the EMA will be illustrated from the vantage point of an ENCePP center. Topics will include preparing a competitive tender, formulating the research question, finding collaborators, and role of EMA. Guidelines for risk minimization studies will be reviewed, and risk minimization studies of antidiabetic agents will be used as examples. 

11:40 Observational Data Collection Studies: Considerations to Minimize Bias

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

Bias is an error in design or execution of a study which produces results that are consistently distorted in one direction because of nonrandom factors. This presentation will provide tips to consider to help minimize the impact of confounding, selection bias or information bias when designing observational data collection studies. By applying proper epidemiologic methods for sampling, analysis and design observational study results can provide robust real-world data that cannot be obtained through the typical clinical trial.

12:05 pm Evolution of Physician and Patient Retention in Post-Marketing and Real-World Research

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

The role of the patient in clinical research and the use of prediction and prevention methods to optimize participation has transformed over the years. As patients are more actively engaged in their healthcare, we see a similar trend with participation in clinical research.  However, there are unique challenges to maintain patient engagement for observational research where treatment is not provided by the study and standard-of-care office visit frequency can vary.  This presentation will highlight strategies to enhance active physician and patient involvement in non-interventional research and present several real world case studies.

UBC12:35 LUNCHEON CO-PRESENTATION: Fido Fights Cancer: Lessons Learned Recruiting Four-Legged Patients in a Registry

Peterson_JanetJanet Peterson, Director, Patient & Physician Services, UBC

Erin SearfossErin Searfoss, Senior Project Manager, Morris Animal Foundation

Each unique patient population requires a customized approach to clinical trial recruitment. As learned during a first-of-its-kind prospective cohort study in Golden Retrievers, recruiting Man’s Best Friend presented its own challenges. Together with The Morris Animal Foundation, UBC discovered that sometimes the best approach to canine recruitment is the grassroots approach. Attendees will benefit from lessons learned from enrolling this very unique patient population and be able to apply these strategies across registries, especially those in rare disease communities.

1:15 Session Break


1:25 Chairperson’s Remarks
Kahn_Steven(2)Steven Kahn, Ph.D., Vice President, Operations Americas, Real World Evidence, Mapi



1:30 Increasing Late Stage Patient Recruitment through Patient-Centered Technologies

Nariman NasserNariman Nasser, CCRP, Digital Strategist, Patient Recruitment & Retention, Roche

Making observational studies more accessible to patients is key to increasing recruitment and retention. In this session we will examine opportunities to better align the study experience to the daily lives of our patients.


1:55 Next-Generation Oncology Registry Management, Big Data Considerations and Applications

Jomol Mathew, Ph.D., Director, Clinical and Translational Informatics, IS, Dana-Farber Cancer Institute

Patient data registries that systematically gather longitudinal clinical data that includes family history, exposure/risk factors, health status, diseases, treatments and outcomes along with genomic/molecular profiles are important in conducting correlative studies and establishing diagnostic and prognostic markers of diseases and treatment outcomes. We will be presenting our experiences and perspectives on systems that we have developed for oncology registries at the Dana Farber Cancer Institute.

2:20 Optimizing Post-Marketing Research – It is Time to Change the Way We do Post-Marketing Research

Nayan T. Nanavati, President, Late Phase Division, BioClinica

There has been a surge in post-marketing clinical studies. Pharma and bio technology industries are conducting more studies with increased complexity in design which often translates into increase costs in conducting post-marketing studies; voluntarily or to satisfy regulatory mandate. Despite this phenomenal growth, our approaches in designing and conducting these complex and expensive studies have been far more traditional, lacks innovation and results in less than optimal outcomes.  Although post-marketing studies must be conducted within the established regulatory framework and on a sound scientific foundation, there is room for us to re-evaluate how we conduct studies for better outcomes.  Harnessing patient power, creating and integrating new technologies and data bases, and better use of social media are a few approaches that may minimize the cost of conducting such studies while addressing challenges associated with post-marketing studies and maximizing its informational value.

2:45 Optimizing Quality and Risk in Managing Observational Research
Khoury_HadyHady Khoury, Vice President, Global Head, Research & Alliance Services, Peri-Approval & Observational Research, Commercialization & Outcomes, ICON

Non-interventional study designs are common for registries and other studies looking to obtain real world data. These studies frequently engage clinical practitioners who are not trial experts and may have limited budgets to support onsite study monitoring. As a result, they must heavily rely on remote site management strategies to detect site-level and systematic operational trends. In this session, we present a variety of quality by design, risk management as well as hub-based centralized site management strategies that can comprehensively provide an optimal cost effective outcome for observational research.  



3:00 Refreshment Break in the Exhibit Hall

4:00 Find Your Table and Meet Your Moderator

4:05 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.

View Detailed Interactive Breakout Discussion Groups 

5:00 Welcome Reception in the Exhibit Hall

6:15 Close of Day

Tuesday | Wednesday | Track Brochure | Full Event Brochure
Stay on for Part 2: Pharmacovigilance 

Wednesday, February 25

7:30 am Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee

8:25 Plenary Keynote Session

9:45 Coffee Break in the Exhibit Hall


10:45 Chairperson’s Remarks

Catherine (Koepper) Connelly, Director, Global Registry Operations, Genzyme, a Sanofi Company

10:50 Using Observational Research and Registries to Support Drug Development across the Lifecycle: Pfizer’s Experience 

Rachel SobelRachel Sobel, Dr.PH., Senior Director, Epidemiology - Team Lead Global Innovative Pharmaceuticals, Pfizer, Inc.

This talk will describe how epidemiologists at Pfizer use observational research and registries to support drug development across the lifecycle, with a focus on how they can be used to better understand the patient populations and safety profiles of drugs to ensure an appropriate benefit-risk balance and enable faster drug approvals and/or maintain medicine licensures. The presentation will cover some of the major regulatory frameworks and provide examples utilized at Pfizer.

11:15 Registry Scope and Stakeholders: Matching Scope Feasibility With Stakeholder Needs and Expectations

Cathy KoepperCatherine (Koepper) Connelly, Director, Global Registry Operations, Genzyme, a Sanofi Company

Registry research continues to proliferate. Registries offer important research value, and associated stakeholders are often associated with multiple registry stages, such as planning and development, patient enrollment and data collection, and data reporting and publishing. While the stakeholders may be the same across stages, their needs and expectations may evolve. We will investigae how to actively support various stakeholders throughout the lifecycle of a registry, while also actively managing overall expectations and program scope.

11:40 CO-PRESENTATION: Value of the Patient Voice: Role of PROs in Pricing and Reimbursement Strategy

Kelly HollisKelly Hollis, MBA, Global Head, Surveys and Observational Studies, RTI Health Solutions


Mark PriceMark Price, MA, MEd, Senior Director, Surveys and Observational Studies, RTI Health Solutions

Patient-reported outcomes (PROs) are an accepted and often actively solicited source of evidence in evaluating and approving pharmaceutical interventions based on their clinical efficacy. Likewise, real world data including PROs plays an important role in the evaluation of the costs and benefits of new technologies. This presentation will include case studies and provide discussion on leveraging the patient voice to support pricing and reimbursement decision-making.

Mapi12:05 Projections of Patient Enrollment in the Post Marketing Environment
Kahn_Steven(2)Steven Kahn, Ph.D., Vice President, Operations Americas, Real World Evidence, Mapi
Post-approval observational studies and registries are different than Phase III programs; this session discusses the implications of applying inappropriate patient and site enrollment projections algorithms: Clinical interventional patient and site projection algorithms from Phase III studies should not be deployed for Phase IV observational programs. While projecting site and patient enrollment in Phase III Randomized Controlled Trials is a common activity and algorithms that are utilized are fairly well established by therapeutic area, these methods will lead to failed expectations in post approval research programs. In this session attendees will gain an understanding of how the different environments impact the projections, such as the influence of third party payers, market uptake, motivations of stakeholders, and real clinical practice. 

12:35 BRIDGING LUNCHEON CO-PRESENTATION: Engage and Retain Patients in Long-Term Observational Studies

Smurzynski_MarleneMarlene Smurzynski, Ph.D., MSPH, Epidemiologist, Quintiles




Reites_JohnJohn Reites, Senior Director, Offer and Product Development, Health Engagement & Communications, Quintiles







Traditionally, real-world and late phase studies require sites to enroll, engage and retain patients and collect and record patient reported outcomes (PRO), which can be burdensome to both sites and patients. Overtime, sites and patients may lose motivation to participate, contributing to high patient dropout rates, increased study costs and site dissatisfaction. This session will focus on innovative approaches for effectively engaging and retaining patients in long-term studies, such as: identifying design and operational considerations with conducting long-term observational research, understanding site and patient retention challenges, and examining engagement strategies and opportunities for improving retention and compliance.

1:15 Session Break/Close of Conference

Tuesday | Wednesday | Track Brochure | Full Event Brochure
Stay on for Part 2: Pharmacovigilance 

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