2018 Archived Content

Cambridge Healthtech Institute’s 7th Annual

Late Stage Research and Observational Studies:

Novel Approaches and Data Sources for Post-Approval Research
February 13-14, 2018 | Hyatt Regency Orlando | Orlando, FL

Non-interventional studies are an integral part of 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 regulatory agencies, payers, health care management organizations, formulary inclusion decision makers, healthcare professionals, and patients. Cambridge Healthtech Institute’s 7th Annual Late Stage Research and Observational Studies: Novel Approaches and Data Sources for Post-Approval Research 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 obtained data to pivotal business and medical decisions. Similarities and differences between clinical and observational studies will be addressed by the top industry experts.

Monday, February 12

9:00 am - 7:30 pm Registration Open

5:00 - 6:15 pm Pre-Conference Plenary Keynote Panel

6:308:30 pm SCOPE’s Kick-Off Networking Happy Hour on the Garden Terrace Hosted by CHI, DrugDev, Exostar, & Praxis

8:30 Close of Day

Tuesday, February 13

7:15 am Registration Open and Morning Coffee

8:20 Opening Plenary Keynotes

9:45 Grand Opening Coffee Break in the Exhibit Hall


10:45 Chairperson’s Remarks

Cathy Critchlow, Ph.D., Vice President, Center for Observational Research, Amgen

10:50 Evolving Role for Real-World Evidence in Facilitating Regulatory, Payer and Provider Decision-Making

Cathy_CritchlowCathy Critchlow, Ph.D., Vice President, Center for Observational Research, Amgen

Increasing use of real world evidence (RWE) to tailor health care decision-making to patients in clinical practice complements evidence obtained from the carefully selected patients enrolled in randomized clinical trials. While growing data availability and sophistication of analytic tools have transformed evidence generation, challenges impeding full realization of benefit from RWE remain. Collectively addressing these challenges and advancing suitable use cases will help guide and enable appropriate impact of RWE.

11:15 Non-Interventional Studies and Pragmatic Clinical Trials to Support Product Value

Christopher_ChinnChristopher Chinn, Head of RWE Strategy for Market Access Health Economics and Value Assessment, Sanofi

Real World Evidence can be defined to include both non-interventional studies - aka observational studies or registry studies – and pragmatic clinical trials. These can provide evidence of interest to payers and inform clinical guidelines. The design and delivery of such studies draws on skills required for RCTs, but raise new challenges. Study teams should be prepared to find appropriate solutions.

11:40 Combining Safety, Efficacy and Pharmacoeconomics Endpoints

Durgesh Bhandary, Senior Director, HEOR, AstraZeneca

This talk will discuss the strategy and logistics of designing and executing an observational study that would incorporate multiple end points to serve major stakeholders such as epidemiologists, drug safety researchers, sales and marketing, treating physicians. 

IQVIA12:05 pm Next Generation Real-World Evidence: Moving the Industry Standard Forwards

Louise Parmenter, PhD, Vice President, Global Head, Scientific Affairs, Real-World Insight Solutions, IQVIA

New Approaches to Generate RWE. Using Data and Insights to Improve Predictability of Delivery.

12:30 Session Break

12:40 Luncheon Presentation: Value of Natural History Studies Throughout the Product Life Cycle

Haley_KaplowitzHaley Kaplowitz, Ph.D., Executive Director, Safety, Epidemiology, Registries and Risk Management, UBC: An Express Scripts Company

Having an in-depth knowledge of the disease or condition for which is drug is being developed seems fundamental, yet the natural history of the disease may not be fully known prior to or during drug development. Further, once a new product enters the marketplace, it is important to characterize the impact on disease natural history. Successful product development and marketing are aided by the collection of natural history data.

1:20 Coffee and Dessert Break in the Exhibit Hall


2:00 Chairperson’s Remarks

Ginger Spitzer, Executive Director, Foundation of Sarcoidosis Research

2:05 Co-PRESENTATION: Value of Conducting Feasibility Studies in Observational Research

Mark_PriceMark Price, Senior Director, Surveys and Observational Studies, RTI Health Solutions

David_RichardsonDavid Richardson, Project Manager, Surveys and Observational Studies, RTI Health Solutions

This presentation will provide justification and case study examples to demonstrate why it is important to spend time and resources up front to conduct feasibility assessments in noninterventional studies and the range of feasibility approaches that could be performed to get the most out of the implementation phase.

2:30 CO-PRESENTATION: Collaborative Approach to Real World Data Collection

Ginger_SpitzerGinger Spitzer, Executive Director, Foundation of Sarcoidosis Research

Winnie_NelsonWinnie Nelson, Pharm.D., MS, MBA, Senior Director HEOR, Mallinckrodt Pharmaceuticals

This presentation will focus on the valuable role of non-profit disease research foundations in securing and managing real world data and collaboration with industry to access the data. The two-part presentation will include review of methods such as patient registries, clinical site networks, collaboration, wearables, and other techniques, and will feature the perspective of industry partners as both collaborators and first-line collectors of data. The “neutral third party” status of the non-profit organizations can enable industry to navigate more easily the issues in logistics to get direct real world data.

2:55 A Crawl, Walk, Run Strategy towards Virtual Studies in Real World Research

Adam Halbridge, Vice President, Business Development, Parallel6, a PRA Health Sciences Company

This presentation will address new and exciting developments in planning and executing real world research studies virtually. Attendees will learn about the reality of conducting virtual studies on a global scale and the role mobile technology can have to optimize recruitment, engagement and retention of patients, while driving better data, decisions and outcomes more efficiently, and at a significant reduction in costs.

3:20 PANEL DISCUSSION: Meeting the Evidentiary Needs of Multiple Stakeholders by Better Non-Interventional Studies

Cathy_CritchlowCathy Critchlow, Ph.D., Vice President, Center for Observational Research, Amgen

Panelists: Ginger Spitzer, Executive Director, Foundation of Sarcoidosis Research

Juliane Mills, Scientific Affairs Director, Real World Solutions, PRA Health Sciences

Christopher Chinn, Head of RWE Strategy for Market Access Health Economics and Value Assessment, Sanofi

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 utilizations 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)?


3:50 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.

5:00 Welcome Reception in the Exhibit Hall

6:30 Close of Day

Wednesday, February 14

7:15 am Registration Open

7:45 Breakfast Presentation: The Next Generation of Site Payments: Technology Do's and Don'ts

Stuart Thiede, President, Payments, DrugDev (An IQVIA Company)

Ineffective site payment processes can irreparably damage the site/sponsor relationship, resulting in the dreaded “one and done” mentality for many sites. However, leveraging a purpose-built technology solution can remove such concerns by ensuring reliable cycle times, efficient processing, real-time reporting, and transparency, all of which drive site satisfaction and simplify the lives of study teams. This session will highlight best practice technology and process models to ensure you achieve your site payment objectives.

8:15 Session Break


8:25 Chairperson’s Remarks

Haley Kaplowitz, Ph.D., Executive Director, Safety, Epidemiology, Registries and Risk Management, UBC: An Express Scripts Company

8:30 CASE STUDY: Leveraging Real World Data in Observational Research to Address Safety Risks

Younus Muhammad, Director, Epidemiology, Worldwide Safety and Regulatory, Pfizer

Observational data from real-world setting are increasingly being used to assess safety risks with pharmaceutical products. This presentation will describe how real world data from routine clinical practice were used to address a safety risk in a special patient population including details around feasibility assessment, regulatory agency interactions, study methods and results, and impact on the product label.

9:00 CO-PRESENTATION: The Use of Real-World Data and Evidence in Clinical Research and Post-Marketing Safety Monitoring

Mary_Jo_LambertiMary Jo Lamberti, Ph.D., Senior Research Fellow, Tufts CSDD, Tufts University

Jill AbellJill Abell, MPH, Ph.D., Team Leader, Real World Evidence, Janssen

Tufts CSDD has recently conducted a study of 30 biopharmaceutical companies to examine insights on the industry’s response to this opportunity including current and planned uses of real-world data, operational issues and return on investment/performance areas impacted by real-world data use. Significant challenges are identified as well as strategies and practices that impact return on investment or performance.

9:40 Real World Evidence: Separating the Hype from the Promise

Charles_MankinCharles Makin, Vice President & Global Head, Late Phase Research, Commercialisation & Outcomes, ICON

Increasing availability of RWE has created justifiable excitement, accompanied by confusion about its true meaning and implications for stakeholders. While RWE is neither the starting point nor the solution, it is a critical component of the value demonstration toolkit. It is not the data that counts, but the insights gained from it. Join us for best practices to ensure you are asking the right questions and using data to guide the pursuit of answers.

10:10 Coffee Break in the Exhibit Hall


11:10 Chairperson’s Remarks

Maria Harrison, Vice President, Real World Solutions, PRA Health Sciences

11:15 Incorporating the Patient Perspective into Drug Development/Real-World Evidence

Emily_FreemanEmily Freeman, Ph.D., Director,HEOR- Patient Reported Outcomes, Abbvie

Patient engagement is a key aspect to improving health outcomes and contextualizing real-world evidence within the pharmaceutical industry. It is imperative to incorporating the patient perspective into drug development beyond patient reported outcomes and subsequent RWE strategies. This session strategizes how to incorporate the patient perspective to develop patient focused drug development activities utilizing concepts from the social/behavioral sciences that focuses on patient activation, patient measurement, and shared-treatment decision making.

11:40 CO-PRESENTATION: Using Exit Interviews with Participants in Randomized Controlled Trials to Collect “The Rest of the Story”

Carla_RomanoCarla Romano, MS, Executive Director, Patient Centered Outcomes Assessment, RTI Health Solutions

Sandy Lewis, BSN, Director, Patient Centered Outcomes Assessment, RTI Health Solutions

This presentation will explore the benefits of conducting qualitative interviews with patients as they complete participation in RCTs. Important information can be collected on safety signals, clinically meaningful change on PRO measures, understanding reasons for drop out, and bringing the patient voice into clinical trials.

12:05 pm Session Break

Syneos Health12:10 pm Bridging Luncheon Presentation: Achieving Evidentiary Equilibrium

David_ThompsonDavid Thompson, Ph.D., Senior Vice President, Real-World & Late Phase, Syneos Health

Achieving Evidentiary Equilibrium - Generating the Right Evidence for the Right Stakeholders at the Right Time Throughout the Clinical/Commercial Continuum

12:50 Coffee and Dessert Break in the Exhibit Hall

1:30 Close of Conference

2018 SCOPE Conference at a Glance


For questions or suggestions about the meeting, contact:

Marina_62x62Marina Filshtinsky, M.D.
Senior Director, Conferences
Cambridge Healthtech Institute (CHI)
T: (+1) 781.972.5496
E: mfilshtinsky@healthtech.com

For partnering and sponsorship information, contact:

IlanaIlana Quigley
Senior Manager, Business Development
Cambridge Healthtech Institute (CHI)
T: (+1) 781.972.5457
E: iquigley@healthtech.com

For media and association partnerships, contact:

RichRich Handy
Senior Director, Marketing
Cambridge Healthtech Institute (CHI)
T: (+1) 781.972.5456
E: rhandy@healthtech.com