Cambridge Healthtech Institute's

Risk-Based Monitoring

April 7-8, 2020


Ensuring quality from the outset of a clinical trial leads to higher quality, lower risk clinical trials. The establishment of appropriate risk assessment policies and clinical quality management systems lays the foundation for successful risk-based monitoring (RBM). Learn more at CHI's 9th Annual Clinical Trial Innovation Summit.


Final Agenda


2:30-5:30 pm Pre-Conference Workshops

Workshop 2: Digital Clinical Development: Tutorial, Case Studies, and Future Readiness

Workshop 3: Inspection Readiness in Changing Global Regulatory Environment

*Workshops are included in your registration. However, please RSVP to reserve your seat.


8:15 am Registration and Morning Coffee

Real World Data, Artificial Intelligence and Analytics to Reshape Clinical Development

9:00 Organizer’s Opening Remarks

Micah Lieberman, Executive Director, Conferences, Cambridge Healthtech Institute (CHI)

9:05 Chairperson’s Remarks (Sponsorship Opportunity Available)

9:10 Digitalization of Clinical Trials: Breakthroughs in Technologies and Data

Peter BergethonPeter Bergethon, MD, Vice President, Head of Digital and Quantitative Medicine, Biogen

Medicine as systems science characterizes the health state by measurable properties. Changes in state over time (dynamics) define trajectories through growth, health and disease. Dynamic transitions define trajectories between health, illness and therapeutic interventions. Capturing time dependence is the power of digital technology. Herein lies the power of digital medicine. We will review specific cases showing how this paradigm is transforming our clinical trial landscape.

9:30 CO-PRESENTATION: The Role of Real-World Data in Creating New Pathways to Biopharma Industry Transformation

Alan LouieAlan Louie, PhD, Research Director, Life Sciences, IDC Health Insights

Charles MakinCharles Makin, Global Head, Real World Evidence Strategy, Biogen

In parallel to other industry best practices, the biopharmaceutical industry is embracing digital transformation as it seeks to better leverage data across the life science ecosystem. In conjunction with these efforts, increasingly available real-world data (RWD) promises to bring new patient-specific data and insights to the industry, data which bring researchers closer to understanding patient-level treatment responses while also opening new channels to engagement. With strong support from regulators, real-world evidence offers significant potential to accelerate new drug discovery and development, improve process efficiencies, and improve patient outcomes over the near term.

9:50 AI in Human Health – Connecting Data Modules from Discovery Biology to Clinical Development

Rangaprasad SarangarajanRangaprasad Sarangarajan, PhD, CSO & Senior Vice President, Clinical and Translational Sciences, Research & Development, BERG

Drug development programs in companies are predicated on biological insights supporting a hypothesis of target and its role in disease etiology. This presentation will focus on the data modules essential for linking discovery biology to early/late stage clinical development and the leveraging of AI in the integration of the various dataflow to make data-driven informed decisions and ensure success. It will outline how to utilize data, AI analytics with RWD-based support for planning drug development to propel products all the way through the clinic.

10:10 Grand Opening Coffee Break in the Exhibit Hall (Sponsorship Opportunity Available)


11:10 Chairperson’s Remarks (Sponsorship Opportunity Available)

11:20 FEATURED PRESENTATION: Developing and Driving a Robust Clinical Development Quality Risk Management Program

Jonathan RoweJonathan Rowe, PhD, Associate Principal, ZS Associates

ICH E6 R2 calls for the establishment of a system to manage quality throughout all stages of the trial process. This presentation will describe elements of a protocol to CSR quality management process that allows sponsors/CROs to understand risks to GCP and proactively mitigate those risks.

11:45 Challenges and Solutions for Delivering High Quality Consistent Data for ICHE6 (R2)/RBM Analytics

Muhammad AliMuhammad Ali, Central Monitoring, Pfizer

This presentation will discuss a intro into risk-based monitoring (RBM), as well as why traditional SDLC methodology is challenging, implementation approaches, conflicting priorities and perspectives, change agent networks, innovations (ex. application of AI/ML), and real-world scenarios.

12:10 Is Your Centralized/Onsite Site Monitoring Program Being Implemented per ICH E6(R2)? How Do You Know?

Linda SullivanLinda Sullivan, Co-Founder & Executive Director, WCG - Metrics Champion Consortium

The revision to ICH E6 has spawned a transformative approach to monitoring safety and data quality at investigational sites. As new centralized/remote/site monitoring models are being implemented, organizations are considering the perplexing question about how to oversee and manage these models. For example, what should be measured and reviewed to ensure the process is being implemented according to the plan? What metrics could be used to provide insights about the process and future process improvements? Members of an industry consortium are developing consensus-based metrics by considering the process steps, critical success factors, key performance questions, and by using the process analysis technique Failure Mode and Effects Analysis (FMEA). This session will provide insights on the progress to date and plans to obtain additional industry feedback.

12:35 pm Transition to Luncheon Presentation

12:40 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:25 Coffee & Dessert Break in the Exhibit Hall (Sponsorship Opportunity Available)


2:00 Chairperson’s Remarks (Sponsorship Opportunity Available)

2:10 Building a Data Sciences Team for Risk-Based Study Execution

Amy NeubauerAmy Neubauer, Director, Data Quality Oversight, Alkermes

When looking to create a team to manage risk-based study execution, what are the skills and experience needed? How can roles and responsibilities be managed across the team to cover everything from performing risk assessments, KRI selection and programming, issue detection/action/escalation, QTL selection and tracking, to documentation of actions? We will share our experience and best practices in building a data sciences team and managing risk-based study execution processes.

2:35 Proactive Risk Assessment for the Development of a Robust Integrated Quality Management Plan

Sheri KussSheri Kuss, Clinical Quality Lead, Clinical Development Quality, Global Product Development, Pfizer, Inc.

Development of a risk management plan should be more than a check box exercise.  It should be a prospective integrated risk plan that connects the dots between protocol development, risk-based monitoring and data review.  A case study will be reviewed during this presentation.

3:00 Review of ICH E8 R1 – The Developing Regulatory Direction and How to Address

Andy LawtonAndy Lawton, Director & Consultant, Risk Based Approach Ltd.

The release of ICH E8 R1 (draft) has cemented the direction that the Regulatory bodies want us to follow, that is towards a Quality by Design process, rather than “Quality by Accident” as some regulators have expressed. A background of the Regulatory presentations, guidances and discussions at meetings will be brought together to give their path of attack initially via RBM and now cemented in a compelling need for QbD. This presentation will take you stepwise through the following topics

  • Regulatory background from “crumbs on the ground” to clear direction
  • From early presentations from 2000 to recent (2019) Regulatory Guidance
  • What is Quality by Design
  • How can QbD principals be incorporated into RBM with an example

3:25 Transition to Keynote

Why Do Trials Succeed and Why do they Fail? Insights from Industry and Academia

3:35 Organizer’s Opening Remarks

Marina Filshtinsky, MD, Executive Director, Conferences, Cambridge Healthtech Institute

3:40 Chairperson’s Remarks (Sponsorship Opportunity Available)

3:45 BEST CLI Trial: Lessons Learned From Execution Of A Complex Multicenter RCT

Alik FarberAlik Farber, MD, Chief, Division of Vascular and Endovascular Surgery, Associate Chair for Clinical Operations, Department of Surgery, Boston Medical Center; Professor of Surgery and Radiology, Boston University School of Medicine

The BEST-CLI trial is the largest RCT ever undertaken to evaluate strategies for treatment of critical limb ischemia (CLI). This study is an international, multispecialty, prospective, multicenter, randomized comparative effectiveness trial of endovascular versus open surgical revascularization in patients with CLI who are candidates for both procedures. The study has been performed at 134 sites and has enrolled 1843 patients. Its execution had had multiple obstacles and challenges that the trial leadership team has successfully navigated.

4:15 CO-PRESENTATION: What Causes Studies to Fail? Understanding Causal Drivers of Operational Success Using Machine Learning

Marcy KravetMarcy Kravet, BS, MBA, Head, Operational Design Center (ODC), Global Clinical Operations, EMD Serono, Inc., an affiliate of Merck KGaA, Darmstadt, Germany

Sylvia MareckiSylvia Marecki, PhD, Design Analyst, Operational Design Center (ODC), Global Clinical Operations, EMD Serono, Inc., an affiliate of Merck KGaA, Darmstadt, Germany

It is currently unknown what drives successful study enrollment despite extensive analysis conducted across the industry, which largely has focused on correlations. The Operational Design Center at EMD Serono has leveraged causal machine learning to analyze hundreds of variables across thousands of clinical trials with the objective of identifying causal drivers of enrollment success, which can be optimized to conduct faster, less expensive trials. Early insights will be shared.

4:45 Welcome Reception in the Exhibit Hall (Sponsorship Opportunity Available)

5:45 End of Day


7:45 am Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee


8:25 Chairperson’s Remarks (Sponsorship Opportunity Available)

8:35 CO-PRESENTATION: Technology Integration- Delivering Real Time Data Access and Oversight

Amy KlawitterAmy Klawitter, Senior Manager, Clinical Management, Otsuka Pharmaceutical Development & Commercialization, Inc.

Lucien AgnantLucien Agnant, Technology Integration Specialist, Clinical Management, Otsuka Pharmaceutical Development & Commercialization, Inc.

By integrating tools such as eSource, eConsent and IRT, sites will experience efficiencies with data entry and sponsors will benefit from having access to near real-time data. The presentation will provide a brief overview of how Otsuka has worked with vendors to integrate systems, the lessons learned, and valuable outcomes.

9:05 Talk to Be Announced

9:35 Solving Protocol Deviations Challenges: from Protocol Creation to CSR

Laura GaluchieLaura Galuchie, TransCelerate Program Lead, Oversight Committee, TransCelerate Biopharma Inc.

  • Clarify the definition of “protocol deviation”
  • Support classification of “important” and “non-important” protocol deviations
  • A toolkit to support multiple processes associated with protocol deviation management from identification through reporting, including several feedback loops

9:50 Coffee Break in the Exhibit Hall (Sponsorship Opportunity Available)


10:40 Chairperson’s Remarks (Sponsorship Opportunity Available)

10:50 PANEL DISCUSSION: Unicorn or Racehorse? Lessons from the Trenches in RBM


Laurie Halloran, CEO, Halloran Consulting Group, Inc.


Catalina OrtizCatalina Ortiz, Principal Business Operations Analyst, Vertex Pharmaceuticals

Chandler_DianeDiane Thornton Chandler, BSN, RBM/Centralized Monitoring Lead, Medtronic

Kevin DouglassKevin Douglass, Associate Director, Process Excellence & Risk Management, Daiichi Sankyo, Inc


The FDA’s guidance on Risk Based Monitoring has been in use since August 2013, and the ICH E6 r2 has recently pushed the issue of using risk-based approaches to streamline the overly complex processes that cost life sciences sponsors so much and add so little. But, given that we tend to wrestle with “the way it’s always been done” – how has the adoption been progressing in both early adopters and companies new to the more expanded use of technology to enhance their adoption of risk-based approaches. On our panel, we will talk practical applications, challenges and lessons learned from companies who’ve managed to break through the promise to reality. They have found their unicorns, and they will share what they did to get there. A few of the key areas for discussion will include:

  • At what point does trial design need to be factored into a risk-based approach?
  • How to get everyone on the same page in start-up: selection of sites and vendors, training of the entire team.
  • The centralized monitoring role: What have they learned through the set up and management of the process and role?
  • What did they wish they know before embarking on this path that will help others?  
  • What is the overall impact to time, cost and quality of the paradigm shift?


11:40 Transition to Breakout Discussions


11:45 Find Your Table and Meet Your Moderator

11:50 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. Bring your pharma, biotech, CRO, site, hospital or patient perspective to each of the discussions below. 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.

12:30 pm Community Networking Lunch in the Exhibit Hall (Sponsorship Opportunity Available)

1:40 Transition to Plenary Keynote

Understanding the Patient Journey to Improve Trials

1:45 Organizer’s Opening Remarks

Kaitlin Searfoss Kelleher, Senior Conference Director, Cambridge Healthtech Institute

1:50 Chairperson’s Remarks (Sponsorship Opportunity Available)

1:55 The Journey from Patient to Advocate to Leader: Changing the Clinical Research Game

Rob LongRob Long, Executive Director, Uplifting Athletes

I will talk about my journey from Division I football and top NFL prospect to cancer patient. My experience of undergoing chemo and radiation and fighting through a battle with brain cancer has enabled me to leverage my story to make a difference in the lives of those affected by rare diseases. The attendees will gain insights into what it means to be a volunteer at the front lines of oncology research and clinical trials. New approaches to partnering with advocates, disease communities and young researchers will be shared. In addition, we will discuss the future of rare disease research and outreach and illustrate where industry and the patient community can go together in collaboration. Read an article published about Rob Long's journey, Ex-Syracuse punter Rob Long takes on cancer, survival and what comes next

NOTE: Uplifting Athletes and Cambridge Healthtech Institute are proud to include some new young researchers in rare disease at this conference. They are our future partners. Advocacy groups interested in nominating young researchers for the Uplifting Athletes Young Investigator Draft can submit to:; Nominations open on November 21, 2020. Click here for additional information on applying for a guest pass directly.

2:10 PATIENT CO-PRESENTATION: The Patient’s Point of View: Clinical Trial Perceptions and Experiences

Annick de BruinAnnick de Bruin, MBA, Director, Research Services, Center for Information & Study on Clinical Research Participation (CISCRP)

Phyllis KaplanPhyllis Kaplan, Trial Volunteer and Patient with T1D

Results from a large-scale global study conducted in 2019 among patients and the public offer robust insights into the latest perceptions of clinical research and enrollment barriers, as well as patient engagement preferences (exploring patient receptivity to virtual trial models and the latest technologies). The 2019 CISCRP Perceptions & Insights study is the largest global study of its kind (over 12,400 responses from around the world, including experiences of 3,600+ prior study participants), offering robust global insights that audience members can directly apply within their own organizations. Learn what information is critical to support the participation decision-making process from the patient’s point of view. Identify key participation elements which matter most to patients and their support network. Determine which convenience-enhancing solutions create the biggest impact on overall experiences.

2:30 CASE STUDY CO-PRESENTATION: Co-Creating with Patients to Create a Better Clinical Trial Experience

Jackie ZimmermanJackie Zimmerman, Patient Advocate

Maura SnyderMaura Snyder, MBA, Global Head, Clinical Trial Engagement, Clinical Insights and Experience, Janssen Pharmaceuticals

Janssen, in partnership with one of our patient advocates, will co-present on an initiative established in 2019 to bring the global patient perspective and input into the Janssen patient engagement plans in immunology. While it’s important to hear about the strides that the industry is making in the areas of patient engagement – it’s more important to hear directly from the patients about their perspective on this. This discussion will provide the audience practical, tangible ways to approach patient engagement. There are a variety of methodologies to approach this patient voice work and we will share one way that we have found to be very beneficial and successful.

2:55 Closing Remarks

Micah Lieberman, Executive Director, Conferences, Cambridge Healthtech Institute

3:00 Close of Conference

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