Cambridge Healthtech Institute's 7th Annual

Late Stage Research and Observational Studies:
( 後期段階の研究と観察研究 )

Novel Approaches and Data Sources for Post-Approval Research

2018年2月13-14日 | Hyatt Regency Orlando | フロリダ州オーランド




Monday, February 12

9:00 am - 7:30 pm Registration Open

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

6:30 - 8:30 pm SCOPE's Kick-Off Networking Happy Hour on the Garden Terrace Hosted by CHI, DrugDev, & 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.

12:05 pm Presentation to be Announced

12:30 Session Break

12:40 Luncheon Presentation to be Announced

Annette Stemhagen, Dr.P.H., FISPE, Senior Vice President, Safety, Epidemiology, Registries and Risk Management, UBC: An Express Scripts Company

1:20 Coffee and Dessert Break in the Exhibit Hall


2:00 Chairperson's Remarks

To be Announced

2:05 Value of Conducting Feasibility Studies in Observational Research

Mark_PriceMark Price, Senior Director, 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

Kathleen_MandziukKathleen Mandziuk, MPH, RN, Executive Director, Scientific Affairs, Real World Solutions, PRA Health Sciences

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: Speakers of the Day

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: A New Way to Pay: SaaS Technology and Outsourcing How You Need It

Stuart Thiede, President, Payments, DrugDev

8:15 Session Break


8:25 Chairperson's Remarks

Annette Stemhagen, Dr.P.H., FISPE, Senior Vice President, Safety, Epidemiology, Registries and Risk Management, UBC: An Express Scripts Company

8:30 Pragmatic Clinical Trials: Bridging the Gap between Research and Care

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

Clinical trial findings that lead widespread change in health care is relatively slow. Since many clinical trials are not relevant to clinical practice, it is difficult to translate results to the real world clinical practice. Tens of thousands clinical trials published each year, yet systematic reviews consistently find that we don't have enough evidence to effectively inform the clinical decisions. Clinical research is more than just traditional RCTs. Pragmatic research is designed with input from health systems and produces evidence that can be readily used in improve care. By engaging health systems, providers, and patients as partners, pragmatic research accelerates the integration of research, policy, and practice.

8:55 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.

9:20 The Use of Real-World Data and Evidence in Clinical Research and Post-Marketing Safety Monitoring

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

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.

9:45 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

Emily Freeman, Ph.D., Director, Risk Management Sciences, Abbvie

11:15 Incorporating the Patient Perspective into Pharmacovigilance

Emily_FreemanEmily Freeman, Ph.D., Director, Risk Management Sciences, Abbvie

Patient engagement is a key aspect to improving health outcomes and effectiveness of risk management activities within the pharmaceutical industry. It is imperative to incorporating the patient perspective into pharmacovigilance and subsequent risk management strategies. This session strategizes how to incorporate the patient perspective to develop patient focused risk minimization activities utilizing concepts from the social/behavioral sciences that focuses on patient activation, patient measurement, and shared-treatment decision making.

11:40 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

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

12:10 Bridging Luncheon Presentation: Achieving Evidentiary Equilibrium

David Thompson, Ph.D., Senior Vice President, Real-World & Late Phase, INC Research/inVentiv 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

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In Memory of Gerald "Jerry" Matczak
Matczak Jerry

Gerald "Jerry" Matczak, lead consultant in clinical innovation at Eli Lilly & Co., was a rare breed in the pharmaceutical world, someone who not only embraced social media, but also listened to activist patients. He has been a part of the SCOPE conference since its inception and won the Patient Engagement Award on Wednesday, the day before his passing. Matczak died suddenly on Feb. 2 at age 54. We will all miss him.

- Cambridge Healthtech Institute (CHI)